getting under our skin · mately $9.1 billion. u.s. sales were down for the quarter, dropping 8.5...
TRANSCRIPT
By Jeffrey Bouley
SAN DIEGO—For Illumina Inc., reproductive health represents “a very exciting market opportu-nity for us for both our array and sequencing products, with seg-ments that go all the way from carrier screening on the one hand to developmental delay in young children on the other”—that sentiment arising out of recent statements by President and CEO Jay Flatley. To that end, the com-pany recently signed a definitive agreement to acquire Redwood City, Calif.-based Verinata Health Inc., a provider of non-invasive tests for the early identification of fetal chromosomal abnormali-ties, for $350 million upfront plus as much as another $100 million in milestone payments
through 2015.Upon completion of the
acquisition, Illumina will pos-sess access to Verinata’s verifi prenatal test, which it calls “the broadest non-invasive prenatal test (NIPT) available today for high-risk pregnancies,” as well as gain “the most comprehensive intellectual property portfolio in the non-invasive prenatal test industry.”
Describing non-invasive pre-natal testing as one of the most rapidly growing areas utilizing next-generation sequencing, Illumina sees the agreement with Verinata as demonstrating its commitment to developing innovative diagnostic solutions and providing its partners with
feBruary 2013 Volume 9, Number 2www.drugdiscoverynews.com
fiNaNce ................................................................... 3
markets.................................................................. 4
editorial/commeNtary ................................... 10
products & serVices ....................................... 30
facts & figures ................................................. 31wh
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Tools & Technology 12
gloBal news 6
DiagnosTics 15
omics & sysTems Biology 21
research & DevelopmenT 24
conTracT research services 27
Getting under our skin
Looking for a prenatal payoffIllumina agrees to acquire Verinata Health for $350 million up front plus $100 million in potential milestones
Pfizer, Halozyme partner on subcutaneous biologics delivery in potential $500 million-plus dealBy Kelsey KausTinen
SAN DIEGO—In pursuit of easing medicine administration meth-ods for patients, Pfizer Inc. and Halozyme Therapeutics Inc. have announced a worldwide collabora-tion and license agreement to devel-op and commercialize biologics for subcutaneous delivery. The partner-ship brings together proprietary biologics from Pfizer’s collections with Halozyme’s Enhanze technolo-
gy, a proprietary drug delivery plat-form based on Halozyme’s patented recombinant human hyaluronidase enzyme, rHuPH20.
Per the terms of the agreement, Pfizer has been granted a world-wide license to develop and com-mercialize products that combine its proprietary biologics, directed at up to six targets, with rHuPH20. The targets may be selected on an exclusive or non-exclusive basis. In return, Halozyme will receive an initial payment of $8 million, a sum that includes an upfront fee for exclusive licenses to two specified therapeutic targets in primary care and specialty care indications, as
well as the right for Pfizer to select up to four additional targets upon the payment of additional fees. If certain development, regulatory and sales milestones are met, Halo-zyme stands to receive additional payments of up to $507 million, and is also eligible to receive roy-alty payments on net sales of any licensed products.
“I am delighted about this oppor-tunity, as it has the potential to enhance Pfizer’s ability to optimize treatments for patients,” Jose Car-los Gutierrez-Ramos, senior vice president of Pfizer BioTherapeutics R&D, said in a statement.
sKin coNtiNued oN page 9
facTs & figures
Europe ranks second in global CRO marketFrost & Sullivan report says European CRO market will generate more than $11 billion in 2018 see page 31
conference preview: society of Toxicology
52nd annual meeting and toxexpo
One-stop toxicology shopAnnual meeting of the Society of Toxicology promises a wide range of toxicology and safety assessment science see page 18
End of the road for hESC opponentsU.S. Supreme Court declines to hear case seeking to restrict federal funding for human embryonic stem cell researchBy amy swinDerman
WASHINGTON, D.C.—Federal funding for scientific and medical research involving the use of human embryonic stem cells (hESCs) will continue unimpeded after the U.S. Supreme Court on Jan. 7 declined to review the controversial case of Sherley v. Sebelius.
It’s been a long road for the plain-tiffs in the case—adult stem cell researchers James Sherley and The-resa Deisher—who filed their lawsuit in 2009. Although their case reig-nited a national debate over hESC research—and even, at one point, temporarily brought federal funding for such research efforts to a halt—their legal effort has been grounded by the high court’s decision.
“We were of course disappointed that the court declined to hear the case, but of course, we were also not surprised,” Samuel Casey,
prenaTal coNtiNued oN page 17
After a four-year, contentious legal battle, Sherley v. Sebelius was put to rest last month when the U.S. Supreme Court declined to review the case.
sTem cell coNtiNued oN page 26
EDITOR’S FOCUS:Sherley v. Sebelius: Where do we go from here?see page 10
With the successful completion of an acquisition of Verinata by Illumina, the verifi test would continue to be offered through Verinata’s CLIA-certified and CAP-accredited laboratory, which will continue to act as a reference laboratory to gather some of the necessary clinical data for future regulatory submissions.
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For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 3Finance
EXTON, Pa.—Nuron Biotech Inc. ended 2012 by securing $80 mil-lion in private deals, including a $30 million equity investment by Healthcare Royalty Partners and a $50 million Synthetic Royalty agreement tied to the future sales of company products. The Synthet-ic Royalty consists of a non-dilutive financing alternative created by HC Royalty, and entitles HC Royalty to receive payments primarily secured by future product sales as well as by the underlying product assets.
“It is unique to find an emerg-ing specialty pharmaceutical com-pany that has near-term access to revenues from well-established commercial products as well as tre-
Nuron nets $80M for vaccine, biologics developmentmendous upside from a promising pipeline of novel drug candidates in treating serious diseases,” Dr. Gregory B. Brown, founding man-aging director of HC Royalty, said in a statement. “We are confident that Nuron can build the infrastructure to expand Meningitec’s market in Europe while capitalizing on new markets with unvaccinated and under-vaccinated populations. In the company’s pipeline, lead candidate
NU100, for example, is a proprietary human interferon beta-1b, which is in a Phase III clinical trial for mul-tiple sclerosis, and may improve long-term clinical efficacy, tolerabil-ity and safety for patients currently requiring therapy with interferon.”
The proceeds from the financing will be used to commercialize and expand Meningitec, a meningitis vaccine that Nuron acquired from Pfizer Inc. in December 2012. Pro-
ceeds will also go towards the clini-cal development of Nuron’s biolog-ics and vaccines for other infectious and neurodegenerative diseases, including HibTITER, a Haemophi-lus b conjugate vaccine, and NU100.
“Nuron appreciates this signifi-cant level of commitment from HC Royalty, which validates the recog-nized market opportunity for our branded products and candidates in development,” Dr. Shankar
Musunuri, founder and CEO of Nuron, said in a press release. “The successful closing of this transac-tion provides us the capital needed to execute our Meningitec commer-cial plan and reach key milestones in advancing our pipeline and in providing new treatment options for patients around the world.”
JMP Securities served as exclu-sive placement agent to Nuron for the financing. ddn
ultragenyx closes $75 million series b roundNOVATO, Calif.—A Series B financ-ing round has raised $75 million for privately held Ultragenyx Pharma-ceutical Inc., with Adage Capital Partners LP, leading the funding round. The round attracted new investors advised by T. Rowe Price Associates Inc., Jennison Associates LLC, funds and accounts managed by subsidiaries of BlackRock Inc., Sanofit-Genzyme, BioVentures and Shire PLC, and existing investors TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also took part.
“We deeply appreciate the sup-port of all our investors, new and existing, and their confidence in our ability to find and efficiently develop compelling new treatments for devastating rare genetic disor-ders,” Dr. Emil D. Kakkis, founder and CEO of Ultragenyx, said in a press release. “This financing trans-action is critical to expanding our efforts to deliver profound novel therapies that benefit even more rare disease patients.”
The funds will be used to fur-ther development of Ultragenyx’s lead drugs for rare genetic diseases, including UX001 and UX003, as well as additional undisclosed pro-grams. UX001 is being developed as a potential replacement therapy for heredity inclusion body myopathy, a severe muscle disease caused by the lack of an enzyme that processes pro-teins and fats that leads to progres-sive muscle weakness between 20 to 30 years of age. UX003 is an enzyme replacement therapy designed to treat mucopolysaccharidosis type 7, an extremely rare, progressive lyso-somal storage disorder that affects many of the body’s systems. ddn
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Markets4 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
Patent cliffs hit Sanofi in Q3PARIS—Sanofi saw its net income for the third quarter of 2012 drop to approx-imately $2.1 billion, a 23-percent decrease compared to the same three months of 2011. Overall sales for the quarter came in at approximately $11.7 billion, up 3.3 percent from the same period in 2011, and revenue from pre-scription drug sales was also up, posting a 1.4-percent increase at approxi-mately $9.1 billion. U.S. sales were down for the quarter, dropping 8.5 percent to just under $3.9 billion, a decrease credited to the effects of generic com-petition for Eloxatin, Sanofi’s drug for the treatment of colon or rectal cancer, and Lovenox, an anticoagulant for the prevention of blood clots and deep vein thrombosis. According to Christopher A. Viehbacher, CEO of Sanofi, “the long-expected impact of generic competition is having its way, but we are really seeing a light at the end of the tunnel,” crediting “the solid performance of our growth platforms … coupled with tight cost control” for allowing the company to limit the effects of the patent cliffs.
Evotec sees 16-percent revenue drop in Q3HAMBURG, Germany—The third quarter of 2012 saw Evotec AG posting revenues of $29.6 million, down 16 percent from the same period in 2011. Total group revenues for the first nine months of 2012 were up to $85.8 million, an 8-percent increase over the $79.8 million reported in the same period in 2011. Operating income for the first nine months remained positive at $3.8 million, down noticeably from the $12.7 million reported in the same nine months in
2011. Evotec’s liquidity, including cash, cash equivalents and investments, equaled $74.7 million at the end of September 2012. Evotec also announced the adjustment of its operating result guidance, which, before impairment and changes in contingent consideration for fiscal year 2012, is expected to be less than that of 2011. The company’s forecast for revenues for fiscal year 2012 remain the same: double-digit growth of company revenues to reach between $117.6 million and $120.3 million.
Bruker posts 7-percent jump in revenueBILLERICA, Mass.—For its third quarter ended Sept. 30, 2012, Bruker Corp. announced revenue of $447.8 million, up 7 percent from the $418.4 million reported in the same quarter of 2011. GAAP net income doubled, up to $39.7 million, or 24 cents per diluted share, compared to $19.8 million, or 12 cents per diluted share, in 2011. Adjusted net income for the quarter was also up, coming in at $47.1 million, or 28 cents per share, a moderate change from the $34.4 million, or 21 cents per diluted share, seen in Q3 2011. Bruker posted revenue of $1.27 billion for the nine months ended Sept. 30, 2012, up 8.3 percent from the $1.17 billion posted for the same period in 2011. GAAP net income for the nine-month period was $64.7 million, or 39 cents per diluted share, an increase over the $53.2 million, or 32 cents per diluted share, seen in the first nine months of 2011. Adjusted net income for the first nine months of 2012 was $91.6 million (54 cents per diluted share), a modest increase over the $87.7 million (53 cents per diluted share) posted in 2011.
By Burrill & Co.
SAN FRANCISCO—As 2013 begins, Burrill & Co.’s latest report looks back on the highlights of 2012 to describe a positive year despite serious economical roadblocks. A total of $71.1 billion in capital was raised by U.S. life-science companies in 2012, up 23.9 per-cent from $57.4 billion in 2011, and when the potential value from partnering transactions is included, 2012’s total jumps to $91.7 billion, compared to $80.4 billion in 2011. Global life-science venture financings rose 23 percent to reach more than $12.4 billion in 2012.
A total of $109.2 billion worth of M&A transactions was announced in 2012, a 31.2-percent drop from the total posted in 2011, and global partnering deals were down for the year as well. The total for global part-nering was valued at $37.6 billion for 2012, down 1.3 percent from 2011.
With eight new drugs gaining U.S. Food and Drug Administration (FDA) approval in December, 2012 ended with a total of 39 new drugs approved, five more than 2011 and the highest number since 1996. Of the eight approved in December, six were granted orphan drug status, and nearly half of those approved in 2012 had orphan drug designations.
“We’ve seen drugmakers embrace orphan drugs for a variety of reasons, including the opportunity to win faster approvals and run smaller, less costly clinical trials, and better chances of success,” says G. Steven Burrill, CEO of Burrill & Co. “The passage in 2012 of new incentives for developing rare disease drugs will only further fuel this trend, partic-ularly as more precision therapies are devel-oped to target subpopulations of patients
December sees 2012 tag out with record drug approvalswith a given disease.”
The Prescription Drug User Fee Act earned renewal in 2012, which aids the FDA in col-lecting fees for reviewing products and pro-vides accelerated approval pathways for com-panies developing therapies for rare diseases. In addition, the Jumpstart Our Business Startups Act, legislation that aims to make it easier for emerging companies to access capital via the public markets, was passed.
The report also forecasts a good year
for life sciences in 2013. The capital mar-ket’s position will likely remain tenuous as the world economy improves. Fundraising should be strong, with $100 billion forecast in 2013, and “corporate venture, disease advocacy groups and philanthropic organi-zations” will likely begin to take the place of traditional venture investors as the latter move toward later-stage deals and away from start-ups. Private financings are forecast to grow 20 percent in 2013.
According to the report, the industry will likely see 25 IPOs completed in 2013, and M&A activity is expected to increase by at least 20 percent. R&D collaborations are expected to continue to make up the bulk of partnering efforts in 2013, and a total of 35 drugs are expected to gain approval for the year. Sequencing and diagnostics will con-tinue to gain popularity, and digital health technologies and cost control will become greater facets of the healthcare industry. ddn
P u B l i C C o m P a n y n e w s
Pharmaceutical and biotech market indices
amex Pharmaceutical index
source: yahoo FiNaNce
400
350
300
250
200
150
100
50
0
363.98
331.61337.8332.25362.19
377.02353.6
372.15
338.09 335.76351.48
385.76
source: burrill & co.
Burrill select 1800
1600
1400
1200
1000
800
600
0
15151571
16231552
16391719
1346 1338 13251404 1389
1444
Burrill mid-Cap Biotech and small-Cap Biotech
source: burrill & co.
2000
1800
1600
1400
1200
1000
800
0
MID-CAPSMALL-CAP
986
1313
10961172
893.95 911.12824.55
941.62
1245
1544
1244
1096 1065
1347
1135
12821284
1453
1219
14781544
347
1096
9893.95
356 1076
829.11 793.9
1009
For the full-year 2012, a total of 16 life-science companies completed U.S. IPOs, the same as completed in 2011. The Burrill Select Index closed the year up 40.5 percent, the largest gain, though the other major
indices also ended the year positively: the Dow Jones Industrial Average was up 7.3 percent, the S&P 500 was up 13.4 percent; and the Nasdaq Composite Index closed up 15.9 percent.
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Global News6 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
ARIAD, NCRI collaborate on Phase III leukemia study
CAMBRIDGE, Mass.—An agreement was struck in early January between ARIAD Pharmaceuticals Inc. and Newcastle University, United Kingdom, on behalf of the U.K. National Cancer Research Institute CML Working Group. The organizations will collaborate on a multicenter, randomized Phase III trial to determine the impact of switching chronic myeloid leukemia patients being treated with a first-line tyrosine kinase inhibitor to ponatinib in cases of suboptimal response or treatment failure. The study’s primary endpoint will be the proportion of patients who achieve major molecular response (MMR) at three years on their first line of therapy, with secondary endpoints including proportion of patients who achieve therapy cessation three years after achieving stable MMR, therapy cost, safety, tolerability, overall survival, progression- and event-free survival and treatment failure at five years.
CEVEC, Yuhan ink license agreement
COLOGNE, Germany—CEVEC Pharmaceuticals and Yuhan Corp. have signed a CAP-Technology license agreement enabling Yuhan to make use of CEVEC’s CAP-T and CAP cell expression systems for the production and development of therapeutic proteins. CEVEC’s CAP-T CAP cells, immortalized cell lines for transient and stable protein produc-tion, are non-tumor origin cell lines that allow for superior protein yields in less time.
“With Yuhan we have won one of the most pres-tigious Asian pharma companies as a customer. We’re very pleased that Yuhan has chosen our CAP and CAP-T expression systems as a pivotal platform to develop therapeutically relevant molecules. I’m convinced that the cells will have a major impact to develop more efficient biologics and will also shorten the timeline to enter clinical phases,” CEVEC CEO Wolfgang Kintzel said in a statement.
Oxford Nanopore completes series of collaborations
OXFORD, United Kingdom—Oxford Nanopore Technol-ogies has announced the completion of agreements with several academic research institutions, includ-ing the University of Cambridge, Boston University, Stanford University, University of Southampton, Brown University and University of Illinois at Urba-na-Champaign. Oxford Nanopore’s current focus is on techniques for nanopore-based analysis through the use of biological and solid-state nanopores.
“The collaborations announced today add to our existing positions in core areas spanning cur-rent and future generations of nanopore technol-ogy,” said Dr. Gordon Sanghera, CEO of Oxford Nanopore, in a press release regarding the deals. “We are pleased to support further innovation in the laboratories of our collaborators, to comple-ment the pioneering work being performed by our own interdisciplinary R&D staff.”
Improving immune responseGerman pharma Phenex joins forces with Janssen Biotech to target autoimmune, chronic inflammatory disordersBy Lori Lesko
LUDWIGSHAFEN, Germany—Aimed at pro-viding an urgent unmet need for disorders such as rheumatoid arthritis, psoriasis and inflammatory bowel disease, Janssen Biotech Inc. and Phenex Pharmaceuticals AG have joined forces to discover compounds that tar-get the nuclear hormone receptor RORγT in the treatment of chronic autoimmune and inflammatory disorders.
Under the terms of an agreement announced Dec. 17, Phenex will receive an
immune coNtiNueD oN page 7
TICKER>> Janssen biotech and phenex
pharmaceuticals to discover compounds that target nuclear hormone receptor rorγt in treatment of chronic autoimmune and inflammatory disorders
>> phenex will receive upfront, milestone payments up to $135 million
Trio Tackles TranslaTional researchThree-way collaboration tackles Parkinson’s diseaseBy iLene schneider
DIJON, France—A year ago, a drug discovery company/pharmacology service provider (Oncodesign) partnered with a pharma-ceutical company (Ipsen) to discover and develop innovative LRRK2 kinase inhibitors
as potential therapeutic agents against Par-kinson’s disease (PD) and other therapeutic areas. A month ago, the collaboration added the expertise of an academic group exploring the roles of LRRK2 and a-synuclein in PD.
Building on Oncodesign’s January 2012 LRRK2 program with advanced Nanocyclix lead molecules with Ipsen, Oncodesign is
trio coNtiNueD oN page 8
“The aim of the program is to create in first instance the molecular tools that allow the validation of LRRK2 as an attractive target in PD, and in second instance LRRK2 inhibitors useful to treat the disease,” says Dr. Philippe Genne, CEO and founder of Oncodesign.
Getting AMP-ed up Daiichi sankyo, amplimmune partner to develop immune modulation therapyBy keLsey kaustinen
TOKYO—A broad strategic collabora-tion was announced early last month between Daiichi Sankyo Co. Ltd. and Amplimmune Inc. for the develop-ment of AMP-110, a therapeutic pro-tein from Amplimmune being devel-oped as a potential immune modula-tion therapy for autoimmune diseases.
Per the terms of the agreement, Dai-ichi Sankyo will pay an option fee of an undisclosed amount as well as more than $50 million to reimburse past and planned research and development costs for the compound, including funds for future development through a Phase II proof-of-concept study. In addition, Amplimmune will be eligible to receive milestone payments dur-ing the collaboration. Amplimmune’s responsibilities will include manufac-turing clinical supplies, handling regu-latory filings and conducting clinical trials through the proof-of-concept study. Both companies expect to col-laborate on further research into the mechanism of AMP-110 and identifica-tion of biomarkers that could predict response to the compound. Through the study, Daiichi Sankyo will have an exclusive option to acquire AMP-110. Should the option be exercised, Dai-ichi Sankyo will have sole responsibil-ity for all future development, manu-facturing and commercialization.
“We are very pleased to be collabo-rating with Daiichi Sankyo on AMP-110,” Michael S. Richman, president and CEO of Amplimmune, said in a press release. “This unique transaction allows Amplimmune to collaborate with an important and well-respected partner and positions the program for an acquisition that will provide signifi-cant value for both Daiichi Sankyo and our shareholders.”
AMP-110 targets the B7-H4 path-way, which is believed to play a sig-nificant role in maintaining tolerance and controlling inflammation by inhibiting T cell-mediated inflamma-
amp coNtiNueD oN page 8
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 7Global News
upfront payment and milestone payments up to $135 million upon meeting specific goals. Phenex will also be eligible to receive tiered royalties and milestones on the global sales of products that arise from the collaboration.
There is little doubt that a huge market exists for these disorders, which seem to come out of nowhere to disarm the body’s immune system. Autoinflammatory diseases are a rela-tively new category of diseases that are differ-ent from autoimmune diseases, according to the U.S. National Institutes of Health. Howev-er, autoimmune and autoinflammatory diseas-es share common characteristics in that both groups of disorders result from the immune system attacking the body’s own tissues, and also result in increased inflammation.
The part of the immune system that orchestrates all of this develops as a person grows, and is known as the acquired immune system. It “remembers” foreign antigens, or proteins, so that it can fight them if they come back. It employs white blood cells called lymphocytes.
In autoinflammatory diseases, the body’s more primitive innate immune system causes inflammation for unknown reasons. Auto-inflammatory disorders are characterized by intense episodes of inflammation, result-ing in such symptoms as fever, rash or joint swelling. These diseases also carry the risk of amyloidosis, a potentially fatal build-up of a blood protein in vital organs.
Researchers from Phenex and Janssen believe the solution is to work collabora-tively to identify compounds that are active against RORγT and optimized for preclini-cal development. Janssen will then have sole
responsibility for the continued development and worldwide commercialization of any compounds that arise from the collaboration.
RORgamma(t), or RORγT, is a nuclear receptor that was recently identified as a key differentiation factor of Th-17 cells—immune cells that produce and secrete Interleukin-17 (IL-17), which is believed to be a key player in chronic autoimmune-related inflammation.
The relevance of the IL-17 pathway has been highlighted by the fact that antibodies that target key cytokines in this pathway have demonstrated impressive efficacy in reducing symptoms in patients with plaque psoriasis.
The effectiveness of inhibiting the IL-17 pathway through small-molecule RORγT inhibitors was recently demonstrated in animal models in two adjacent publications in Nature.
“The upfront payment and near-term mile-
stones that may be achieved through this col-laboration are quite important for Phenex, as the funds received will make it possible for us to both collaborate with Janssen on RORγT and continue the clinical development of our proprietary FXR program through the next few years,” says Thomas Hoffmann, Phenex’s chief financial officer.
Under the terms of this agreement, “Phenex will be able to fund its operations and does not expect to seek further equity financing,” says Hoffman. “Phenex maintains a favorable and exciting position in its ability to both help the patients who could benefit from RORγT-based therapies and provide our shareholders with a satisfactory return on their investments.”
Murray McKinnon, vice president and head of Immunology Discovery at Jans-sen Research & Development, has been
impressed with Phenex’s particular expertise.“We have been aware of Phenex’s exper-
tise in the nuclear hormone receptor field for some years and have followed their RORγT program with interest,” McKinnon tells ddn. “Phenex brings strong expertise and capa-bilities in nuclear receptor biology to the collaboration, as well as a strong portfolio of chemical modulators of RORγT function. We believe our combined scientific efforts increase our overall probability of success.”
Even the latest, most sophisticated drugs do not help everyone, he notes.
“Despite the successes of antibody-based therapeutics in rheumatoid arthritis, psoria-sis and inflammatory bowel disease (Crohn’s disease and ulcerative colitis), there remains unmet need in patients who exhibit an inad-equate response, or who are non-responsive, to these therapeutics,” McKinnon says. “Oral-ly available molecules may have advantages in patient compliance for those who cannot tolerate—or are afraid of—injections.”
McKinnon defines “success” in this part-nership as “a robust, mutually beneficial sci-entific collaboration founded on rigorous sci-ence that enables Janssen to test the hypothe-sis in the clinic and ultimately advance novel therapeutic options for patients.”
Successful external collaborations “con-tinue to enhance our focus of driving strong science in the Immunology,” McKinnon adds.
“Ultimately, novel, new medicines will position the Janssen Immunology Thera-peutic area for future success beyond our current portfolio of biologics in address-ing unmet needs in autoimmune diseases, and satisfy patient and healthcare provider needs—while strengthening our industry-leading position,” he says. ddneditconnect: e021304
immunecoNtiNueD From page 6
“Ultimately, novel, new medicines will position the Janssen Immunology Therapeutic area for future success beyond our current portfolio of biologics in addressing unmet needs in autoim-mune diseases, and satisfy patient and healthcare provider needs—while strengthening our industry-leading position,” says Murray McKinnon, vice president and head of Immunology Discovery at Janssen Research & Development.
aligneD in oncologyChugai Pharma and Debiopharm announce an exclusive license agreement for anti-cancer agent FF284By Jeffrey BouLey
TOKYO—Under a deal announced Jan. 8, Chugai Pharmaceutical Co. has entered into a licensing agreement with Lausanne, Swit-zerland-based Debiopharm Group around a compound, FF284, discovered by Chugai that is about to enter the clinical development process for oncology indications.
Under the license agreement, Chugai will grant Debiopharm an exclusive license to develop and commercialize FF284 (known as Debio 1347 within Debiopharm) in all countries worldwide, including Japan. Under the same agreement, Chugai will grant a non-exclusive license to develop and commercialize a companion diagnostic for the FF284/Debio 1347 compound.
According to the terms of the license, Debiopharm and Chugai will collaborate to conduct a Phase I dose escalation study. In return for the license, Chugai will receive an undisclosed upfront fee as well as milestone and royalty payments from Debiopharm.
“We are delighted that we have concluded a license agreement for the development and marketing of FF284 with Debiopharm” said Tatsuro Kosaka, president and chief operat-ing officer of Chugai, in the announcement about the deal. “We will continue to coop-erate with Debiopharm in order to deliver FF284 to cancer patients as soon as possible.”
“We are happy to combine forces with Chugai for the development of Debio 1347,” added Rolland-Yves Mauvernay, president and founder of Debiopharm Group. “Due to its profile, Debio 1347 is expected to become a tailored treatment with high anti-tumor activity, which is very promising.”
Contacted via email, Debiopharm didn’t have much to add beyond what was noted in the news release, though David Deperthes, director of business development and licens-
ing, did note that Chugai first proposed the idea of such a collaboration at the BioAsia 2012 meeting, with due diligence conducted in summer 2012 and “negotiation started straight away after due diligence.”
Deperthes notes that “Debiopharm is always looking for innovative targets in oncology, and Chugai proposed a very robust and convincing preclinical package,” adding that “Debiopharm had the possi-bility to put resources rapidly to develop this project combining personalized medi-cine and clinical development. Chugai requested a rapid commitment. Both par-ties were aligned.”
Chugai Pharmaceutical, a member of the Roche Group, is one of Japan’s leading research-based pharmaceutical companies and touts its strengths in biotechnology
products. Specifically, Chugai is working to develop innovative products that may satisfy unmet medical needs, mainly focusing on the oncology area.
Debiopharm Group is a Swiss global bio-pharmaceutical group of companies founded in 1979, with a focus on the development of innovative prescription drugs that target unmet medical needs, with its area of focus, like Chugai’s, also on oncology. Debiopharm in-licenses, develops and/or co-develops promising biological and small-molecule drug candidates having reached clinical development Phases I, II and III—as well as earlier-stage candidates at times. Debio-pharm is also active in the field of companion diagnostics, with a view to progressing in the area of personalized medicine. ddneditconnect: e021307
“Due to its profile, Debio 1347 is expected to become a tailored treatment with high anti-tumor activity, which is very promising.”Rolland-Yves Mauvernay, president and founder of Debiopharm Group
Global News8 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
working in a research collaboration with the Laboratory for Neurobiology and Gene Ther-apy (LNGT) at the department of neurosci-ences at the Katholieke Universiteit Leuven (KU Leuven). The objective of the collabora-tion is to evaluate Oncodesign’s compounds in multiple pharmacology models for PD.
The Ipsen-Oncodesign collaboration ema-nated from Oncodesign’s discovery of novel macrocyclic LRRK2 inhibitors with attractive profiles. Ipsen entered into an option-based collaboration on this target. In this collabo-
ration, Oncodesign uses its Nanocyclix plat-form expertise to advance the program, while Ipsen contributes its know-how in central nervous system (CNS) disorders.
“Our partnership with Ipsen on the dis-covery of novel therapeutic agents in Par-kinson’s disease has advanced to a stage where we can use further in-depth expertise and advanced pharmacology models to posi-tion our leads,” says Dr. Jan Hoflack, chief scientific officer and head of Oncodesign’s discovery activities. “We are very excited to collaborate with KU Leuven to advance our understanding of both our inhibitors and the LRRK2 target.”
According to Dr. Philippe Genne, CEO and founder of Oncodesign, “the group of Veerle Baekelandt at KU Leuven is an expert team in PD in general, and more specifically related to LRRK2 and alpha-synuclein. The group was initially involved in a feasibility study of early Oncodesign LRRK2 inhibitors in which the investigators confirmed the potency and selectivity of the compounds in advanced cellular models. Now that the Oncodesign/Ipsen program is advancing well, the group is again involved in testing the novel compounds in advanced in-vitro and in-vivo models.”
Oncodesign will be responsible for the partnership to advance the drug discovery program. KU Leuven will perform the bio-logical evaluation of the best compounds, in a non-exclusive way. Other external part-nerships with key opinion leaders in PD are being set up related to the LRRK2 program, according to Genne.
The goal is to advance the science related to PD and LRRK2, especially related to a link with alpha-synuclein. In addition, the col-laboration will profile the Oncodesign com-pounds in advanced models to assess their potential in a translational manner.
The overall deliverables goal of the proj-ect is to select a LRRK2 inhibitor candidate compound for preclinical and clinical devel-opment by the end of 2013. The next goal is to establish clinical proof of concept for a LRRK2 inhibitor in a Phase IIa study. Ipsen will do the clinical development.
“We are delighted that our partnership with Oncodesign is moving forward as planned with the objective of developing
therapeutics for Parkinson’s disease patients,” says Dr. Claude Bertrand, executive vice pres-ident of R&D and chief scientific officer at Ipsen. “The Oncodesign collaboration with LNGT will greatly accelerate the progress of this research program.”
Bertrand adds, “Parkinson’s disease is a serious condition with high unmet medical needs where patients are seeking improved care and quality of life. Today, there is no treatment targeting the underlying patho-genetic mechanism leading to progressive deterioration in those patients.”
According to Genne, “LRRK2 is one of the most exciting ‘drugable’ targets for PD because of its genetic linkage with famil-ial cases of PD. Nevertheless, it is still an exploratory target that requires significant validation. The aim of the program is to cre-ate in first instance the molecular tools that allow the validation of LRRK2 as an attractive target in PD, and in second instance LRRK2 inhibitors useful to treat the disease.”
The collaborators estimate the commercial
potential to be medium to high, depending on the utility of LRRK2 inhibitors in sporadic PD as well as in familial cases of PD. ddneditconnect: e021305
triocoNtiNueD From page 6
TICKER>> oncodesign, ipsen and
researchers at Ku Leuven probe LrrK2 kinase inhibitors as potential therapeutic agents against parkinson’s disease, other therapeutic areas
>> oncodesign will use Nanocyclix platform to advance program; Ku Leuven will perform biological evaluation of best compounds; ipsen to undertake clinical development
“Our partnership with Ipsen on the discovery of novel therapeutic agents in Parkinson’s disease has advanced to a stage where we can use further in-depth expertise and advanced pharmacology models to position our leads.”Dr. Jan Hoflack, chief scientific officer and head of Oncodesign’s discovery activities
tory reactions. AMP-110 has been designed to mimic B7-H4’s natural ability to induce a co-inhibitory pathway that reduces inflam-matory T cells, and the compound has dem-onstrated potency in animal disease models. Daiichi Sankyo and Amplimmune intend to begin a Phase I clinical study of AMP-110 for the treatment of an undisclosed autoimmune disease indication sometime in the first half of this year.
Dr. Gary Fanger, senior vice president and chief operating officer at Amplimmune, says that Daiichi Sankyo emerged as an ideal part-ner for moving the AMP-110 program for-ward, a “very committed partner interested in helping to develop drugs that expand the course of medicine for patients with autoim-mune disease.”
“They had a very strong visionary com-ponent to their thinking related to drug development in the autoimmune space, and that correlated well with our perspectives, because this AMP-110 drug has a very novel
mechanism of action,” says Fanger. “And in talking with them and their scientific group and their clinical group, we really liked the alignment around the possibility of develop-ing drugs with novel mechanisms of action. So they had a wealth of creativity around drug design that we really liked.”
Thanks to the novelty of AMP-110’s mech-anism of action, the program is thought to
have the potential to treat a wide range of autoimmune diseases, to the point that Fanger says one of the toughest challenges was to narrow in on an indication. In consid-ering potential markets and unmet medical need, Fanger says the areas they are most interested in exploring with AMP-110 are rheumatoid arthritis, lupus, multiple scle-rosis and Sjögren’s syndrome.
Fanger adds that this partnership repre-sents the first time the two companies have worked together, and calls it a good fit for Amplimmune’s growth strategy. The option asset buyout approach of this partnership, he says, allows Amplimmune to balance its needs and the needs of pharma companies, and helps “to ultimately accelerate returns for our investors.” The company is consider-ing additional partnerships along this same format for the other biological therapeutics in its pipeline.
“This collaboration strengthens our com-mitment to working with partners that are at the forefront of science,” Dr. Glenn Gorm-ley, global head of R&D and senior execu-tive officer of Daiichi Sankyo, said in a state-ment. “Immune modulation therapy is one of the exciting areas of autoimmune disease research that has the potential to meet an unmet medical need. As a global pharma innovator, identifying and meeting unmet medical needs is an important part of Daiichi Sankyo’s mission. We are excited to start with the first trial.” ddneditconnect: e021306
ampcoNtiNueD From page 6
“Immune modulation therapy is one of the exciting areas of autoimmune disease research that has the potential to meet an unmet medical need. As a global pharma innovator, identifying and meeting unmet medical needs is an important part of Daiichi Sankyo’s mission.”Dr. Glenn Gormley, global head of R&D and senior executive officer of Daiichi Sankyo
Dr. Claude Bertrand, executive vice president of R&D and chief scientific officer at Ipsen, notes that “Parkinson’s disease is a serious condition … where patients are seeking improved care and quality of life,” but there is still “no treatment targeting the underlying pathogenetic mechanism leading to progressive deterioration in those patients.”
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 9GLOBAL NEWS
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Pfi zer licenses spinal muscular atrophy program from Repligen
pFIZER ALSO RECENTLY ENTERED into an exclusive worldwide licens-ing agreement with Repligen Corp. to advance Repligen’s program for the treatment of spinal muscular atrophy (SMA), an orphan neurodegenerative genetic disease that presents early in life.
The program includes RG3039, a small-molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies. Under the terms of the agreement, Repligen is entitled to receive up to $70 million from Pfi zer, starting with an upfront payment of $5 million and total potential future milestone
payments of up to $65 million, well as royalties on any future sales of SMA compounds developed under the agreement.
Under the terms of the agreement, Repligen is responsible for completing the fi rst two cohorts of an active Phase I trial evaluating RG3039 in healthy volunteers, which it anticipates will occur during the fi rst quarter. Repligen will also provide certain technology transfer services to Pfi zer, which will then assume full responsibility for the SMA program moving forward, including the conduct of any registration trials necessary for product approval. Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in Europe.
“There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options,” said Jose Carlos Gutierrez-Ramos, senior vice president of Pfi zer BioTherapeutics R&D, in a statement. “This partnership will combine our expert capabilities in advancing mol-ecules for genetic diseases with Repligen’s leading SMA program.”
The SMA program was originally in-licensed from Families of SMA. ddn
skincoNtiNueD From page 1
Halozyme’s Enhanze platform is based on rHuPH20. The enzyme removes the limita-tions on the volume of biologics that can be administered subcutaneously by “tempo-rarily [degrading] hyaluronan, a structural component of the subcutaneous space that is just beneath the outside surface of the human skin,” the company notes on its web-site. This creates a window of opportunity for the administration of larger injectable biolog-ics such as monoclonal antibodies, allowing molecules as large as 200 nanometers to pass through the subcutaneous space, the com-pany claims.
“We look forward to working with Pfi zer to apply Enhanze to these exciting targets,” Dr. Gregory Frost, president and CEO of
Halozyme, said in a press release. “Enhanze enables biologics to be delivered as a simple subcutaneous injection.”
Frost notes that following its acquisition of Wyeth, Pfi zer has emerged as a strong part-ner in biologics. The company, he says, is one known for its strength in oral medications and small molecules, but in recent years, Pfi zer’s work with recombinant proteins has been growing as well.
“When you think of some of the fi rst tar-gets that we’re collaborating on, which are primary care and specialty markets, Pfi zer has historically been a leader in these sorts of areas as well, from the commercial per-spective. So the collection of those two really made for an exciting partnership for us and a really good fi t,” says Frost.
The Pfizer collaboration is one of sev-eral Halozyme has established based on its Enhanze platform—with other partners like Roche, Baxter, ViroPharma and Intrexon—and Frost says he considers the approach a “win-win” for patients in seeking to simplify medicine administration. Great medicines and advancements have been made, he notes, and trying to turn those medicines into easy-to-use products for patients is very important, with the added benefi t of reduc-ing healthcare costs.
“Biologics, things like monoclonal anti-bodies today, they’re great medicines. We’ve made, as an industry, so much progress on them. But they are not proteins that you can take as a simple pill. We would all agree that a pill you take once per month would be nirvana—unfortunately, science is just not there,” says Frost. “Oral medications still have a very strong place in the industry, but when you start developing things like monoclonal antibodies, there’s been a tremendous shift away from the classic intravenous infusion towards more simplifi ed methods of adminis-tration. So self-administration for patients is something which is a trend that the industry is very much going toward.” ddneditconnect: e021302
“Oral medications still have a very strong place in the industry, but when you start developing things like monoclonal antibodies, there’s been a tremendous shift away from the classic intravenous infusion towards more simplifi ed methods of administration. So self-administration for patients is something which is a trend that the industry is very much going toward.”Dr. Gregory Frost, president and CEO of Halozyme Therapeutics
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Editorial10 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
ThaT’s noT fair! or is iT?By PEtEr t. kissingEr
a number of accusations in the 2012 elections—on both sides—struck me as extrapolations that were then rebroadcast out of con-text. I then began to
wonder if the very notion of fairness was worthy of study, or if the word had any substantive meaning at all beyond complexion or the weather.
Is it fair that a runner is faster and thus wins a race? Is it fair that if you study harder, you get a better grade? Is it unfair that others are smarter than you? Is it fair that you have two parents who love you and others do not? Is it unfair that you can’t stop smoking? Disease is unfortunate, but is it unfair? Does fair have anything at all to do with luck or diversity? Would there then be no luck or diversity if everything were fair? Are dif-ferences to be avoided or celebrated? What is a fair share—only something others should pay, but not you? Who decides?
In my youth, fair implied “not cheating.” We spoke of fair play or a fair ball in baseball. The word had nothing to do with outcomes achieved (or not) when the rules of the game were fol-lowed. If we had a fair chance, we could lose with disappointment, but without complaint. It was unfair to trip someone in a race or to copy
homework. It was fair to lose a race or get an “F” in math. A girl was near-perfect on every test in my 9th grade math class. I did not think this unfair, but I did find it annoying, so I stud-ied harder, but never got close. More recently,
a son struggled with physics. He said this was due to the fact that he was not Chinese, and that this was unfair. Neither his mother nor I could change his genome at that stage. Thus, a totally unanticipated consequence of Nixon engaging with China more than 40 years ago impacted our family. That damn Nixon and the one we’ve unfairly joked was Uncle Henry Kissinger.
I have recently become fond of overrepresented minority groups and
why they are overrepresented. I am now neither. I contend that underrepresented minority groups would do well to study the former and emulate those characteristics proven to work. This would indeed be both a smart and fair strategy. Com-plaining about the overrepresented minorities is, in my view, inappropriate. The best of them didn’t use unfair tactics to achieve their success over a curmudgeon like me.
Today, I note two totally irreconcilable fairness concepts, both widely held. In the first of these, the winner or achiever is recognized and reward-ed. That’s thought to be fair. In the second, fair
implies equal outcomes, an equal share of the recognition or rewards. The second is appeal-ing to egalitarians and engenders the thought of “entitlement” as in “all get a blue ribbon, and that’s only fair.’’ One could argue this second view carries back to the American Revolution, but making that extrapolation is not accurate. “All men are created equal” is a phrase counter-ing birthrights to royal positions, not the guaran-tee of equality of result. The choice of words in the Declaration of Independence was less than precise and subject to misinterpretation. The expressed right was to life, liberty and the pursuit of happiness. Any guarantee of achieving happi-ness destroys it and replaces meritocracy with a dystopia. Slavery complicated the Declaration, but we worked through that (slowly).
What do you think is the meaning of the word fair? Today, it seems to be a loaded four-letter word (an F-bomb?) used with convenient impre-cision. As with the more popular F-bomb, we can drop this one on any number of occasions. In this respect, most of us seem to follow F. Scott Fitzger-ald’s notion that “the test of a first-rate intelli-gence is the ability to hold two opposed ideas in the mind at the same time and still be able to function.” This quote is from an essay, “The Crack-Up,” which was published in 1936 in Esquire as the world was doing exactly that, and so seems to be doing once more. As now, we were indecisive and
fair coNtiNueD oN page 11
Peter T. Kissinger, Purdue University
Sherley v. Sebelius: where do we go from here?By aMy swindErMan
I n the 1960s, McCulloch-Till discovered stem cells while studying the effect of radiation on the bone marrow of mice. In the 1980s, two groups of researchers derived embryonic stem cells from mouse
embryos. In the 1990s, James Thomson devel-oped a technique to isolate and grow human embryonic stem cells (hESCs) in a cell culture. In 2006, Shinya Yamanaka successfully trans-formed human fibroblasts into pluripotent stem cells using four genes with a retroviral system.
With each groundbreaking discovery, thou-sands of people—from the world’s top scien-tists to average Joes—began to question some of the ethical, moral and social aspects of stem cell research, and in 1995, American lawmakers attempted to address those concerns in the form of Dickey-Wicker, an appropriations bill rider that prohibited the National Institutes of Health (NIH) from using government funds for the cre-ation of human embryos for research purposes, or for research in which human embryos are destroyed. In the early 2000s, President George W. Bush issued an executive order restricting the use of federal funds for hESC research. A few years later, his successor, President Barack Obama, reversed that order.
Five decades into the stem cell era, this promis-ing area of research is still very much under a cloud of uncertainty. So where do we go from here? In 2009, a pair of adult stem cell researchers sued the government over Obama’s executive order in a controversial case that effectively reignited the debate over hESC research. After a four-year court battle, the case found its way to the U.S. Supreme Court, which kicked off 2013 by declining to hear the case. And so, the uncertainty continues.
In our cover story, “End of the road for hESC opponents,” we bring you this news, along with some very interesting interviews with the plaintiffs in Sherley v. Sebelius, adult stem cell
researchers James Sherley and Theresa Deisher, as well as their attorney, Samuel Casey, man-aging director and general counsel for the Law of Life Project. All three of these opinionated people have always been generous with ddn in granting us revealing interviews, and although they seem a bit fatigued by their long court volley, once more they did not hold back.
“What we have going on right now with hESC research is the civil rights movement of the 21st cen-tury, a debate on whether science is going to enslave and manipulate the embryo,” says Casey. “They are the new slaves. They are too small to vote. We are reopening the door on human subject experimentation, something that until now, only World War II closed the door on. I have spent the last 10 years trying to point out the truth, and this is where we today.”
Casey says the plaintiffs won’t continue to pursue their cause in court, and a legislative remedy or renewed examination of Dickey-Wicker may be needed to end the debate—“but ultimately, language is plastic and will be manip-ulated depending who has the political power,” opines Casey.
“Obama is largely ruling on executive order now, because you can’t get anything through Con-gress,” he alleges. “It is a great disappointment to live in a country where a president’s executive order can be deemed to abdicate your rights.”
For Sherley, his focus will be on his stance that human embryos are living human beings, and thus, the government should not be funding research that uses them.
“The one thing I want—although I don’t think he’d ever do it—is for [NIH Director] Francis Collins to come out, put his stake down and answer the question of whether human embryos are living human beings. If he says yes
to that question, we should not be funding this research,” says Sherley.
Sherley, who has authored hundreds of op-eds and letters to lawmakers on the subject, says he is considering compiling them into a collection
of writings.“I’d also love to get back into a
university setting, where I can have a larger imprint on young minds, but I think that writing will be the best use of my time,” says Sherley, who was a professor at the Massachusetts Institute of Technology from 1998 to 2007. “Something I am interesting in writing is some scholarly work on what happened in Roe v. Wade. I think we have to go back and look at that
decision as scientists. When we look at the case, there is no science in it. Our case was like that, too. When we are talking about hESC research, we are talking about abortion. We need to change the way we treat the unborn and embryos.”
No matter how the debate continues to play out, Deisher maintains that adult stem cell research will come out the victor.
“It’s not rocket science,” says Deisher, who heads AVM Biotechnology, an adult stem cell research firm in Seattle. “hESCs form tumors, illicit immune rejection and are outrageously expensive. We need to inform the American people that adult stem cells are a better alter-native. We can do this work without exploiting other humans.”
Meanwhile, until scientists and the govern-ment can reach more of a common ground on the issue, you can read more about these individuals, their case and their cause on our website, www.drugdiscoverynews.com, as well as our blog, www.drugdiscoverynews.com/blog. We invite you to weigh in on the debate on our blog, because isn’t it about time we get serious about it? ddn
Amy Swinderman, ddn Chief Editor
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 11Editorial
coMMentAry: The times, they are a changin’ in the biopharma market By siMon wEBstEr, avacta analytical
T he biopharmaceutical devel-opment community has success-fully developed a broad range of highly effective therapies for a wide variety of serious and debili-
tating diseases. However, developing biophar-maceuticals is inherently an extremely chal-lenging and commercially risky undertaking, often working to tight timelines and typically resource-constrained. For many, this means adopting a pragmatic approach to develop-ment, where the goal is to get the product to a point where its clinical performance and analytical characterization are “good enough” to satisfy the regulatory authorities and get to market.
There are a number of drivers simultane-ously working to move the biopharmaceuti-cal industry into this increasingly competi-tive era, including: the availability of multiple innovator products to treat the same indica-tions; the emergence of biosimilar products and development of a framework by which they can get approval; arguably, an increas-ingly demanding regulatory environment; evolving scientific understanding that also highlights areas of ignorance; and emerging markets such as those in Asia.
As a result, healthcare refunders, prac-titioners and patients will have an ever-increasing choice about which drug to buy and can increasingly be expected to base their purchasing decisions on factors such as relative price, convenience and cost of administration, effectiveness and degrees of adverse reactions. Both innovator and bio-similar developers will need to adapt in order to compete effectively in this new environ-ment. A number of aspects of the develop-ment process, although already very impor-tant, will become increasingly critical to the commercial success of the resulting products.
It would seem that both originators and biosimilar developers will need to do more—and do it better, faster and more cheaply—if they are to succeed in the new marketplace. However, for many, the economic reality in the current climate may be that there will be little, if any, additional resources available to achieve these ambitious goals. New strate-gies and ways of working will be required; developers will need to be more open to new ideas and approaches, and to embrace new and emerging technologies that conservative attitudes may previously have prevented. Working harder is probably not enough, or in many cases, possible. Developers will need to work smarter.
Every stage of the development and manu-facturing process has potential for optimiza-tion, and a wide range of innovative tech-nologies and strategies are already available.
Embracing new technology, however, brings with it some risk and fear of the unknown. If current practices are considered good enough, why risk something new? As a result, many innova-tions have struggled to gain acceptance; this may be the time to reconsider some of these, in the drive to move beyond “good enough” and toward “the best things can be.” As these inno-vations, both relatively developed and just emerging, are too numerous to detail here, a few areas where the development pro-cess might be advanced to improve prod-uct competitiveness have been selected for discussion.
speeding development: start right, move fasterA key potential cause of slow and expensive development can be early selection of a can-didate molecule that, while efficacious and specific, is fundamentally unstable and con-sequently difficult to develop, manufacture and achieve adequate storage stability in a convenient dosage form. Picking the right candidate is, however, easier said than done. Early in development, both time and protein material are typically in short supply, limiting the number of candidates that can be investi-gated and the extent of analytical character-ization that is possible for each.
To overcome these issues, analytical screening technologies are needed which are rapid and automated (high-through-put), consume very little material and also make analytical measurements that are in some way predictive of how the protein will behave during manufacture and subsequent storage. Such technologies allow candidate protein molecules to be screened for “devel-opability”—or, in the context of this article, “competitiveness”—in addition to basic therapeutic function. If the measurements made have some predictive power, the risk of progressing too far with a molecule which is ultimately incompatible with modern, cost-effective platform manufacturing processes, or which can’t be made suitably stable for long-term storage in a convenient dosage form, is reduced. The same small-volume, high-throughput approach also offers the potential to rapidly and cost-effectively test the efficacy of protein engineering efforts to improve stability earlier in development, and to identify optimum solution conditions for processing and storage.
A tough, stable molecule, selected from the outset to be inherently compatible with modern platform manufacturing pro-cesses and for which the successful results
of long-term storage stability studies can be (almost) assured, has a better chance of progress-ing through development more rapidly, costing less to develop and reaching the market soon-er. Once at market, the selected and/or engineered-in attributes should result in lower manu-facturing costs, longer shelf life and potentially other benefits to the customer, increasing the
molecule’s chances of commercial success. Instrumentation suitable for this type of stability screening is readily available from a wide range of vendors at comparatively modest cost, and a number of the largest biopharmaceutical developers are already embracing this approach.
Analysis as a route to market … and as a roadblockAnalytical characterization plays a pivotal role in biopharmaceutical development and manufacture, and takes on a new importance with the emergence of biosimilar products. Therapeutic proteins are highly complex entities, and fully characterizing them is challenging, or indeed arguably not possible, with current technology. This presents a key challenge to biosimilar developers, who need to demonstrate to the regulatory authorities that their product is “highly similar” to the marketed reference product.
U.S. and European regulators have indicated that the scope and extent of expensive and time-consuming clinical testing they will require will depend on the apparent similarity, based on initial physiochemical characterization. The authorities recognize the current limitations of the analysis of proteins and recommend the use of a wide range of analytical measure-ments, each of which may have limitations, but which, when combined, build as complete a picture—or “fingerprint”—of the molecule as possible. This is sometimes described as a “totality-of-the-evidence” approach. It is therefore potentially highly advantageous for biosimilar developers to present as comprehen-sive a physiochemical analytical comparison of their new proposed product and the reference material, rather than taking a more limited, “play-it-safe, bare-minimum” approach.
A range of both new and established, but traditionally underused, analytical tech-niques are available which can add consid-erably to a convincing picture of similarity. Embracing new analytical technologies is not without risk, but the potential rewards of a faster, cheaper route to market could be sig-nificant for biosimilar developers. The corol-lary, of course, is that innovative developers may wish to build a more comprehensive
analytical fingerprint of their “reference” product to demonstrate dissimilarity of a potential biosimilar with their material.
new technologies to meet new challengesTwo related areas where innovation may poten-tially improve the competitiveness of a product are the way it is administered to the patient (delivery) and the use of stabilizing additives to improve stability and shelf life (formulation). However, for many time- and cash-pressed developers, the concept of thoroughly optimiz-ing the protein formulation or adopting new formulation technologies for improved long-term storage stability or delivery properties may be seen as a luxury. Additionally, biosimi-lar developers may consider any deviation from the originator formulation as a risk that will need explaining to the regulatory authorities.
However, in a newly competitive market, improved formulation may be a route to achieving a market advantage. This thinking can already be seen with a move away from lyophilized products—which need reconsti-tuting and administering by healthcare pro-fessionals—toward liquid formulations, pre-filled syringes and patient self-administration. The technical challenge of this shift is some-times considerable and not free of risk for the developer, but in this example, the benefits to the customer are significant, going beyond simple convenience and extending to overall cost savings for the healthcare provider.
For innovator and biosimilar developers alike, delivery and formulation provide not only improved product performance, but also the potential to engineer useful additional intellectual property protection into prod-ucts where the patent on the basic therapeu-tic protein has expired. There are multiple companies around the world with innova-tive formulation and delivery technologies that offer the potential reward of increased product competitiveness and improved IP position. Maybe now is their time to shine.
good enough isn’t good enough anymoreA host of innovative approaches and technol-ogies exist that have the potential to improve almost every stage of the process of develop-ing and manufacturing biopharmaceuticals. Embracing them, however, is not without risk to developers, and in an environment where the status quo is considered good enough, they struggle to gain acceptance. A change in the market dynamics for biopharmaceuticals may, however, mean this attitude will have to change and opportunities for innovation will increase. In the meantime, for all the small companies with the innovative analyti-cal, formulation, delivery and manufacturing technologies—hang in there! ddn
Simon Webster is chief scientific officer and co-founder of Avacta Analytical, a specialist contract research organization providing biophysical characterization services and innovative instrumentation to the biopharmaceutical market.
let our troubles grow. Fair deserves a rest. A more precise set of words could include meritorious, disciplined and ethical.
We learn the most from failure, and the more we fail, the more we learn from trying. To hide this fact behind self-esteem does not
serve our students well and prepare them for the real world. Keeping score in sports is encouraged, and we learn to handle dis-appointments to try again. Why are we so reluctant to face the inevitable disappoint-ments in other endeavors? Inventing an excuse called “that’s-not-fair” doesn’t serve us well.
We are in an industry where many spend
a career and never work on a drug that is approved for sale. Our hit rates are extreme-ly low, yet the cause justifies trying. That’s noble and fair. What is unfair in our game is fiddling with the data to increase our odds. John Adams famously noted in 1770 that “facts are stubborn things; and whatever may be your wishes, our inclinations or the dictates of our passion, they cannot alter
the state of facts and evidence.” It’s sad to note the number of Lance Armstrongs in politics, science and business. Making stuff up is cheating. That’s not fair. ddn
peter t. Kissinger is professor of chemistry at purdue university, chairman emeritus of basi and a director of chembio Diagnostics, phlebotics and prosolia.
faircoNtiNueD From page 10
Simon Webster, CFO, Avacta Analytical
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tools & technology12 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
Bruker teams with Erasmus Medical Center
BREMEN, Germany—An exclusive licensing agree-ment has been established between Bruker and Erasmus Medical Center for the rapid testing of beta-lactamase activity using MALDI-TOF technol-ogy. The new method is fully compatible with the Bruker MALDI Biotyper system used for MALDI-TOF mass spectrometry-based identification of micro-organisms. No financial details were disclosed.
“We have been working with MALDI-TOF mass spectrometry for many years originally in the field of clinical proteomics, but more recently the increased usage of the MALDI-TOF approach for microbial identification also became an area of interest for us … With their outstanding expertise in mass spectrometry and clinical microbiology market presence, we think that Bruker is the right partner to bring such novel assays to the mar-ket,” Dr. Theo Luider, head of the Laboratories of Neuro-Oncology, Department of Neurology, Eras-mus University of Rotterdam, said in a statement.
Titian, Labcyte unite technologyLONDON—Titian Software and Labcyte Inc. have announced the integration of Titian’s Mosaic sample management suite, which manages and tracks sample libraries with improved sample preservation and viability, with Labcyte’s Access workstation and POD automation platform, both of which incorporate the company’s Echo liquid handler. The combination enables rapid, accurate and fully traceable automated workflows for high-quality assay development and miniaturization.
“We have been working in close collaboration with the team at Labcyte to integrate the Mosaic web-based sample ordering facilities to the Access workstation. One of the key objectives was to give users the convenience of requesting low-volume assay-ready plates, with these being prepared automatically with very high precision,” Edmund Wilson, CEO of Titian, said in a press release.
Hamilton breaks ground on new facility
FRANKLIN, Mass.—Hamilton Storage Technologies has announced the establishment of a new facility meant to deal with the growing business for the company’s automated sample management and storage systems. The new facility will become headquarters for the Sample Management divi-sion and the East Coast competence center for Hamilton Co. The establishment will replace two existing facilities in Hopkinton and Milford, Mass.
“The company selected Franklin primarily because of its proximity to biotechnology commu-nities and skilled talent pool in the greater Boston area. By more than quadrupling our space, we can bring all of our operations under one roof and still have plenty of room to grow, while providing a more collaborative and productive work environment for our employees,” said Matt Hamilton, vice president of Hamilton Storage Technologies, in a statement.
Shifting from manual to automaticRoche, PSS unite to develop fully automated emulsion PCR instrumentBy Kelsey Kaustinen
MATSUDO, Japan—In pursuit of streamlining sample prep workflows, Roche and Precision System Science Co. Ltd. (PSS) have signed an exclusive agreement for the development and manufacture of a fully automated emul-sion PCR instrument for Roche’s portfolio of next-generation sequencing platforms. The instrument will support Roche’s GS Junior and GS FLX+ systems in addition to its next-generation sequencing platform currently in development. No details were disclosed as to the financial terms of the deal.
“This partnership aims to address one of the key needs of sequencing customers. The automated solution will not only improve the efficiency of laboratory workflows, but also increase the reproducibility of results by eliminating manual workload,” said Dan Zabrowski, head of Roche Applied Science, in a press release. “This development pro-gram has made great progress over the last
year, and we are looking forward to work-ing with PSS because they offer outstanding expertise and have a strong track record in
developing fully automated solutions.”According to Thomas Schinecker, head of
auto coNtiNueD oN page 13
Roche has united with PSS to develop a fully automated emulsion PCR instrument for Roche’s next-generation sequencing platforms. The agreement builds on a long-term relationship between the companies.
Market share for Bio-Rad; focused direction for MorphoSysBio-Rad acquires MorphoSys’ AbD Serotec for approximately $70 million in a cash dealBy Jeffrey Bouley
HERCULES, Calif.—Bio-Rad Laboratories Inc. announced Jan. 10 that it had completed its acquisition of AbD Serotec, a division of Mar-tinsried, Germany-based MorphoSys AG, for about $70 million in U.S. dollars. The news that an acquisition deal was in the works and awaiting customary closing conditions had come just a little less than a month previous.
Touting the strengths of the newest mem-ber of the Bio-Rad family, the company notes that AbD Serotec “is one of the world’s lead-ing antibody manufacturers,” offering more than 15,000 antibodies, kits and accessories. In addition, AbD Serotec has an ISO 9001- and ISO 13485-certified production facility in the United Kingdom and facilities in Ger-many and the United States as well.
Or, as Bio-Rad President and CEO Norman Schwartz put it, “With AbD Serotec’s com-prehensive catalog of antibodies, we are able to offer our customers total assay solutions
that can be validated on many of our research platforms for Western blotting, multiplex protein expression, ELISA and cell sorting.”
When the deal was first announced Dec. 16, Brad Crutchfield, president of Bio-Rad’s Life Science Group, repeated much the same sentiment as Schwartz, but also noted, “We are impressed with the quality of AbD Serotec’s products as well as the company’s reputation. With this acquisition, Bio Rad will have access to a comprehensive catalog of antibodies ... in addition, we will be able to exploit a powerful in-vitro technology to accelerate future antibody generation.”
According to the terms of the transaction, Bio-Rad acquired all of MorphoSys’ AbD
aBd coNtiNueD oN page 14
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acquires abD serotec, a division of Morphosys, for $70 million
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124 and counting Venerable WHEATON Industries acquires chromatography vial supply company MicroLiter Analytical SuppliesBy lloyd dunlap
MILLVILLE, N.J.—WHEATON Indus-tries has acquired MicroLiter Analyti-cal Supplies Inc., of Suwanee, Ga., in a move that adds a specialty brand of precleaned chromatography sample vials and accessories for use with auto-samplers to WHEATON’s product mix.
WHEATON, which will celebrate the 125th anniversary of its corporate found-ing in 1888 next year, brought scale, cash flow and global reach to the marriage, says company president Wayne Brinster.
“MicroLiter, a leading brand in high-precision chromatography, is a strate-gic step in strengthening WHEATON’s
vial coNtiNueD oN page 14
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 13tools & technology
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Roche Sequencing Solutions, this agreement builds on a “long-term reciprocal relationship” between the two companies, who began collaborating on automated DNA extraction 15 years ago. Roche and PSS successfully developed and marketed nucleic acid purification products as a result of that agree-ment. In addition, Schinecker notes, “PSS has developed Roche’s MagNA Pure LC 2.0 System and MagNA Pure Compact System, which are flexible nucleic acid iso-lation and purification platforms that utilize the PSS proven ‘Mag-tration technology.’”
“PSS has been very instrumen-tal in establishing themselves as experts in a niche market of new automation system concepts,” says Schinecker. “PSS manufactures sys-
tems that automate sample prepa-ration process before gene, protein or immunological analysis—such as fully automated DNA extractor, mainly based on the PSS’ patented ‘Magtration technology’—and it supplies those systems/instruments to worldwide markets. In addition, PSS also produces the reagents and the plastic consumables used in these automated systems.”
At present, manual upfront prep-aration of genomic samples is time-consuming and complicated, but the new instrument will serve to automate the entire emulsion PCR process. By streamlining the work-flow, the hands-on time required
autocoNtiNueD FroM page 12
“Preparing samples for sequencing is a complex, high-skill process that continues to hamper broader use of the technology. We believe that together, PSS and Roche will be able to develop an automated instrument that helps overcome this obstacle and promote greater use of advanced sequencing systems.”Hideji Tajima, president of Precision System Science
will drop from several hours to only a few minutes.
In addition to the instrument itself, PSS will also be developing and manufacturing the accompany-ing consumables, vessels and tips. The product will enable communi-cation with a laboratory network or LIMS “to download sample input data directly into the Automation Instrument and upload subsequent run information,” says Schinecker, and will have features such as a touch screen, integrated PC, solid/
liquid waste management system and RFID readers for identifica-tion of reagents and consumables. Roche will oversee the develop-ment process and provide PSS with feedback.
The instrument, says Schinecker, will fully automate the entirety of the emulsion PCR process, includ-ing emulsion generation, amplifi-cation, emulsion breaking, bead enrichment and “the annealing of the sequencing primers on a PSS-developed liquid-handling robot.”
By eliminating the manual work-load, reproducibility of results will increase as the chance for human error or variation decreases.
“To obtain ready-to-sequence DNA beads with copies of clonally amplified DNA, users will only have to place reagents such as capture beads and primers along with the prepared DNA library and select the desired platform,” he explains.
“We are delighted that PSS and Roche are expanding their long-standing relationship in automated
DNA extraction to the challenging field of DNA sequencing,” Hideji Tajima, president of PSS, said in a statement. “Preparing samples for sequencing is a complex, high-skill process that continues to hamper broader use of the technology. We believe that together, PSS and Roche will be able to develop an automated instrument that helps overcome this obstacle and pro-mote greater use of advanced sequencing systems.” ddneditConneCt: e021310
tools & technology14 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
Serotec research reagent and diag-nostic business, comprising the AbD Serotec segment of MorphoSys AG as well as the subsidiaries MorphoSys UK Ltd., MorphoSys AbD GmbH and MorphoSys U.S. Inc. In addition, the transaction includes a fully paid-up, non-exclusive license agreement to use MorphoSys’ HuCAL technology for diagnostic and research purposes.
Following the announcement of the deal in December, Dieter Feger, senior vice president at AbD Serotec, reached out to customers via the company’s web-site to say that AbD Serotec welcomed the news and “sees this as a great oppor-tunity to work with one of the world’s leading life-science and clinical diag-
nostics companies. We will continue to serve you with the same commitment to quality and customer service; in that regard, nothing will change.”
For its part, the former parent company of AbD Serotec is looking at the sale of the research and diagnos-tic antibody company as a chance to increase its focus on therapeutics.
“We are making great progress in our therapeutic antibody pipeline, which is a huge potential value driver for MorphoSys,” according to Dr. Simon Moroney, CEO of MorphoSys. “With the sale of AbD Serotec, we will bring a laser-like focus to driving growth in this, our area of core competence.”
Jens Holstein, chief financial offi-cer of MorphoSys, added in the com-pany’s news release about the sale that “Bio-Rad provides AbD Serotec and its employees with an ideal environment in which to exploit its potential in the research and diagnostic arena.”
Zacks Investment Research was also upbeat about the acquisition deal, writ-ing in an investor’s note that the acqui-sition “will enhance Bio-Rad’s Life Science Research segment as well as its mainstay Clinical Diagnostic fran-chise” and expects the transaction to strengthen Bio-Rad’s market position.
“With an improved portfolio, Bio-Rad should be able to gain an edge over its competitors,” Zacks noted. “Also, higher revenues and substantial goodwill enhancement seems to be on the cards for the company due to the complementary portfolio of AbD Sero-tec. We are rather optimistic regarding Bio-Rad’s ability to accelerate growth via inorganic means.” ddneditConneCt: e021308
aBdcoNtiNueD FroM page 12
AlliAnce of A lifetimeAlmac and TTP Labtech partner on fluorescence lifetime technology for screening applicationsBy amy swinderman
CRAIGAVON, Northern Ireland—Two leaders in instrumentation, Almac and TTP Labtech, have joined forces to give researchers an innovative tool to perform screening appli-cations without the bottleneck of false hits.
This improved screening performance comes in the form of fluorescence lifetime technology (FLT), a reading modality that offers a robust, antibody-free, homogeneous assay platform that enables researchers to avoid interference from fluorescent com-pounds within a screening library. The offer-ing combines Almac’s FLEXYTE assay with TTP Labtech’s Ameon system.
Developed for peptide engineering and chemical synthesis and design, Almac’s FLEXYTE assay platform offers solutions for many major therapeutic target classes, including kinases, proteases, phosphatases, DUB and an increasing number of epigenetic targets. The platform, which is still in devel-opment, harnesses the power and potential of FLT to provide an efficient and economical platform for screening and profiling.
TTP Labtech touts its Ameon system as “the next generation of FLT reader technol-ogy.” By offering real-time decay curve analy-sis, the Ameon system provides speed, preci-sion and data quality for FLT assays that can be readily integrated into high-throughput screening workflows.
The combined solution represents a poten-tial game changer for any researcher perform-ing screening applications, says Dr. Wayne Bowen, chief scientific officer of TTP Labtech, which is based in Cambridge, England.
“There is some pain in the industry with some of the technologies we have now. People don’t want to admit it, but until a new technol-
ogy comes along, you are stuck there. I certain-ly see FLT being regarded as the best screening application compared to some of the existing technologies we have now,” says Bowen.
To overcome those hurdles and bring a better solution to the market, “this area of research needed two well-known industry players get into this market,” says Bowen.
“One reason this technology has not yet come into fruition is that there has not been an offering that is strong in both a reader and reagents. This is paramount—you must have that,” he says. “If people are going to reconfigure their screening applications to FLT, that is a big undertaking. You need cred-ibility to adopt these types of technologies, a sense of security.”
Both companies tout the experience and market-leading position of the other.
“Almac is a leader in FLT reagents, so we are very pleased to be hooking up with them. We like to work with the best,” says Bowen.
TTP Labtech has an equally strong reputa-tion for introducing novel technologies in the instrumentation market, says Dr. Robert Grun-
dy, director of research alliances at Almac.“They are a trusted, recognized brand in
the instrumentation world,” Grundy says. “They are very easy to work with in the sense that they embrace new ideas and are very dogmatic in their approach to technology development. It is very important to Almac that we are complemented by a company that is well-known to people and trusted.”
Because neither company likes to second-guess the market, the combined offering is in beta testing and will be until summer.
“We want to supply a product that people can use, and meet their specific demands,” says Bowen. “We will test this at a few sites and get feedback before launching it later this year.”
The partners are targeting pharma com-panies with large-screening capabilities and interest, and service providers who are look-ing to provide screening services, says Grundy.
“We believe this technology will enable them to get more out of their compound libraries, and do it for less and more quickly,” he says. ddneditConneCt: e021311
According to TTP Labtech, by offering real-time decay curve analysis, the company’s Ameon system provides “a revolutionary combination of speed, precision and data quality” for Almac’s FLEXYTE assays.
“With AbD Serotec’s comprehensive catalog of antibodies, we are able to offer our customers total assay solutions that can be validated on many of our research platforms for Western blotting, multiplex protein expression, ELISA and cell sorting.”Norman Schwartz, president and CEO of Bio-Rad
leadership in chemical analysis products. To that end, we welcome MicroLiter’s dedicated customers and advocates,” says Brinster. “We’re looking forward to a syn-ergistic relationship in which we can better support scientists and researchers through efficiencies and product line expansion.”
Brinster notes that WHEATON cur-rently has a manufacturing operation in Europe and a presence through distribu-tors in Asia, South American and Africa, and points out that MicroLiter, founded in 1996 to provide high-quality consum-able products to the autosampler market, is recognized for its focus on supplying pre-cleaned, sample containment prod-ucts processed in Class 10,000 cleanroom facilities. Its product line includes 12x32mm glass autosampler vials, inserts, septa and closures, and the MicroLiter Plate Sampling System, a patented design that provides a 96-well, multi-array sample-handling for-mat for chromatography autosamplers.
“We have had a line of chromatography products for several years,” Brinster notes. “It’s a competitive line but the buying deci-sion can hinge on things like packaging or cap design. MicroLiter, on the other
hand, is regarded by customers as having products that are effective in background reduction, which is particularly important in the high-precision area. You can get the job done without it, but the absence of competing peaks makes the job easier.”
MicroLiter’s focus is on offering high-per-formance autosampler vials and accessories. To underline this, “we do not consider any vial clean that enters our facility. To ensure cleanliness we have installed a cleaning pro-cess that results in very low particulate vials. As you know, gamma-radiating products give you clean dirt. Dirt, however, is still dirt in chromatography. MicroLiter believes we have an overall higher standard of prod-ucts because we control the cleanliness of the products we sell, not our suppliers,” the company website states.
Brinster points out that WHEATON is supporting continued product development
and service improvements with its supply chain expertise and additional sales support from its established team of specialists. “We didn’t buy MicroLiter to roll them up,” Brin-ster states. “We’ll capitalize on volume pur-chasing and other similar economies, but MicroLiter will continue to deal with its cus-tomers from its very good operational base.”
Kim Gamble, founder and president of MicroLiter Analytical Supplies, adds: “We are delighted to become part of WHEATON, a brand that honors the work of scientists.”
Gamble will remain involved with the business, providing consultative and leadership support. As part of the acquisi-tion, the Instrument Top Sample Prepara-tion products and patents were retained by prior management, as this portion of MicroLiter’s business did not fit the WHEATON business model. ddneditConneCt: e021309
vialcoNtiNueD FroM page 12
“MicroLiter, a leading brand in high-precision chromatography, is a strategic step in strengthening WHEATON’s leadership in chemical analysis products. To that end, we welcome MicroLiter’s dedicated customers and advocates.”Wayne Brinster, president of WHEATON Industries
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DIAGNOSTICSFor more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 15
Lab21 divests S.C. operationsCAMBRIDGE, United Kingdom—Personalized medi-cine and clinical diagnostics specialist Lab21 Ltd. has announced the sale of its South Carolina-based laboratory operations to Reedy Acquisitions Corp., formed by existing local management and South Carolina-based investors. The transaction removed a loss-making business from the Lab21 Group and was completed at the end of 2012. No financial details were disclosed.
“After three years of developing the clinical service business in South Carolina, the Group has taken the decision to sell the laboratory opera-tions in Greenville and Columbia. This sale does not impact our core strategy to focus on personal-ized medicine, and Lab21 will continue to focus on diagnostic content strategy opportunities within the U.S. market,” Susan Lowther, chief financial officer of Lab21, said in a press release.
Verastem selects LabCorp for CDx partner
CAMBRIDGE, Mass.—Verastem Inc. and Laboratory Corporation of America Holdings (LabCorp) have inked an agreement for the validation of biomark-ers for VS-6063, Verastem’s lead focal adhesion kinase inhibitor, in the development of a compan-ion diagnostic. The biomarkers will be tested in clinical studies in patients with ovarian cancer and mesothelioma, including a VS-6063 meso-thelioma study expected to begin this year. The compound is designed to target and kill cancer stem cells through the inhibition of FAK signaling.
“We believe the approximately 50 percent of mesothelioma patients lacking merlin may be particularly responsive to treatment with FAK inhibitors,” Dan Paterson, Verastem’s vice president, head of Corporate Development and Diagnostics, said in a statement. “LabCorp is the perfect partner to progress our research on merlin into a companion diagnostic for VS-6063.”
SeraCare sells biorepository business to Precision
Health HoldingsMILFORD, Mass.—SeraCare Life Sciences, a port-folio company of Linden Capital Partners, has announced the sale of its biorepository, biobank-ing and laboratory services business to Chevy Chase, Md.-based Precision Health Holdings Inc. The transaction includes the transfer of SeraCare’s biorepository assets and employees, and financial details were not disclosed. SeraCare was advised by BroadOak Partners LLC and Kirkland & Ellis LLP.
“The sale of our Biorepository business is an important element in our growth strategy for SeraCare,” Charles Mamrak, CEO of SeraCare, said in a press release regarding the deal. “We can now more freely serve our growing customer base in the in-vitro diagnostics market and exe-cute on our mission of improving quality of life by advancing a global understanding of disease.”
Lilly, Dako make powerful companionsCDx collaboration aims to help speed oncology products to market By Jim Cirigliano
INDIANAPOLIS—Eli Lilly & Co. Inc. and Dako, an Agilent Tech-nologies company, have agreed upon a master framework for a collaboration on the develop-ment and commercialization of companion diagnostics (CDx) for Lilly’s oncology product pipe-line. These diagnostic tests will help to identify patients who are most likely to benefit from oncol-ogy medicines currently under development by Lilly.
The framework also defines and outlines the terms of the collaboration between Dako and Lilly in such as way that contrac-
tual negotiations for current and future companion diagnostics projects between the two compa-nies can be executed more swiftly.
Under the agreed-upon frame-work, Lilly will be exclusively responsible for the development and registration of the thera-peutics, including clinical trials. Dako/Agilent will be responsible for developing and registering the CDx assay/system solution.
“Companion diagnostics are cornerstones of personalized medicine,” says Susanne Munk-sted, director of PharmDx Com-mercial Management at Dako. “Dako has a strong heritage
developing companion diagnos-tic tests in collaboration with pharmaceutical companies.”
Dako has previously announced similar partnerships to develop CDx with AstraZeneca PLC, Bristol-Myers Squibb Co., OSI Pharmaceuticals LLC, Amgen
and Genentech, among others. Dako is responsible for developing and commercializing HercepTest, which was the first tissue-based CDx in oncology in 1998.
Creating CDx for Lilly’s oncolo-gy pipeline will help its scientists
dako coNtiNueD oN page 16
Creating companion diagnostics for Lilly’s oncology pipeline will help its scientists to create tailored therapies, which is one of the major pillars of the company’s current R&D strategy.
CDx deal is thrice as nice Already partnered on the development of a companion diagnostic test for lymphoma, Eisai and Epizyme bring Roche Molecular Diagnostics into the foldBy amy Swinderman
CAMBRIDGE, Mass.—Seeking to bring a third party into their agreement to develop and com-mercialize a companion diagnos-tic (CDx) test for patients with genetically defined cancers, Eisai Co. Ltd. and Epizyme Inc. have selected Roche’s Molecular Diag-nostics unit to supply the requi-site technology and platform.
On Jan. 7, the companies announced a collaboration to develop an in-vitro polymerase chain reaction (PCR)-based CDx test, with the goal of enabling the identification of lymphoma patients with non-wild type EZH2, including the Y641 mutation. The CDx will be used to detect the EZH2 mutation in subjects to determine enroll-ment eligibility for dosing with a compound that Epizyme and Eisai are developing.
“Roche is a company that pos-sessed world-class cutting-edge
technology and know-how, and has a proven history of compan-ion diagnostic approvals,” says Lynn W. Kenney, senior director of corporate communications for Eisai, of the company’s decision to bring Roche into the fold.
Epizyme and Eisai announced their collaboration in March 2011. That agreement gave Epizyme $6 million up front, with the potential to earn more than $200 million in additional research, development and sales milestones, and up to double-digit royalties.
Eisai agreed to fund 100 per-cent of the R&D costs through human proof-of-concept, at which point Epizyme has the right to opt into a profit-shar-ing and co-commercialization arrangement for the United States.
In a September 2012 Nature Chemical Biology paper, “A selec-
roChe coNtiNueD oN page 16
QIAGEN plus fourQuartet of deals helps boost company’s diagnostics for personalized healthcareBy lloyd dunlap
HILDEN, Germany—QIAGEN has entered into four separate agreements that add multiple biomarkers and manufacturing capability to the company’s pipeline of diagnostics for personal-ized healthcare applications. QIAGEN intends to develop new diagnostics to guide treatment decisions (including companion diagnostics paired with medicines) based on these biomarkers for use in therapeutic areas such as rheumatoid arthritis, lung cancer and colorectal cancer.
Most of these assays will be designed to run on the QIAsym-phony Rotor-Gene Q (RGQ) modular laboratory workflow auto-mation system, as well as QIAGEN’s next-generation sequencing workflows currently in development. RGQ is a real-time PCR cycler with a proprietary rotary design that the company claims offers the highest reliability and quality of results due to the more homogenous temperature distribution in the reaction chamber.
“These new agreements add further depth to our extensive development portfolio of biomarkers with potential to provide valuable diagnostic information as well as personalized guid-ance for treatment decisions. The opportunity to create a new, improved paradigm in the important and vast field of rheuma-toid arthritis is very exciting, and the other agreements further deepen our pipeline in oncology,” says Peer M. Schatz, CEO of QIAGEN. “QIAGEN’s global leadership in co-developing person-alized healthcare solutions in partnership with pharmaceutical and biotechnology companies has become a key growth driver for our business. Our diagnostics are delivering molecular infor-mation to transform medical care for a wide range of diseases.”
The four developments include a strategic equity investment in Drug Response Dx GmbH with the option to obtain world-wide rights to biomarkers for evaluation of rheumatoid arthri-
four coNtiNueD oN page 16
diagnostics16 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
tive inhibitor of EZH2 blocks H3K27 meth-ylation and kills mutant lymphoma cells,” Epizyme reported that lymphomas with genetic alterations of EZH2 require EZH2 enzymatic activity for proliferation, and suggested that EZH2 is a driving oncogene in these cancers and therefore an important therapeutic target.
Ultimately, if the compound is successful in clinical studies and receives regulatory approval, the parties hope to make a compan-ion diagnostic available to help physicians identify patient candidates with the EZH2-
related mutation who may be likely to benefit from the approved drug.
“Our collaboration with Epizyme and Eisai highlights Roche’s position as the partner of choice for the development and commercial-ization of companion diagnostics for person-alized healthcare,” said Paul Brown, head of Roche Molecular Diagnostics, in a statement. “We are excited to be developing a diagnostic test that will support Epizyme and Eisai in the development of an EZH2 inhibitor as a new therapeutic for patients with genetically defined lymphomas.”
The companies declined to comment on the commercial potential of the test. ddnediTConneCT: e021313
to create tailored therapies, which is one of the major pillars of the company’s current R&D strategy.
“Tailored therapies may help to identify patients who are most likely to respond to a specific therapy and, just as impor-tantly, which patients will not,” says Eva Groves, communications manager at Lilly. “For patients, this means a patient-specific treatment regimen and, hopefully, improved outcomes. For payers, this means they can be more confident of the value for which they are paying, because our studies will be designed to demonstrate the ben-efits to specific patient populations.”
The agreement plays to the goal shared by both companies of expediting oncology medicines into the hands of patients with unmet medical needs.
“It is Dako and Agilent’s aim to build strategic partnerships with companies who have a global presence and the strength and market leadership within oncology to drive therapeutic products through devel-
opment, registration and commercializa-tion,” says Munksted.
“Companion diagnostics play an impor-tant role in our tailoring efforts, and we’ll continue to partner with leading diag-nostics companies like Dako in an effort to bring our investigational medicines, paired with diagnostics, to patients who are waiting,” says Groves.
“I warmly welcome Lilly, and I am excited about what we may accomplish together for the benefit of patients world-wide,” Dako CEO and Agilent Senior Vice President Lars Holmkvist said in a media release announcing the collaboration. “This agreement heralds our mutual com-mitment to fighting cancer.”
Lilly manufactures such well-recognized pharmaceuticals brands as Cialis, Cymbal-ta, Methadone and Prozac, among many others, and employs approximately 38,000 people worldwide. Dako is a Denmark-based cancer diagnostics company that provides antibodies, scientific instruments and software pathology laboratories. ddnediTConneCT: e021312
dakocoNtiNueD From page 15
Passing the transplantation diagnostics torchImmucor acquires LIFECODES business from Hologic Inc.By Jeffrey Bouley
NORCROSS, Ga.—Reportedly combining best-in-class transfusion diagnostics and best-in-class transplantation diagnostics, Immucor Inc., a provider of automated instrument-reagent systems to the blood transfusion industry, announced in January a definitive agreement to acquire the LIFECODES busi-ness, which includes a leading position in transplantation diagnostics, from Hologic Inc.
Under the terms of the agreement, Immu-cor will purchase the outstanding shares of the LIFECODES business for $85 million in cash, plus an earn-out provision totaling $10 million in cash based on the achievement of certain financial targets in calendar 2013. The transaction is subject to the satisfaction of cus-tomary closing conditions, including appli-cable regulatory approvals, and is expected to close in the first calendar quarter of this year.
The addition of LIFECODES is expected to strengthen Immucor’s position in the global in-vitro diagnostics market by creating a sin-gle source for transfusion- and transplanta-tion-related testing.
“The LIFECODES business is recognized as a worldwide leader in transplantation diag-nostics,” noted Immucor in announcing the acquisition plans, “which represents a $400 million market that is growing at mid-to-high single digits.”
“Everything we do at Immucor is about helping anyone who needs blood get the right blood safely. The LIFECODES business will extend our mission to organs and stem cells,” said William A. Hawkins, president and CEO of Immucor, in announcing the deal. “The clear market advantage here is that custom-ers anywhere in the world will have a single source for proven diagnostics that has a core competency in antigen typing and antibody screening and identification.”
For its part, Hologic—a developer, manu-facturer and supplier of premium diagnos-tics, medical imaging systems and surgical products dedicated to serving the healthcare needs of women—sees the sale of LIFE-
CODES as a kind of new beginning.Hologic acquired the LIFECODES busi-
ness unit as part of its acquisition of Gen-Probe Inc., which was completed in August 2012. LIFECODES sells molecular and anti-body-based assays in the markets of trans-plant diagnostics, specialty coagulation and transfusion medicine, but from the start it was not necessarily seen as a good fit with Hologic. In fact, the company notes that the sale of LIFECODES was taken into consider-ation when the Hologic issued financial guid-ance for fiscal year 2013 on Nov. 12, 2012.
“Our sale of LIFECODES will enable Hologic to focus our resources on diagnos-tics opportunities that are more in line with our fundamental growth strategies,” said Rob Cascella, president and CEO of Hologic, in announcing the plans to sell LIFECODES. “We are committed to reducing our debt bal-ance and plan to use proceeds from the sale of LIFECODES for that purpose.”
According to Immucor, the LIFECODES business is much like Immucor itself in that it “represents 30 years of specialty diagnostic innovation, including molecular methods, to better serve laboratories in the United States and internationally.”
Also like Immucor, The LIFECODES busi-ness specializes in pre-transplant human leukocyte antigen (HLA) typing and screen-ing to ensure the most compatible match between patient and donor, as well as post-transplant patient monitoring to aid in the identification of graft rejection, Immucor notes, and LIFECODES also offers other immune-monitoring products.
“We are excited to welcome LIFECODES to the Immucor team,” said Hawkins. “Hos-pital labs and blood banks rely on best-in-class technology and support to improve their operations, and achieve better health-care outcomes. With the LIFECODES acqui-sition, Immucor will be well-positioned to help both transfusion centers and HLA labs anywhere in the world improve patient safe-ty by ensuring the most compatible transfu-sion and transplantation.”
Neither Hologic nor Immucor could be reached for additional comment about the acquisition. ddnediTConneCT: e021315
roChecoNtiNueD From page 15
tis (RA) patients to guide treatment with TNF-alpha inhibitors, which are widely prescribed for treating RA. Therapy with TNF-alpha inhib-itors is successful in approximately 60 to 70 percent of RA patients, but treatment failure does not become apparent for about six months. A companion diagnostic to predict which RA patients are likely to respond to TNF-alpha inhibitors would improve quality of life for patients through tailored and ear-lier use of the most effective drugs, while also decreasing costs due to failed treatments.
Drug Response Dx, based in Hennigsdorf, Germany, is being
financed at this stage by QIAGEN and High-Tech Gründerfonds GmbH, a German investor in early-stage companies and a co-founder of Drug Response Dx.
In a second move, QIAGEN has agreed to license exclusive world-wide rights from Insight Genetics Inc., a molecular diagnostics com-pany in Nashville, for the RET, ROS1 and DEPDC1 biomarkers for use in non-small cell lung cancer (NSCLC), the most common form of lung cancer. Collectively, RET, ROS1 and DEPDC1 mutations are estimated to be present in up to about one in 10 of all NSCLC cases, but so far there are no regulatory-approved commercial tests to reli-ably and efficiently identify these biomarkers. Previously, QIAGEN
and Insight entered a licensing and co-development partnership for companion diagnostics focusing on the ALK gene, which several phar-maceutical companies are targeting for new anticancer treatments. The development of the EM4-ALK assay for use on the QIAsymphony auto-mation system is progressing well, says Dr. Thomas Theuringer, QIA-GEN’s director of communications. Mutations of ALK are implicated in NSCLC and other malignancies, and several compounds known as ALK inhibitors are in clinical trials with one ALK inhibitor already on the market.
Also, QIAGEN Marseille (QIA-GEN’s subsidiary previously known as Ipsogen) has licensed exclusive worldwide rights to a biomarker
for mutations of the HSP110 gene, which allows for identification of specific types of colorectal cancer, from Inserm Transfert, the tech-nology transfer arm of the French National Institute of Health and Medical Research (INSERM) in Paris. QIAGEN intends to develop the biomarkers into a diagnostic test for routine use in the workup of colorectal cancer patients.
Diagnosing HSP110 mutations holds promise for enabling doctors to identify so-called microsatellite instability (MSI), which occurs in about 15 percent of all colorectal cancers. Patients with MSI have a more favorable outcome. On the other hand, about one-third of these patients with MSI have Lynch syndrome, an inherited form of
colorectal cancer linked to a higher risk of developing a second cancer.
Finally, QIAGEN has entered into a multiyear development and license agreement with IntelligentMDx under which the latter will design, develop and manufacture several undisclosed CE-marked and FDA cleared diagnostic tests for use on QIAGEN’s flagship QIAsymphony RGQ automated platform. The undisclosed assays being developed by IntelligentMDx are planned to be incorporated into QIAGEN’s grow-ing menu of molecular diagnostic assays and will be distributed by QIAGEN worldwide. QIAGEN has also retained the rights to assume manufacturing of the assays pursu-ant to volume considerations. ddnediTConneCT: e021314
fourcoNtiNueD From page 15
Collaborating since early 2011 on the development of therapeutics targeting EZH2, an epigenetic enzyme, for the treatment of lymphoma and other cancers in genetically defined patients, Epizyme and Eisai have brought Roche into their agreement to develop an in-vitro PCR-based companion diagnostic test.
diagnosticsFor more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 17
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the most advanced technologies for improved patient care and, as Flatley noted in the news release about the deal, it builds “on the recent acquisition of BlueGnome Ltd. and our expertise in next-gen-eration sequencing [and] further establishes Illumina as a leader in reproductive health.”
Available through a physician, the verifi test analyzes cell-free fetal DNA naturally found in a pregnant woman’s blood to look for missing or extra copies of chromosomes, or aneuploidies.
“We entered this market initially four or five years ago with our cyto-genetics arrays. And now in conjunc-tion with BlueGnome we’re working on future generations of these cyto-genetic chips,” Flatley said at a JP Morgan Global Healthcare Confer-ence in January as he discussed the Verinata acquisition deal. “We plan an FDA submission of these arrays with our iScan. That’s been delayed for about a quarter so rather than Q4 it looks like that’s going to happen now in the first quarter.”
But Flatley doesn’t see Verinata’s prenatal testing as the end of the line for reproductive diagnostics, also saying at the JP Morgan confer-ence, “We think there are two other future segments in reproductive health. One is in fertility factors and many of these we believe will have genetic underpinnings and sequencing will be applicable—and in the long run, probably the biggest market opportunity here in newborn screening, which we think over time will transition to sequencing from today’s tests that are based on mass spec and hor-mone testing.”
The JP Morgan conference, how-ever, also served as a venue for some degree of tension, as Sequenom Chairman and CEO Harry Hixson expressed confusion over Illumi-na’s decision to acquire Verinata, according to various media reports, since it would make Illumina the owner of Sequenom’s competitor in the non-invasive fetal aneuploidy testing space. Reportedly, though, Hixson added in his comments that Flatley assured him that their com-panies’ relationship would not be affected by the acquisition.
“Together, Illumina and Verinata are well-suited to drive the adop-tion of the non-invasive prenatal testing market. With approximate-ly 500,000 high-risk pregnancies annually in the United States and an estimated four million pregnan-cies in total, there is a clear need for such tests,” said Dr. Jeffrey Bird, executive chairman and CEO of Verinata Health. “Given the recent American College of Obstetrics and Gynecology and Society of Mater-nal and Fetal Medicine joint opin-ion that recommended cell-free DNA prenatal testing as a first or second trimester option for women at increased risk of aneuploidy, we
believe more physicians will be adopting NIPT.”
The verifi test will continue to be offered through Verinata’s CLIA-certified and CAP-accredited labo-ratory, which will continue to act as a reference laboratory to gather some of the necessary clinical data for future regulatory submissions.
Zacks Investment Research, in issuing a research note about the acquisition, points out that Illumi-na “continues to demonstrate its strength in the genotyping market
with its high-margin next-genera-tion sequencing platform,” despite ongoing difficulties in obtaining research funding at many compa-nies and institutions that would use Illumina’s products. Relatively undeterred by this hiccup in the economies of research efforts and seeking growth all the same, Illu-mina has, Zacks notes, “made sev-eral acquisitions and entered into partnerships to tap this growing [genotyping] market.” ddnediTConneCT: e021303
Illumina has its eye on the reproductive health testing market, and with the acquisition of Verinata, it would possess access to the company’s verifi prenatal test, which Illumina calls the “the broadest non-invasive prenatal test available today for high-risk pregnancies.”
18 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.comSOT Annual Meeting and ToxExpo
SOciETy Of TOxicOlOgy52nd Annual Meeting and ToxExpo
March 10-14, 2013 n Henry B. Gonzalez Convention Center n San Antonio, Texas
One-stop toxicology shopAnnual meeting of the Society of Toxicology promises a wide range of toxicology and safety assessment scienceBy Jeffrey Bouley
SAN ANTONIO, Texas—According to the Society of Toxicology (SOT), its annual meeting is the largest gathering of its type. So, it only makes sense for the biggest thing in toxicology to take place in a state known for things being grandiose and often physi-cally large—Texas. Specifically, the meeting will take place in San Antonio, home of the Alamo, at the Henry B. Gonzalez Convention Center from March 10-14.
“We’re excited about this meeting because it is the biggest of its kind in the world,” says Dr. William Slikker Jr., 2012-13 president of the SOT and the director of the U.S. Food and Drug Administration’s National Center for Toxicological Research. “It’s an event that draws attendance from all across the United States, but more than that, about 20 percent of attendees attend from places out-side the U.S., as far away as Australia, China, Latin America and Africa. We truly appreci-ate the opportunity each year to share the latest science of toxicology literally to the whole world.”
Based on past attendance, some 7,300 or more people from more than 50 nations will attend SOT’s show for the scientific program, ToxExpo and the various social and networking events. Slikker says there are already 2,500 abstracts, so “the entire science of toxicology will be on display and covered at the meeting.”
The five-day event will revolve around
five scientific themes: Application of Sys-tems Biology to Toxicology; Biomarkers for Exposure Assessment, Safety Evaluation and Translational Medicine; Effects of Nanomate-rials on Biological Systems; Molecular Basis of Genetic Variability and Susceptibility to
Toxicants; and Regulatory Science: Advanc-ing New Approaches for Hazard Identifica-tion and Risk.
“The truly exciting thing is that we have one-stop shopping for toxicology and safety assessment over the several-day program because of the size of our offerings and the significance of the people who present and attend,” says Slikker. Also exciting, he adds, is
an entirely new offering this year, with SOT’s Scientific Program Committee sponsoring the inaugural Frontiers for Toxicology ses-sion at the annual meeting in San Antonio.
“We’re trying to start a tradition of bring-ing in a speaker who’s cutting-edge in their area of professional life or their scientific interest, and see how they can apply it to our area of toxicology,” Slikker explains. “Wheth-er its cutting-edge approaches people aren’t all that aware of in toxicology or areas of sci-ence, technology and practice that directly impact toxicology, we want to bring those perspectives into our program. That new ‘Frontiers’ program is something our SOT vice president, Lois Lehman-McKeeman, has worked very hard on this year.”
For the 2013 annual meeting, the Fron-tiers for Toxicology session will be “Systems and Computational Biology As Foundations for Toxicology Research.” As SOT describes the program, “systems and computational approaches are holistic methods to elucidate and understand the complex interactions among components of a biologic response network and are central to the comprehen-sive understanding of all biological processes. The field requires the integration of concepts
SoT coNtiNueD oN page 19
2012 SOT AwArD recipieNTSAchievement AwardDonna D. Zhang, Ph.D.University of Arizona
Arnold J. Lehman AwardJoe L. Mauderly, D.V.M.Lovelace Respiratory Research Institute/ National Environmental Respiratory Center
Congressional Science Leadership AwardU.S. Rep. Rush Holt (D-N.J.)House Committee on Education and the Workforce/ House Committee on Natural Resources
Best Postdoctoral Publication AwardMaryse Lemaire, Ph.D.Institute for Medical Research, Montreal, QuebecXuefeng Ren, Ph.D.The State University of New York at BuffaloNisha S. Sipes, Ph.D.U.S. Environmental Protection Agency, Research Triangle Park, N.C.
Distinguished Toxicology Scholar Award Ernest Hodgson, Ph.D.North Carolina State University, Raleigh
Education AwardJohn H. Duffus, Ph.D., D.Sc.Heriot Watt University’s Edinburgh Centre for Toxicology
Founders AwardJohn A. Moore, D.V.M.Global Senior Scholar ExchangeOrish Ebere Orisakwe, Ph.D.University of Port Harcourt, NigeriaJesus Olivero-Verbel, Ph.D.University of Cartagena, Colombia
Leading Edge in Basic Science AwardMyung-Haing Cho, D.V.M., Ph.D.Seoul National University, Korea
Merit AwardCurtis D. Klaassen, Ph.D.University of Kansas Medical Center
Public Communications AwardMartin A. Philbert, Ph.D.University of Michigan School of Public Health
Translational Impact AwardJohn G. Benitez, M.D., M.P.H.Vanderbilt University, Nashville, Tenn.
Translational/Bridging Travel AwardXuemei Huang, M.D., Ph.D.Penn State Hershey Medical Center/Penn State University
Endowment Fund Undergraduate Educator AwardSue M. Ford, Ph.D.St. John’s University, Jamaica, N.Y.
AstraZeneca Traveling Lectureship AwardBhagavatula Moorthy, Ph.D.Baylor College of Medicine, Houston, Texas
Colgate-Palmolive Grants for Alternative Research Mingzhu Fang, Ph.D.University of Medicine and Dentistry of New JerseyJennifer Freeman, Ph.D.Purdue UniversityMelanie Adler, Ph.D.University of Wuerzburg, Germany
Syngenta Fellowship Award in Human Health Applications of New Technologies Benjamin Moeller, Ph.D. University of North Carolina at Chapel Hill
“The truly exciting thing is that we have one-stop shopping for toxicology and safety assessment over the several-day program because of the size of our offerings and the significance of the people who present and attend.”Dr. William Slikker Jr., 2012-13 SOT president
Among the many old Catholic missions in San Antonio, the most famous is the Alamo, which was originally called the Misión San Antonio de Valero. The famous Battle of the Alamo took place from Feb. 23-March 6 in 1836, and it remains one of the most celebrated military engagements in Texas history as well as a symbol of patriotic sacrifice for many people.
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For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 19
March 10-14, 2013 n Henry B. Gonzalez Convention Center n San Antonio, Texas
The theme’s the thing
T he Society of Toxicology (SoT) has arranged its symposium sessions, workshop sessions, roundtable ses-sions and other special sessions around five themes at the annual meeting, to illustrate the core contributions of toxicology scientific efforts to the world.
Application of Systems Biology to ToxicologyRecent technological advances allow the study of multiple interacting networks in cellular systems and facilitate studies of how such complex networks respond to toxicants. The integrated application of genomics, pro-teomics, metabolomics, computational mod-eling and bioinformatics to cell-specific and organ-specific toxicity as well as to broader questions in toxicology continues to devel-op. Application of these technologies will provide for systems to improve predictive toxicity tools, enable more complete under-standing of the mechanisms underlying the toxicity of pharmaceutical agents and envi-ronmental chemicals and facilitate the inter-rogation of disease etiology and prevention.
Biomarkers for Exposure Assessment, Safety Evaluation and Translational Medicine The development of biomarkers that can be applied to assessing exposure, predicting toxicity, defining mechanisms of toxicity and improving translation of preclinical and clinical toxicity has impacted how toxicol-ogy research is carried out. Developing the basic biology and analytical tools to support biomarker identification, development and validation is critical to the successful incor-poration of biomarkers in all areas of toxicol-ogy research.
Effects of Nanomaterials on Biological Systems Research in the toxicology of nanomaterials has expanded along with the application of this technology in material science research and development. factors that influence the potential for toxic responses and identifica-
tion of relevant target organs for exposure and toxicity are critical to the development of cogent and reliable risk assessment for these materials. Basic, applied and regulatory science must converge in order to address the needs for this class of materials that will advance understanding of potential impacts on human and environmental health.
Molecular Basis of Genetic Variability and Susceptibility to Toxicants Many toxicants alter gene expression and many types of toxicities can be affected by variation in gene expression or genetic poly-morphisms. Similarly, age-dependent gene expression can influence toxic responses and epigenetic perturbations influence heritable gene expression. Both genetic and epigenetic differences can influence the individual’s response to pharmaceuticals and environmental chemicals. it is recog-nized that single nucleotide polymorphisms directly affect genetic differences on rates of metabolism, but for other responses, such as behavior, the connections are more complex. linking genetic, epigenetic and environmen-tal variables with exposure data is essential to accurately define potential beneficial or adverse effects of chemicals and to assess disease susceptibility and prevention.
Regulatory Science: Advancing New Approaches for Hazard Identification and Risk Assessment Regulatory science encompasses the sciences used to evaluate the safety, efficacy, quality and performance of any product. Advance-ments in regulatory science will facilitate the development and evaluation of innova-
tive new products. As we modernize the tools used to assess the potential risks from drugs, environmental chemicals, food and other products, we must also consider the global applications of such methods and strategies to drive better risk assessment decisions. This theme is intended to foster session content that will provide for perspective on ongoing efforts to improve hazard identification and risk assessment with emphasis on how best to coordinate these efforts for more consistent regulatory practices around the world. ddn
from biology and physiology, computer sci-ence and applied mathematics, as well as physics and engineering.”
Toxicology also is a multidisciplinary sci-ence, SoT notes, and application of systems and computational approaches can help in sorting out the dynamic nature of toxic responses and the complexities therein.
“in light of the broad utility of systems biology approaches to toxicology and risk assessment, the goal of this session is to fea-ture eminent scientists who have made semi-nal contributions and advances in systems and computational biology,” notes SoT. The speakers in the session include: Trey ideker of the University of california San Diego, Avi Ma’ayan of the Mount Sinai School of Medi-cine, laszlo Urban of the Novartis institutes for Biomedical Research and Robert Murphy of carnegie Mellon University. The frontiers session will take place March 13 from 9 a.m. to 11:45 a.m. in grand Ballroom c1 at the convention center.
Another highlight of the program for Slik-ker is the Plenary opening lecture, in which Bruce A. Beutler, director of the center for the genetics of Host Defense at the Universi-ty of Texas Southwestern Medical center, will present a lecture titled, “genetic Analysis of innate immune Sensing,” a topic that Slikker describes as being “so very important to toxi-cology and also to drug discovery generally.”
in addition, Slikker points out, this year’s annual meeting stands out for being the first at which continuing medical education (cME) credits can be earned by attending some of the scientific program presentations.
“We are hopeful that this area will expand and we can offer more courses for cME credit at future meetings,” Slikker says. “it’s a really broad opportunity for us, allowing people to go in depth to build on their background or to get exposure to new areas.”
Slikker sees no reason that the meeting won’t draw the roughly 7,300 people it did last year, and perhaps more, since early reg-istration numbers have been tracking con-sistently with the levels seen for the 2012 annual meeting.
“i just want to emphasize that one of the great opportunities at the SoT meetings is that we tend to have roughly equal represen-tation from academia, government, Ngos
and industry,” Slikker notes, “so it offers a wonderful chance to build collaborations and network with a global community.” ddnEDITCONNECT: E021328
SOTcoNtiNueD From page 18
SOT Annual Meeting and ToxExpo
The celebrated Paseo del Rio, or the River Walk, is a winding, 2.5-mile-long waterside path that meanders through downtown San Antonio and is lined with shops, restaurants and other attractions.
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San Antonio boasts a population of 1.14 million and a zoo that is reportedly the third-largest in the nation, among many other attractions. The climate tends to be mild much of the year, with an average high in March of 70 degrees Fahrenheit and an average low of 48. It is the original home of the Frito and Cheeto (the Frito-Lay headquarters is currently in Plano, Texas, though), and is the birthplace of Pace Picante Sauce and possibly even chili itself.
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About the SOTt
he Society of Toxicology (SoT) is a professional and scholarly organiza-tion of scientists from academic institutions,
government and industry, represent-ing the great variety of scientists who practice toxicology—the study of the adverse effects of chemical, physical or biological agents on living organ-isms and the ecosystem, including the prevention and amelioration of such adverse effects—in the United States and abroad. The SoT’s stated commitment is to creating a safer and healthier world by advancing the science of toxicology. The society pro-motes the acquisition and utilization of knowledge in toxicology, aids in the protection of public health and facili-tates disciplines. The SoT has a strong commitment to education in toxicol-ogy and to the recruitment of students and new members into the profession.
The SoT was founded in 1961 as a not-for-profit scientific society and is governed by a 13-person elected coun-cil and managed by an administrative office in Reston, Va., near Washington, D.c. The society’s activities are assist-ed by the efforts of nearly 46 elected and appointed committees, subcom-mittees and task forces. in addition, SoT has established 27 specialty sec-tions that may propose sessions for the annual meeting, exchange informa-tion through its newsletter, Commu-niqué, present awards and participate in other scientific activities.
in addition, SoT has 18 regional chapters that sponsor regular local meetings throughout the year. The purpose of the regional chapters is to foster scientific exchange at a local level. ddn
20 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.comSOT Annual Meeting and ToxExpo
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Expo actionA major feature of SOT’s annual meeting is its ToxExpo.Exhibit hours are until 4:30 p.m. Monday through Wednes-day, with doors opening at 9 a.m. the fi rst day and 8:30 a.m. the next two days. in addition to the standard exhibit hall hours and poster presentation times, one hour of dedicated networking time has been allot-ted in the scientifi c program for attendees to visit with exhibi-tors. ToxExpo Time will take place on Tuesday, March 12, from 12:30 p.m. to 1:30 p.m.
Attendees can also enjoy their chance at a $500 prize drawing each day of ToxExpo, in the form of an American Express gift card. Attendees need only drop their business cards in the ToxExpo prize drawing boxes found in all booths designated as Diamond level Sponsors.
And, as SoT notes, ToxExpo isn’t just those three days dur-ing the annual meeting—it also continues throughout the year at ToxExpo.com, the soci-ety says, “for all your toxicolo-gy-related science information and data as well as informa-tion about current exhibitors … ToxExpo is a rich resource for the working scientist, the decision maker, the student or anyone looking for the best products and services that toxi-cology has to off er.” ddn
Why the SOT annual meeting mattersT HE SOCIETY OF Toxicology (SOT)
notes that its annual meeting is the largest toxicology meeting and exhi-
bition in the world, with an expected atten-dance this year of more than 7,300 scientists from academia, government and industry, hailing from various countries worldwide.
“From the plenary opening lecture and featured lectures to the wide range of sci-entifi c sessions and continuing education courses, the annual meeting offers an unpar-alleled depth of analysis and relevant toxi-cological issues,” according to the society. “From basic to advanced topical issues, the thematic approach provides each attendee an opportunity to learn about emerging fi elds. Whether you are speaking in or chair-ing a session, honoring a colleague as the recipient of an SOT award or collaborating with your peers at an SOT event, this meet-ing has something for every attendee.” ddn
CONTINUING EDUCATION OFFERINGSTHE CONTINUING EDUCATION offered
by SOT at the annual meeting spans a wide range of courses covering estab-lished knowledge in toxicology, as well as
new developments in toxicology and related disciplines. Courses can be applied toward certifying and licens-
ing board requirements and may also be used for recer-tifi cation with the American Board of Toxicology, with both basic and advanced course topics are offered.
The courses are as follows: n Recent Developments in Cardiovascular Physiology-Based Toxicology (Basic)
n A Refresher of Immunoglobulin and Fc-Receptor Biology and Advances Related to Therapeutic Antibody Development (Basic)
n Basic Principles of Human Risk Assessment (Basic) n Approval of Biosimilar Monoclonal Antibodies: Scientifi c, Regulatory and Legal Challenges (Basic)
n The What, When and How of Nonclinical Support for an IND Submission (Basic)
n The Practice and Implementation of Neural Stem Cell-Based Approaches to Neurotoxicology (Basic)
n Toxic Effects of Metals (Basic) n Advances in Nanotoxicology—Challenges (Advanced)
n Gonadal Development, Function and Toxicology (Basic) n The REACH Regulation and Safety Assessment Approaches for Chemicals That Come in Contact with the Skin (Basic)
n T4: Tools and Technologies in Translational Toxicology (Advanced)
n Understanding Toxic Neuropathy in Drug Development: Both Clinical and Nonclinical Perspectives (Advanced)
n Weighing in on Nutrition—Essential Concepts for Toxicologists (Basic)
All courses will be held on Sunday, March 10, at the Henry B. Gonzalez Convention Center. ddn
BRIEFS
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Omics & systems BiOlOgyEvotec, Apeiron ink cancer
immunotherapy agreementHAMBURG, Germany—Evotec AG has announced a research collaboration with Apeiron Biologics to develop immunomodulatory lead compounds for treating cancer. Apeiron Biologics will bring to the table its expertise in in-vitro and in-vivo pharma-cology, while Evotec will handle the medicinal chemistry and chemical proteomics. No financial terms for the partnership were disclosed. The collaboration results from the success of a phe-notypic high-throughput screen commissioned by Apeiron Biologics to Evotec.
“We were excited about the outcome of the primary screen and look forward to refining the hit compounds in this collaboration applying our immunological know-how. There is no doubt that immunomodulatory compounds like these carry huge therapeutic and commercial potential,” Dr. Hans Loibner, CEO of Apeiron Biologics, said in a press release.
Weill Cornell’s Tres Cantos project targets TB
NEW YORK—Weill Cornell Medical College has received funding from the Tres Cantos Open Lab Foundation to support a Weill Cornell microbiolo-gist’s two-year research project at the Tres Cantos Open Lab in Spain, which will focus on measur-ing and analyzing the permeability of chemical compounds into Mycobacterium tuberculosis bac-teria, which cause tuberculosis. The researcher in question, Dr. Saki Raheem, will work as an Open Lab Scientist alongside researchers from GlaxoSmithKline PLC. This is the second project by Weill Cornell in the Tres Cantos Open Lab. The new project will focus on the identification of chemical principles that could be used to trans-form potential chemical inhibitors into active drugs, with the goal of revealing the relationships between cell permeability and anti-tuberculosis activity using metabolics technology.
Sigma donates RNA libraries to the RNA Institute
ST. LOUIS—Sigma-Aldrich Corp. has announced the donation, through Sigma Life Science, of several collections of small RNA molecules to the RNA Institute of the University at Albany. The donation includes Sigma Life Science’s MISSION Target ID human miRNA and ncRNA Library, LentiElite Mouse shRNA Library, as well as human and mouse esiRNA and LentiPlex Pooled shRNA Libraries. The libraries, consisting of hundreds of thousands of small RNAs, will allow researchers to study the expression and regulation of genes in human and mouse genomes.
“This generous contribution from Sigma-Aldrich avails our Institute with a rich resource generally afforded only to large pharmaceutical corporations,” Dr. Paul Agris, director of The RNA Institute, said in a press release.
Shire acquires Lotus Tissue Repair, gains potential therapy for rare skin diseaseBy Kelsey Kaustinen
LEXINGTON, Mass.—Shire PLC has further expanded its portfolio of orphan disease therapies with the signing of an agreement to acquire privately held Lotus Tis-sue Repair Inc., a biotechnology company developing the first and only protein replacement for the treatment of the orphan disease dystrophic epidermolysis bullosa (DEB). Currently, no approved treatment options exist for the disease other than palliative care.
Though no specific details were released, the acquisition consists of an upfront payment as well as certain additional payments if specified safety and development milestones are met.
The acquisition is subject to cus-tomary government approvals.
Epidermolysis bullosa (EB) is a set of rare genetic diseases characterized by extremely fragile skin, and patients suffer recurring blister formation from even minor amounts of friction or trauma. Of these diseases, DEB is one of the more severe, and in extreme cases of DEB, patients may also suffer internal blistering of the mouth, esopha-gus, upper airway, lower gastro-intestinal tract and genitourinary tract. In the recessive form of the disease, severe blistering and scarring occurs on the hands, feet and joints. By the time a
deB coNtiNueD oN page 23
Shire has acquired Cambridge, Mass.-based Lotus Tissue Repair for an undisclosed amount. Lotus Tissue Repair is currently developing a protein replacement therapy for the treatment of dystrophic epidermolysis bullosa, a rare genetic disease.
My antisense is tinglingStrategic alliance between AstraZeneca and Isis aims to develop next-generation RNA therapeutics By Jim Cirigliano
LONDON—AstraZeneca PLC and Isis Pharmaceuticals Inc. have announced a strategic alli-ance focused on discovering and developing next-generation anti-sense therapeutics against five cancer targets. As a key element of the alliance agreement, Astra-Zeneca will be granted an exclu-sive license to develop and com-
mercialize ISIS-STAT3Rx, Isis’ promising new product currently in early clinical trials in patients with advanced lymphomas.
Under the terms of the agree-ment, Isis will receive from Astra-Zeneca $25 million on signing, plus a $6 million payment in the second quarter of 2013 as long as the research program persists at that time. In exchange, Isis has granted AstraZeneca an exclu-sive license to develop and com-mercialize ISIS-STAT3Rx and a preclinical program, as well as an option to license products devel-oped under a separate research program. AstraZeneca will be
isis coNtiNueD oN page 22
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develop next-generation antisense therapeutics against five cancer targets
>> isis received $25 million at signing, is eligible for milestone payments later
>> antisense therapies target proteins involved in disease processes by destroying rNa that create them
From platform to clinicPartnership between Heptares Therapeutics and Cubist Pharmaceuticals targets GCPRs for unmet needs in acute careBy ilene sChneider
WELWYN GARDEN CITY, U.K.—Heptares Therapeutics is a pio-neer in the discovery and development of G protein-coupled receptors (GPCRs). Cubist Pharmaceuticals Inc. has expertise in the development and commercialization of products that address significant unmet medical needs in the acute care and hospital environment. Together, the two companies will collaborate on the research and discovery of new medicines targeting GPCRs, membrane proteins involved in a broad range of biological processes and diseases.
The agreement gives Cubist exclusive worldwide rights to research, develop and commercialize products generated from the collaboration. The collaborative research agreement will focus on up to two GPCR drug targets selected by Cubist. For the first target, Heptares will receive $5.5 million upfront and up to approximately $4 million in research funding, plus mile-stones and royalties. Cubist also has the option to nominate a second GPCR target at a later point in the collaboration, which Heptares will work on according to predetermined financials.
According to Malcolm Weir, Heptares’ CEO, GPCRs play a central role in many biological processes and are linked to a wide range of disease areas. GPCRs involved in CNS and meta-bolic diseases are perhaps the best characterized, and there are a number of very attractive commercial product opportunities, “so it made sense for Heptares to focus its initial efforts in this area to build its internal pipeline,” says Weir.
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OMICS & SySteMS BIOlOgy22 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
Autism spectrum under studyPopulation Dx, Toronto’s Hospital for Sick Children discover gene variants for autism spectrum disorder By lori lesKo
MELVILLE, N.Y.—Population Diagnostics Inc. (PDx) recently announced the results of its collaboration with the Hospital for Sick Chil-dren (SickKids) in Toronto on the discovery of a collection of gene variants associated with autism spectrum disorder (ASD).
Their findings, reported Dec. 12 in the journal Genes/Genomes/Genetics (G3), could open the door of early detection diagnostic tests and more personalized treatment for those suffering from the social, behavioral and learning disabilities of autism.
“Both Population Diagnostics and SickKids have been independently engaged for several years in finding genes—and the mutations within them—that cause autism,” Peggy S. Eis, chief technology officer at PDx, tells ddn. “We both use the same gene discovery approach, so it was a natural fit to collaborate on autism.”
The partners first identified copy number variants (CNVs) that are exclusively found in autism patients, which in subsequent genetic analysis studies enabled them to uncover the full spectrum of mutations within the set of ASD-associated genes.
“We are currently analyzing the data fur-ther using additional methods that reveal even smaller sized genetic variants, most
of which would likely be missed by other microarray methods,” says Eis. “Important-ly, because we are using a microarray that screens for CNVs in all regions of the genome, we can discover ASD mutations that occur within introns and in regions between the genes, which often harbor regulatory DNA sequences that can alter gene expression (i.e., can increase or decrease the amount of pro-tein made from the gene).”
Intronic and intergenic regions are com-pletely missed in exome sequencing studies, says Eis. A subset of the genes described in the study have already been linked to neu-rodevelopment (e.g., synapse formation) and neurochemistry (e.g., neurotransmitter receptors). For example, the gene SYAP1 (synapse associated protein 1) that was found to be mutated in three ASD patients is known to impact learning and memory in a fly model for the corresponding gene, she says.
“In order to find genes that are causative or linked to ASD, the ASD data were inter-preted using our company’s control cohort data, which was generated on genomic DNA samples obtained from 1,000 individuals without ASD or any other major diseases or conditions,” Eis says.
Discovering these ASD-associated genes via CNVs that occur only—or almost exclu-sively—in ASD patients is the critical first step, Eis says. Because there are at least 1,000-fold fewer CNVs in a person’s genome compared to single-nucleotide variants, it is far more efficient to first assess CNVs in the
genomes of ASD cases versus controls.“Our gene discovery approach can be
applied to any disease that has a genetic basis, including Alzheimer’s, Parkinson’s, schizo-phrenia, endometriosis, peanut allergy and progressive multifocal leukoencephalopathy (PML), which is a rare but deadly brain disease that occurs in a small percentage of patients on immunosuppressive therapies,” Eis says.
In some cases, targeting a biological path-way that is impacted by mutations in one of several genes may be possible, and in other cases drugs will have to be developed that target a single gene, she says.
“While this sounds rather daunting, for-tunately, there has been significant progress in drug discovery methods that will enable development of targeted therapeutics,” Eis says. “There are a handful of targeted drugs on the market today in other diseases, so the drug development technologies already exist for targeted medicines.”
In addition to discovering 16 novel genes associated with autism, “this study highlights the general importance of analyzing genomes specifically for CNVs, which is a type of genet-ic variant that can disrupt, delete or generate multiple copies of a gene,” says primary inves-tigator Dr. Stephen Scherer of the Hospital for Sick Children.
“Several interesting new autism risk genes have been highlighted in this study,” Scherer says. “We now need to go back and perform more validation work in larger case and con-
asd coNtiNueD oN page 23
responsible for all further development and commercialization of ISIS-STAT3Rx apart from the ongoing clinical trial, which Isis will complete.
Isis is eligible to receive additional mile-stone payments subject to achieving pre-defined clinical success criteria for ISIS-STAT3Rx and preclinical milestones for the other programs. Isis is also eligible to receive downstream development and approval mile-stone payments, license fees for research pro-gram targets and royalties on sales from prod-ucts that are successfully commercialized.
The agreement also includes a research com-ponent; there are three undisclosed research targets that the agreement allows AstraZeneca the option to license at a later time.
ISIS-STAT3 is designed to inhibit the pro-duction of a protein called “signal transducer and activator of transcription 3” (STAT3), which is implicated in a number of different cancers and appears to play a critical role in tumor growth and survival. The new drug uses Isis’ most current technology and next-generation chemistry (dubbed Generation 2.5) to produce potent new therapeutics that will initially be examined for their applica-tion against lymphoma, including diffuse large B cell lymphoma, which is an area of high unmet need in the medical community. Other oncology applications will be explored.
“AstraZeneca has evaluated ISIS Genera-tion 2.5 antisense technology in-house for oncology, and the data generated was very compelling with improved potency com-pared with earlier antisense chemistry,” says Susan Galbraith, head of AstraZeneca’s Oncology Innovative Medicines unit.
Isis became interested in a large phar-maceutical partner when it became clear that its new drug could have broad-ranging implications. The company sought to license their product to an organization that had the wherewithal to conduct broad evaluations of the drug’s efficacy and potential applica-tions, and the ability to move the drug swiftly forward through the development process.
The STAT3 protein is widely viewed as a promising target for treatment of numerous diseases. It has been shown to be hyperactive in a variety of cancers, including brain, lung, breast, head and neck, bone, liver, myeloma and lymphoma.
“We feel AstraZeneca brings significant experience in oncology, and our partnership will serve to enhance our oncology franchise, which will lead to innovative new drugs and seeing STAT3 evaluated in a broader clini-cal program than we could have done on our own,” says Amy Blackley, associate director of corporate communications at Isis.
“While STAT3 is implicated in a number of different cancers, the focus in our ongoing clinical study is to evaluate the effectiveness of our drug in hematologic malignancies, such as lymphoma,” Dr. Brett Monia, senior vice president of antisense drug discovery at Isis, said of the trials in a news release. “We have worked closely with AstraZeneca to design a rapid path to the market for ISIS-STAT3Rx in these patient populations.”
In early clinical trials, two patients with dif-fuse large B cell lymphoma showed evidence of tumor shrinkage, suggesting that ISIS-STAT3 is potent enough to achieve reduction of the STAT3 protein in tumor tissue.
Beyond the ISIS-STAT3 product, the part-nership is designed to be an alliance for the discovery and development of antisense ther-
apeutics against several cancer targets. Anti-sense therapies target the proteins involved in disease processes by destroying the RNA involved in creating these proteins.
“When the genetic sequence of a gene is known to cause a disease, it is possible to syn-thesize a strand of nucleic acid—DNA, RNA or a chemical analogue—that binds to the mes-senger RNA produced by that gene and effec-tively turning that gene ‘off,’” says Galbraith. “The Isis discovery platform develops specific therapies that bind to mRNA and inhibit the production of disease-causing proteins.”
“We’ve structured a deal that will enable us to enhance our oncology franchise and work with a partner that is knowledgeable and experienced with taking oncology prod-ucts to market,” says Blackley. ddneditConneCt: e021317
isiscoNtiNueD From page 21
AstraZeneca will be responsible for the development and commercialization of ISIS-STAT3Rx, Isis Pharmaceuticals’ promising new product for advanced lymphoma.
The GPCR superfamily is the larg-est and single most important family of drug targets in the human body. GPCRs are expressed in every type of cell in the body, where their function is to transmit signals from outside the cell across the membrane to signaling pathways within the cell, between cells and between organ systems.
Heptares is constantly talking to com-panies about targets in many indications.
“Cubist was one such company, a partner-of-choice and clear business leader in a space in which certain GPCRs could be medically very impor-tant,” Weir says. “Discussions moved forward very rapidly with a focus on GPCRs with significant medical prom-ise in the acute care space.”
Steve Gilman, Cubist’s executive vice president of research and develop-ment and chief scientific officer, says, “Heptares is a recognized leader in the GPCR field, and we are pleased to col-laborate in the pursuit of potential new GPCR drug candidates. This partner-ship underscores our commitment to develop a robust pipeline of novel prod-ucts that address high unmet medical needs in patients with acute diseases. We bring expertise in the discovery, development and commercialization of therapies used in settings in and around the hospital where acutely ill patients are treated for days or weeks—versus the months or years of treatment with a chronic care therapy.”
He adds, “Cubist’s acute-care chan-nel strategy embraces multiple thera-peutic areas in the development and commercial areas of its business, while our discovery and preclinical research remains focused on antibiotics as well as non-opioid pain and inflammation.”
An industry pioneer in GPCR struc-ture-based drug design (SBDD), Hep-tares has built a special capability for discovering novel molecules that target historically “undrugable” or challenging GPCRs, according to Weir. Heptares’ integrated discovery platform includes proprietary technologies for engineer-ing stabilized GPCRs (known as StaRs) in their natural pharmacological con-formations, identifying previously unknown chemistries for GPCR pro-tein-drug interactions and deploying advanced fragment-based approaches to GPCR target space for the first time.
In the partnership, Heptares will focus on creating leads using its SBDD platform (structure, chemistry and pharmacology) for Cubist to take into development. Cubist will be responsi-ble for preclinical and clinical develop-ment, as well as regulatory and com-mercial activities on a global basis. The goal is to discover and develop new medicines that target the first (and potentially a second) GPCR selected.
“The initial target is one that both companies believe is well suited to addressing with Heptares’ SBDD plat-form and also that a new medicine hit-ting this target will make a very impor-tant difference in the lives of patients,” Weir concludes. ddneditConneCt: e021318
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trol cohorts to determine the truly bona-fide autism risk genes. We also need to figure out how they work in brain development.”
Unfortunately, there are not yet any effective drugs that treat autism, he says.
“However, with our new gene discover-ies, pharmaceutical targets are now known and companies are developing drugs for them,” Scherer says. “The progress looks very encouraging.”
“Collectively, these newly discovered genes from our collaboration with Sick-Kids, along with novel genes from our finer-scale analysis that will be reported in a future paper, represent a significant por-
tion of the unexplained genetic contribu-tion to autism and greatly contribute to our understanding of the underlying genetic causes of autism,” Eis says. ddneditConneCt: e021319
asdcoNtiNueD From page 22patient reaches their 20s or 30s, DEB leads
to the development of aggressive squamous cell carcinomas in areas of chronic wound-ing, and these often metastasize and lead to death. One of the primary causes of DEB is the dysfunction or deficiency of type VII collagen (C7).
Dr. Philip J. Vickers, global head of research and development at Shire Human Genetic Therapies (HGT), notes that the global incidence of DEB is roughly one per million, for an approximate total of 1,275 people with the recessive form of DEB and more than 200 with the dominant form of the disease.
“DEB is one the most devastating orphan diseases, severely impacting the lives of patients and their families, many of whom have few or no treatment options other than palliative care,” said Vickers in a press release. “rC7 protein replacement therapy has the potential to provide a first-in-class disease-modifying treatment for these chil-dren. We plan to apply our proven ability to develop protein replacement therapies for rare genetic diseases to progress rC7 as a pos-sible groundbreaking treatment that offers hope to patients with DEB.”
Lotus Tissue Repair’s lead product candi-date is a proprietary recombinant form of C7 (rC7), an intravenous protein replacement therapy, for which Shire’s HGT business will take over further development. The com-pound is in late preclinical development, and preclinical findings show that as a protein replacement therapy, rC7 is potent, long-lasting and specifically retained in the skin after intravenous injection. The treatment complements Shire’s own existing work in EB, which includes ABH001, a regenerative medicine product Shire is investigating as a dermal substitute therapy for the treatment of non-healing wounds in EB patients.
“This acquisition of Lotus Tissue Repair by Shire further underscores the potential of this proprietary rC7 technology to dramati-cally change the treatment landscape for DEB patients and their families,” Dr. Mark de Souza, founding CEO of Lotus Tissue Repair, said in a statement. “We are thrilled that this program will become part of the innovative pipeline at Shire and the com-pany’s growing commitment to this patient community.”
Following the acquisition, Vickers notes that de Souza will join Shire’s HGT team at the Lexington campus as a consultant, add-ing that Shire “will be engaging additional consultants who were key to the progress made by Lotus.”
“Shire Regenerative Medicine has experi-ence focusing on developing and delivering solutions that support the body’s natural healing process, and will use this expertise to develop ABH001 as a skin substitute for EB,” says Vickers. “At HGT, we are approaching the treatment of [DEB] from a complemen-tary angle as an intravenous protein replace-ment, with rC7 as a potential treatment for the systemic manifestations of the disease. This indication leverages our proven protein replacement expertise and is aligned with the HGT focus on the orphan disease space. In addition to scientific synergies, we antici-pate that the cross-business unit focus on EB will lead to operational synergies, including coordinated links with patient associations and centers of excellence.” ddneditConneCt: e021316
deBcoNtiNueD From page 21 “Several interesting new autism risk genes have been
highlighted in this study,” says primary investigator Dr. Stephen Scherer of the Hospital for Sick Children. “We now need to go back and perform more validation work in larger case and control cohorts to determine the truly bona-fide autism risk genes. We also need to figure out how they work in brain development.”
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24 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
BioMarin acquires ZacharonSAN DIEGO—BioMarin Pharmaceuticals Inc. has announced the acquisition of Zacharon Pharmaceu-ticals, a privately held company founded in 2004. The acquisition is priced at $10 million up front, with the potential for additional payments if clinical, regu-latory and commercial milestones are met. Zacharon targets multiple forms of lysosomal storage disease and rare forms of cancer through small-molecule drugs that target glycans, with compounds in its pipeline for conditions such as Tay Sachs, metachro-matic leukodystrophy and acute spinal cord injury.
“The acquisition of Zacharon will further expand our glycobiology expertise and will sup-port our lysosomal storage disease drug develop-ment efforts,” said BioMarin CEO Jean-Jacques Bienaimé in a statement. “We are committed to investing in our advancing pipeline, which has evolved through a combination of internal devel-opment and targeted acquisitions, such as this.”
Neuroscience focus of AstraZeneca, Vanderbilt deal
NASHVILLE, Tenn.—Vanderbilt University has joined forces with AstraZeneca PLC in a research deal to identify potential treatments for psychosis and other psychiatric symptoms associated with diseases such as Alzheimer’s and schizophrenia. AstraZeneca will make an upfront payment of research funding to Vanderbilt for two years, as well as milestone payments and royalties on sales of any drugs resulting from the collaboration. In return, AstraZeneca has exclusively licensed rights to compounds developed by the Vanderbilt Center for Neuroscience Drug Discovery (VCNDD).
Dr. Mike Poole, vice president of the AstraZen-eca Neuroscience Innovative Medicines Unit, said the combination of “AstraZeneca’s deep experi-ence in drug development and translational sci-ence with VCNDD’s expertise in drug discovery is an important step toward bringing new medicines forward for people who are suffering from neuro-degenerative diseases.”
MacroGenics, Gilead pair up on DART project
ROCKVILLE, Md.—A license agreement was estab-lished between MacroGenics and Gilead Sciences in early January to develop and commercialize Dual-Affinity Re-Targeting products aimed at up to four undisclosed targets. Per the agreement, Gilead will pay up to $30 million total in license fee payments, as well as potentially $85 million in preclinical milestones across the four programs. MacroGen-ics is also eligible for approximately $1 billion in milestone payments if all four programs reach the required clinical, regulatory and commercialization milestones, and stands to receive tiered royalties on future net sales. In return, Gilead holds exclusive worldwide rights for three of the programs, and will fully fund MacroGenics’ research activities for the four programs.
A LEAP forward for stem cell researchCollaboration gives scientific team access to private techBy Jim Cirigliano
LA JOLLA, Calif.—The Sanford-Burnham Medical Research Institute, a world leader in stem cell research, has entered into a collaboration agreement with leading synthetic biology com-pany Intrexon Corp. The partner-ship will focus on rapidly accel-erating induced pluripotent stem cell (iPSC) research by giving world-class researchers access to innovative, proprietary technol-ogy platforms that are not widely available.
The agreement grants Sanford-
Burnham access to Intrexon’s Laser-Enabled Analysis and Pro-cessing (LEAP) instrument and RheoSwitch Therapeutic System (RTS). In exchange, according to the terms of the agreement, Intrexon may gain commercial and intellectual property rights resulting from technological advances made under the aus-pices of the collaboration.
Sanford-Burnham is currently building the largest collection of iPSCs in the world from individ-ual patients and volunteers.
The LEAP instrument is Intrexon’s proprietary automat-ed system that provides high-throughput cell imaging and pro-
leap coNtiNueD oN page 25
Sanford-Burnham and its partners are pursuing iPSCs for a variety of prospective uses, including drug development or potentially using culture-born cells to treat disease directly.
Stem cell developers Give CuresCET, John Paul II Medical Research Institute to develop stem cell biobankBy lori lesko
CORALVILLE, Iowa—Aimed at overcoming obstacles to offering personalized medicine, as well as accelerating the search for effec-tive treatments, human stem cell biomanufacturer Cellular Engi-neering Technologies Inc. (CET), and its nonprofit arm, the John Paul II Medical Research Insti-tute (JP2MRI), have launched a partnership to develop a private stem cell biobank to create more than 5,000 patient and disease-
specific stem cell lines.While CET is a for-profit bio-
tech company that manufactures stem cells from normal volun-teers and from patients with clin-ical disease to facilitate the drug discovery process for the general scientific community, JP2MRI focuses on clinical and transla-tional research, administrates clinical proposals and recruits patients for research protocols, but works with private hospitals and clinics around the country to procure tissue and collect clini-cal data on patients.
The objective of this venture is to create a database of private-practice physicians who will refer patients and/or serve as collection
sites to procure tissue for produc-ing patient and disease-specific stem cell lines, and to create stem cell lines that can be used by the general scientific community.
“The project is ongoing and indefinite,” says Dr. Alan Moy,
co-founder and CEO of CET and founder and scientific director of JP2MRI. “We currently have several hospitals and clinics that are participating in our program. The institute launched a program
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a strategic fit Sutro and Celgene collaborate on ADCs in $500 million-plus dealBy ilene sChneider
SAN FRANCISCO—Sutro Biopharma will collaborate with Cel-gene Corp. to design and develop novel antibody drug conju-gates (ADCs) and bispecific antibodies for two undisclosed targets and to manufacture a proprietary Celgene antibody. The deal could be worth more than $500 million to Sutro, which will receive a substantial upfront payment, an equity investment in the company and payments for the completion of research, development and regulatory milestones, plus roy-alties on product sales.
“The collaboration between Sutro and Celgene arose due to the strong strategic fit for both companies, as Sutro has built a platform for creating the next-generation ADC and bispecific products Celgene seeks to develop and commercialize,” says William Newell, CEO of Sutro.
Newell explains that Sutro has developed a cell-free bio-chemical protein synthesis technology that allows the creation of “best-in-class ADCs and bispecific antibodies,” which are made up of pieces of two separate antibodies and can bind to two different antibody-producing antigens.
Celgene, a global biopharmaceutical company, “has a world-class reputation for the discovery of breakthrough medicines and has significant expertise in development and commer-cialization of therapeutic products in various disease areas,” Newell adds.
More than 300 clinical trials at major medical centers use compounds from Celgene. Investigational compounds are being studied for patients with incurable hematological and solid-tumor cancers, including multiple myeloma, myelo-dysplastic syndromes, chronic lymphocyte leukemia, non-Hodgkin’s lymphoma, myelofibrosis, small-cell lung cancer and prostate cancer.
Dr. Thomas Daniel, Celgene’s president of global research adC coNtiNueD oN page 25
The biobank stem cell lines to be developed by CET and JP2MRI will serve as models to better predict drug therapy outcomes in patients and help bring new treatments to market sooner and at lower cost.
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 25ReseaRch & Development
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cessing. The instrument images cells right in their plates, where computational software is able to identify cell types based on their shape and chemistry. Undesired cell types can be targeted and eliminated in situ by way of a laser system.
“The laser fires 1,000 shots per second to eliminate undesirable cells without touching cells of interest,” says Dr. Fred Koller, vice president of business development at Intrex-on and co-inventor of the LEAP instrument. “We have the only technology today that’s able to purify adherent cells directly in the dish.”
The LEAP instrument will allow greater efficiency of iPSC cultures, and will allow cell lines to be developed much more quick-ly. Standard methods typically require two plates to generate one cell line.
“The bottleneck for progress has been how many colonies you can generate with limited technicians,” says Dr. Yang Liu, manager of the Stem Cell Research Institute at Sanford-Burnham. “Thanks to this collaboration, we will have access to software that will enable high-throughput plates—allowing the pos-sibility of multiple cell lines on a single plate.”
Using the LEAP instrument to develop iPSCs promises a vastly improved method because researchers will have no need to take cells out of the well.
“This will allow us to save reagents, while also reducing the risk of contaminations,” says Liu.
The LEAP instrument will help researchers to achieve the next step in stem cell develop-ment, which is cell differentiation. The typical process of transitioning iPSCs into a desired cell type results in an impure culture that con-tains roughly 10 parts the desired cell type and
leapcoNtiNueD From page 24
90 parts something else. The laser-based pro-cessing affording by the LEAP instrument will allow researchers to purify the cells directly in the adherent state, yielding a sample contain-ing only the desired cell type.
“If we can achieve this, we can create and label a pure sample of a desired cell type,” says Liu. This is especially exciting for the prospect of growing pure pancreatic and liver cells, among others.
The agreement also provides Sanford-Burnham access to Intrexon’s RTS technol-ogy, a proprietary ligand-activated biological “switch” that allows researchers to turn gene
expression on or off. This will allow Sanford-Burnham scientists to regulate when certain genes are expressed in cells, which has several promising applications with regard to iPSCs.
“We’re interested in the RTS technology because it will help us to turn genes on or off in stem cells that have been transplant-ed,” Dr. Evan Y. Snyder, director of Sanford-Burnham’s Stem Cell Research Center and Stem Cell and Regenerative Biology Program, said in a media release about the agreement. “For example, it can be used for therapeutic protein expression in stem cells that home to and help eradicate brain tumors.”
Sanford-Burnham is a nonprofit medical research institute with locations in California and Florida. Its Stem Cell Research Center, nicknamed the “Stem Cell Core,” provides resources and services to researchers at orga-nizations around the world. Sanford-Burn-ham and its partners are pursuing iPSCs for a variety of prospective uses, including drug development or potentially using culture-born cells to treat disease directly.
Intrexon is a privately held biotechnology company focused on the industrial engineer-ing of synthetic biology. ddnediTConneCT: e021322
and early development, says, “We look for-ward to working with the team at Sutro and to exploring their platform’s potential to accelerate the discovery and development of superior multifunctional biologics.”
Celgene was interested in applying Sutro’s platform to generate many types of proteins and using Sutro’s cGMP manufacturing facil-ity, thus reducing the time for the preclinical development, according to Daniel.
Sutro’s antibody-based therapeutics for targeted cancer therapies will “significantly extend the clinical impact of current oncol-ogy therapeutic approaches and are beyond what can be envisioned with current, cell-based expression technologies,” Newell says. Sutro’s biochemical synthesis technology “allows the rapid and systematic exploration of many protein drug variants to identify drug candidates.” Once these product candidates are identified, production can be rapidly and predictably scaled up to commercial levels.
“Sutro aims to successfully complete the research, development and regulatory mile-stones in the Celgene collaboration,” Newell says. “Additionally, Sutro continues to develop its own pipeline and to establish additional collaborations with pharma and biotech com-panies interested in developing next-genera-tion ADCs and bispecific antibodies.”
Sutro will be responsible for product design and production of preclinical materi-als and will make clinical batches of a Cel-gene-developed “naked” antibody that works without carrying a drug. ddnediTConneCT: e021320
adCcoNtiNueD From page 24
26 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.comReseaRch & Development
managing director and general counsel for the Law of Life Proj-ect—a pro-life law firm represent-ing the plaintiffs—tells ddn. “The court grants a hearing in about one of every 4,000 cases. But we are still disappointed that the court didn’t take this opportunity to solve two big problems that we have across the country, which are that courts are issuing preliminary
injunctions, but then changing their minds; and that the president is using executive orders to trump federal statutes.”
Casey’s clients filed their law-suit after President Barack Obama issued an executive order lifting restrictions placed on federal fund-ing of hESC research by his prede-cessor, President George W. Bush. The order was followed by new U.S. National Institutes of Health (NIH) guidelines for hESC research and a public comment period.
Sherley and Deisher sued the government, alleging a failure to review and respond appropriately to public comments, challenging the NIH’s interpretation of the Dickey-Wicker amendment and arguing that Obama’s order intensified com-petition for government dollars, making it more difficult for them to get funding for their own research, using only adult stem cells.
A long court volley ensued. In October 2009, Judge Royce C. Lamberth of the U.S. District
Court for the District of Columbia granted the government’s motion to dismiss the suit on the ground that the plaintiffs lacked standing. However, in June 2010, the D.C. circuit court reversed this decision, finding that the plaintiffs had alleged sufficient competitive injury, and granted a preliminary injunction against federal hESC funding. The NIH appealed this injunction, and just a few weeks later, the Court of Appeals issued a stay while the appeal was pending. Although the
appeals court recognized that Dick-ey-Wicker was “ambiguous,” it ulti-mately deferred to the NIH’s inter-pretation that it could fund research using stem cells from embryos that were not actually destroyed in the course of that research.
The plaintiffs then filed a peti-tion for writ of certiorari with the Supreme Court, arguing that the government failed to respond in any way to the 30,000 comments made on the NIH’s new guidelines.
The Supreme Court made no
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called Give Cures that is recruiting patients and doctors nationwide. The ultimate objective is to part-ner with and make available data and cell lines to academia, govern-ment and industry to advance their respective drug discovery efforts.”
The cell lines for the biobank are derived solely from adult stem cells, thus sparing the partnership the controversy associated with the use of human embryonic stem cells. Stem cell donations to the bank will come directly from patients recruit-ed from private practice doctors and private hospitals.
The project also enables drug testing on patient-specific stem cells, in contrast to the currently used models involving animal test-ing and clinical trials that are vastly more expensive, time-consuming and less effective, says Moy.
The biobank stem cell lines will serve as models to better predict the outcome of drug therapy in patients and dramatically advance research to bring new treatments to market sooner and at lower cost.
While the main objective of the partnership is “to create a patient and physician registry, recruit patients, harvest tissue and create stem cell lines, once the lines are developed, it is anticipated that we will partner with other stakehold-ers working on their own drug dis-covery platforms,” Moy says. ddnediTConneCT: e021323
Jp2mricoNtiNueD From page 24
sTem CellcoNtiNueD From page 1
comment in denying the petition, which is not an unusual move for the court. Important consid-erations for accepting a case for review include the need to resolve disagreements among lower courts about specific legal questions, or to consider issues that seem to be of importance to the public. That wasn’t the case here, says Hank Greely, a Stanford University Law School professor who has closely followed the case on his Center for Law and the Biosciences blog.
“Even if the Supreme Court thinks a lower court has it wrong, if there is not disagreement in the lower courts, they let it go,” Greely says. “As for the second instance, although this is a politically charged issue, I am not sure the court feels like it needs to go out of its way to look for another one.” ddnediTConneCT: e021301
BRIEFS
ContraCt researCh serviCes
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 27
Synteract acquires Harrison Clinical Research
SAN DIEGO—Synteract has announced the sign-ing of a definitive agreement under which it will acquire Harrison Clinical Research (HCR), an international contract research organization headquartered in Munich, Germany, with opera-tions in Europe, Israel, the United States and South America. The transaction gains Synteract a clinical inpatient unit in Germany, as well as a clinical research training center in Belgium. The combined company will be led by Wendel Barr, CEO of Synteract, and will maintain its connec-tion with emerging to midsize biopharmaceutical companies. Dr. Francisco Harrison, chairman and founder of HCR, will be a member of the execu-tive team and the board of directors. HCR’s cur-rent management will remain in place. Financial details were not released.
CHOP, BGI target pediatric diseasesPHILADELPHIA—The Children’s Hospital of Phila-delphia (CHOP) and BGI have teamed up to form BGI@CHOP, a partnership geared to discover the genes responsible for rare and common pediatric diseases using next-generation sequencing. The large-scale human genome sequencing and bio-informatics analysis will take place at the new Joint Genome Center at Children’s Hospital. This partnership combines BGI’s expertise with whole-genome sequencing and analysis with CHOP’s biobank and experience in clinical phenotyping.
“We are very excited about this new partner-ship with BGI. It is an ideal collaboration between two world-class institutions. The ultimate goal is to change the way we diagnose and treat dis-eases that affect children and families, and our work with BGI is an important first step toward that end,” Dr. Philip Johnson, chief scientific offi-cer at CHOP, said in a press release.
Catalent targets biosimilars with UMN Pharma
SOMERSET, N.J.—An agreement has been inked between Catalent Pharma Solutions and UMN Pharma Inc. to provide UMN Pharma with a range of biosimilar cell lines through the use of Catalent’s proprietary GPEx technology. Per the agreement, UMN and its subsidiary UNIGEN will produce biosimilars using Catalent’s GPEx cell lines. Sepa-rately, UMN Pharma will assemble an alliance of pharmaceutical companies for product develop-ment, including clinical trials, marketing and sales, and plans to begin several biosimilar projects with the allied companies for the Asian market. “There are great synergies between UMN Pharma and Catalent in terms of technologies, business structure, speed and culture. Harness-ing Catalent’s broad biologics development and cell construction expertise gives UMN Pharma a unique platform to grow our biosimilars business globally,” said Masahiro Michishita, executive chairman of UMN Pharma.
Second time around LGC acquires bioanalytical sciences business from Quotient BioresearchBY JEFFREY BOULEY
MIDDLESEX, U.K.—For the second time in two years, LGC Ltd. has acquired a business unit from Cam-bridgeshire, U.K-based Quotient Bioresearch, this time the division formerly known as Quotient Bioana-lytical Sciences.
“We’re delighted to have acquired Quotient Bioanalytical Sciences, a high-quality business which is com-plementary to our existing activities,” said Jeremy Cook, managing director of LGC’s Health Sciences division, in an official statement about the early-January acquisition. “We continue to develop our range of first-rate services in the pharmaceutical, biotechnology and agrochemical sectors by offering our customers a unique mix of techni-cal experience, leading-edge facilities and knowledgeable people.”
This facility, as it happens, is located in Fordham, Cambridgeshire—the same location as HFL Sport Science, which was the business unit LGC previously acquired from Quotient in December 2010.
HFL, one of the world’s largest independent providers of drug sur-veillance, doping control and research activities to human and equine sports, was—like the bioanalytical sciences division—also touted in terms of its ability to “complement LGC’s current analytical service offerings,” but it was also highlighted for its ability to “extend” those offerings.
The newest acquisition will form part of LGC’s Health Sciences division and, admittedly, will probably also be an opportunity to expand as well as complement, as it will “enable LGC to provide an enhanced range of products and services to the pharmaceutical sector including bioanalysis, materi-als science and reference materials, amongst others,” according to LGC.
Lgc coNtiNueD oN page 28
Controlling the flow of informationPAREXEL acquires regulatory information management provider LiquentBY LLOYd dUnLap
BOSTON—PAREXEL Intl. Corp. has acquired all of the outstanding equity securities of Liquent Inc., a global provid-er of regulatory information management (RIM) solu-tions. The purchase price was approximately $72 million (which was adjusted at clos-ing to reflect Liquent’s cash, indebtedness and working capital balances), and was funded through the expan-sion of one of the company’s existing credit facilities. Liquent provides an integrated platform of software solutions for regulatory submis-sions and product registration manage-ment, as well as a range of complementary
business process outsourcing capabilities. Prior to the sale, Liquent was owned by Mar-lin Equity Partners. Liquent was founded in 1994, and its clients include more than 200 biopharmaceutical and life-science compa-nies. With headquarters in Horsham, Pa., and additional offices in the United Kingdom,
Germany and India, the company employs nearly 300 individuals.
“Liquent is a very strong player in the impor-inFO coNtiNueD oN page 28
The addition of Liquent strengthens PAREXEL’s Perceptive Informatics and its regulatory solution offerings, and enables PAREXEL to provide a stronger end-to-end range of software and outsourcing solu-tions across development, regu-latory and commercialization, says Josef von Rickenback, chairman and CEO of PAREXEL.
A deal with ‘significant efficiencies’CNS acquires business assets of longtime partner BELTASBY KELsEY KaUstinEn
BRISBANE, Australia—Clinical Network Services (CNS), an Australian contract research organization (CRO) that offers integrated development services for the planning, implementation and delivery of Phase I and II trials, has announced the acquisition of the business assets of BELTAS, an Auckland, New Zealand-based CRO and longtime partner of CNS. The acquisition was officially completed at the end of 2012, and BELTAS’ related con-tracts and clinical staff were transferred to CNS. No financial terms were disclosed.
“2012 has been a tremendous year for CNS and has seen us grow significantly over
the last 12 months, expand our regional CRA base so we can better service local sites in Perth and Melbourne as well as further bed down our BioDesk services,” Russell Neal, managing director of CNS, said in a press release. “This acquisition continues our goal of widening our capa-bilities across Australia and New Zealand, and by bringing together these two highly skilled clinical teams with years of collec-tive experience, we are able to offer our clients a truly regional solution.”
Neal says that the acquisition is one that the company has been considering for some time “in response to the growth in the num-ber of CNS’ clients looking to capitalize on other opportunities local to Australia.”
“Bringing [New Zealand] into the broader Australian solution is not a new concept, but as CNS has been reliant on our partner
BELtas coNtiNueD oN page 29
TICKER>> clinical Network
services, a cro, acquires beLtas’ business assets
>> beLtas is based in auckland, New Zealand and is a longtime partner of cNs
contract research services28 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
tant and growing market of regula-tory information management tech-nology and services solutions,” says Josef von Rickenback, chairman and CEO of PAREXEL. “The addition of Liquent strengthens PAREXEL’s Per-ceptive Informatics and its regulatory solution offerings and enables PAR-EXEL to provide a stronger end-to-end range of software and outsourc-ing solutions across development, regulatory and commercialization. In the end, this benefits clients because it allows PAREXEL to provide a more complete suite of offerings.”
Through Liquent’s flagship soft-
ware platform, InSight, PAREXEL’s clients will have access to compre-hensive regulatory agency submis-sion planning, viewing, tracking, publishing and registration man-agement throughout the entire lifecycle of a medicinal entity, says von Rickenback.
“We expect the acquisition will also benefit the PAREXEL Consult-ing and Medical Communications Services business, where we will be able to leverage Liquent’s signifi-cant expertise in regulatory infor-mation management outsourcing through its Liquent Direct solu-tions,” he adds.
Clients are looking for fewer stra-tegic partners that can provide a broader range of service and technol-ogy solutions, von Rickenback notes.
“PAREXEL has a full range of clinical outsourcing services and ... clinical software capabilities, and Liquent has done the same thing from a regulatory perspective. Liquent will now be part of a full
range of capabilities for clients and will be afforded the opportunity to get involved earlier in the develop-ment process,” he says.
For a year or so, Liquent will largely continue to operate as a distinct service line within PAR-EXEL’S Perceptive Informatics
group. Integration teams will be established to evaluate the appro-priate points of future integration to maximize the combined services and technologies to serve current and prospective customers. The Liquent management team will continue to run their operation and PAREXEL “welcomes their exper-tise and the value their employees bring to PAREXEL’s clients and employees,” the company states.
Perceptive Informatics is the technology subsidiary of PAREXEL and provides eClinical solutions that help customers maximize the benefits of an integrated clinical trial technology suite by provid-ing flexible software-as-a-service,
or SaaS, applications. The subsid-iary offers a portfolio of products and services that includes medical imaging services, interactive voice response systems, clinical trials management systems, web-based portals, systems integration and patient diary applications.
In conjunction with the acqui-sition, PAREXEL also updated its financial guidance for the fiscal year. The company anticipates reporting consolidated service rev-enue in the range of $415 million to $420 million for the second quarter of the fiscal year, and in the range of $1.675 billion to $1.695 billion for fiscal year 2013. Of the increase, the Liquent acquisition is expected to contribute a small amount of ser-vice revenue in the second quarter and between $17 million and $23 million in service revenue during the second half of fiscal year 2013. The previous consolidated service revenue guidance was $400 million to $410 million for the second quar-ter and $1.63 billion to $1.66 billion for the fiscal year. ddnEditcOnnEct: E021324
inFOcoNtiNueD From page 27 TICKER
>> pareXeL acquires Liquent for approximately $72 million
>> Liquent provides software solutions for regulatory submissions and product registration management
Not your usual CRO arrangementU.S.-based biopharma Onconova partners with Indian CRO GVK Biosciences on development of cancer drugsBY amY swindERman
HYDERABAD, India—It’s not uncommon for U.S.-based drug developers to seek the services of a contract research service provider abroad, but it is novel for a company to align itself with a service provider that contributes intellectual prop-erty of its own to a drug discov-ery target. That’s the unique drug discovery model put in place last month by Onconova Therapeutics, a U.S.-based biopharmaceutical com-pany focused on the development of novel small molecules for oncology, and Indian contract research orga-nization (CRO) GVK Biosciences.
On Jan. 9, the companies announced that they have entered into a novel joint endeavor to develop new cancer drugs. The partnership will be based in the United States and will align research priorities and tech-nological expertise from both com-panies to facilitate moving certain Onconova oncology assets from early discovery to the clinical development stage. Dr. E. Premkumar Reddy, the
scientific founder and director of Onconova and a world-renowned molecular oncologist, will oversee the biology and biomarker aspects of the partnership. Onconova will provide two discovery targets with early chemical equity, while GVK BIO will use its multidisciplinary discovery platform to advance these programs through lead optimization and Investigational New Drug (IND) candidate selection.
GVK will gain an increasing share of the programs as they advance, up to a 50/50 split based on achieve-ment of milestones and funding brought into the joint partnership. Onconova retains the rights to buy back the programs. Financial terms of the partnership were not released.
“We have used CROs for some of our clinical trials, but not in this capacity,” says Ben Hoffman, direc-tor of public and investor relations for Onconova. “This is a unique agreement in that GVK will openly serve as a partner that is helping us to develop our assets. They were highly motivated to help us move these projects forward.”
Founded in 1998 as a chemistry platform company, Onconova has an internally developed pipeline of mostly small-molecule agents based on a portfolio of nearly 150 differ-ent chemical phenotypes. The com-pany is now focused on pursuing novel pathways to specific types of cancer, improving drug efficacy and
overcoming drug resistance and the burden of chemotherapy toxicity.
Onconova’s most advanced prod-uct, rigosertib, is being tested in a Phase III pivotal trial under a Special Protocol Assessment from the U.S. Food and Drug Administration for myelodysplastic syndromes (MDS), a type of blood cancer. Onconova is developing rigosertib in partner-ship with Baxter Intl. for Europe and SymBio Pharmaceuticals for Japan and Korea, and retains com-mercial and development rights in the rest of the world.
Rigosertib has been granted orphan drug status for MDS in both the United States and Europe. A New Drug Application submission for rigosertib’s initial indication for MDS is projected for 2014. Oncon-ova is also developing rigosertib as a treatment for metastatic pancre-atic cancer. The drug is currently being tested in a Phase III trial (with a planned interim analysis)
for this indication. In addition, Onconova is target-
ing unmet needs in blood cancers and solid tumors to ensure rapid approval and first-to-market advan-tage for its advanced products.
GVK BIO works with more than 200 pharmaceutical and biotech companies across multiple service offerings, has a scientific pool of more than 2000 scientists and has an IP-generating capability that has delivered INDs to other clients.
“The demands for integrated and outcome-based research deals are increasing in the service business,” said Manni Kantipudi, CEO of GVK, in a press release announcing the partnership. “This announce-ment shows GVK BIO’s commit-ment to new and innovative models for drug discovery with partners. Onconova is a leading oncology company, and GVK BIO is happy to partner with them.” ddnEditcOnnEct: E021327
“We have used CROs for some of our clinical trials, but not in this capacity. This is a unique agreement in that GVK will openly serve as a partner that is helping us to develop our assets.”Ben Hoffman, director of public and investor relations for Onconova
Under the terms of the deal, Quotient and LGC have “committed to collaborate as preferred partners on the provision of Bioanalytical Sci-ences,” and this will reportedly enable clients to continue to benefit from the early devel-opment services offered by the Quotient group, including clinical trials, metabolism and radiolabeling.
For now at least, the Bioan-alytical Sciences business will continue to trade using the “Quotient” name, even though it is under new ownership.
“LGC’s focus on complex analytical chemistry, high-quality science and service delivery make them a natural home for our business,” said Dr. Steve Pleasance, manag-ing director of Quotient Bio-analytical Sciences, after the announcement of the acqui-sition. “Both our organiza-tions have reputations built upon quality and innovation, and LGC’s global presence will help support the contin-ued growth of services to our customers.”
The Quotient deal isn’t the only recent expansion news from LGC. Shortly before acquiring Quotient Bioanalyti-cal Sciences, LGC acquired in mid-December the trade and assets of Bio Senate, a sup-plier of microbiology profi-ciency testing and reference materials based in Bury, U.K. The business was merged with LGC Standards, a division of LGC Group and a leading global supplier of reference materials and proficiency test-ing schemes. Bio Senate sup-plies a range of microbiology proficiency testing schemes focused on the pharmaceuti-cal, food and water, as well as a range of microbiology refer-ence materials, sterile matrix materials and quality-control strains to more than 700 labo-ratories worldwide—thus, it is considered highly complemen-tary to LGC Standards’ existing proficiency testing business.
In mid-January, right after the Quotient deal, LGC wid-ened its U.S. market share by investing in a genom-ics laboratory expansion in Beverly, Mass., near Boston. This move initiates nucleic acid extraction services from LGC to customers in North America, and is intended both to complement exist-ing LGC genotyping services in Berlin and London and to allow improved research and development support to North American researchers. ddnEditcOnnEct: E021326
LgccoNtiNueD From page 27
“PAREXEL has a full range of clinical outsourcing services and ... clinical software capabilities, and Liquent has done the same thing from a regulatory perspective. Liquent will now be part of a full range of capabilities for clients and will be afforded the opportunity to get involved earlier in the development process.”Josef von Rickenback, chairman and CEO of PAREXEL
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 29contract research services
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to achieve this to date, we recog-nized that there were significant efficiencies such an acquisition would effect and thus improve our client service yet further … I am delighted to say we have been able to achieve this very quickly and effectively,” he adds.
The acquisition includes most of BELTAS’ business assets, though BELTAS has retained some busi-ness functions, says Neal, includ-ing local training services. Among the acquired assets are BELTAS’ ongoing New Zealand studies and its clinical research team.
“The fact that all of the team have been working under CNS project managers for, in some cases, sev-eral years, has allowed for a very smooth and seamless transition,” says Neal. “This has been evidenced by positive support we have already received from our clients.”
BELtascoNtiNueD From page 27
“Bringing [New Zealand] into the broader Australian solution is not a new concept, but as CNS has been reliant on our partner to achieve this to date, we recognized that there were significant efficiencies such an acquisition would effect and thus improve our client service yet further.”Russell Neal, managing director of CNS
Neal notes that CNS has always sought to “have a deep understand-ing of just what can be delivered by our region and how that benefits our clients’ global product devel-opment strategies.” Along those lines, he says, this transaction fits “extremely well” into CNS’ growth strategy, which is “to add capabili-ties that first and foremost lever-age that local capability in a way that directly and positively adds value to our clients’ investment in this region and expedites their
development timelines.” Adding CNS capabilities in New Zealand, says Neal, “clearly met this strat-egy criteria.”
CNS and BELTAS have been partners for several years, Neal notes, adding that “establishing committed, quality partnerships has very much been CNS’ strategy since our inception in the 1990s, both locally and internationally.”
“Working together in this man-ner has always been recognized as a sound business precursor to
establishing closer relationships until such time as to unlock front and back office efficiencies requires combining the entities,” he says. “This has very much been the case with CNS and BELTAS, but spe-cifically in respect to our clinical services. As BELTAS have other business units such as training that aren’t part of CNS’ core business, it also made sense for BELTAS to continue such services in their own way whilst allowing CNS to contin-ue to focus solely on the provision
of our intelligent product develop-ment services.”
CNS caters to virtual, small- and medium-sized biotech companies, and offers services such as product development and regulatory affairs planning and development, clinical planning and study startup, moni-toring, project management, data management/biostatistics, medical consultancy/monitoring, medical writing, bioanalytical services and safety reporting. ddnEditcOnnEct: E021325
Products & services30 Drug Discovery News | | February 2013 For more information, visit www.DrugDiscoveryNews.com
Rapid multiplexed imagingTTP LabtechTTP Labtech introduces the acumen hci system, for more productive high-through-put imaging. The system offers fast imag-ing for multiplex assays in up to 1,536-well plates, with open-source image format for flexible image analysis. The acumen hci can image and analyze the wells of 96- to 1,536-well plates in eight minutes, at a res-olution of 0.5 µm/pixel, and with up to three lasers, multiplexing is easy in a wide range of fluorescent dyes. The system incorpo-rates TTP Labtech’s proprietary cellista software, and is compatible with micro-scope slides using the widefield objective.
TTP Labtech+44 1763 262626www.ttplabtech.com
Metabolic biomarker kitsPerkinElmerPerkinElmer announces the launch of a trio of kits from its AlphaLISA assay platform, including C-peptide, leptin (mouse) and FGF21, fibroblast growth factor 21, a protein involved in pathways that control glucose, lipid, bile acid, phosphate and vitamin D metabolism. The kits require small sample volumes, 1 to 5 µl, and enable biomarker assays to be run in less than three hours.
Perkinelmer(800) 762-4000www.perkinelmer.com
Fast, simple 96- and 384-channel pipettingINTEGRA BiosciencesINTEGRA Biosciences introduces the INTEGRA VIAFLO 384, a handheld 384-channel electronic pipette with lower sample and reagent use. Users can choose between two 384-channel pipet-ting heads that cover volume ranges of 0.5 to 12.5 µl and 5 to 125 µl, and the unit is fully compatible with VIAFLO 96-channel pipet-ting heads for easy switching. The system features INTEGRA’s Touch Wheel pipette user interface for simple programming and comes with 10 predefined pipetting modes.
iNTegra biosciences+41 (0)81 286 95 30www.integra-biosciences.com
Automated pipetting systemsEppendorfEppendorf introduces the epMotion P5073 and M5073 workstations for a variety of automated liquid handling applications. The epMotion M5073 automates DNA purifica-tion, with short setup time and elution vol-umes as low as 25 µl. The epMotion P5073 helps to automate the PCR setup workflow in 96- or 384-well formats, and features four step-by-step guided PCR assistants for work involving normalization, dilution,
High-pressure ion chromatography systemThermo Fisher Scientific Inc.The Thermo Scientific Dionex ICS-5000+ Reagent-Free HPIC system is designed to boost ion chromatography separa-tion capabilities, operating at continuous pressures of up to 5,000 psi at analytical flow rates. With the system’s high back-pressure tolerance, users can increase flow rates for maximized throughput while retaining electrolytic eluent generation and suppression. The system features a proprietary all-PEEK polymer flow path for metal-free inertness, and can utilize 4 µm-particle-size IC columns at high pres-sures. The unit can perform simultaneous analyses of two samples or two-dimen-sional analyses of a single sample.
Thermo Fisher scientific inc.(800) 678-5599www.thermofisher.com
Versatile image analysis systemSyngeneSyngene introduces the PXi Touch range of high-resolution, multi-application image analysis system for accurate imaging of chemiluminescent and fluorescent blots and 1-D and 2-D gels stained with any vis-ible or fluorescent dye. The compact sys-tem features a built-in touch screen and processor, and comes with a high-reso-lution 4-million, 6-million or 8-million pixel camera with a large fixed aperture lens. The PXi Touch can be used with Syngene’s UV and blue light transilluminators, and white, IR, red, blue or green epi lighting.
syngene(800) 686-4407www.syngene.com
Flexible microplate storageThermo Fisher Scientific Inc.Thermo Fisher Scientific Inc. announces the launch of the Thermo Scientific VALet, a benchtop robot that enables configurable laboratory automation. The system is designed for multiple hotel positioning for customizable, high-den-sity microplate movement and storage, and features a four-axis plus servo-gripper. Random or sequential storage access provides a capacity of up to 45 or 120 microplates, respectively, and thanks to its Thermotor technology, the VALet can be controlled from a computer.
Thermo Fisher scientific inc.(800) 678-5599www.thermofisher.com
Purified DNA, cDNA offeringsAMSBIOAMSBIO introduces several additions to its collection of genomic DNA and cDNA products derived from human normal and disease-state tissue types, including can-cer and normal tissue cDNA arrays. Each array’s cDNA is synthesized from high-quality total RNA from pathologically veri-fied tissues, and each array comes with clinical information and QC data normal-ized and validated with ß-actin in two qPCR analyses. AMSBIO’s newly introduced genomic DNA is isolated from FFPE tissue, and its 96-well genomic DNA plates enable high-throughput gene profiling studies in automated gene analysis systems.
aMsbio+44 (0) 1235 232100www.amsbio.com
Fully automated blood fractionationHamilton RoboticsThe Microlab easyBlood STARlet worksta-tion from Hamilton Robotics is a fully auto-mated system for blood fractionation in biobanking applications. The high-through-put system features state-of-the-art imag-ing technology, pipetting capabilities and powerful sample tracking software, and is compatible with LIMS. Pipetting speed can be adjusted via the software, and the system comes with all required supplies.
Hamilton robotics(800) 648-5950www.hamiltonrobotics.com
setup reactions and composing master-mixes. Both units have six SBS positions and feature a large color touch screen.
eppendorf(800) 645-3050www.eppendorf.com
Small-diameter UHPLC mediaPhenomenex Inc.Phenomenex Inc. introduces its new 1.3-micron UHPLC columns, the latest addi-tion to its Kinetex core-shell product line. The 1.3-micron features efficiency levels over 400,000 plates per meter, with easy method transferability and scalability to any of the other Kinetex particle sizes. The columns offer increased sensitivity and performance, as well as high resolution.
Phenomenex inc.(310) 212-0555www.phenomenex.com
Washer performance testingBioTekThe 405 Touch and 405 LS Microplate Washers from BioTek are now available with its patented Verify Technology, a fast, reliable way to identify clogged washer manifold tubes. By using an ultrasonic sen-sor, Verify can pinpoint clogged dispense and aspirate tubes. A preprogrammed quality-control routine fills microplate wells and confirms fluid levels, aspirates the wells, then measures again, and any discrepancies indicate a blockage.
bioTek(888) 451-5171www.biotek.com
Single-platform phenotypic screeningGenedataThe Genedata Screener for High Content Screening (HCS) covers all stages of screening analysis, from cell-level data to final campaign results. The system supports multiple HCS image acquisition and analysis systems, eliminating data
Flexible open platformSingulex Inc.The Erenna Immunoassay System, part of Singulex Inc.’s Singulex Solution, includes high-definition immunoassays and a full range of custom services such as assay development, sample testing and antibody derivatization. Singulex’s high-definition, single-molecule counting technology enables quantification of biomarkers with LLoQ to femtogram levels.
singulex inc.(888) 995-6955www.singulex.comVisit us at ToxExpo 2013 at booth #666
F E AT U R E D
analysis bottlenecks and offering instant image access. Genedata Screener uni-fies HCS data analysis workflow and enables interactive definition and analy-sis of cell populations, and features a stand-alone image viewer for down-stream applications access to images.
genedata(877) 436-3872www.genedata.com
Blue light LED illuminatorsBiometraBiometra has expanded its offering to include illuminators with high-performance blue light LEDs for gel documentation pur-poses. Unlike UV tables, the illuminators pose no risk of sample damage during illu-mination, and blue light excitation is appli-cable for fluorescent dyes for nucleic acid or protein stains, with excitation wave-length around 470 nm. The illuminators come in two versions: BLstar9 for mini gels, and BLstar 16 for gels up to 16 cm x 20 cm.
biometra+49 551 50686-0www.biometra.com
Modular flow reactor systemsUniqsisUniqsis introduces a new portfolio of modular flow systems based on its Binary Pump Module (BPM), a stand-alone two-channel high-pressure reagent deliv-ery system, and FlowSyn flow reactor technology. Uniqsis’ FlowSyn Auto LF can automate multi-step, multi-reagent experiments without supervision. The lat-est BPM module, the Polar Bear Plus Flow reactor module, is capable of maintaining temperatures from -40°C to +150°C with-out cardice or liquid nitrogen. The BPM is available in three flow paths—PTFE, stainless steel and Hastelloy —and is capable of pumping up to 100 ml/min and operating at up to 200 bar.
uniqsis+44-845-864-7747www.uniqsis.com
AMRI .......................................................................3 www.amriglobal.com
Applied Photophysics Ltd. ...............................17 www. photophysics.com
Aptagen ..................................................................9 www.aptagen.com
Bio-Rad Laboratories, Inc. ...............................32 www.bio-rad.com
Cambridge Healthtech Institute .....................23 www.healthtech.com
Exiqon .....................................................................5 www.exiqon.com
INDIGO Biosciences .........................................26 www.indigobiosciences.com
Offenberger & White, Inc. ................................29 www.offwhite.com
Panasonic Healthcare
Company of North America ..........................2 www.panasonic.com/biomedical
R&D Systems, Inc. .............................................25 www.RnDSystems.com
Rockland Immunochemicals, Inc. .................13 www.rockland-inc.com
Singulex, Inc. ......................................................20 www.singulex.com
Thermo Scientific Pierce
Protein Research Products .................Cover www.thermoscientific.com/pierce
A D V E R T I S E R ’ S I N D E x
For more information, visit www.DrugDiscoveryNews.com February 2013 | | Drug Discovery News 31Facts & Figures
Drug Discovery News (usPs 024-504) is published monthly by old river Publications LLc, 19035 old Detroit road, suite 203, rocky river, oH 44116; 440-331-6600. Periodical postage paid at cleveland, ohio and additional mailing offices. Publisher assumes no responsibility for unsolicited material or prices quoted in the magazine. contributors are responsible for proprietary classified information. ©2013 by old river Publications. all rights reserved. reproduction, in whole or in part, without written permission of the publisher is expressly prohibited. back issues, when available, cost $7 each within the past 12 months; $12 each prior to the past 12 months. back orders must be paid in advance by check. Drug Discovery News is distributed without charge in North america to qualified drug discovery research professionals. Paid subscriptions to those not qualified cost $65 annually to the u.s. and canada and $150 to all other countries. all payments must be made in u.s. funds drawn on a u.s. bank. Publications mail agreement no. 41401058 return undeliverable canadian addresses to Po box 503, rPo west beaver creek, richmond Hill, oN L4b 4r6. For subscription services, including subscription information, please call 215-785-5196. PosTMasTer: send address changes to Drug Discovery News, Po box 3100, Langhorne, Pa 19047-8800.
A s modernization in drug development technologies continues to enable contract research organizations (CROs) to offer cost-effective, complete solutions for drug manu-facturers, Europe ranks second after the United
States in the global CRO market, according to a recent market research report by Frost & Sullivan.
According to the report, “Analysis of the European Contract Research Outsourcing Markets: Opportunities in the Rapidly Changing Clinical Drug Development Outsourcing Market Across Europe,” Europe accounts for 26 percent of the global CRO market share. A rise in CROs in Eastern Europe and Asia are hindering the Western European market growth due to economic constraints. The European CRO market generated $6.07 billion in 2011. The market is expected to reach approximately $11.54 billion in 2018 at a CAGR of 9.6 percent from 2011 to 2018.
The report examines the European regions of Germany; France; the United Kingdom; Italy; Spain; the Benelux region composed of Belgium, the Netherlands and Luxembourg; and the Scandinavian region composed of Denmark, Norway and Sweden.
Key end-user groups of CRO services include pharmaceutical and biotechnology drug developers, government-controlled agencies of health and universities. Frost & Sullivan’s list of pharma CRO customers includes Sanofi, Bristol-Myers Squibb Co., GlaxoSmithKline PLC, Prodia Clinical Laboratory, ASLAN Pharmaceuticals, Debiopharm Group, EMD Millipore, Shire Specialty Pharmaceuticals and Transpogen Biopharmaceuticals.
Among the key industry participants reviewed by Frost & Sullivan are Quintiles, Charles River Laboratories, Covance, Icon, PRA International, PAREXEL International, INC Research, Theorem Clinical Research, Pharmaceutical and Product Development, PharmaNet/i3, CONET, Pierrel Research Italy and Harrison Clinical Research. According to the report, the top 10 CROs contribute to 55.5 percent of the total CRO market. Covance leads the CRO market share in the European market with 10.5 percent, followed closely by Quintiles with 9.5 percent.
Frost & Sullivan notes that exclusive service offerings along niche therapeutic areas are the major driving factors within this market. These segments include cardiovascular diseases, central nervous system disorders, endocrine and metabolic disorders, infectious diseases, oncology and other diseases such as respiratory infections, ophthalmology, specialty disorders, orphan diseases and neurology.
Service contracts and impactful acquisition activities help build market position, a com-mon trend seen among all top CROs. Most of the top CROs expand operations along prime and low-cost geographic locations to help build a globalized service offering. ddn
all information and data courtesy of Frost & sullivan. For more information about Frost & sullivan’s reports, e-mail [email protected].
Europe ranks second in global CRO market
CNS Disorders Therapeutics Segment: Impact of Key Market Trends
Cardiovascular Therapeutics Segment: Impact of Key Market Trends
Endocrine and Metabolic Disorders Therapeutics Segment: Impact of Key Market Trends
Oncology Therapeutics Segment: Impact of Key Market Trends
High Impact
Low Impact
Projected Impact on the Cardiovascular
Disease Segment
High Growth Impact
CertaintyLow High
Medium Growth Impact
Low Growth Impact
Global tests for declaring safety and effi cacy promote CROs
Focus on retaining partnerships with Big Pharma clients for availing newer contracts for the future
Investment from the biopharmaceutical drug manufacturers to contract research organizations due to the rise in market demand
High Impact
Low Impact
Projected Impact on the CNS Disorders Therapeutics
Segment
High Growth Impact
CertaintyLow High
Medium Growth Impact
Low Growth Impact
Strong market growth potential for the future from CNS disorders segment like pain, fi bromyalgia and diabetic nerve pain
Lack in established disease diagnosis and disease management procedures within several CNS disorders promotes research contracting
Heavy investment from Big Pharma/biopharma clients for Alzheimer’s research
High Impact
Low Impact
Projected Impact on
the Endocrine and Metabolic
Disorders Therapeutics
Segment
High Growth Impact
CertaintyLow High
Medium Growth Impact
Low Growth Impact
Clinical trials and testing for developing improvised anti-diabetic diagnostics and drugs for treatment contribute towards market growth
EMA regulatory laws on anti-diabetic drug development attract clients to adopt outsourcing to meet the demands due to wide range of service offerings by contract research organizations.
Specialized testing offerings like neuro-behavioural activities, toxicokinetic favor outsourcing options over in-house activities
High Impact
Low Impact
Projected Impact on
the Oncology Therapeutics
Segment
High Growth Impact
CertaintyLow High
Medium Growth Impact
Low Growth Impact
Globalization of contract research organizations promotes end-result comparative analysis by co-relating data across different geographies, providing access to a wider range of product molecules
Data management outsourcing helps better systemization of trials and testing results within this segment
Focusing on niche factors in analyzing a tumor and formulating trials, testing and product strategy based on it helps contract research organizations gain popularity against in-house developmental activities
Job # 13-0236 Publication DDN Trim Size 10.625” x 13.875” Run Date 01/01/13
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