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April 2019 / 1

Q2 2019 Outlook Report

Q2 2019

OUTLOOK REPORT

Find out more about the full report

https://pages.pharmaintelligence.informa.com/meddevicetracker-Q2-outlook-demo-request

April 2019 / 2


Summary In this report, we cover catalysts from 25 drugs, devices and diagnostics expected to occur in Q2 2019. For each drug, the likelihood of Phase/PDUFA review success and overall Likelihood of Approval (LOA) given their particular phase, drug class, and disease group are provided. For the first time, these data points were provided using a combination of Pharmapremia, Informa’s drug development benchmarking product utilizing Biomedtracker’s LOA data to assist in informed decisions about drug pipeline prioritization, partnerships, and acquisitions, and drug approval data from Biomedtracker. The results of the catalysts highlighted in our Early 2019 Outlook Report can be found on Page 4. At the end of this report, we have included a list of Large Impact catalysts through early 2019. The catalyst list is also provided in Excel by downloading the supplemental material at the top of this page. Like our report? Have any questions or feedback? Please let us know at [email protected].

About the Author Biomedtracker is an independent research service that offers proprietary clinical assessments of developmental drugs within a comprehensive and intuitive drug information database. Clients from the pharmaceutical, biotech, and investment industries rely on Biomedtracker for its insight on the likelihood of approval, commercial potential, and future data and regulatory catalysts for drugs within the competitive landscape of every important disease and indication. Over recent years, Biomedtracker has become the leader in providing objective information alongside evidence-based clinical assessments and investment research on pipeline drugs worldwide. For more information on getting direct access to Biomedtracker, please email [email protected]. Meddevicetracker is an all new medtech intelligence platform that provides clients with real-time data and analysis on medical devices and diagnostics. From the people behind Biomedtracker, comes an event-driven research service for the medical device and diagnostic marketplace. For access to Meddevicetracker please contact your sales representative or email [email protected].

Disclaimer Copyright © 2019 Sagient Research This report is published by Sagient Research (the Publisher). This report contains information from reputable sources and although reasonable efforts have been made to publish accurate information, you assume sole responsibility for the selection, suitability and use of this report and acknowledge that the Publisher makes no warranties (either express or implied) as to, nor accepts liability for, the accuracy or fitness for a particular purpose of the information or advice contained herein. The Publisher wishes to make it clear that any views or opinions expressed in this report by individual authors or contributors are their personal views and opinions and do not necessarily reflect the views/opinions of the Publisher.

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Contents Outcomes of Biomedtracker's Large Impact Drug Catalysts from the Early 2019 Outlook Report ............... 4

NKTR-181 for Chronic Low Back Pain (CLBP) (NKTR) ..................................................................................... 6 Maribavir for Cytomegalovirus (CMV) Infection (Antiviral) (TAK) .................................................................. 7

Dengvaxia for Dengue Fever - Vaccines and Treatments (SNY) ..................................................................... 8

Visomitin for Dry Eye (Ophthalmology) (Mitotech) ....................................................................................... 9 Humacyl for End-Stage Renal Disease (ESRD) (Humacyte) .......................................................................... 10

Bremelanotide for Female Sexual Arousal Disorder (AMAG) ...................................................................... 11 Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP) (CMTA) ....................................................... 12

Ibrexafungerp for Fungal Infections - Systemic (SCYX) ................................................................................ 13 Jakafi for Graft vs. Host Disease (GVHD) (INCY) ........................................................................................... 14

IFX-1 for Hidradenitis Suppurativa (IFRX) .................................................................................................... 15

Tivicay/Epivir for HIV / AIDS (GSK) ............................................................................................................... 16 AXS-05 for Major Depressive Disorder (MDD) (AXSM) ................................................................................ 17

GSK2857916 for Multiple Myeloma (MM) (GSK)......................................................................................... 18 Selonsertib for Non-Alcoholic Steatohepatitis (NASH) (GILD) ..................................................................... 19

Skyrizi for Psoriasis (ABBV) .......................................................................................................................... 20 Beraprost 314d for Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension (PH) (UTHR) ... 21

Ofev for Scleroderma (Boehinger Ingelheim) .............................................................................................. 22

TransCon Growth Hormone for Short Stature / Growth Hormone Deficiency (ASND) ............................... 22 Zolgensma for Spinal Muscular Atrophy (NVS) ............................................................................................ 23

Relugolix for Uterine Fibroids (MYOV) ........................................................................................................ 24 DBLG1 System for Diabetes Mellitus, Type I (Diabeloop) ............................................................................ 26

FoundationOne for Solid Tumors (RHHBY) .................................................................................................. 27 Acquisition of BTG plc by Boston Scientific ................................................................................................. 28

Acquisition of Spark Therapeutics by Roche................................................................................................ 29 Q2 2019 Large Impact Drug Catalysts………………..…..………………..………………………………………………………………30

Q2 2019 Large Impact Device/Diagnostics Catalysts……………………………………………………………………………..…40

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Outcomes of Biomedtracker's Large Impact Drug Catalysts from the Early 2019 Outlook Report

Occurred Date

Lead Company Product Market Catalyst

Did LOA Predict

Outcome

LOA Before Outcome

LOA After Outcome

2/6/2019 MacroGenics Margetuximab Oncology Phase III SOPHIA - Top-Line Results Yes 36%

(1% Above Avg.) 43%

(8% Above Avg.)

2/6/2019 Sanofi Cablivi Hematology PDUFA for BLA - First Review Yes 89%

(5% Above Avg.) 100%

(Same As Avg.)

2/14/2019 Motif Iclaprim (IV) Infectious disease PDUFA for NDA - First Review No 98%


(5% Above Avg.)

2/19/2019 Intercept Ocaliva Endocrine Phase III REGENERATE - Top Line

Results Yes

62% (2% Above Avg.)

63% (3% Above Avg.)

2/21/2019 Zosano Qtrypta Neurology Phase III M207-ADAM - Top-Line

Results Yes 57%

(5% Above Avg.) 57%

(5% Above Avg.)

2/28/2019 Horizon Teprotumumab Ophthalmology Phase III - Top-Line Results N/A1 51%

(Same As Avg.) 61%

(10% Above Avg.)

2/28/2019 Novavax ResVax Infectious disease Phase III Prepare - Top-Line Results Yes 55%

(6% Below Avg.) 51%

(10% Below Avg.)

3/5/2019 Johnson & Johnson

Spravato Psychiatry PDUFA for NDA - First Review Yes 93%


(Same As Avg.)

3/12/2019 Aerie Rocklatan Ophthalmology PDUFA for 505(b)(2) NDA - First

Review Yes 88%


(Same As Avg.)

3/20/2019 Jazz Sunosi Neurology PDUFA for NDA - First Review Yes 87%


(Same As Avg.)

3/22/2019 Recro N1539 Neurology PDUFA for NDA - Second Review No 86%

(3% Above Avg.) 78%

(5% Below Avg.)

3/26/2019 Novartis Mayzent Neurology PDUFA for NDA - First Review Yes 87%


(Same As Avg.)

3/28/2019 Astellas Enfortumab

Vedotin Oncology Phase II EV-201 - Top-Line Results Yes 37%

(2% Above Avg.) 43%

(8% Above Avg.)

3/28/2019 Gilead Filgotinib Autoimmune/ immunology

Phase III FINCH 1 - Top-Line Results/ Phase III FINCH 3 - Top-Line Results

Yes 69%


(13% Above Avg.)

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4/2/2019 Evoke Gimoti Gastroenterology

(non inflammatory bowel disease)

PDUFA for NDA - First Review No 92%

(2% Above Avg.) 91%

(1% Above Avg.)

1No prior LOA change

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Drugs NKTR-181 for Chronic Low Back Pain (CLBP) (NKTR)

Drug Company Partner(s) Indication(s) Date Range Expected Catalyst(s)

NKTR-181 Nektar Therapeutics N/A Chronic Low Back

Pain (CLBP) 05/28/2019 PDUFA for NDA - First Review

Phase Disease Group Drug Class Group/Class Phase Success

Group/Class LOA (PTS)

BMT LOA Opinion

NDA Neurology NME 84.3% 53.0% Above NKTR-181 is a mu-opioid analgesic investigational drug candidate engineered using Nektar's small molecule polymer conjugate technology. The FDA has granted NKTR-181 Fast Track status for the treatment of moderate to severe chronic pain. In July of 2018, the FDA confirmed acceptance of the New Drug Application for NKTR-181 for the treatment of chronic low back pain in adult patients new to opioid therapy. A PDUFA (Prescription Drug User Fee Act) target action date of May 28, 2019, has been assigned by the FDA. Results from the pivotal Phase III SUMMIT-07 trial support the NDA for NKTR-181 and establish its efficacy for the treatment of CLBP. In the SUMMIT-07 trial, opioid-naive patients were enrolled and treated with NKTR-181. Patients’ average pain scores dropped by 65% (from 6.73 to 2.32 points) in the open-label titration period. Those who experienced sustained pain reduction were then randomized to either continue on NKTR-181 or receive placebo during the double-blind 12-week treatment period. The study’s primary endpoint was met with average pain scores at week 12 (compared with randomization baseline) increasing by 1.46 points in the placebo group compared with 0.92 points in the NKTR-181 group (P-Value vs. Placebo = 0.0019). Key secondary endpoints were also achieved in this trial as a statistically significant proportion of patients on NKTR-181 experienced pain reductions greater than 50% compared to placebo (51.1% versus 37.9%; p=0.001). As the FDA continues efforts to combat the opioid epidemic, NKTR-181 holds the potential to be a new treatment option for individuals with CLBP. Although still targeting opioid receptors, NKTR-181’s small molecule–polymer conjugate technology slows entry of the drug to the central nervous system, thus preventing the euphoria often associated with common opioids. Results from the human abuse liability study confirmed that NKTR-181’s abuse potential was similar to placebo and significantly lower than oxycodone’s, thus demonstrating that NKTR-181 could be a safe, non-addictive treatment option. In a pre-NDA meeting with the FDA, the agency confirmed that Nektar has an adequate abuse potential assessment data package, and that together with the safety results, the data appeared to be adequate to warrant a discussion of a less restrictive scheduling than Schedule 2.

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Maribavir for Cytomegalovirus (CMV) Infection (Antiviral) (TAK)


Maribavir Takeda

Pharmaceutical Company Ltd

GlaxoSmithKline Cytomegalovirus (CMV) Infection

(Antiviral)

04/01/2019 - 06/30/2019

Phase III 303 Refractory -

Top-Line Results



BMT LOA Opinion

III Infectious disease NME 70.4% 62.3% Above

Shire is currently developing maribavir, an oral benzimidazole riboside, to treat cytomegalovirus (CMV) infection in transplant recipients, an indication with an unmet need. Originally, maribavir was developed by GlaxoSmithKline and licensed to ViroPharma worldwide, excluding Japan in 2003. ViroPharma later merged with Shire in 2014 and was acquired by Takeda in 2019. The U.S. Food and Drug Administration (FDA) granted maribavir Orphan Drug Designation and Fast Track status for the prevention of CMV infection in February 2006 and February 2007, respectively. A Phase III trial studying the efficacy and safety of maribavir for the prevention of CMV in patients who have undergone allogenic stem cell treatment was initiated in September 2006, and a second Phase III trial assessing maribavir in patients who had undergone liver transplantation with a high-risk of developing CMV disease was initiated in July 2007. However, maribavir suffered a setback as top-line results from the first of the two Phase III trials with patients who had undergone allogenic stem cell transplantation failed to reach its primary (incidence of CMV disease within 6 months post-transplant) and secondary (rate of initiation of anti-CMV treatment within 6 months) efficacy endpoints. Due to these disappointing results, the company suspended the second Phase III trial in February 2009. Since then, the developmental focus of maribavir has switched from the prevention of CMV disease to the treatment of CMV infections. In May 2011, the FDA granted Orphan Drug Designation for the treatment of CMV in at-risk patients. Furthermore, in January 2018, maribavir was granted Breakthrough Therapy Designation by the FDA based on the data from two Phase II studies initiated in June 2012. One of the Phase II studies was evaluating maribavir in 120 patients with CMV infection resistant or refractory to (val) ganciclovir or foscarnet. The study reported positive results where the primary endpoint was met with 67% of patients treated twice daily for 42 weeks (either 400mg or 1200mg) achieving undetectable plasma CMV DNA within six weeks of treatment. Following the trials, the company met with the FDA and received feedback on how to move forward to Phase III. The pivotal Phase III SHP620-303 trial comparing the efficacy of maribavir to investigator-assigned treatment in transplant recipients with CMV infections resistant or refractory to prior CMV treatment initiated in December 2016. In March 2017, another pivotal Phase III study, SHP620-302, was initiated, and is studying maribavir in comparison to valganciclovir for the treatment of CMV infection in stem cell transplant recipients. Shire anticipates top-line data from the Phase III SHP620-303 trial in the second quarter of 2019.

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Dengvaxia for Dengue Fever - Vaccines and Treatments (SNY)


Dengvaxia Sanofi N/A Dengue Fever - Vaccinces and

Treatment 05/01/2019 PDUFA for BLA -

First Review



BMT LOA Opinion

BLA Infectious Disease Vaccine 100.0% 82.0% Below

Dengvaxia is a vaccine for dengue fever, a mosquito-borne viral disease that is most prevalent in Latin American and Asia-Pacific countries. Dengvaxia is a tetravalent vaccine comprised of four monovalent, chimeric live-attenuated vaccines. Each chimeric virus within Dengvaxia is constructed by replacing the pre-membrane and envelope proteins of the yellow fever virus 17D strain backbone with those of a single dengue virus serotype (DEN-1/2/3/4). All four serotypes are included in the vaccine in order to stimulate pan-serotype immunity and reduce the likelihood of antibody-dependent enhancement (ADE) of the disease, a phenomenon in which patients with pre-existing immunity to one serotype develop more severe disease after subsequent infection with another, previously unencountered serotype. In December 2016, Sanofi Pasteur filed a Biologics License Application (BLA) application to the US Food and Drug Administration (FDA). In October 2018, the FDA granted Dengvaxia priority review as it would represent a medical prevention tool against dengue fever, including severe dengue, which is considered an unmet medical need. The FDA set a Prescription Drug User Fee Act action date of May 1, 2019. The BLA includes efficacy, immunogenicity, and safety data from 16 studies, including two Phase III and one Phase IIb efficacy studies (CYD15, CYD14, and CYD23, respectively), three immunogenicity and safety studies in adults aged 18–45 years (CYD47, CYD28, and CYD22), and one study to support manufacturing consistency (CYD17). CYD15 was a randomized, placebo-controlled, observer-blind, multicenter study that evaluated vaccine efficacy, immunogenicity, and safety of Dengvaxia in subjects aged 9–16 years in Puerto Rico and Latin America. Study CYD14 was identical to CYD15 in study design, but enrolled children aged 2–14 years in the Asia-Pacific region. A meta-analysis of the pooled Phase III data demonstrated that Dengvaxia was 65.6% effective against symptomatic dengue infection within the first 25 months of follow-up in children aged >9 years. However, concerningly, there was an increased risk of hospitalization due to severe dengue in children aged <9 years (who are more likely to be dengue-naive) compared to placebo recipients at three year’s post vaccination, leading to the vaccine’s approval in other markets being limited to individuals aged >9 years. An additional analysis of patients with up to six years of follow up data later showed that patients who were seronegative at baseline were also at increased risk of severe dengue infection compared to placebo recipients. While Dengvaxia’s demonstrated efficacy and safety in individuals aged >9 years is expected to be sufficient for it to gain US approval, In March 2019, an FDA Advisory Panel Meeting proposed a narrower label for the vaccine (9–17 years) than that which Sanofi was pursuing (9–45 years), due to insufficient bridging safety data in adults. The panel also raised concerns over the lack of a commercially-available test to identify patients who are seronegative at baseline and therefore should not be vaccinated. Thus

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there is a risk that the FDA will not approve the vaccine until such a test becomes available, which Sanofi hopes will occur in 2020. Visomitin for Dry Eye (Ophthalmology) (Mitotech)


Visomitin Mitotech S.A. Essex Dry Eye (Ophthalmology)

04/01/2019 - 06/30/2019

Phase III - Top-Line Results



BMT LOA Opinion

III Ophthalmology NME 42.4% 30.1% Above Visomitin is a topical ophthalmic formulation of SkQ1, a small molecule which efficiently brings the active antioxidant plastoquinone into mitochondria to prevent damage from reactive oxygen species (ROS). Mitotech is developing Visomitin for the treatment of dry eye syndrome. In December 2011, Visomitin was approved in Russia after a series of clinical trials. In early 2012, Mitotech filed an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) to run a program of similar studies in the US in anticipation to file a New Drug Application (NDA) in 2020. In April 2014, Mitotech initiated a Phase II trial to assess the safety and efficacy of Visomitin in patients with moderate to severe dry eye syndrome in a controlled adverse environment (CAE). The primary endpoints of the study were fluorescein staining and worst dry eye syndrome at day 29. The trial completed patient enrollment in May 2014 with 90 patients. Results of the trial were positive and Visomitin demonstrated superiority over placebo in terms central and total corneal fluorescein staining at week four as well as ocular discomfort scores at week four. In January 2015, Mitotech met with the FDA for an end of Phase II meeting to discuss the development of Visomitin in a Phase III trial. Prior to the initiation of the pivotal trial, Mitotech announced a partnership with Essex Bio-Investment for Visomitin’s Phase III clinical program in dry eye. Subsequently, the Phase III VISTA-1 trial initiated across the US in October 2018, with an expected enrollment of 450 patients with moderate to severe dry eye. The trial will feature three arms: high dose Visomitin, low dose of Visomitin, and a placebo. Primary endpoints include central corneal staining change and grittiness change from baseline to day 57. Top-line results for VISTA-1 are expected in the second quarter of 2019. Mitotech anticipates seeing similar positive results as with the previous Phase II trial and clinical studies in Russia.

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Devices DBLG1 System for Diabetes Mellitus, Type I (Diabeloop)

Device Company Partner(s) Market(s) Date Range Expected Catalyst(s)

DBLG1 System Diabeloop SAS Cellnovo Endocrine 04/01/2019 - 06/30/2019 Product Launch (EU)

The DBLG1 System is an artificial pancreas solution designed to automatically monitor and regulate blood-sugar levels in people with Type I Diabetes (T1D) through the delivery of insulin. The DBLG1 System consists of the Diabeloop handset, a Cellnovo insulin pump and a Dexcom G5 continuous glucose monitor. Every 5 minutes, a glucose measurement is transmitted via Bluetooth technology to the handset. DBLG1 artificial intelligence analyzes data in real time and takes into account the patient’s physiology, history and data entries (meals or exercise) to determine the correct dose of insulin to administer. Results for the DBLG1 System were first released in February 2017 at the Advanced Technologies & Treatments for Diabetes conference (ATTD). In this study, thirty-eight type I diabetes (T1D) patients on insulin pump therapy participated in the randomized crossover study. With patients participating in various situations such as intensive exercises, gastronomic dinners or rest, the DBLG1 System was able to reduce the time in hypoglycemia, to decrease average blood glucose (BG) level and double the percent-time spent in 80-140 mg/dl BG range during the night, compared to a sensor-augmented pump. In June 2018, top-line results from a 68-patient home setting study were presented at the American Diabetes Association's 78th Scientific Sessions. The study found that the DBLG1 closed-loop system was efficient regarding metabolic outcomes. The time spent in the 70-180 mg/dl range was 69.3% compared to 56.6% for the open-loop system CGM. The time in hypoglycemia (<70 mg/dl) was 2.1% for the Diabeloop and 4.1% for the open-loop CGM. In November 2018, Diabeloop announced that it has received the CE marking for the DBLG1 System, and announced that the company anticipates launching the device in Europe in the second quarter of 2019. When launched, this device will significantly improve the quality of life for T1D patients because it can predict glucose levels and engage the insulin pump at an optimal rate, and ultimately prevent hypoglycemia events. This self-learning and highly customizable system can reproduce the insulin-dispensing functions of a pancreas and adapt to the patient’s physiology and lifestyle. One of the mental burdens of T1D is continuously checking blood sugar levels and being self-aware of symptoms of hypoglycemia after manually administering a dose of insulin. The DBLG1 System is a breakthrough that reduces the need for patients to self-administer insulin, and alleviates the worries associated with falling into hypoglycemia.

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FoundationOne for Solid Tumors (RHHBY)

Device Company Partner(s) Market(s) Date Range Expected Catalyst(s)

FoundationOne Roche Holding AG

Chugai, Mirati Oncology 01/01/2019 -

06/30/2019 FoundationOne Japanese Reimbursement Decision

FoundationOne CDx is a next-generation sequencing device used to detect various aberrations across 324 genes in patients with solid tumors. FoundationOne CDx also serves as a broad companion diagnostic used to identify those patients who may benefit from treatment with one of 17 approved targeted therapies used in the treatment of non-small cell lung cancer, melanoma, colorectal cancer, ovarian cancer, or breast cancer. The assay identifies the actionable molecular growth drivers of these cancers, helping oncologists match patients with relevant targeted therapeutic options. Foundation Medicine (acquired by Roche in July 2018) received US Food and Drug Administration (FDA) approval for FoundationOne CDx in November 2017. In March 2016, the Centers for Medicare and Medicaid Services (CMS) issued a final National Coverage Determination (NCD) stating that patients with Stage III and Stage IV, metastatic, recurrent, relapsed, or refractory solid tumors who receive next-generation sequencing testing with FoundationOne CDx would be eligible for coverage. In December 2018, Foundation Medicine received approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for FoundationOne CDx as a comprehensive genomic profiling test for all solid tumors and a broad companion diagnostic for individuals living with advanced cancer. The MHLW is expected to issue a reimbursement coverage decision in the first half of 2019. Following the approval, the reimbursement decision is an important step in improving personalized oncology care in Japan. If positive, this coverage decision for FoundationOne CDx is expected to improve access to MHLW-approved targeted therapies and immunotherapies, as well as clinical trials, for patients with cancer.

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Company Transactions Provided by Strategic Transactions

Acquisition of BTG plc by Boston Scientific On November 20, 2018, Boston Scientific Corp. (BSX) agreed to the terms of a cash offer to acquire BTG plc, a UK maker of minimally invasive products for cancer and vascular conditions and acute care specialty pharmaceuticals, for 840 pence per share ($10.76; a 38% premium to the 10-day pre-announcement trading average), a total equity value of £3.3 billion ($4.2 billion). The transaction, the highest-value medtech M&A deal of 2018, is expected to close in the first half of 2019 and has already received backing from the company's directors and BSX's three largest shareholders: Invesco Asset Management, Novo Holdings, and Woodford Investment Management (which collectively hold 33%). Established in 1981 through the merger of the National Research Development Council and the National Enterprise Board, BTG currently has three main businesses. Interventional medicine, its largest, was established in 2010 when BTG bought UK interventional oncology company Biocompatibles International (for $287 million) to start owning and developing its own products. This unit now offers both oncology and vascular product lines. Over the past six years, BTG itself was an active acquirer, building up the interventional medicine business by scooping up various interventional players, including most recently Novate Medical, maker of the Sentry inferior vena cava filter, for up to $150 million (in September 2018). BTG's specialty pharmaceutical business is made up of acute care antidotes to treat envenomation by certain snakes and overexposure to heart and cancer medications and toxins, with products including DigiFab (digoxin immune Fab (ovine) for digoxin toxicity or overdose; CroFab (crotalidae polyvalent immune Fab (ovine) for North American pit viper envenomation; and Voraxaze (glucarpidase) for methotrexate toxicity. Finally, its licensing business is an income-generating model that receives royalty revenues related to products subject to BTG intellectual property and license agreements. With advantages for both, BSX will notably benefit from BTG’s product revenues (over 90% from the US) while BSX's broad geographic reach can better position BTG to launch products globally. BSX also expects to realize significant cost synergies and to augment its capabilities in cancer and pulmonary embolism. BSX CEO Michael Mahoney said the acquisition will enable the company to scale its capabilities in diagnosing and treating cancer, and projects BTG's interventional medicine business will add more than $400 million of revenue to BSX's peripheral interventions division during 2019. For the six months ended Sept. 30, 2018, BTG recorded sales of $495.7 million, approximately $340 million of which was from product sales--with interventional medicine generating a little over half of that amount (or $172 million: $111 million from oncology/$59 million from vascular) and $168 million from pharmaceuticals--and the rest from licensing revenues. On February 25, 2019, BSX completed a public offering of $4.3 billion aggregate principal amount of its senior notes and intends to use a portion of these net proceeds to help finance the BTG acquisition.

April 2019 / 29


Acquisition of Spark Therapeutics by Roche In late February 2019, Roche announced it signed a definitive agreement to acquire public gene therapy firm Spark Therapeutics for $4.8 billion in cash on a fully diluted basis. The $114.50 per share price represents a 128% premium to the ten-day market average. Roche commenced the tender offer in early March and the period was set to expire on April 3, 2019 but was later extended until May 2, 2019. The acquisition is expected to close in the second quarter of 2019. Post-transaction Spark would operate as an independent entity. Spark was created in October 2013 as a spin-off from Children’s Hospital of Philadelphia. The start-up went public just fifteen months after its founding, netting $172 million. In its short history, Spark has become a commercial-stage company and brought to market the first gene therapy for an inherited disease. Luxturna (voretigene neparvovec) is an adeno-associated virus vector-based gene therapy approved in the US and Europe for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. (Under an early-2018 tie-up, Novartis holds exclusive worldwide rights to the drug outside of the US.) In its pipeline, Spark also has gene therapies for other ophthalmic diseases, in addition to hemophilia, lysosomal storage disorders, and neurodegenerative diseases. Most of the excitement surrounding Spark’s pipeline stems from its gene therapies for hemophilia A, an indication that Roche entered at the end of 2017 with the approval of Hemlibra (emicizumab). Spark’s lead asset in this space is SPK-8011 (expected to commence Phase III trials in 2019), followed by Phase I/II SPK-8016. Prior to the announcement, Roche hadn’t been much of a player in the gene therapy arena, including CAR-T, with the exception of some smaller deals and a relatively larger alliance – worth $1.38bn – with SQZ Biotechnologies in October 2018. Should Roche successfully acquire Spark, the Big Pharma would have a front row seat in the growing gene therapy space, which has had several high-priced biopharma deals just this year. In January, Janssen Pharmaceuticals and MeiraGTx teamed up in a potential $440 million agreement to create gene therapies for inherited retinal diseases, and two months later Biogen penned an $800 million deal to acquire ophthalmic gene therapy developer Nightstar Therapeutics. Gene therapies are getting a lot of attention because they offer the potential for a long-lasting and even permanent effect via a one-time treatment for diseases with few options.

Q2 2019 Large Impact Device/Diagnostics Catalysts

Company Symbol Device Indication Phase Catalyst Title Expected Date Range Link

Abiomed, Inc. ABMD Impella ECP Congestive Heart Failure (CHF) and Cardiomyopathies Preclinical First-In-Human Study to Start Now-04/30/2019 137490

AtriCure, Inc. ATRC AtriClip LAA Exclusion System Atrial Fibrillation/Flutter Approved AtriClip PRO•X Product Launch

(US) Now-04/30/2019 139577

AtriCure, Inc. ATRC EPi-Sense Coagulation Device Atrial Fibrillation/Flutter IDE IDE - CONVERGE 1-Year

Follow-Up Results Now-04/30/2019 125229

AtriCure, Inc. ATRC Synergy Ablation System Atrial Fibrillation/Flutter Approved DEEP Pivotal Trial Enrollment

Completion Now-04/30/2019 108938

AtriCure, Inc. ATRC cryoICE Cryoablation Probes

Dysrhythmia (Arrhythmia) Approved FROST Study - Patient Enrollment Completed Now-04/30/2019 141028

AtriCure, Inc. ATRC cryoICE Cryoablation Probes

Atrial Fibrillation/Flutter IDE IDE - ICE-AFIB Study to Start Now-04/30/2019 146961

Biosensors International Group Ltd.

BioFreedom Coronary Artery Disease IDE Asian STEMI Registry to Start Now-04/30/2019 144466

Biosensors International Group Ltd.

BioFreedom Coronary Artery Disease IDE Biofreedom Observation Registry to Start Now-04/30/2019 144604

CryoLife, Inc. CRY E-liac Aortic Aneurysm Development Outside U.S.

PLIANT Study - Enrollment Completion Now-04/30/2019 130403

CryoLife, Inc. CRY PerClot Hemostasis IDE Patient Enrollment Completion Now-04/30/2019 144710

Decision Diagnostics Corp. DECN GenChoice! Diabetes Mellitus, Type I Development GenChoice International

Launch Now-04/30/2019 141460

Decision Diagnostics Corp. DECN GenChoice! Diabetes Mellitus, Type II Development GenChoice International

Launch Now-04/30/2019 141479

Decision Diagnostics Corp. DECN

GenPrecis! Precision Monitoring System

Diabetes Mellitus, Type I Development International Product Launch Now-04/30/2019 136955

Decision Diagnostics Corp. DECN

GenPrecis! Precision Monitoring System

Diabetes Mellitus, Type II Development International Product Launch Now-04/30/2019 136969

Decision Diagnostics Corp. DECN GenChoice! Diabetes Mellitus, Type II Development 510(k) Approval Decision Now-04/30/2019 139956

DexCom, Inc. DXCM

DexCom G6 Mobile Continuous Glucose Monitoring (CGM) System

Diabetes Mellitus, Type I Approved DexCom 6 Second Generation Transmitter Launch Now-04/30/2019 143231

Edwards Lifesciences Corp. EW CardioValve Cardiac Valve Surgery Development AHEAD-US Study to Start Now-04/30/2019 118067

Edwards Lifesciences Corp. EW FORMA Cardiac Valve Surgery IDE Enhanced System Trial to Start Now-04/30/2019 138738

Edwards Lifesciences Corp. EW INSPIRIS RESILIA

Heart Valve Cardiac Valve Surgery Approved INDURE Study to Start Now-04/30/2019 146006

Edwards Lifesciences Corp. EW CardioValve Cardiac Valve Surgery Development Cardiovalve Feasibility Study

to Start Now-04/30/2019 146526

Edwards Lifesciences Corp. EW Cardioband Cardiac Valve Surgery IDE TriBAND Post Market Study to

Start Now-04/30/2019 147682

Insulet Corporation PODD Omnipod Horizon System Diabetes Mellitus, Type I IDE Pivotal Trials to Start Now-04/30/2019 127688

Insulet Corporation PODD Omnipod Horizon System Diabetes Mellitus, Type I IDE IDE3 Trial Completion Now-04/30/2019 141253

Lemaitre Vascular, Inc. LMAT XenoSure Biologic Patch Other Cardiovascular Surgery Approved Approval (Australia) Now-04/30/2019 124486

Lemaitre Vascular, Inc. LMAT XenoSure Biologic Patch Other Cardiovascular Surgery Approved XenoSure China Study Patient

Enrollment Completion Now-04/30/2019 145641

Merit Medical Systems Inc. MMSI WRAPSODY Stent

Graft Renal Disease / Renal Failure Development Phase I Feasibility Study to Start Now-04/30/2019 145092

MRI Interventions, Inc. MRIC ClearAblate System Pancreatic Cancer Development Product Launch (U.S.) Now-04/30/2019 132857

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