panmetics presentation
TRANSCRIPT
ANTYRAThe Next Generation of
BiologicalsNon-Confidential 1
PanMetics
For Additional Information:Neil Goldstein, Ph.D.
Chief Scientific [email protected]
Website: www.panmetics.com
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BiologicalsNon-Confidential 2
• PanMetics is a newly formed biotechnology company developing
a powerful array of novel, targeted therapeutic leads against
cancers with high mortality rates and poor therapeutic options
such as Pancreatic Cancer.
• PanMetics has an exclusive license to develop a novel scaffold,
PanM-429, as a drug lead and as a vehicle for delivery of
therapeutic payloads.
Strategic Goals
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Company Overview
•Cancers such as Pancreatic are unmet medical needs with poor survival rates.
•IGF-1R is a validated target for Pancreatic and other cancers.
•PanM-429 is Mini-protein which acts as an IGF-1R antagonist and inhibits the growth of established human pancreatic cancer tumors in animal models.
•PanM-429 is also a “core scaffold” used to generate other therapeutic drug leads including Bi-specifics, which inhibit multiple targets, and as a delivery vehicle for payloads such as small molecule drugs, siRNA and other biological therapeutics (Cell Penetrating Peptides [CPPs]).
•With a $10M tranched investment, PanMetics will complete Phase 1 clinical trials for at least 2 drug leads over a 3 year timeframe.
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Cost Basis & Rationale
•PanMetics is looking for an initial investment $10M for 3 years to complete the following:
• Complete a Phase 1 trial for PamM-429 alone or in combination with approved drugs• Generate and characterized PanM-429 derivatives (Bi-specifics and CPPs)• Identify 1-2 PanM-429 derivatives for clinical development• Complete 2nd Phase I trial for a PanM-429 derivative with an approved drug
•The investment can be paid in tranches and additional funding be based on •Go/No Go decisions
•PanMetics believes that the drug leads generated from these studies are valuable commodities and will generate strong interest from pharmaceutical companies who are constantly looking for novel drug leads and ways to deliver their own products in a highly specific manner.
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Exit Strategy
•PanMetics believes that its focused drug development strategy will be amply rewarded through investment from both the financial community and pharmaceutical licensing deals.
•Expected exit strategies include:
• Buyout by a large pharma or biotech company• M&A with a biotech with synergistic technologies (e.g., siRNA delivery)• IPO/Reverse Merger under the appropriate conditions
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The Medical Opportunity
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• The Insulin-like growth factor (IGF) signaling axis is a focus for developing targeted drugs for treatment of indications including cancer.
• Preclinical research and clinical investigations have implicated the IGF type 1 receptor (IGF-1R) and its ligands IGF-I and IGF-II in the development and progression of a number of human cancers including pancreatic, ovarian, colorectal, prostate and hematopoietic tumors.
• Targeting the IGF-1R receptor represents an important new approach in cancer therapy. PanMetics intends to use its PanM-429 core scaffold to generate drug leads which will inhibit the IGF-1R cancer axis.
The Target: IGF-1R
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PanM-429 is a Novel IGF-1R Antagonist
• PanM-429 is a member of a class of novel, highly specific bioactive Mini-Proteins called Peptimetics which act as agonists or antagonists and can be generated for any disease indication.
• PanM-429 is an IGF-1R antagonist with high affinity and selectivity
• Because of its high specificity and biological activity, PanM-429 can be used as a therapeutic in its own right as well as in combination with approved drugs for Pancreatic and other cancers.
• In addition to its value as a drug lead, PanM-429 can also be used to generate novel derivatives such as Bi-specifics and CPPs to deliver payloads such as small molecule and biological drugs.
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PanMetics’ R&D Goals
•Fully characterize PanM-429 as a therapeutic lead in animal models alone and in combination with approved drugs such as gemcitabine (Gemzar) and Tacerva
•Generate novel derivatives of PanM-429 including Bi-specific and CPPs.
• Bi-specifics which inhibit multiple targets such as IGF-1R/EGFR & IGF-1R/FGFR1
• Cell Penetrating Peptides to deliver a vast array of therapeutics including small molecules and biologicals such as, but not limited to, siRNA and antisense oligonucleotides
•Characterize these entities in vitro and in the appropriate animal models
•Identify 2 drug leads for clinical development (manufacturing, PK/ADMET)
•Complete two Phase 1 clinical trials by the end of Year 3 for these 2 drug leads
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PanM-429: A Drug Lead for Treating Pancreatic Cancer
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PanM-429: A Mini-Protein which Inhibits the IGF-1R
• Affinity = ~300nM = ligand (IGF-1)
• In vitro properties– Blocks IGF-I induced phosphorylation of IGF-IR– Inhibits IGF-IR/IR hybrid receptors– Specificity for IGF-1R vs IR– Inhibits IGF-I induced proliferation in cell assays
• Inhibits tumor growth in vivo• Predicted to weakly immunogenic (by analysis)• Indication: Pancreatic Cancer (& other cancers)
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PanM-429 inhibits the Growth of Pancreatic Cancer Cells in Animals
Injections (ROI = SC): 4/week x 3
* Control Vehicle 429-300 429-50 G12-300
**
Established Tumors >100mm3
Control
Tumor Growth Tumor Weight
Control Vehicle 5-FU 429 300 429 50
Tu
mo
r W
eig
ht (g
)
0.0
0.5
1.0
1.5
2.0
2.5
DAYS
0 5 10 15 20 25 30
Tu
mo
r V
olu
me
(m
m3)
0
500
1000
1500
2000
2500
3000
ControlVehicleANT-429 300ANT-429 50ANT-G12 300
429 300
429 50
PanM-429 inhibits the growth of established MiaPaCa tumors growing in nude mice
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PanM-429 as a Scaffold for Novel Therapeutic Derivatives
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Generation of Bi-specifics with PanM-429
5mer Linker429 Sequence Target Binding Sequence
Pan vs. 2nd Target (EGFR, FRGR1, etc.)
5mer Linker429 Sequence 15mer Random Sequence
• Screen in ELISA vs IGF-1R & 2nd Target
• Move to secretory vector for transfections studies
• Synthesize HP and test in cell-based assays
• Determine efficacy in animal models
• Library can be used for any 2ary target
Screen
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PanM-429 as Bi-specific Cell Penetrating Peptide
‣PanM-429 internalizes into various cell types including Pancreatic
Cancer
‣PanM-429 can be used to deliver therapeutic payloads such as
siRNA, antisense, biologicals and small molecules.
‣PanM-429 conjugated to a payload can be considered “Bi-specific”
because both the Peptimetic and the payload have biological
activity.
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PanM-429 Conjugated to a Payload
5’ or 3’ linkage to PanM-429Utilize well established chemistry
PanM-429 is cationic and help shield net negative charge on siRNA and other biologicals
• Enzymatic• Chemical• Endosomal
• siRNA (single or ds)• Antisense•Small Molecule Drugs•Biologicals
Chemical Linker [Stable or Cleavable ]PanM-429
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The Investment Opportunity
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Year 1 Year 2 Year 3
• Full Characterization of PanM-429
• Develop PanM-429 Bi-specifics (with EGFR and FGFR1)
•Develop PanM-429 delivery conjugates with gemcitabine & cis-platin
•Develop PanM-429 delivery conjugates with Kras antisense
•Develop PanM-429 directed liposomes with Kras siRNA
•Animal studies with PanM-429
•Begin Manufacturing Lead 1
•SBIRs to supplement investor funding
• Complete Phase 1 for Lead 1 and full data package (Q3)
• Lead 2 IND
•Complete Phase 1 for Lead 2 and full data package (Q4)
• Business Development : partnering deal(s) for drug leads 1&2 and/or other PanM-429 derivatives in pre-clinical
•Pre-clinical development of additional leads
• Continue animal studies with PanM-429 and derivatives
• PK and ADMET Lead 1
•Lead 1 IND (Q4)
•Begin Manufacturing Lead 2
•Develop a full pre-clinical package for partnering and licensing discussions with pharma/biotech companies
Business Development opportunities
PanMetics’ Business Strategy
$10M Seed Investment: Tranched based on milestones
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Risks:Unanticipated technical issues/delays Slower than forecasted development timelines Slower than expected BD licensing deals
Potential: Faster than expected development timelines for PanM-429 &
derivatives Rapid characterization of Lead 1: e.g., PanM-429 Rapid development of Lead 2: e.g., PanM-429 –gemcitabine
conjugate Identification of additional leads with therapeutic potentialEarly licensing/partnering deal brings non-dilutional funds
and clinical expertise into company
Business Development: Risks & Potential
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Competitive Landscape for IGF-1R Cancer Drugs
Drug Company Clinical Trial Cancer(s) Target(s) Type Continuing?
XL228 Exelixis Two Phase 1s
(updated 2011)
CML IGF-1R, src,
BCR-ABL
Small
molecule
Both Studies
terminated?
OSI 906 OSI Phase I ACC IGFR, IR Small
Molecule
ongoing
AMG-479 Amgen/Takeda Phase I/II
Phase III (+
Gemzar)
Multiple solid
tumors
Pancreatic
IGFR MAB ongoing
R 1507 Roche Phase I Sarcoma IGFR MAB Stopped
development
Figitumumab Pfizer Phase I Multiple solid
tumors
IGFR MAB Stopped
development
BMS-754807 BMS Phase I Multiple solid
tumors
IGFR, IR Small
molecule
ongoing
MK0646 Merck Phase I +/-
Gemzar+/-
erlotinib
Pancreatic IGFR MAB ongoing
Cixutumumab
(IMC A12)
Lilly (from
ImClone)
Phase I +
temsirolimus
Multiple solid
tumors
IGFR MAB ongoing
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PanMetics Has the Therapeutic Edge
•PanMedics believes that PanM-429 and its derivatives will be effective alone and in combination with any approved drugs to increase the therapeutic index for IGF-1R expressing cancers such as Pancreatic.
•PanM-429 is cheaper to produce than monoclonal antibodies and vaccines (=decreased COGs).
•PanM-429 Bi-specifics will inhibit multiple targets thereby increasing selectivity, potency and therapeutic index.
•PanM-429 conjugated to small molecules and biologicals will be able to target internal targets inaccessible to monoclonal antibodies
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Assets and Capabilities
PanM-429 has already shown efficacy in vivo
PanM-429 Bi-specific and delivery modules are available &
tested in vitro
Hugh pipeline potential
Proven scientific approach
Strong IP base, proprietary reagents and know-how poised
to move 2 Leads through clinical trials within 3 years
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BiologicalsNon-Confidential 23
PanMetics
For Additional Information:Neil Goldstein, Ph.D.
Chief Scientific [email protected]
Website: www.panmetics.com
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BiologicalsNon-Confidential 24
PanMetics: 5 Year Plan•Primary Opportunities: Years 1-3
• Move 2 drug leads through Phase I Clinical Trials
•Secondary Opportunities: Years 1-3• Continued pre-clinical development of additional PanM-429 derivatives for
partnering/licensing and downstream INDs
•Round B financing = $7.5M: Years 4-5• Move additional drug leads into Phase I Clinical Trials (from PanM-429 derivative
pool)• Identify new targets for Pancreatic Cancer and generate novel Peptimetic
antagonists for pre-clinical development• Develop antagonists for additional high mortality/untreatable cancers
Phase II Clinical study(s): With additional financing for Years 4-5 ($15-20M) PanMetics would be able to move one of its drug leads into a Phase II trial. It is anticipated that this would be done in partnership with a large pharmaceutical/biotech company