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1 從國際觀點論台灣新藥創新研發策略及產學合作機制 : 以 RSV Vaccine 為例顧曼芹博士 M. Sherry Ku, Ph.D., R.Pharm Kus_98@yahoo.com TWi Biotechnology Inc. 生醫創新研發技術商化論壇 _ 暨 _ 第九屆國家新創獎說明會議 April 19, 2012

2 Presentation Outline Introduction Current US New Drug Trend Open Innovation Model Unmet Medical Needs End-Game Based Project Selection Process High Barrier RSV Vaccine NHRI-Twi Biotech RSV Strategy Q&A 2

3 EvaluatePharma World Preview 2016 Prediction Slower Worldwide Prescription Drug Sales Total $785bn in 2016; 2.9% (CAGR) over ; Over $267bn of Sales at Risk from Patent Expirations ; Slower Global Pharmaceutical R&D Growth 2.3% CAGR to $145bn by % CAGR, 2009 down 0,9% to 124bn Biotech Products Fail to Overtake Small Molecule within Top 100 Drugs by 2016 partly due to Biosimilar entries 3

4 2011 US FDA in Review: A turning point from conservatism on Drug Safety to patient needs??? 1. CBER integrated into CDER. 2. Estimated 2010 based on 9 approvals by August 4

5 2011 US FDA 35 NME Approval Mostly from Specialty Pharma on Rare Diseases 13 from Large Pharma, 23 from smaller firms 10 orphan Drugs (paitent population < 200,000) 16 Priority Review (Review in 6 Months) 13 Fast Track (Rolling review and Frequent FDA consultation) 3 Accelerated Approval (by pass clinical end point (survival) but surrogate (shrink tumor)) 2 with companion diagnostic kits Xalkari ALK NSCLC (Pfizer/Abbott) Zelboraf Braf Melanoma (Genetech) FDA Draft Guidance, In Vitro Companion Diagnostic Devices, issued on July 14,

6 2011 US FDA 35 NME Approval Mostly Small Molecules but No nanoparticles 27 Small molecules, 7 biologicals, 1 polypeptide 7 Biologicals including 1 vaccines and 1 diagnostic 7 Cancer drugs (3 Kinase, 2 Mab, 2 NCE) 3 antiviral 2 antibiotics 1 head lice 2 imaging agent, 1 cosmetic agent 2 DPI 1 prodrug Drug delivery 6

7 Why even the most efficacious therapy do not have 100% response rate Ref: Spear et al Clinical applications of pharmacogenetics. Trends in Mol. Med. 7; ,2001 4/9/2012 M.Sherry Ku 生醫創新研發技術商化論壇 7

8 Open Innonvation Model

9 Product Life Cycle Curve Determination of Net Present Value Time to market Technology available Technical expertise Proximity Quick reaction Proven quality system High capacity Low cost Low cost 12/16/2010 學界研發策略及與產業合作模式 9 9

10 Product-related Deals by Stage of Development Source: MedTRACK, pulled 2/5,

11 Phase 2 Attrition due to Lack of Efficacy Create Opportunity for In-Licensing of Phase 2 POC Compounds 4/9/2012 4/19/2012 M.Sherry Ku Ku Opportunity 生醫創新研發技術商化論壇 in Specialty Pharma 11 11

12 Big Pharma s Collaborative Open Source: Nature Medicine may 2009 Innovation Model 12 12

13 Principal Investigators in US IND are 41% from foreign countries in 2007 Murray M. Lumpkin, FDA Deputy Commissioner of international Programs Within the next 3 years, upto 65% of clinical studies will be conducted outside the US. 35% remains in US. 13

14 Project Selection based on Unmet Medical Needs

15 Cardiovascular disease has been treated successfully and few options remain wanted. Unmet needs No interest 1. Heart Failure drugs 1. Fibrates 2.Atrial Fibrillation 2. PPAR 3. Anti-coagulants 3.ACE inhibitors 4. Anti-platelet agents 4. B-blockers 5.Adrenergic agents 6. Heparins 7.Treatment of acute Myocardial Infarction 8.Treatment of restenosis 15

16 Why Diabetic Mellitus has many treatment options but remains an area of interest??? Merck is interested in: 1. Liver targeting insulin 2.Non injectable insulin 3.Best in class GLP-1 4.Non-PPAR insulin sensitizer 5.Glucose dependent insulin secretagogue (GDIS) 6.B-cell protection 7.Combine c/metforminin Merck has no interest in: 1. B-cell transplant 2. Non-GDIS 3. PPAR 4.Any lead with unknown molecular target 5. Neutraceutical TWi AC-201 meet 4 criteria 16 16

17 Project/Target Selection Process

18 Project Selection Due Diligence End Game based Marketable Approvable Clinical Study Feasible Animal Model based on Medical Practice Market protection by IP or Trade secret or Contracts Move toward Individualized Medicine for a subset of population 18

19 End-Game Project Selection Process Project Selection Clinical Feasibility Animal Model Design In-Vitro Screening Lead Optimization Tech Transfer Medical Needs Established Market Size New Market Building (Herceptin was an orphan Drug) Patient Availability Study Duration Study Size Clinical End Points Clinical Safety Risk Translatable to Human Biomarker Available Dosing Regimen Milligram Potency Toxicity Evaluable in Animals Organ (target vs Vital) Distribution Fast Translatable Nanomolar Potency Clinical End Points Clinical Safety Risk Oral Bioavailability Metabolic Stability Biological half life Dosing Frequency Dose- Formulation Small Molecules Proteins Vaccines 19

20 RSV Vaccine Story

(Microsoft PowerPoint - ku \245\315\302\345\254\343\265o\262\243\276\307\260\323\244\306 RSV example.ppt [\254\333\256e\274\322\246\241])

$(Microsoft PowerPoint - ku \245\315\302\345\254\343\265o\262\243\276\307\260\323\244\306 RSV example.ppt [\254\333\256e\274\322\246\241])$ 從國際觀點論台灣新藥創新研發策略及產學合作機制 : 以 RSV Vaccine 為例顧曼芹博士 M. Sherry Ku, Ph.D., R.Pharm Kus_98@yahoo.com TWi Biotechnology Inc. 生醫創新研發技術商化論壇 _ 暨 _ 第九屆國家新創獎說明會議 April 19, 2012 Presentation Outline Introduction Current