BioEntrepreneurship: Exploiting Early Staged Discoveries


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Strong patent protection is essential for a start-up biotechnology company and can be a valuable company asset. However, it is also expensive, with costs ranging from tens to hundreds of thousands of dollars over time. This session will focus on how to get the most out of your patent dollars.

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BioEntrepreneurship: Exploiting Early Staged Discoveries

  1. 1. Exploiting Early Staged Discoveries BioEntrepreneurship Konrad Powell-Jones Manager, Medical/Biotechnology December 4, 2006
  2. 2. Innovations at University of Toronto(IUT) Established in 1980 (Innovations Foundation) • Goal: maximize the impact of > $2 million spent every day on research at UT and the affiliated teaching hospitals (such as SWCHSC) • IUT services now focused on the UT community • We help researchers and businesses capitalize on unique opportunities
  3. 3. What is Innovation? The process of making improvements by introducing something new (Oxford Dictionary) The successful exploitation of new ideas (Dept. of Trade and Industry, UK)
  4. 4. How do you exploit your early stage discovery? Protect it (patents, copyright, trade secrets) then Further developmental research then Out-License it or “Ever feel you’re on the verge of an Create a Start-Up incredible breakthrough?” company
  5. 5. Technology Transfer Business In Canada Develop 60% have TT offices Financing University Protect Discovery
  6. 6. The Process in Summary Preliminary Prior Discovery/Observation Art Analysis Preparation of Invention Disclosure Preparation of brief description and preliminary claims Review by I.P. Committee Review Proposed Disclosure and Prior Art with Patent Counsel Decision to YES Prepare Patent YES Assessment Application Patentability NO NO Additional YES Studies Prepare Application Patent Assess Inventorship Counsel NO Continuations Submission of Provisional Application Divisionals etc. File
  7. 7. What are the Commercialization Options? License out to existing company Start a new company
  8. 8. But what will you encounter… ……in the real world!!!!
  9. 9. The Big Picture Unmet Medical Aging Needs Population Biomedical Discoveries Knowledge Translation Innovation Stagnation Payers Perspectives Regulatory R&D Perspectives Productivity Escalating R&D Costs
  10. 10. Industry Productivity vs. Investment “The Challenge” Total R&D Investment ($Billions) $35 $30 $25 $20 $15 $10 # NCEs $5 60 40 20 $0 0 2001 2003 2002 1984 1970 1971 1972 1973 1975 1976 1977 1978 1982 1985 1986 1987 1988 1989 1992 1994 1995 1997 1999 2000 1974 1979 1980 1981 1983 1990 1991 1993 1996 1998 Source: 2004 PhRMA Annual Survey, 2003/2004 PAREXEL’S Pharmaceutical Industry Sourcebook
  11. 11. Development Process: Bottleneck to Delivery of New Products Compound Success Compound Success Years Rates by Stage Rates by Stage 0 0 5,000–10,000 2 2 Discovery 5,000–10,000 Preclinical Testing Screened (2–10 Years) Screened Laboratory and 4 4 Animal Testing 250 6 6 Enter Preclinical Phase I 20–80 Healthy Volunteers Used to Testing 8 8 Determine Safety and Dosage Phase II 100–300 Patient Volunteers 10 Used to Look for Efficacy 10 Phase III and Side Effects 5 1,000–5,000 Patient Volunteers 12 Used to Monitor Adverse Enter Clinical 12 Reactions to Long-Term Use Testing 14 FDA Review Approval 14 Additional Post- Marketing Testing 16 1 16 Approved by the FDA Net Cost: $802 Million Invested Over 15 Years Source: PhRMA Pharmaceutical Industry Profile 2003, Chapter 1: Increased Length and Complexity of the Research and Development Process. And DiMasi, JA, Hansen, RW, Grabowski, HG. “The Price of Innovation: new estimates of drug development costs.” J of Health Economics. 2003:22:151-185.
  12. 12. Patients and Physicians Waiting for Treatments 8% of compounds entering Phase 1 will make it to market, down from 14 % fifteen years ago Cost of development are escalating Failures due to lack of safety and/or efficacy Inability to predict failures
  13. 13. News Flash: Lipid Drug Study Halted Pfizer’s CETP inhibitor which increases HDLs causes serious adverse events. Pfizer stops 15,000 patient Torcetrapid study, withdraws drug!! 81 patients taking Torcetrapid and Lipitor died cf. 51 taking Lipitor alone Torcetrapid patients also showed an increase in angina, CHF and clogged arteries.
  14. 14. Improving Development Sciences New Biomarkers and Surrogate Endpoints Safety biomarkers ! Pharmacogenetics Efficacy Surrogate biomarkers ! New Imaging techniques endpoints X ! Target therapies Patients ! Responders/non responders Predictive New imaging disease techniques ! Markers of drug metabolisms models ! Predictive evaluation of safety ! Effective translation to clinical practice Personalized Medicine
  15. 15. Improving Development Sciences Predictive Animal Models Advancing Innovative Clinical Trials : Learning trials versus empirical trials Improving Measurement of Patient Responses : Variation in individual response Correlation with biomarkers Measuring Patient Preferences Identify the overall benefits of therapies
  16. 16. “Mind The GAP” …how do we narrow it?
  17. 17. Hurdles to Commercialization Invention published prior to patent filing Prior art Un-validated discovery Market too small Competition too advanced Market not ready for invention
  18. 18. Possible Solutions Academic Partnerships and Collaborations Government funding (CIHR and NSERC) Industrial Partnerships and Collaborations
  19. 19. Useful Documents UofT Confidential Disclosure Form Assignment Agreement Technology Owner’s Agreement Confidentiality Agreement Material Transfer Agreement Inter-institutional Agreement Service Agreement Research Contract License Agreement
  20. 20. Case Study Professor Min Zhuo 4 gene targets (pain and fear memory) In vitro and in vivo data Patent and literature searches R&D strategy Commercialization strategy Protect intellectual property Pursue commercialization
  21. 21. Collaborations in place Mouse models Medicinal chemistry sessions SMART HTS Facility CIHR POP1 Grant Toxicology studies
  22. 22. NeoBrain Pharma Profile Focused on small molecule drug discovery for treating diseases of the cns. Founded by the Innovations at the University of Toronto and Professor Min Zhuo (Physiology UofT).
  23. 23. 3 Areas of Indication acute persistent, neuropathic and chronic inflammatory muscle pain. delaying opioid tolerance fear memory and anxiety
  24. 24. Drug Discovery Programs lead program focused on the inhibition of a specific neuronal adenylyl cyclase (nAC) isoform. NB001 has been identified as one such non-competitive nAC inhibitor. discovery of inhibitors of a specific protein kinase, which when combined with opioids reduces analgesic tolerance to chronic treatment. drug discovery program focused on treating anxiety conditions associated with contextual and auditory fear memory by the selective inhibition of specific glutamate receptor subunits.
  25. 25. Proof-of-Concept Data Models Mouse models of neuropathic pain Knockout mouse models Electrophysiological data Antisense data Small molecule data Behavioural data Preliminary toxicology data
  26. 26. Example of neuropathic pain AC inhibition (i.p.) in neuropathic pain Vehicle 120 NB001 0.1mg/kg NB001 1.1mg/kg 100 80 60 * 40 (% Response) Mechanical allod 20 0
  27. 27. Effective for neuropathic pain when applied orally in rat! NB001 orally at 1 mg/kg/3ml 120 100 80 60 40 20 0 Mechanical allodynia (% res Before 45min 2hr 4hr 6hr Time
  28. 28. Comparison with other pain medicine Cumulative effect of gabapentin on withdrawal responses on day 7 after CPN ligation (n=4 C57 mice; vonFrey filamnet 1.65) 100 80 60 40 20 Number of responses in 5 trials in % 0 D ay 7 30 10 1 30 m ho im t tm n ot ua tr gf am ial n la te af0 3f0 3 /krg tem tem rg rg m m /k /k g g
  29. 29. Intellectual Property -Adenylyl Cyclase program: US and Chinese patent applications filed on the specific adenylyl cyclase target and the class of inhibitory compounds -Protein Kinase program: A US patent application filed. -Fear memory/Anxiety Program: A US patent application filed
  30. 30. Financial Requirements Over the next three years, NBPI plans to complete IND-enabling preclinical studies for NB001 for pain, to advance another drug to preclinical development for anxiety and to identify a lead compound for a third indication. NBPI requires financing of US$5 million to support operations through 2009. Financing will be used to advance the drug discovery programs, execute licenses to its intellectual property, build its management team, pursue business development efforts, and complete a subsequent round of financing to allow entry into clinical trials.
  31. 31. Ongoing R&D activities $150,000 awarded from CIHR Proof-of- concept grant Initial PK and toxicity tests for leading compound NB001
  32. 32. Remember If you have doubts seek advice Public disclosure of research results is a fundamental act of scholarship The choice is yours, but publicly disclosing before you have given consideration to patenting and commercialization may, in the long run, remove an option you wish you had kept
  33. 33. Contact Information Konrad Powell-Jones Phone: 416 - 978 - 5730 Email: Patent, Publish, Prosper!