Real Time Coverage @BIOConvention #BIO2019: Chat with @FDA Commissioner, & Challenges in Biotech & Gene Therapy June 4 Philadelphia
Reporter: Stephen J. Williams, PhD @StephenJWillia2
Fireside Chat with Dr. Norman Sharpless, Acting Commissioner, US Food and Drug Administration
109AB, Level 100
- taking patient concerns and voices from anecdotal to data driven system
- talked about patient accrual hearing patient voice not only in ease of access but reporting toxicities
- at FDA he wants to remove barriers to trial access and accrual; also talk earlier to co’s on how they should conduct a trial
Digital tech
- software as medical device
- regulatory path is mixed like next gen sequencing
- wearables are concern for FDA (they need to recruit scientists who know this tech
Opioids
- must address the crisis but in a way that does not harm cancer pain patients
- smaller pain packs “blister packs” would be good idea
Clinical trial modernization
- for Alzheimers disease problem is science
- for diabetes problem is regulatory
- different diseases calls for different trial design
- have regulatory problems with rare diseases as can’t form control or placebo group, inhumane. for example ras tumors trials for MEK inhibitors were narrowly focused on certain ras mutants
Realizing the Promise of Gene Therapies for Patients Around the World
103ABC, Level 100
How Early is Too Early? Valuing and De-Risking Preclinical Opportunities
109AB, Level 100
- global network of liaisons who are a dedicated team to research potential global startup partners or investments. Pfizer has a separate team to evaluate academic laboratories. In Most cases Pfizer does not initiate contact. It is important to initiate the first discussion with them in order to get noticed. Could be just a short chat or discussion on what their needs are for their portfolio.
Question: How early is too early?
Luc Marengere, TVM: His company has early stage focus, on 1st in class molecules. The sweet spot for their investment is a candidate selected compound, which should be 12-18 months from IND. They will want to bring to phase II in less than 4 years for $15-17 million. Their development model is bad for academic labs. During this process free to talk to other partners.
Dr. Chaudhary, Biogen: Never too early to initiate a conversation and sometimes that conversation has lasted 3+ years before a decision. They like build to buy models, will do convertible note deals, candidate compound selection should be entering in GLP/Tox phase (sweet spot)
Merck: have MRL Venture Fund for pre series A funding. Also reiterated it is never too early to have that initial discussion. It will not put you in a throw away bin. They will have suggestions and never like to throw out good ideas.
Michael Hostetler: Set expectations carefully ; data should be validated by a CRO. If have a platform, they will look at the team first to see if strong then will look at the platform to see how robust it is.
All noted that you should be completely honest at this phase. Do not overstate your results or data or overhype your compound(s). Show them everything and don’t have a bias toward compounds you think are the best in your portfolio. Sometimes the least developed are the ones they are interested in. Also one firm may reject you however you may fit in others portfolios better so have a broad range of conversations with multiple players.
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