Agenda
3:00 – 3:25 PM - Registration and Networking 3:25 – 3:30 PM – Welcome remarks by Dr. Mark Batshaw 3:30-3:45 PM - Overview of the Vamorolone drug development program by Dr. Eric Hoffman 3:45-4:25 PM. The regulatory path of Vamorolone: Insights and innovations from parallel FDA and EMA approvals. Panel Chair: Daniela Drago Panelists: Jesse Damsker, Eric Hoffman, John McCall, Kanneboyina Nagaraju, John van den Anker [ReveraGen], Paula Clemens (University of Pittsburgh), Lauren Morgenroth (CEO TRiNDS LLC) Topics: - Role of pre-clinical efficacy studies - Pediatric first-in-patient studies (4 to <7 years); choice of stage of disease - Clinical trial design (dose ranging n=48; double-blind placebo and prednisone-controlled n=121) - Effective and rapid pivot during COVID to remote assessments - Choice of novel primary outcome (time to stand from supine) - Effective leveraging of International Academic Clinical Trial Consortium (CINRG) - Discordant discussions with the FDA and EMA - EMA PIP - enabling a broad age label at initial approval (FDA 2 years and older) - Joint FDA-EMA inspection of Sponsor - Contrasts of the FDA and EMA labels for Vamorolone - Hand-off to marketing partner at NDA (Santhera) 4:25-4:35 PM Coffee break 4:35-5:20 PM. The venture philanthropy business model: Shared risk, shared benefit, and giving back to the non-profit sector. Panel Chair: Sharon Hesterlee, Muscular Dystrophy Association Panelists: Eric Hoffman, Jesse Damsker, John McCall [ReveraGen], Paula Clemens (University of Pittsburgh) Topics: - Definition of venture philanthropy model; MDA's experience - ReveraGen venture philanthropy model: where the $75M of non-dilutive funding came from - The importance of 'de-risking' as a component of the model - The importance of NIH NCATS TRND in early-stage de-risking - The importance of federal sources of support (NIH, DoD, EU) - Paying forward to the international non-profit research community (2/3rd of ReveraGen proceeds to non-profits) - While this clearly worked, can it be done again? 5:20 – 5:50 PM Fireside Chat | Dr. Batshaw + Dr. Hoffman
5:50 -7:30 PM | Networking reception |