Join Children’s National Hospital for an enlightening symposium that shines a spotlight on the remarkable journey from academia to successful FDA and European Commission approvals of the drug AGAMREE® for the treatment of DMD in children. This symposium is dedicated to recognizing the importance of academic entrepreneurship in advancing breakthroughs for rare diseases and how collaboration with small businesses and the industry played a crucial role in the process.

 

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