Developing Meaningful Endpoints for CNS-Targeting Therapies

Developing Meaningful Endpoints for CNS-Targeting Therapies

In response to requests from drug development professionals to help identify CNS endpoints appropriate for the regulatory review process, and to accelerate the development of CNS-targeting DM therapies, IDMC-11 and the Myotonic Dystrophy Foundation will jointly host a multi-panel discussion to inform the development of patient-centric CNS endpoints for DM therapies. The session, entitled Developing Meaningful Endpoints for CNS-Targeting Therapies, will begin with a presentation on outcome measure development to help the lay audience understand the importance and role of endpoints in drug development and approval, and frame the goals for the session. Panel presentations will then be followed by moderated audience discussion and participation from U.S. Food and Drug Administration (FDA) professionals.

The meeting format will mirror the very successful Patient-Focused Drug Development meeting hosted by MDF with the FDA in September 2016. Panels will be seated with people affected by DM, industry professionals and academic investigators. The meeting will be moderated by James Valentine, who also moderated the September 2016 PFDD meeting.

Session discussion will include:

  • The patient and caregiver perspective on the most prevalent and burdensome CNS impacts of myotonic dystrophy and what clinically meaningful benefit of a therapy would include
  • Exploration of CNS endpoints used in other similar diseases and CNS symptoms for possible relevance in DM drug development
  • Moderated discussion with audience participation regarding the pros and cons of possible CNS endpoint options, what options appear to be most promising, where resources could be best invested and next steps for moving forward
  • The session will be followed by a lunch discussion among families, industry and academic researchers that will include feedback and a report out at the end of lunch 

A publication summarizing the workshop is also planned.