Thank you to all who joined Cantor’s Neurology & Psychiatry Conference. With today being World Mental Health Day, and given the recent approval of a new drug to treat ALS (Lou Gehrig’s disease), it was fascinating to hear some of the leading experts in these fields discuss new treatments and therapies to help battle these debilitating conditions.
Senior Biotech Analyst, Charles C. Duncan, Ph.D. hosted a KOL lunch with Dr. Jeremy Shefner, M.D., Ph.D., Chair of the Department of Neurology and Associate Director for Barrow Neurological Institute.
• Discussions focused on the debilitating nature of ALS, limited number of therapies available, key considerations in designing a trial, and optimism from the recent approval of RELVRYIO
• Selecting suitable endpoints capturing therapeutic benefit has become a key topic, with many trials using ALSFRS-R, but breathing and extended survivability gaining more traction
• Biomarkers that track disease progression or treatment outcome have been looked to as a de-risker for ALS therapies in development, but more research is needed to establish its relationship to symptom improvement
• Although more work needs to be done, there is optimism as Dr. Shefner opines that there is possibly increased flexibility of the FDA that we believe bodes well for the “next-generation” of therapies