Surfing the CNS Pipeline: Ripples, Swells, or Waves


  • Ginger S. Johnson, PhD, CEO, Defined Health


  • Harry Tracy, PhD, Founder and President of NI Research and the author of NIR’s bimonthly publication, NeuroPerspective.

While  media attention tends to be focused upon CNS clinical misses, particularly when they involve  Alzheimer’s disease modifying therapies, several CNS programs have recently generated data that portend paradigm shifts in the treatment of diseases with significant unmet need. Novel treatments have shown proof-of-concept and more in Spinal Muscular Atrophy, Super-Refractory Status Epilepticus, Rett Syndrome, Treatment-Resistant Depression, Progressive MS, and rare pediatric epilepsies.  Looking forward, there are additional clinical-stage programs that promise to change the standard of care and reset the bar for compounds that follow (e.g., those of GW Pharma, Alder Pharma, AveXis, Quartet Medicine, Wave Life Sciences). This is in contrast to some high-profile programs which seem more likely to be additions to the current and still marginal standard of care than true clinical breakthroughs.

Please join us for a webinar discussion between Ginger Johnson, PhD, of Defined Health and Harry Tracy, PhD, in which we will explore some of these programs and why we consider them major clinical events for catalyzing renewed interest in the CNS pipeline:

  • What does the data really say?
  • How will these novel treatments alter the standard of care?
  • What is the likely impact on the competitive landscape?
  • What are potential pricing and market access considerations?