NeurologyLive® Mind Moments®

114: Phase 3 PHOENIX Study, AMX0035, and Amylyx's Future Drug Development


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Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice.


In this episode, Justin Klee and Josh Cohen, cofounders and cochief executive officers of Amylyx Pharmaceuticals, sat down at the recently concluded AAN Annual Meeting to discuss the results from the pivotal phase 3 PHOENIX trial (NCT) of AMX0035 (Relyvrio) in patients with amyotrophic lateral sclerosis (ALS). The duo provided clarity on the findings, positive takeaways from the disappointing data, and the lessons learned in ALS drug development. In addition, the two provided commentary on the future plans of the therapy in other tauopathies like progressive supranuclear palsy and neurologic conditions like Wolfram syndrome. Furthermore, Klee and Cohen shared thoughts on the company's drug pipeline, including AMX0114, an antisense oligonucleotide in development for patients with ALS. 

Looking for more neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page.

Episode Breakdown:
  • 1:05 – Decision behind removing AMX0035 from market
  • 3:00 – Lessons from PHOENIX, AMX0035 drug development program
  • 6:45 – Unraveling PHOENIX study data 
  • 10:00 – Neurology News Minute
  • 12:10 – Potential of AMX0035 in other tauopathies, neurologic conditions
  • 15:00 – Outlook of antisense oligonucleotide AMX0114 in ALS

    The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

    • Diazepam Buccal Film’s Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD
    • Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS
    • FDA Approves Sprinkle Formulation of Neurocrine Biosciences’ Valbenazine for Tardive Dyskinesia or Huntington disease Chorea

    • Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.
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