Topline results from global phase III PHOENIX trial of AMX 0035 in amyotrophic lateral sclerosis.- Amylyx Pharma
Amylyx Pharmaceuticals, Inc.., announced topline results from PHOENIX, a global, 48-week, randomized, placebo-controlled Phase III clinical trial of AMX 0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine] ; (Relyvrio in the U.S., Albrioza in Canada) in people living with amyotrophic lateral sclerosis (ALS).
PHOENIX did not meet its primary endpoint of reaching statistical significance (p=0.667) as measured by change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at Week 48, nor was there statistical significance seen in secondary endpoints . Amylyx plans to present the data from PHOENIX at an upcoming medical meeting and will publish the results in a medical journal later this year.
Amylyx will continue to engage with regulatory authorities and the broader ALS community, including ALS specialists and other multidisciplinary experts, people living with ALS, and advocates, to discuss the results from PHOENIX within the next eight weeks and make informed decisions. Amylyx intends to share plans for (Relyvrio/Albrioza in ALS, which may include voluntarily withdrawing Relyvrio/Albrioza from the market. At this time, (Relyvrio/Albrioza and its related patient support program will continue to be available for people living with ALS. Amylyx has voluntarily decided to pause promotion of the medication during this time.
"We are surprised and deeply disappointed by the PHOENIX results following the positive data from the CENTAUR trial. Our main priority at the moment is sharing the information with people living with ALS and their treating physicians; this is part of our continued commitment to them and our mission. Over the next eight weeks, our team will continue to engage with regulatory authorities and the ALS community to discuss the results from PHOENIX. We will be led in our decisions by two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us. On behalf of the entire Amylyx team, we are grateful to the ALS community and for the dedication of trial participants, investigators, and study site teams. With data collected from 664 participants in PHOENIX, we are certain there will be important learnings that will help inform future ALS research. We are steadfast in our commitment to the ALS community and our mission, including with AMX 0035 where it has shown potential in neurodegenerative diseases such as Wolfram syndrome and progressive supranuclear palsy, and with AMX 0114, our investigational antisense oligonucleotide targeting calpain-2, in ALS,” said Justin Klee and Joshua Cohen, Co-CEOs of Amylyx.
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