Amylyx Pharmaceuticals Announces Publication of New CENTAUR Trial Analyses Further Demonstrating Significant Survival Benefit with AMX0035 in People with ALS
- An analysis using the rank-preserving structural failure time model (RPSFTM), a method frequently employed in oncology to account for placebo crossover, estimated a 10.6-month longer median survival duration for AMX0035 participants
- Participants randomized to receive AMX0035 and who continued into the open-label extension (OLE) phase showed an 18.8-month longer median survival duration than participants who never received AMX0035 in a subgroup analysis
Excerpt from the Press Release:
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the publication of long-term survival analyses of the Phase 2 CENTAUR trial adjusting for treatment crossover of the placebo group. The post hoc analyses suggested a larger survival benefit for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) when adjusting for placebo crossover ranging from 10.6 to 18.8 months compared with 6.9 months seen in the original prespecified intent-to-treat (ITT) analysis in participants with amyotrophic lateral sclerosis (ALS). These results are published in the peer-reviewed medical journal, Muscle & Nerve.
“Trials that incorporate placebo-to-active treatment crossover are critically important in rapidly fatal diseases like ALS, however, this design may underestimate the clinical effect of investigational therapies,” said Machelle Manuel, Ph.D., Head of Global Medical Affairs of Amylyx. “Fortunately, statistical models, like the RPSFTM, allow us to adjust results for treatment crossover. Here, similar to the prespecified ITT analysis, we observed a significant survival benefit for AMX0035 in the survival analyses adjusting for treatment crossover, potentially offering those living with ALS and their families hope of sharing more memories and milestones together.
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