Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) on Wednesday decided to discontinue the ORION program of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with progressive supranuclear palsy (PSP).
PSP is a rare, degenerative brain disorder causing issues with balance, movement, eye movements, speech, swallowing, mood, and cognition.
AMX0035 did not show differences compared to placebo on primary or secondary outcomes at Week 24.
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Based on these results, the company will discontinue the Phase 2b trial and open-label extension and will not initiate the Phase 3 portion of the program.
Safety data were consistent with safety data from prior studies, and AMX0035 continued to be generally well-tolerated.
Amylyx says its highest priority and focus remain on the pivotal Phase 3 LUCIDITY trial of avexitide, with enrollment expected to be completed in 2025 and topline data anticipated in the first half of 2026.
The company is also continuing development of AMX0035 in Wolfram syndrome and progressing AMX0114 in amyotrophic lateral sclerosis (ALS), with early cohort data from the Phase 1 LUMINA trial expected in 2025.
In July, Amylyx Pharmaceuticals presented new exploratory analyses from its Phase 2 PREVENT and Phase 2b trials of avexitide, an investigational GLP-1 receptor antagonist, at the Endocrine Society’s annual meeting.
In the Phase 2b trial, a 90 mg once-daily dose of avexitide, now being evaluated in the Phase 3 LUCIDITY trial, achieved a 64% reduction in the rate of moderate to severe hypoglycemic events. More than half of the participants experienced no such events during treatment.
Amylyx expects its cash runway to extend through the end of 2026.
In March 2024, Amylyx shared topline results from the PHOENIX Phase 3 trial of AMX0035, also known as Relyvrio in the U.S. and Albrioza in Canada, for 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.
Price Action: AMLX stock is trading lower by 0.66% to $9.10 at last check Wednesday.
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