Amylyx Pharmaceuticals has announced that it will discontinue the ORION program of AMX0035 for progressive supranuclear palsy (PSP), following results indicating no meaningful differences between the drug and placebo in primary or secondary endpoints at Week 24, according to the company. Based on these findings, the company said that it will halt the Phase 2b trial and its open-label extension, and will not move forward with the Phase 3 portion of the program. Safety data were consistent with prior studies, and AMX0035 remained generally well-tolerated, the company stated.
The company said that its primary focus will now shift to the pivotal Phase 3 LUCIDITY trial of avexitide for post-bariatric hypoglycemia, with enrollment expected to finish in 2025 and topline results anticipated in the first half of 2026. The company also continues development of AMX0035 in Wolfram syndrome and is advancing AMX0114 in ALS, with early cohort data from the Phase 1 LUMINA trial expected in 2025.
The company said its cash runway is expected to extend through the end of 2026.
