Amylyx(US:AMLX) Businesswireยท2024-03-08 12:00Core Insights - Amylyx Pharmaceuticals announced topline results from the PHOENIX trial, which did not meet its primary endpoint of statistical significance in ALSFRS-R total score change at Week 48 (p=0.667) [1][5] - The company plans to engage with regulatory authorities and the ALS community to discuss the trial results and may consider withdrawing RELYVRIO/ALBRIOZA from the market [2][3] - AMX0035 was well-tolerated in the trial, with no new safety signals reported, reinforcing its favorable safety profile [5] PHOENIX Trial Results - The PHOENIX study enrolled 664 adults with ALS, randomized to receive either AMX0035 or placebo, alongside standard-of-care treatments [4][9] - No significant differences were observed in secondary endpoints, and the treatment did not show efficacy in participants who met CENTAUR trial criteria [5] - European participants completing the trial have the option to enroll in an ongoing open-label extension phase [5][9] Future Plans and Community Engagement - The company will share the PHOENIX results with the ALS community and regulatory authorities over the next eight weeks [2][3] - Amylyx remains committed to ALS research and is exploring AMX0035's potential in other neurodegenerative diseases, including Wolfram syndrome and progressive supranuclear palsy [3][6] Ongoing Studies - The ORION trial of AMX0035 in progressive supranuclear palsy is ongoing, with topline results expected in 2025 or 2026 [7] - Preliminary data from the HELIOS trial in Wolfram syndrome is anticipated in the second quarter of 2024 [7] About AMX0035 - AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol, approved as RELYVRIO in the U.S. and ALBRIOZA in Canada for ALS treatment [19][20] - The drug targets neurodegenerative pathways by mitigating endoplasmic reticulum stress and mitochondrial dysfunction [6][19]