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CAMBRIDGE, Mass. - Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX), a $713 million market cap biotech company whose stock has surged over 330% in the past year, announced Wednesday it is discontinuing its ORION program for AMX0035 in progressive supranuclear palsy (PSP) after the drug failed to show differences from placebo in a Phase 2b trial. According to InvestingPro analysis, the stock appears overvalued at current levels.
The company said AMX0035 did not demonstrate efficacy compared to placebo on primary or secondary outcomes at the 24-week mark, prompting the decision to halt both the current trial and its open-label extension. Amylyx will not proceed with the planned Phase 3 portion of the program. With a Financial Health Score of FAIR from InvestingPro, the company maintains strong liquidity with a current ratio of 8.72.
Safety data remained consistent with previous studies, with the drug continuing to be "generally well-tolerated," according to the company’s press release.
"We set a high bar for AMX0035 in PSP and made a commitment to base our decision-making on the totality of the data and the potential for clinically meaningful outcomes," said Camille L. Bedrosian, Chief Medical Officer at Amylyx.
The company will now focus on its Phase 3 LUCIDITY trial of avexitide for post-bariatric hypoglycemia, with enrollment expected to complete in 2025 and topline data anticipated in the first half of 2026. Amylyx is also continuing development of AMX0035 in Wolfram syndrome and advancing AMX0114 in ALS.
Progressive supranuclear palsy is a rare, fatal neurodegenerative disorder affecting approximately seven in 100,000 people worldwide. The condition impacts movement, balance, eye movements, and speech, with patients typically having a life expectancy of six to eight years after diagnosis.
Despite this setback, Amylyx maintains its expectation that current cash reserves will extend through the end of 2026. Analyst targets range from $8 to $17 per share, reflecting mixed sentiment on the company’s prospects. For deeper insights into AMLX’s financial health and growth potential, including exclusive ProTips and comprehensive analysis, check out the detailed Pro Research Report available on InvestingPro.
In other recent news, Amylyx Pharmaceuticals announced the discontinuation of its ORION program for the drug AMX0035 in adults with progressive supranuclear palsy (PSP). The decision followed the Phase 2b trial results, which showed no significant differences compared to a placebo in primary or secondary outcomes. Consequently, the company will not proceed with the planned Phase 3 portion of the program. Meanwhile, Mizuho has raised its price target for Amylyx Pharmaceuticals to $12, citing an improved outlook for the drug avexitide in post-bariatric hypoglycemia (PBH). TD Cowen and Citi have both reiterated a Buy rating for the company, emphasizing avexitide’s potential based on recent data presented at the ENDO conference. The data showed significant reductions in hypoglycemia events with avexitide. Additionally, Goldman Sachs has resumed coverage of Amylyx with a Buy rating, highlighting avexitide as a "highly de-risked" asset following successful Phase 2 studies. These developments underscore a shift in focus towards avexitide for Amylyx Pharmaceuticals.
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