Amylyx Pharmaceuticals discontinues ORION program for AMX0035 in PSP

Published 27/08/2025, 12:14
Amylyx Pharmaceuticals discontinues ORION program for AMX0035 in PSP

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX), currently trading at $9.16 and showing a remarkable 330% return over the past year, announced Wednesday that it is discontinuing the ORION program evaluating AMX0035 in adults with progressive supranuclear palsy (PSP). According to InvestingPro analysis, the company maintains a FAIR financial health score, with analysts maintaining a bullish consensus on the stock despite today’s news. According to a statement in a press release, the company reported that AMX0035 did not demonstrate differences compared to placebo on primary or secondary outcomes at Week 24 of the Phase 2b trial. With a market capitalization of $713 million and analyst price targets ranging from $8 to $17, detailed analysis available in the comprehensive InvestingPro Research Report suggests the stock is currently fairly valued.

As a result, Amylyx will discontinue both the Phase 2b trial and its open-label extension, and will not initiate the planned Phase 3 portion of the program. The company stated that the safety data observed in the trial were consistent with results from prior studies of AMX0035.

AMX0035 is a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine). The ORION program was designed to assess the efficacy and safety of this therapy in adults living with PSP, a rare neurodegenerative disease.

This information is based on a statement provided in a Securities and Exchange Commission filing by Amylyx Pharmaceuticals .

In other recent news, Amylyx Pharmaceuticals has been the focus of several analyst reports highlighting its drug avexitide, which targets post-bariatric hypoglycemia (PBH). Mizuho has raised its price target for Amylyx to $12.00 from $8.00, citing an improved market share outlook for avexitide. TD Cowen reiterated a Buy rating, emphasizing the drug’s potential, supported by Phase II data showing significant reductions in hypoglycemia events. Citi also maintained its Buy rating and $12.00 price target after a key opinion leader event that discussed avexitide’s benefits and the challenges in current PBH management. Goldman Sachs resumed coverage with a Buy rating and a $10.00 price target, describing avexitide as "highly de-risked" after successful Phase 2 studies. Additionally, Citi initiated coverage with a Buy rating, recognizing Amylyx’s promising pipeline, which includes avexitide and other assets like AMX0035 and AMX0114, addressing various medical needs. These developments underscore the growing optimism among analysts regarding Amylyx’s potential in the pharmaceutical market.

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