Amylyx Pharmaceuticals has discontinued a clinical programme evaluating a neuronal cell death inhibitor as a result of disappointing data when tested as a treatment for a rare brain disorder.
Amylyx’s AMX0035, a combination therapy of sodium phenylbutyrate and taurursodiol, failed to show a difference from placebo in the Phase IIb ORION trial (NCT06122662). Along with this stage of the study being discontinued, the US biotech has also shelved plans for a Phase III portion.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
ORION was evaluating AMX0035 as a potential treatment for progressive supranuclear palsy (PSP), a rare and progressive neurological condition. It belongs to a group of disorders called atypical parkinsonism, in which symptoms can be mistaken for Parkinson’s disease. Patients have difficulties with balance, vision, and speech, amongst other movement-based problems.
AMX0035, which Amylyx planned to administer orally for a year, failed to meet primary and secondary endpoints in ORION. Both endpoints were assessing the drug’s ability to impact disease progression via the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS). The company shelved the trial after data at 24 weeks showed no differences when compared to placebo.
Amylyx’s chief medical officer Camille Bedrosian said: “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 for those living with PSP.
“While we are disappointed in these results, we believe these data will inform the PSP trial literature, as well as deepen scientific understanding of this devastating disease.”
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataAmylyx confirmed that it will continue AMX0035’s development in Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss.
AMX0035 was briefly approved in 2022 based on Phase II data in the US and Canada to treat amyotrophic lateral sclerosis (ALS), known under the brand names Albrioza and Relyvrio, respectively. However, Amylyx pulled the drug from the market after Phase III results showed no treatment benefit. It was a difficult time for Amylyx, which was forced to reduce its workforce by 70% as it redirected resources to other clinical programmes.
No mentions of restructuring were made by the company in the wake of ORION’s discontinuation, with a cash runway expected to extend until the end of 2026.
Amylyx’s attention will now turn towards its Phase III LUCIDITY trial of glucagon-like peptide 1 receptor (GLP-1R) agonist avexitide. The company is evaluating the drug as a treatment for post-bariatric hypoglycaemia (PBH), a complication of blood sugar levels following weight loss surgery. The first patient was dosed in May, with topline data slated for the first half of 2026.
