Sodium Phenylbutyrate/Tauroursodeoxycholic Acid

Amylyx Pharmaceuticals said Wednesday that it's discontinuing a clinical trial programme for AMX0035 (sodium phenylbutyrate/taurursodiol) after it did not show a benefit versus placebo in a Phase IIb trial of adults living with progressive supranuclear palsy (PSP).The latest disappointing results for AMX0035 come after Amylyx last year pulled the drug — then marketed under the brand Relyvrio for amyotrophic lateral sclerosis — from US shelves following its failure in a confirmatory trial.In the ORION programme, Amylyx reported that AMX0035 "did not show differences compared to placebo on primary or secondary outcomes at Week 24." As a result, the company is discontinuing the study and will not progress to a previously planned Phase III portion."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," said Chief Medical Officer Camille Bedrosian. "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."The news didn't seem to disappoint investors — in fact, shares of Amylyx ticked up about 3% on Wednesday — as the company's new entrant in the metabolic disease space seems to be the main attraction now.Last year, it acquired avexitide from Eiger BioPharmaceuticals for $35.1 million. The candidate — a GLP-1 receptor antagonist, rather than agonist — has received breakthrough therapy status from the FDA for both post-bariatric hypoglycaemia and congenital hyperinsulinism. The drug is designed to mitigate hypoglycaemia by decreasing insulin secretion and stabilising glucose levels."Our highest priority and focus remain on the pivotal Phase III LUCIDITY trial of avexitide, with enrollment expected to complete in 2025 and topline data anticipated in the first half of 2026," said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. They added that AMX0035 will remain in development for Wolfram syndrome.

Amylyx has disctontinued the Phase IIb stage of the study and shelved plans for a Phase III portion. Credit: vilai12 seehalath via Shutterstock.com. 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. 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.” GlobalData Strategic Intelligence 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 GlobalData Learn more about Strategic Intelligence Amylyx 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. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now