Eiger BioPharmaceuticals, Inc.

Amylyx has discontinued the phase 2b trial and is tossing plans for the phase 3 portion of the study.\n Amylyx’s withdrawn-from-market Relyvrio has failed to make an impact on primary or secondary endpoints in a rare neurodegenerative disease, prompting the company to discontinue the program.Oral therapy Relyvrio, which Amylyx is again referring to as AMX0035, was tested in progressive supranuclear palsy (PSP), a fatal and rapidly progressing condition that impacts mobility, eye movements, swallowing and speech. Currently, there aren’t any approved treatments for the disease.Amylyx’s phase 2/3b study was measuring AMX0035’s impact on disease progression and severity using a 28-item, condition-specific scale. The phase 2 portion of the trial found no difference in patients receiving AMX0035 compared to placebo at 24 weeks, according to an Aug. 27 company release.Given the results, the company has discontinued the phase 2b trial, plus a related open-label extension study. Amylyx has also terminated plans for the phase 3 portion of the study.The company noted that AMX0035 was generally well-tolerated, with safety data consistent with prior studies.“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,” Amylyx Chief Medical Officer Camille Bedrosian, M.D., said in the release. “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.” AMX0035 is a combination of sodium phenylbutyrate and taurursodiol that was previously sold as Relyvrio and Albrioza for amyotrophic lateral sclerosis (ALS). The therapy had notched FDA approval in September 2022 based on phase 2 evidence that it significantly slowed the loss of physical function in patients with ALS.But a few months later, Amylyx said Relyvrio had failed a phase 3 trial, missing all primary and secondary endpoints measuring functional improvement in ALS. Shortly thereafter, Amylyx chose to pull the drug from the market. Amylyx still believes that the drug could offer benefits in Wolfram syndrome, a rare genetic disease that impacts about 3,000 Americans. The company is currently testing the therapy among 12 patients in an open-label phase 2 study.The company’s highest priority, however, is avexitide, a potentially first-in-class GLP-1 in development for post-bariatric hypoglycemia. The asset was purchased from Eiger Biopharma in 2024 and is currently being evaluated in a phase 3 trial, dubbed Lucidity. Patient recruitment for the study is expected to wrap by the end of this year, with topline data slated for 2026.The company currently expects its cash runway to last through the end of 2026. 

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.

iStock, tomozina AMX0035—approved as Relyvrio in 2022 for amyotrophic lateral sclerosis but voluntarily pulled from the market last year—was unable to distinguish itself from placebo in a mid-to-late-stage trial of progressive supranuclear palsy. Amylyx Pharmaceuticals is halting clinical development of AMX0035 in progressive supranuclear palsy, a rare neurodegenerative disease. This discontinuation, announced Wednesday , comes after AMX0035 failed to significantly distinguish itself from placebo in the Phase IIb/III ORION trial at a 24-week follow-up. The study, which had a target enrollment of 110 patients, primarily assessed the drug candidate in terms of disease progression. AMX0035 also failed to outperform placebo in terms of secondary outcomes, including improvements in motor aspects of activities of daily living. The drug candidate was generally well-tolerated, according to Amylyx, with a safety pro with that in prior studies. Given these findings, the biotech will terminate the open-label extension phase of ORION and will no longer push through to its Phase III portion. Affecting about 7 of every 100,000 people, progressive supranuclear palsy (PSP) is a rare neurodegenerative disorder that can turn fatal if left unchecked. Patients with this disorder suffer from dysfunctional movement, gait and balance. PSP also compromises swallowing and speech. Overall, the life expectancy among PSP patients runs from about six to eight years after initial diagnosis. While Amylyx will no longer test AMX0035 for PSP, it will still continue assessments for the drug in Wolfram syndrome, with an update expected later this year . Amylyx’s GLP-1 therapy avexitide will remain its “highest priority” moving forward, co-CEOs Josuha Cohen and Justin Klee said in a prepared statement on Wednesday. Avexitide is currently being studied for post-bariatric hypoglycemia (PBH) and the company expects to complete enrollment into the Phase III LUCIDITY trial this year. Topline findings from that study are expected in the first half of 2026. Amylyx acquired avexitide from Eiger BioPharmaceuticals in July 2024 for $35 million . At the time of the purchase, the asset had already been tested in five trials for PBH, with mid-stage studies demonstrating significant reductions in hypoglycemic events. Aside from avexitide, Amylyx is also advancing the antisense oligonucleotide AMX0114 for amyotrophic lateral sclerosis (ALS). Early cohort results from the Phase I LUMINA study are expected later this year, Cohen and Klee said in the company’s statement. Wednesday’s mid-stage stumble comes as Amylyx tries to regain its footing in the market after voluntarily withdrawing its ALS therapy Relyvrio. The drug, which was approved in September 2022 , failed a Phase III trial in March 2024, unable to significantly improve functional performance of patients versus placebo.