Amylyx Pharmaceuticals recently made the tough decision to discontinue the clinical development of AMX0035 for progressive supranuclear palsy (PSP), a rare neurodegenerative disorder. AMX0035, previously known as Relyvrio and approved for amyotrophic lateral sclerosis (ALS) in 2022, failed to demonstrate superiority over a placebo in a mid-to-late-stage trial for PSP. The Phase IIb/III ORION trial, designed to evaluate disease progression in 110 patients over 24 weeks, concluded that AMX0035 did not meet the desired endpoints.
Patients with progressive supranuclear palsy, a condition that affects around 7 out of every 100,000 individuals, experience challenges with movement, balance, swallowing, and speech. The average life expectancy for PSP patients ranges from six to eight years post-diagnosis. Despite being well-tolerated and exhibiting a safety profile consistent with previous studies, AMX0035 did not show significant improvements in motor function or daily living activities compared to the placebo, prompting Amylyx to halt further development for this indication.
While the discontinuation of AMX0035 for PSP is a setback, Amylyx remains committed to evaluating the drug for Wolfram syndrome, with updates anticipated later this year. The company’s focus will now shift to advancing its GLP-1 therapy, avexitide, which is currently undergoing clinical trials for post-bariatric hypoglycemia (PBH). Amylyx obtained avexitide through the acquisition of Eiger BioPharmaceuticals in 2024 for $35 million, building on promising results from mid-stage studies that demonstrated a reduction in hypoglycemic events.
In addition to avexitide, Amylyx is progressing with the development of AMX0114, an antisense oligonucleotide, for the treatment of ALS. Early results from the Phase I LUMINA study are expected later this year, marking a significant step in the company’s efforts to address neurodegenerative conditions. The decision to halt the AMX0035 program for PSP aligns with Amylyx’s strategic realignment following the withdrawal of their ALS therapy, Relyvrio, which faced challenges in a Phase III trial in 2024.
As Amylyx navigates the evolving landscape of neurodegenerative disorders, the company remains dedicated to advancing innovative therapies that address unmet medical needs. While the discontinuation of AMX0035 for PSP represents a setback, the ongoing development of avexitide for PBH and AMX0114 for ALS underscores Amylyx’s commitment to pioneering treatments for challenging conditions. Looking ahead, the biotech industry eagerly anticipates the results of upcoming clinical trials and the potential impact of these novel therapies on patients’ lives.
- Amylyx discontinues clinical development of AMX0035 for progressive supranuclear palsy (PSP) due to failure to meet endpoints in Phase IIb/III trial.
- Company shifts focus to advancing GLP-1 therapy avexitide for post-bariatric hypoglycemia (PBH) and antisense oligonucleotide AMX0114 for ALS.
- Decision aligns with strategic realignment post-withdrawal of ALS therapy Relyvrio, signaling Amylyx’s commitment to innovative treatments for neurodegenerative disorders.
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