Amylyx Pharmaceuticals is still betting on former ALS drug Relyvrio (AMX0035), which it pulled from the US and Canadian markets this year, securing an Orphan Drug Designation for it from the European Medicines Agency (EMA) in Wolfram Syndrome.
The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating conditions where the product may represent a significant benefit over existing treatments.
Amylyx already has Orphan Drug Designation for AMX0035 in the US, which it secured from the US Food and Drug Administration (FDA) for Wolfram syndrome back in 2020.
In addition to Wolfram Syndrome, Amylyx is also investigating the drug in progressive supranuclear palsy (PSP).
AMX0035 is Amylyx’s proprietary, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the US).
Wolfram syndrome is a rare, genetic neurodegenerative disorder characterized by childhood-onset diabetes, optic atrophy and deafness. It often leads to severe complications, including neurodegeneration, and has a significantly reduced life expectancy. The syndrome affects approximately one in 500,000 people worldwide, making it a rare but severe condition.
Related: Amylyx Purchases Investigational GLP-1 Drug Avexitide in Auction Deal
Amylyx recently presented positive data from an interim analysis of its Phase II HELIOS study. The study involves eight patients with Wolfram syndrome who upon evaluation at Week 24, exhibited improved pancreatic function and glycemic control after received AMX0035. Most of the participants also reported some improvement in vision.
In a press release, Amylyx said that in Wolfram syndrome, “progressive decline and worsening of outcomes would have been expected on all measures, so disease improvement or stabilization alone is clinically meaningful.”
Amylyx said it expects to report topline data from all 12 participants in the HELIOS study at Week 24 this fall.
There are currently no approved treatment options for Wolfram syndrome, underscoring the high unmet need.
While continuing to pursue AMX0035, Amylyx is also looking to bolster its developmental pipeline with other assets. Last month, it got into the GLP-1 game, picking up a Phase III ready GLP-1 receptor antagonist from Eiger BioPharmaceuticals in an auction for $35 million. The company is looking to develop the drug, which differs from diabetes and obesity GLP-1 agonist drugs like Ozempic, in rare metabolic disorders.
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