In June, Amylyx Pharmaceuticals acquired the rights to Eiger Biopharmaceuticals’ experimental glucagon-like peptide-1 (GLP-1) receptor antagonist, avexitide, for $35.1 million. Avexitide was previously developed for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). The acquisition is a notable strategic action by Amylyx following the failure of its lead amyotrophic lateral sclerosis (ALS) therapy, Relyvrio (sodium phenylbutyrate and taurursodiol), formerly known as AMX0035, says GlobalData.
In March, Amylyx announced that Relyvrio, a neuroprotective agent, failed to achieve its primary efficacy endpoints in the global Phase III PHOENIX trial for ALS. Relyvrio/Albrioza was first approved in the US and Canada in September 2022 for the treatment of ALS. Following the monumental setback, on April 4, the company announced the asset’s discontinuation, sending its shares to crash by 85 per cent on the same day.
Momna Ali, Healthcare Analyst at GlobalData, states, “Amylyx’s acquisition of avexitide marks its entry into the drug development field for metabolic diseases, a departure from its prior focus on neurodegenerative diseases. This shift in strategy comes after Relyvrio’s disappointing efficacy results from the drug’s confirmatory trial. Prior to acquiring avexitide, Amylyx was not completely unfamiliar with the metabolic space, as AMX0035 was also being investigated for Wolfram syndrome.”
Wolfram syndrome is an inherited condition that generally includes childhood-onset, insulin-dependent diabetes mellitus, and on April 10, Amylyx released interim results from its ongoing HELIOS trial, a 12-participant, open-label Phase II study, aimed at investigating the safety and tolerability of AMX0035 for Wolfram syndrome.
The preliminary data from an analysis of eight participants who completed a 24-week course of treatment indicate that AMX0035 administration resulted in enhanced total C-peptide response, which is significant as that is a validated objective laboratory measure of pancreatic beta cell function and glycaemic regulation.
“Despite the recent setbacks, the change in direction presents a ray of hope for Amylyx,” adds Ali.