Amylyx explores new indications for Relyvrio after ALS trail setback, with early success in Wolfram syndrome and other neurodegenerative diseases
In March 2024, Amylyx Pharmaceuticals announced that Relyvrio, a neuroprotective agent, failed to achieve its primary efficacy endpoints in the global Phase III PHOENIX trial for amyotrophic lateral sclerosis (ALS).
Relyvrio/Albrioza was first approved in the US and Canada in September 2022 for the treatment of ALS. However, following the monumental setback, on April 4, the company announced the asset’s discontinuation, sending its shares crashing 85 per cent the same day. The impact of this has been detrimental for Amylyx; however, the company is exploring other indications in which its only asset may excel, and that seems to be bearing some fruit, says GlobalData.
Amylyx is planning to develop new treatment options for neurodegenerative diseases. More specifically, AMX0035 is being investigated in Wolfram syndrome, Alzheimer’s Disease (AD), and progressive supranuclear palsy (PSP).
Key opinion leaders (KOLs) interviewed by GlobalData expressed that both CENTAUR and PHOENIX trials conducted by Amylyx are “very-well done studies”, however, finding a treatment for ALS is not an easy task due to its complicated disease aetiology and pathophysiology.
Momna Ali, Healthcare Analyst at GlobalData, comments, “When Relyvrio was initially approved, there was a swirl of controversy anyway. A plunge as deep as 85 per cent in shares and having to part ways with 70 per cent of the workforce is a very difficult position to be in.”
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 endocrinological and neurological function, namely Wolfram syndrome.
Ali adds, “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.”
Moreover, AMX0035 demonstrated favourable outcomes across multiple key parameters in the HELIOS trial, including a mean reduction of 0.26 per cent in HbA1c levels following 24 weeks of treatment. Continuous glucose monitoring revealed an average improvement of 7.1 per cent in the absolute time spent within the target glycemic range.
Ali concludes, “Despite the recent setbacks, the investigation of its lead asset AMX0035 in Wolfram syndrome presents a ray of hope for Amylyx.”
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