Just one week after pulling its lead candidate Relyvrio from the market for amyotrophic lateral sclerosis (ALS), Amyyx Pharmaceuticals has announced positive interim results from its Phase II trial of the candidate in another rare disease, Wolfram syndrome.
Amylyx has announced Relyvrio (AMX0035) has shown clinically meaningful changes in key outcomes of a Phase II trial of the candidate in patients with Wolfram syndrome. The announcement comes amid a storm for the company which had to pull the candidate from the market for ALS, for which it received approval from the FDA in September 2022, after a confirmatory Phase III trial failed to meet any of its endpoints.
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The Phase II HELIOS trial (NCT05676034) is an open-label study designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function.
The primary endpoint of the trial measures changes from baseline in C-peptide, an established, objective laboratory measure of pancreatic beta cell function and glycemic control, assessed using a Mixed Meal Tolerance Test (MMTT). Secondary and exploratory outcomes include the measurement of other diabetic responses and other domains affected by the disease.
Interim data from eight of the 12 patients showed that AMX0035 had a clinically meaningful effect on key outcomes measuring the progression of diabetes, visual decline, and overall disease burden in adult participants living with Wolfram syndrome.
The safety profile of AMX0035 in HELIOS was consistent with prior safety data. AMX0035 was generally well-tolerated. The majority of adverse events (AEs) were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The most common AE was diarrhoea.
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By GlobalDataDespite the positive news, the company’s stock took a fall of 7.8%, from $2.67 at market close on 9 April to $2.46 at market close on 10 April.
AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol. Preclinical studies show the candidate may reduce cell death and improve cellular function. The candidate is also being investigated for progressive supranuclear palsy (PSP).
Wolfram syndrome landscape
Wolfram syndrome is an inherited rare disease which affects around one in every 770,000 people worldwide.
Patients generally suffer from childhood-onset insulin-dependent diabetes and progressive optic atrophy. Patients can also develop diabetes insipidus, sensorineural hearing loss and autonomic nervous system degeneration.
There are currently no approved treatments that can delay or reverse the progression of Wolfram syndrome. Instead, symptoms are managed with other approved drugs, such as insulin to manage diabetes symptoms.
