Amylyx shares complete Phase 2 data for withdrawn ALS drug in rare genetic disorder

Amylyx is making strides to repurpose its former ALS drug, focusing on its potential to treat other conditions. Recently, the company shared data reinforcing earlier findings that suggested promising outcomes for a rare genetic disorder known as Wolfram syndrome.

Wolfram syndrome, typically emerging in childhood, is characterized by symptoms commonly associated with diabetes, such as high blood sugar and excessive urine production, and can progress to severe complications including blindness and deafness.

Amylyx conducted a Phase 2 study of its drug, AMX0035, to assess its effectiveness by measuring the average levels of an amino acid called C-peptide in patients after a 24-week period. C-peptide levels are traditionally used to gauge the health and functionality of pancreatic cells in diabetes patients, as noted by the NIH.

To evaluate patient responses to AMX0035, Amylyx administered a “mixed meal tolerance test” to 12 participants, where they consumed a standardized liquid meal in the morning, and their C-peptide levels were monitored over time. Typically, non-diabetic individuals show a peak in C-peptide levels after 30 minutes, while Wolfram syndrome patients exhibit a delayed peak at around 120 minutes.

After 24 weeks of AMX0035 treatment, the results showed that patients' C-peptide responses improved at the 120-minute mark by an average of 3.8 ng/mL compared to their baseline levels. These positive results continued to enhance at the 36 and 48-week marks, according to Amylyx.

However, one patient in the study did not meet the trial’s inclusion criteria, as they possessed only one of the two alleles necessary to confirm Wolfram syndrome. When this patient's data was excluded, the remaining 11-patient dataset revealed an even more significant improvement in C-peptide responses, with an average increase of 20.2 ng/mL at the 120-minute mark compared to baseline levels.

During an investor call, co-CEO Josh Cohen mentioned that the company plans to implement more stringent genetic screening measures in a Phase 3 trial to avoid similar discrepancies in the future. Amylyx is in ongoing discussions about the design of this future study and is contemplating whether to seek accelerated approval. More details about the study will be disclosed next year.

Previously, AMX0035 had received approval for ALS treatment under the brand name Relyvrio. However, the drug did not succeed in a subsequent Phase 3 trial, leading Amylyx to voluntarily withdraw it from the market. This failure resulted in a significant decline in the company’s stock and subsequent layoffs. Despite these setbacks, the company's shares saw a rise of up to 7% on Thursday.