Amylyx is trying to move forward with its effort to turn its former ALS drug into a treatment for other conditions, and data released Thursday build on a small interim dataset from earlier this year that showed signs of improving outcomes in a rare genetic disease.
Wolfram syndrome usually manifests in childhood and patients experience symptoms typically seen in diabetes (high blood sugar, high urine production) that may progress to blindness and deafness.
The Phase 2 study of Amylyx’s drug, AMX0035, measured whether patients’ average levels of an amino acid called C-peptide increased after 24 weeks. C-peptide response rates are traditionally used to determine the health of diabetes patients’ pancreatic cells and how well they function, according to the NIH.
In order to look at how patients responded to AMX0035, Amylyx asked 12 patients to take a “mixed meal tolerance test,” in which patients ingest a “standardized liquid meal” in the mornings with their C-peptide responses measured over time. Usually, C-peptide responses peak after 30 minutes in non-diabetic individuals, but Wolfram syndrome patients’ levels peak more slowly, after about 120 minutes.
After 12 patients took AMX0035 for 24 weeks, the drug improved C-peptide responses at the 120-minute mark by an average of 3.8 minutes by ng/mL compared to baseline levels. Response rates continued to improve at 36 and 48 weeks, Amylyx said.
But one of the patients did not meet the trial’s inclusion and exclusion criteria, possessing just one of the two alleles necessary to categorize the disease. Amylyx also presented an 11-patient dataset removing this individual, showing improved C-peptide responses at the 120-minute mark of an average of 20.2 minutes by ng/mL compared to baseline levels.
In an investor call Thursday afternoon, co-CEO Josh Cohen said the company would look to implement stricter genetic screening measures in a Phase 3 trial to prevent this error from happening again.
The company is continuing to discuss what a study might look like, including whether it will seek accelerated approval, and will reveal more details next year.
AMX0035 was previously approved to treat ALS under the brand name Relyvrio. The drug eventually failed a Phase 3 study and forced Amylyx to voluntarily withdraw it from the market. That failure caused a huge plunge in the company’s stock and led to layoffs. Shares $AMLX were up as much as 7% Thursday.