The neurodegenerative-diseased-focused biotech Amylyx Pharmaceuticals announced that it had received orphan drug designation for AMX0035, a proprietary blend of sodium phenylbutyrate and tauroursodeoxycholic acid.
The Cambridge, Mass.-based company won that designation to treat Wolfram syndrome, a rare genetic disease associated with diabetes, progressive optic atrophy, hearing loss and severe neurological disabilities.
The prognosis for patients diagnosed with the disease is poor. Many Wolfram syndrome patients die by around 35 years of age.
There was no treatment for the disease until researchers identified that the endoplasmic reticulum membrane dysfunction is a hallmark of the disease.
AMX0035 is intended to target endoplasmic reticulum stress, and preclinical data suggest that the drug could potentially stop optic nerve atrophy in Wolfram syndrome patients.
“Orphan drug designation will support us as we explore AMX0035 in treating this rare and underserved population living with a life-threatening disease that has no approved therapies,” said Kent Leslie, chief scientific officer at privately held Amylyx, in a statement.
The company is also investigating the use of AMX0035 to treat amyotrophic lateral sclerosis and Alzheimer’s disease.
Amylyx had announced that Alzheimer’s patients receiving the drug had a 44% reduced mortality compared to trial volunteers who received placebo over a three-year period. The journal Muscle & Nerve published results from that study.