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.
“Wolfram is a debilitating syndrome in need of new treatments,” said Kent Leslie, global head of R&D and chief scientific officer at Amylyx.
The company began focusing on the syndrome after the mother of a patient connected with Wolfram syndrome expert Dr. Fumihiko Urano. The physician had been evaluating Amylyx compounds in his lab.
“Early evidence supports the potential of AMX0035 to target the underlying cellular pathology,” Leslie said. “The commitment to finding new potential therapies from the patients, caregivers and clinicians of this rare disease spearheaded this encouraging progress.”
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,” Leslie said.
The company is also investigating the use of AMX0035 to treat amyotrophic lateral sclerosis and Alzheimer’s disease.
Amylyx had announced that ALS 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.
“It has been inspiring to meet patients and families affected by Wolfram Syndrome,” concluded Josh Cohen, co-CEO and cofounder of Amylyx.
“We will press ahead with their support and hope to enter clinical studies in the near future,” added Justin Klee, the other co-CEO and co-founder of the company. “We are committed to improving the lives of patients with Wolfram syndrome and know they are waiting.”