Amylyx Pharmaceuticals has dosed the first subject in the Phase ll HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) to treat Wolfram syndrome (WS).
The exploratory open-label proof of biology trial will evaluate the safety and tolerability along with various measures of endocrinological, neurological and ophthalmologic function of AMX0035.
Topline results from the trial are expected to be released next year.
Amylyx co-CEOs Joshua Cohen and Justin Klee said: “The WS community is in critical need of treatments to potentially improve their outcomes and standard of day-to-day living.
“We believe AMX0035 has scientific potential in several neurodegenerative diseases.
“We look forward to continuing to work to study AMX0035 in additional populations, including WS, and advancing progress in collaboration with the WS community including clinicians, researchers, and those living with the disease and their caregivers.”
Recently, researchers from Washington University School of Medicine in the US, along with Amylyx, unveiled preclinical data investigating the potential of AMX0035 for the treatment of WS.
These data showed the characterisation of a pathogenic variant in the WFS1 gene, thereby identifying a platform for additional genotype-phenotype review.
They also offered initial proof-of-concept for developing AMX0035 as a therapy to treat WS.
Besides, the study showed that administration of AMX0035 enhanced WFS1 protein expression, increased insulin secretion as well as inhibited cell death in β cells with the WFS1 c.1672C>T, p.R558C variant.
It was also found that AMX0035 postponed the onset of the diabetic phenotype in vivo in the Wfs1-knockout mouse model of WS.
In February this year, Amylyx announced the completion of subject enrolment in the Phase III PHOENIX trial of AMX0035 in amyotrophic lateral sclerosis (ALS) patients.