Phase II clinical data of AMX0035 in the treatment of Wolfram syndrome at 48 weeks shows good efficacy

Recently, Amylyx Pharmaceuticals announced its development of AMX0035 in the treatment of Wolf Positive results at week 48 of the Phase II open-label HELIOS clinical trial in adults with ram syndrome. This data covers key disease indicators such as pancreatic function, blood sugar control, and vision. The results are highly consistent with the efficacy at 24 weeks, showing the stability and sustainability of the treatment effect. These research results have been officially released at the Joint Congress of the European Society of Pediatric Endocrinology and the European Society of Endocrinology in Copenhagen, Denmark, and have attracted attention from the industry.

Wolfram syndrome is a rare single-gene inherited neurodegenerative disease caused by mutations in the WFS1 gene, resulting in progressive damage to multiple system organs. This disease is closely related to endoplasmic reticulum stress and manifests as the onset of childhood diabetes, accompanied by various symptoms such as optic atrophy, hearing loss, and neurodegeneration, which may eventually lead to severe respiratory failure. Currently, there are no approved treatments for this disease in the United States, and patients face a major treatment dilemma.

AMX0035 is a compound oral drug containing sodium phenylbutyrate and taurine diol. The design concept is to simultaneously target two key cellular pathological pathways, endoplasmic reticulum stress and mitochondrial dysfunction, to slow down nerve cell death and degenerative changes from the root. Amylyx believes that the combination has a synergistic mechanism of action and is more effective in preventing neurodegenerative progression than a single-target drug. The drug was previously approved for the treatment of amyotrophic lateral sclerosis as Relyvrio, but was withdrawn from the market due to unsatisfactory phase III trial results. At present, AMX0035 is being actively explored for its therapeutic potential in Wolfram syndrome and progressive supranuclear palsy, and has obtained orphan drug designation from the US FDA and the European Union.

HELIOSThe Phase II clinical trial is a single-center, open-label design, mainly evaluating the safety and efficacy ofAMX0035 in adult patients with Wolfram syndrome. Patients were treated for up to 144 weeks, during which efficacy was monitored by multiple measures of endocrine, neurological and visual function. 48Weekly data comes from1010 patients who met the criteria for genetic diagnosis showed continued improvement in pancreatic beta cell function, stable or improved blood sugar levels, no deterioration in vision status, and overall symptom burden reduced or remained stable. In addition, the safety profile is good, with no serious adverse reactions. This data will provide a scientific basis for the design of subsequent phase III trials and promote the drug to become a potential treatment option for Wolfram syndrome.

In summary, AMX0035has demonstrated sustainable and effective therapeutic potential in improving key physiological functions in patients with Wolfram syndrome through a multi-target synergistic mechanism. Although it is still in the clinical trial stage, its stable efficacy and good safety bring new hope to patients with rare diseases. With the accumulation of more clinical data, AMX0035 is expected to become an important breakthrough in the treatment of Wolfram syndrome in the future, improving the quality of life and prognosis of patients.

References:‘Amylyx Pharmaceuticals Announces Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome’, press release. Amylyx Pharmaceuticals, Inc. official website; published on May 12, 2025.