Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome
- Recently published preclinical data demonstrate initial proof-of-concept for the therapeutic development of AMX0035 (sodium phenylbutyrate and taurursodiol) in Wolfram syndrome
HELIOS is an exploratory open-label proof of biology study assessing the effect of AMX0035 safety and tolerability, and various measures of endocrinological, neurological and ophthalmologic function. Amylyx anticipates topline results from HELIOS in 2024.
More information on the HELIOS clinical study can be found at www.clinicaltrials.gov, NCT05676034.
Researchers from the
Administration of AMX0035improved WFS1 protein expression, increased insulin secretion, and inhibited cell death in β cells with the WFS1 c.1672C>T, p.R558C variant. AMX0035 also prevented cellular death in patient-derived neuronal progenitor cells. Gene enrichment analysis revealed that treatment with AMX0035 ameliorated organelle dysfunction, mitophagy, endoplasmic reticulum (ER) stress, and apoptosis.
- Furthermore, AMX0035 delayed the onset of the diabetic phenotype in vivo in the Wfs1-knockout mouse model of Wolfram syndrome.
“WS is a rare, progressive, and often fatal neurodegenerative disease. Preclinical data showed that a combination treatment of two chemicals, namely AMX0035, may restore cellular functioning in a cellular model of WS,” said
“The WS community is in critical need of treatments to potentially improve their outcomes and standard of day-to-day living,” said
Amylyx announced that the FDA granted orphan drug designation to AMX0035 for the treatment of WS in
About Wolfram Syndrome
Wolfram syndrome (WS) is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of WS include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of WS. The prognosis of WS is poor, and many people with the disease die prematurely with severe neurological disabilities.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the Phase 2 clinical trial of AMX0035 for the treatment of WS, including expectations regarding the timing of trial results, and the potential of AMX0035 as a treatment option for WS and its potential to address unmet needs and improve outcomes for the WS community. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities, the likelihood of preclinical results to be predictive of clinical trial results, Amylyx’ ability to execute on its strategy, regulatory developments, expectations regarding the timing of regulatory review of AMX0035, Amylyx’ ability to fund operations, and the impact that the COVID-19 pandemic, global macroeconomic uncertainty and geopolitical instability will have on Amylyx’ operations, as well as those risks and uncertainties set forth in Amylyx’
Amylyx Media Team