Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome
Preliminary results from HELIOS anticipated in the second half of 2024
“The unmet need for people living with WS remains significant. WS is progressive, often fatal, and difficult to diagnose, with current treatments focused mostly on helping to address symptoms,” said
HELIOS is an open-label proof of biology trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with WS. Data from this initial study will inform potential future Amylyx trials for individuals with WS.
More information on the HELIOS clinical study can be found at www.clinicaltrials.gov, NCT05676034.
“Little progress has been made in the treatment of WS, and we are excited by the potential we have been seeing in the preclinical work with AMX0035,” 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.
About AMX0035 / RELYVRIO®/ ALBRIOZA™ / ALBRIOZA®
AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the
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, the potential of AMX0035 as a treatment for ALS and the Company’s plans to explore the use of AMX0035 for other neurodegenerative diseases including Wolfram Syndrome, the timelines for the HELIOS study in WS, and expectations regarding our longer-term strategy. Any forward-looking statements in this press release 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: Amylyx’ ability to fund operations, the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its commercial and regulatory strategy, regulatory developments, expectations regarding the timing and outcome of EMA’s review of AMX0035 for the treatment of ALS, Amylyx’ reliance on third parties, including to conduct clinical trials and manufacture products, and the effect of global economic uncertainty and financial market volatility caused by economic effects of rising inflation and interest rates, the COVID-19 pandemic, geopolitical instability, changes in international trade relationships and military conflicts, as well as the risks and uncertainties set forth in Amylyx’