CAMBRIDGE, Mass.— Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced interim data from the ongoing Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome, a rare, progressive genetic disease impacting approximately 3,000 people in the U.S. The...
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CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced it has completed enrollment of its Phase 2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS), a rare genetic disease that typically results in neurodegeneration and premature death, which has no...
CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. today announced that the first participant has been dosed in ORION, a randomized, double-blind, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of progressive supranuclear palsy (PSP). The Phase 3 trial will enroll approximately 600 participants...
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced that the first participants have been dosed in the Phase 3 Study A35-004 (PHOENIX) (NCT05021536), a global trial evaluating the safety and efficacy of AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) in people with amyotrophic lateral sclerosis (ALS)....
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced its intention to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO)) for the treatment of amyotrophic lateral sclerosis (ALS). The decision by the company to submit the application in...
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced the closing of an oversubscribed $135 million Series C financing, led by Viking Global Investors, to support the clinical development and potential launch plans for lead product candidate AMX0035, for the potential treatment of ALS, and further research and development in other neurodegenerative...
CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced plans to submit a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in the first half of...
CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced that two abstracts detailing next steps in the development of the Company’s investigational antisense oligonucleotide (ASO), AMX0114, for the potential treatment of amyotrophic lateral sclerosis (ALS) and findings from a pharmacokinetic and pharmacodynamic study of AMX0035 (sodium phenylbutyrate [PB] and...
Quebec City, Canada – In people with amyotrophic lateral sclerosis (ALS), changes in neurons appear to activate immune cells. Lowering the inflammation could reduce the symptoms of the disease, according to a study led by Chantelle Sephton, a professor at Université Laval’s Faculty of Medicine. ALS is caused by the...
HCC is the fifth most common cancer in the world and the third most frequent cause of cancer-related death. Only about 20% of HCC patients are eligible for surgical resection. Therefore, RFA has been used increasingly as a safe technique for treating hepatic tumors. However, for hypervascular HCC, RFA appears...