CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the presentation of early safety and tolerability data from its Phase 1 LUMINA trial of AMX0114 and results from ongoing work characterizing biomarkers of AMX0114 target engagement at the 36th International Symposium on ALS/MND (MNDA) held from December...
treatment News
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...
Data at Week 48 demonstrated that treatment with AMX0035 led to sustained stabilization or improvement in multiple outcomes related to disease progression, including pancreatic function, glycemic control, vision, and overall symptom burden AMX0035 continued to be generally well-tolerated in all participants Week 48 data and discussions with FDA will inform...
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...
AMX0114 is an Amylyx-developed antisense oligonucleotide designed to target calpain-2, a key contributor to the axonal degeneration pathway in ALS Phase 1 LUMINA trial of AMX0114 underway; early cohort data expected in 2025 CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) today announced that the U.S. Food and Drug Administration...
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...
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...
St Louis, Missouri – By recruiting the immune system to combat tumor cells, immunotherapy has improved survival rates, offering hope to millions of cancer patients. However, only about one in five people responds favorably to these treatments. With a goal of understanding and addressing immunotherapy’s limitations, researchers at Washington University...
Arlington, TX – Lung cancer is not the most common form of cancer, but it is by far the deadliest. Despite treatments such as surgery, radiation therapy and chemotherapy, only about a quarter of all people with the disease will live more than five years after diagnosis, and lung cancer...