– Additional research centers now enrolling patients in Australia and New Zealand – Company has achieved 50 percent of target enrollment in global Phase 3 trial LONDON – AMO Pharma Limited (“AMO Pharma”), a privately held biopharmaceutical company focusing on rare childhood-onset neurogenetic disorders with limited or no treatment options, today announced the...
treatment News
CHICAGO, Ill. — Amphix Bio, a pre-clinical stage company developing a new therapeutic modality for regenerative medicine, received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its lead candidate AMFX-200 for treatment of acute spinal cord injury (SCI). SCI is a devastating condition often leading to...
LINKÖPING, Sweden – AMRA announced today that its magnetic resonance imaging (MRI) based method has sufficient sensitivity to detect disease progression at the muscular level in ReDUX4: a multicenter Phase 2b clinical trial testing the safety and efficacy of Losmapimod in treating facioscapulohumeral muscular dystrophy (FSHD). The study introduced a powerful new method in...
DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce today that the U.S. Food and Drug Administration (“FDA”) has granted orphan...
AMSTERDAM — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT’s gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP). Orphan Drug Designation for AIP entitles AMT to...
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...
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. (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...
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)....