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Amylyx Pharmaceuticals Announces Publication of Data Showing Randomization to AMX0035 Prolonged Tracheostomy/Ventilation-free Survival and Reduced Occurrence of First Hospitalization
Adding to previously reported overall functional and survival benefit data for AMX0035, these findings support effect of AMX0035 on disease progression in
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nAdding to previously reported overall functional and survival benefit data for AMX0035, these findings support effect of AMX0035 on disease progression in ALS\n\n\n\nRandomization to AMX0035 resulted in a lower occurrence of death or tracheostomy/permanent assisted ventilation by 49% and first hospitalization by 44% over the Phase 2 trial and duration of follow-up\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the publication of long-term prespecified analyses assessing the occurrence of key events in addition to death from the Phase 2 CENTAUR trial including tracheostomy, permanent assisted ventilation (PAV), and first hospitalization. Data analyses demonstrated that early administration of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) resulted in a lower occurrence of tracheostomy/PAV and delayed first hospitalization during the trial and follow-up period in participants with amyotrophic lateral sclerosis (ALS). These results are published in the peer-reviewed medical journal, Journal of Neurology, Neurosurgery and Psychiatry.\n\n“People living with ALS often require medical interventions like mechanical ventilation as the disease progresses and impacts the muscles of respiration,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Associate Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These interventions come at a great cost, both personally and financially, for people living with the disease and developing therapies that can help delay these interventions can have a positive impact on their lives.”\n\nThe analyses encompassed the occurrence of the following key events from the point of randomization in the Phase 2 CENTAUR trial through a cutoff date of July 20, 2020 (longest post-randomization follow-up, 35 months): death (all-cause), tracheostomy (either for respiratory distress or airway clearance), PAV (defined as non-invasive ventilation >22 hours/day for >7 days), and hospitalizations specifically for ALS-related procedures (placement of a feeding tube, tracheostomy for management of secretions or respiratory support, or diaphragm pacing syst...