Combination Phenylbutyrate/Taurursodiol Early Reduced Key Clinical Events in Amyotrophic Lateral Sclerosis 

As published in the Journal of Neurology, Neurosurgery and Psychiatry, prespecified analyses of long-term data show that earlier treatment with combination sodium phenylbutyrate (PB) and taurursodiol (TURSO) (AMX0035; Amylyx Pharmaceuticals, Cambridge, MA) affected the clinical course of amyotrophic lateral sclerosis (ALS). Participants in the phase 2 CENTAUR trial (NCT03127514) who had PB/TURSO vs placebo treatment in the 24-week double-blind period had longer times until tracheostomy, permanent airway ventilation (PAV), and first hospitalization after diagnosis observed during the open-label extension portion of the trial. 

As of the analysis cutoff with the longest follow up of 35 months, median key event-free survival duration was 4.8 months longer in participants originally randomized to PB/TURSO vs placebo, and median tracheostomy/PAV-free survival duration was 7.3 months longer.
 
“We’re encouraged by the positive data that we continue to collect from the CENTAUR study on the potential benefits of AMX0035 for people with ALS, including a lower occurrence of first hospitalization based on data collected up to 35 months following administration,” said Machelle Manuel, PhD, head of global medical affairs, Amylyx. “These results suggest that AMX0035, if approved, may help extend the time before greater care needs arise—a meaningful benefit for those living with ALS and their loved ones. We will continue to explore other potential benefits of AMX0035 during the follow-up period and plan to evaluate its potential in other neurodegenerative diseases.”  

Overall, reported rates of adverse events and discontinuations were similar between people treated with PB/TURSO and placebo during the double-blind period; however, gastrointestinal events occurred with greater frequency (≥2%) in those who received PB/TURSO.