The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital spoke to the CENTAUR open-label extension and AMX0035’s development.
What we’re interested in is to look at these outcomes in the context of the open-label extension. That will provide more data to really be able to tell if AMX0035 has an impact on survival.
Recently, NeurologyLive spoke with Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Massachusetts General Hospital, about the publication of phase 2/3 data on AMX0035, an investigational combination agent for amyotrophic lateral sclerosis (ALS).
The data from the trial, dubbed CENTAUR, revealed that the sodium phenylbutyrate–taurursodiol combination was safe and well-tolerated by patients with ALS. It also displayed signs of efficacy, with positive changes displayed on the ALS Functional Rating Scale-Revised (ALSFFRS-R). However, there is still a need to assess the agent’s efficacy further, as the trial was not powered to show efficacy on the crucial measures for ALS, such as lung function and muscle strength. Although, Paganoni noted that the numbers did trend in AMX0035’s favor.
In this interview, Paganoni, who was the lead investigator on CENTAUR, detailed the status of the open-label extension study of AMX0035 and its analysis, and expanded on what she hopes future assessments might reveal about the agent’s impact on survival in ALS.
Paganoni S, Macklin EA, Hendrix S, et al. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020;383:919-930. doi: 10.1056/NEJMoa1916945