We are excited to share some promising data regarding the CENTAUR trial evaluating AMX0035 in people with ALS on overall survival analysis, published today (October 16, 2020) in the neuromuscular disease journal Muscle & Nerve.
The survival analysis followed each CENTAUR participant for up to three years from the start of the trial. AMX0035 is the first ALS therapeutic to demonstrate both a statistically significant survival and functional benefit in ALS in a randomized controlled trial. The analysis found that the group of participants who were randomized to AMX0035 at baseline had a 44% lower risk of death compared to the group who originally received placebo over the duration of follow-up.
As previously reported in the New England Journal of Medicine, the CENTAUR study also met its primary efficacy end point of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) versus placebo over a six-month period. AMX0035 was generally well tolerated with a manageable safety profile.
Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital, and also our keynote speaker at the upcoming Les Turner Symposium on ALS on November 9, was encouraged by the data.
“In this trial, we have seen promising functional and survival benefits,” she said. “To add, these results were seen on top of baseline use of approved ALS therapies. I am so excited about the potential of this drug for the people living with ALS.”
The Scientific Advisory Council (SAC) of the International Alliance of ALS/MND Associations states, “With regard to self-treatment regimens, the SAC strongly encourages individuals to speak with their ALS physician before considering. These promising results only pertain to compounded PB and TUDCA at the concentrations and purity tested in AMX0035.”
Please see the links below for more information about the analysis and to register for upcoming webinars that explain AMX0035 and the CENTAUR trial in more detail.
Amylyx Pharmaceuticals Announces Publication of CENTAUR Survival Data – October 16, 2020
NEALS Webinar: Wednesday, October 21 at 1pm CST
“Long-Term Survival of Participants in the CENTAUR Trial,” a webinar with Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR Trial, to discuss the ongoing results of the CENTAUR Trial.
Amylyx Webinar: Monday, October 26 at 6pm CST
Amylyx will be hosting a patient/caregiver webinar to present the CENTAUR data and answer questions. Click here to register.
We will continue to monitor and share information in the months ahead on the rapidly changing results coming from ALS scientific and clinical research.
Together toward a cure,
Andrea Pauls Backman, Chief Executive Officer