Sabrina Paganoni, MD, PhD: AMX0035 Phase 2 Performance in ALS

“The randomized trial was relatively short, only 6 months, and so it’s very important to consider its results in combination with the open-label extension that provides longer term outcomes­­.”

Two agents, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), make up the investigational combination approach to amyotrophic lateral sclerosis (ALS) from Amylyx Pharmaceuticals. PB has been used to treat urea cycle disorders and TUDCA to treat dissolve gallstones and some forms of liver disease for some time.

The combination was recently investigated for the treatment of ALS in the phase 2 CENTAUR trial (NCT03127514), which included more than 100 patients and was conducted by Sabrina Paganoni, MD, PhD, co-director, Neurological Clinical Research Institute, Massachusetts General Hospital, and colleagues. The full results of the study are expected to publish before the end of the year, but in the meantime, Paganoni provided her insight to NeurologyLive in a recent interview.

Paganoni discussed her takeaways from the positive top-line data to this point and why this trial was so important for the ALS community. Additionally, she noted how the ongoing open-label extension data will continue to inform the group about AMX0035 in this population, and what the extension will be observing in the study population.