The main goal was to slow decline on a 48-point ALS scale of 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting, and breathing. In 24 weeks, patients who received a placebo decreased by 2.32 points more than those who took the drug combination, which resulted in a slower 25% decline during that period for patients. who have received the treatment.
The open-label extension study involved 90 of those patients, 34 of whom were in the placebo group, who started taking AMX0035 approximately seven months after those who had started taking AMX0035. The researchers told the FDA that those who received the longest treatment had an average of 4.8 months longer before being hospitalized, having a ventilator, or dying.
“This is the first time we see a benefit in both function and survival in an ALS clinical trial,” said Dr. Sabrina Paganoni, principal investigator of the clinical trial and specialist in neuromuscular medicine at Massachusetts General Hospital’s Healey Center for ALS
“If access is delayed, patients in my clinic today may never be given the time and function they could have. Delaying access is not a risk we should take, “said Dr. Paganoni said.
FDA reviewers, however, identified many problems with both the Phase 2 clinical trial and the open label extension. They stated in the disclosure documents that the identified benefit was “statistically borderline and may not be persuasive enough to allow for a single study-based determination of efficacy.”
Dr Emily Freilich, leader of the FDA team, said at the hearing that the results of the clinical trial suggesting that the drug slowed the decline on the functional scale of ALS were not “very persuasive” and that the trial’s secondary measures, including muscle strength, respiratory capacity and whether patients were hospitalized – were not “generally in favor” of the benefit.
FDA officials said evidence, even from the extension study, doesn’t show that the therapy can help patients live longer. Dr Freilich said patients who received placebo and never switched to AMX0035 survived for a median of 1,295 days, while patients who received AMX0035 survived for more than 96 weeks for a median of 1,237 days.