Rise and shine, everyone, another busy day is on the way. This morning is getting off to a fabulous start, though, as a delightfully warm sun is enveloping the subdued Pharmalot campus, where the official mascot is scurrying about the grounds and the sounds of the street are increasingly wafting through our windows. As always, we are quaffing some cups of stimulation — golden French toast is our choice du jour — and assembling some tidbits for your pleasure. So, time to get cracking. Hope you have a smashing day, and do stay in touch. We always enjoy your tips and insights. …
U.S. Food and Drug Administration staff indicated that results from a single clinical trial of an experimental ALS drug developed by Amylyx Pharmaceuticals (AMLX) “may not be sufficiently persuasive” to support approval, STAT writes. The Office of Neuroscience reviewers criticized the data, which showed the drug demonstrated a modest slowing of disease progression but no survival benefit, as well as the conduct of the study. But the agency noted it can exercise “regulatory flexibility” to approve drugs for serious diseases with unmet medical needs. The FDA is convening a panel on Wednesday to review the trial and vote on whether the data are strong enough to support approval.