Ann: Positive Interim Study Results from OLE Study in ALS, page-34

My son is a biostatistician (and has a PhD in biotech) but has never been involved in clinical trials or the pharmaceutical industry. He has no knowledge of PAA other than today's announcement, which I asked him to comment on. His response, FWIW, is as follows:

"It’s a small number of patients and crucially the matched controls are from historical data and not a simultaneously studied group that were randomised into the control group (I think it has to be this way for phase i and for a fatal disease). They certainly show better survival on their treatment than the matched controls, but how they did the matching matters a lot. Maybe there have been other improvements to MND treatment that their patients received that the matched controls didn’t - you just have to take it on faith that the controls are a good match."