Not to mention a slam-dunk for approval for NTM :-)
The trial failed to meet its primary endpoint - more patients on Arikace than in the placebo arm achieving a one-point reduction in bacterial density.
All that would be needed for that to happen was a freak number of patients in the placebo arm suddenly showing an improvement - after six(?) months of non-improvement before the study.
The primary endpoint WOULD have been the number of patients converting to culture-negative - but they didn't think it likely that could happen after just twelve weeks of therapy.
11 out of 44 converted to culture-negative - a full 25%. A massive result.
Enough for Insmed now to apply for Breakthrough Therapy designation.
Don't be scammed by attempts to pass this off as a failure - it's a resounding success.
After reading the "Guidance for Industry Expedited Programs for Serious Conditions––Drugs and
Biologics" document at the FDA website, I have to agree with them that Arikayce qualifies as a Breakthrough Therapy.
Arikayce has shown, IMO, that "the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”
This is exciting! It looks like my calls are worthless, but that money only would have gotten me 75 more shares, which is why I went with options in the first place.