Zynerba Pharmaceuticals (ZYNE)

[lumpy9200]

Totally agree that it's going to take time now. Also agree with your point that it would take great results to move the stock higher. I really expected a gradual move higher throughout the day Tuesday, as investors looked past the bad headline and realized that Zygel being statistically significant for 80% of patients in the trial could be good enough for approval. I was wrong on that thought clearly.

I do wonder something though, and am curious if anyone has insight into this: has a drug ever gotten approval after failing to meet its primary endpoint in the clinical trial, but the secondary info showed it warranted approval for a specific subset of the patient group? It's not like we're talking 10% of all patients....this was 80% of the trial participants and 60% of the world's population with FX. And this is a condition with zero drugs currently approved to treat it. But will the FDA care, or simply say "run another trial with ONLY patients with full methylation", and then we'll talk.

There is something that bothers me here, in the name of full disclosure. After all the testing and first two phases of the clinical trials, it never became clear to management that good results were only happening with the sickest patients (well, not "sickest" but rather with more of the condition)? And if 80% of the trial participants were all in that group, they didn't think to maybe have the primary endpoint be catered to them?? It's possible they thought the worse off the person, the worse Zygel would do. But if that's the case, then why stack your trial with 80% of those people? I would think that after a deep dive into the phase II trial results, it would become evident that those full methylation patients were getting the most benefit. I'd have then gone to the FDA and asked to change the primary endpoint of the phase III trial to be "show stat sig benefit to those with full methylation, and secondary endpoints of showing benefit to those less afflicted by FX". Would the FDA have allowed them to change the criteria? Maybe not, but it had to be worth a shot.

Fragile X: zero approved drugs on the market, and Zygel shows real clinical benefit to 60% of all people who have it. And you can test BEFORE being prescribed the drug to see if you're in that 60% or not. If the trial was set up differently, we're looking at slam dunk approval and ZYNE stock through the roof. Disappointing to think of it that way. I'm holding tight though, hoping they do get approval for the 60% and really hoping for good things with ASD.

All just my opinion, as always