Synaptogenix Inc (SNPX)

[InTheTrenches]

I think what could really push this thing forward is the fragile X Society. Incredibly well organized group of motivated parents who will have no problem pushing the FDA to expedite any potential cures. They can gain a lot of media coverage. Even though results are likely a ways off, I'm very excited to get this kicked off. I posted well over a year ago that I felt Fragile X might be an easier route to approval.

Very well-said, coach.

If we see positive results, I could see the potential for this to gain big momentum while the society/parents push the FDA. This could become similar to SRPT and their treatment (eteplirsen) for Duchenne Muscular Dystrophy. If successful in the Phase 1 trial, I could easily see the potential for Breakthrough Designation, which would be the fastest path to any approval, through a single placebo-controlled phase 2 trial.

It would be interesting if they add brain scans (Yale study/paper?) to this trial, to actually measure if synapses are normalizing and growing.

One caveat--if bryostatin hasn't been used in kids before (has it?), they may go very slowly in the dosing (dose-escalation?) component of the trial.

Another caveat--it may take 3 months or more to design a trial, assuming meetings and input from the FDA via the orphan drug designation program.