Ahh, there it was , bo....In the TB 4 arm 8 out of 22 pts got total wound healing and closure. That is a huge win. because they don't state how many got "partial" and not "full" closure.
BUT?? An issue? You can also see that 5 pts got closure on the placebo. Which is odd, because the placebo is probably just some water based jelly or whatever base was used with TB 4 comparator. but there is no drug that works that for EB, otherwise it would be out there already, if a placebo gel got 5. But if a placebo gel worked, all you would need is KY Jelly to treat EB....but all we read is that it is impossible to treat?
We need more detailed info. Especially on the placebo end. However???? It was a 7 year trial. The FDA now only asks for EB an OPEN LABEL PHASE 3 approval trial (see Shire). if RGRX is held to teh open label that Shire seeks approval on...for only a REDUCTION in wound....then if TB 4 healed totally and safely 8 of 22 patents?? It is a massive success!
If teh FDA just looks at TB 4, just as they will what they approved w/ Shire.....curing 8 pf 22 is a massive win and an approveable drug. And RGRX has stated that they may keep and market TB 4 for EB 100% themselves. The pts and hospital centers to treat EB are easily reached via EB databases. Hardly any marketing/advertising needed.
Ahhhh, maybe figured it. On feb 6, 2013 the FDA CHANGED the trial protocol AFTER the trial was done, with the important (efficacy) outcome changed to THIS:
Number of Participants Whose Wounds Have Healed [ Time Frame: 56 days ] [ Designated as safety issue: No ]
Wound healing means that the wound has closed without any drainage.
Since the trial was COMPLETED, they had to report what was found on placebo. but on Feb 6 2013 (see clinicaltrialsdotgov), the FDA basically threw OUT the placebo end of it..and the secondary outcome (efficacy) measure simply became "number of patients whose wound healed". So by that NEW FDA approved measure, TB 4 healed 8 out of 22 EB patients! A huge win. But the measure was only for wounds all closed and healed. We got 8 out of 22 healed. But they don't say how many pts got what Shire looks for in thier FDA blessed EB Phs 3, which is only a REDUCTION in wound size. I'd like to know if many pts saw a REDUCTION in wound with TB 4, but not totally healed. If TB 4 did that in many, and healed 8.... it woudl be seen as almost a miracle drug for EB kids....either cures the wound or reduces it greatly.
if this get's out and is EXPLAINED better by RGRX, our short has a HUGE problem. He is a MM, and he is "naked" short...I bet 2-3 million shares worth. Of 70,000 shares trade Friday, he shorted 55,000 - on the BID too!
Also, it would be nice to see which dose of tb4 worked best. It may be that all of the higher dose resulted in closure, it which case it really would be a huge win. Its hard to say without details. But, considering company has been given up for dead, shorts risk has just increased greatly....
Funny, I was just going to post that same thought. There are THREE doses of TB 4 in EB trial:
"Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days"
If they had 22 pts on TB 4, call it 7 w/ 0.01%, 7 w/ 0.03% and 8 on 0.1%. if I had to guess, the 7 pts at the TB 4 level 0.01% didn't heal. Better chances at 0.03% and 0.01%..of which there were about 15 pts. And 8 were totally healed. That is huge win, IMO. Also, the protcol measures were ONLy for complete healing - closed wounds. There is no "mark" for partial or "almost" full healing. The protocol says the wound has to be fully closed for efficacy. It will not count if the blister is "almost" closed" or over half "closed". we just don't know all details.
If our TB 4 Phase 2 was okayed.... as Shires EB Phase 3 design (by the FDA).... with no placebo and Open label...the results shown by TB 4 in EB would be a huge win, and our shares would zoom higher. We need an explanation of the 5 pts placebo response. If the FDA review ignores our placebo (as they do with SHire in Phs 3 for EB), then we have a real winner, and IMO, THIS is the news RGRX was doing the "band aids" for. Not a partner deal....but they go for EB approval on the Phs 2..if they can explain better. And RGRX has stated before they probably won't "partner" EB. Because the specializing hospitals are so few, the EB docs are few.... and there is a complete EB Patient Registry Database...RGRX can market it 100% themselves..very easily. What will the FDA do???