To be clear, the FDA's issue is with the prior leadership... They/he made many of the questionable calls on the trial shift and his attitude and approach with the FDA was toxic. It seems that Dr. Kay has taken a very different approach. That said, the trial was done when he took the wheel. Remember... The FDA did collaborate on the confirmatory trial design. It does include 2 placebo arms (45 and 53) The eteplirsen arm does not have a placebo arm design. This would seem to indicate some real flexibility.
The 6-7 votes was actually an 11-2 win. Every "no vote" sited trial design as the real issue. Every "no vote" agreed that the drug produced dystrophin. (2 or the 7 said they believed the amount was too low to be meaningful) 1 of the 7 "no votes" said: "It's clear that the drug produces dystrophin, there is no clear threshold for how much is needed to deliver benefit, and it's real clear that the patients are getting a benefit. That one should have been a clear YES vote. The wording of the question confused the real issue. Q7 was a vote for full approval... No one expected this one to pass.
Farkas is so nearvous, he can't speak. He's way out there on his claims and the experts know this. The technical problems seem to be getting worse...
All of that has little to do with the question on the table. Although these examples demonstrative the value of the RCT approach, they do not fit into the rare disease space where the boys are dying and placebo controlled arms are nearly impossible to enroll and likely medical unethical!
It important to note Dr. Temple's known bias... He has been a long-time, very valued player at the FDA. That said, he has also been very rigid in his old school views of maintaining the rule of "2 placebo controlled trials" to secure approval. He has also made statements regarding his concerns over the AA pathway and the risks of speeding these processes along. All in all, a solid contributor, but not willing to take moderated risks to speed access to care for rare diseases and unmet needs.
We have just heard from 2 of the leading world experts in DMD, as well as the hyper focused clinical team from Sarepta. And of course, the most passionate advocate in the world. Now we will listen carefully to an FDA staff member who is trained in opthamology. He does reportably have a relationship with a DMD expert, Dr. Hoffman. His friend Hoffman has been an outspoken opponent to Sarepta's drug over the years. This motivation could be rooted in the fact that his underlining research and testing methods are not being used by the company... Aside from that, Dr. Farkas's interpretation of the same data set that the experts just reviewed seems to be very different in the Briefing Documents. I ask, where did he develop a bias or how does his expertise allow him to land on different conclusions?
So... Invest $100K for a chance at making $400-500K. The downside is lose $30-50K. I used this same investment poker strategy with an HGSI investment in 2009. It worked just fine! $1.00 to $34.(.52 cents on the low) With SRPT, I've seen too many pops from $4.00 to $15, $15 to $45, back to $15, back to $54, back to $18, back to $40, repeat, repeat, repeat... (Ironically, .52 cents was also the trading low for AVII before the 6x reverse split.)Could this be the last ride? You decide. Good luck!
Outcomes for speculative investors: If you invest $100K in SRPT your outcomes in May could include...
1) FDA Ad Comm and FDA totally reject the NDA and give no hope for a future approval. (This seems unlikely, as the FDA has partnered with the company to design and implement the confirmatory trial and suggested the company use Natural History data to evaluate effect.) That said, anything can happen... PPS Target: $7
2) FDA issues a CRL or further delays the approval until the confirmatory data fully validates the findings from the Phase 2 trial and 4 year continuation data. (This seems possible, but the strength of safety data coupled with efficacy in 10 of 12 boys should warrant a strong look.) PPS Target: $7 then a slow recovery to the $15 range as data trickles out.
3) Grant AA and closely partner with the company to monitor ongoing confirmatory trial and additional exon trials to make sure that no more disease progression occurs before the "rest of the story" is known. PPS Target: $55 pop within days and a steady climb to the $75-90 range. M&A talks could quickly change these targets and any additional news on the EU market progress would blow this stock up.
Let's hope not. $75-100 would be the value range, based on the US market potential. The EU market access is still unknown, so any value there would be difficult to peg.
I don't believe the stock can be pushed any higher than $16-18 without substantial news. That said, I believe that AA surprise may come in the next 2-3 weeks (without an Ad Comm). This news would torch the shorts and send the pps back to the $45-55 level within 2 days. Once the analysts weigh in, it will move higher. Outside factors include: EU access news and M&A discussions. Either of these bring new highs to the story.
That about sums it up. I've reviewed a number of briefing docs in my day and have never seen a set that were positive. By design, they point out the weakest elements in the NDA to promote discussion. It's not uncommon for some of the areas challenged to already be clarified. These documents are drafted and written over the course of months... Let's hope the rational minds prevail and give these boys a chance. Other therapies will likely be added to etep to extend their "legs" and life.
Gary, Generally speaking, Farkas is doing his job. I don't like the mis-representation and cherry picking of the data, but his job is to point out the challenging issues that need discussion. As for his dirty little secret... He is somewhat chummy with one Dr. Eric Hoffman. EH has been mixing up the waters for AVII/SRPT for years, as they do not use his dystrophin measuring process. It's not the gold standard, yet. All in all, I believe they both know that this drug is producing dystrophin and adding benefit. The drop may be a blessing for many.