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My thoughts on SRPT stock going forwardNot sure how people can say the Drisa briefing docs were bad but "could have been worse". Really? They essentially say all 3 trials failed and that there is no evidence of exon skipping or dystrophin production. To boot, they said a few kids almost died from AE. The FDA has already made it's decision on Drisa. The AdCom is a sham. The voting questions are proof of this. Four questions. Three of them are based on each of the three trials w/ leading questions saying how shitty the results were. They then ask if these trials help or hurt approval chances. Almost unanimously the voters will say either neutral or hurts the way the questions are worded. The final question on Dystrophin is a no brainer that it will be voted negative. The drug doesn't produce any! If the FDA wanted AdCom cover to approve this drug, they would have had a question that said "Based on totality of the data, do you think there is evidence of efficacy?" or "Based on totality of the situation, should Drisa be approved?" How will BMRN spend hours talking about pooling all 3 trials w/ their data mining matter if there is no voting question that lets the panel voice their opinion on this? Further, how will patient and advocate testimonials, no matter how persuasive, matter to these 4 voting questions? They don't. The FDA has removed this variable. This has potential repercussions for SRPT, too, and I'll discuss that later. So what has the FDA decided? Clearly I think they have decided to reject. Everything points to this. BMRN has been cocky with them and has done nothing they asked. The trials show no evidence of efficacy despite being tested in 100s of boys. LARGE N is not so great if LARGE N fails. I put a 90% chance Drisa is rejected and a 10% chance it gets approved w/ a boxed warning. It's immoral and unethical to give this drug to someone. I would not give Drisa to my son. I'm not talking my book, this is the truth. Ask yourself the same question...low chance of efficacy with high chance of major AE plus painful weekly injections. If this was sugar water, the risk/benefit would be better! At least with sugar water you would get the mental placebo affect which has been proven to help patients w/o risking any safety. As far as Tuesday goes, I don't know what we will learn. It seems like a pointless exercise. The votes on all 4 questions will be universally negative. We will hear BMRN talk about their crazy data mining and we will hear the FDA bash everything just like they did in the docs. At the end of they day, nothing will make it seem like they will approve this drug. BMRN PDUFA date will be big, obviously. If it's rejected, SRPT will go up huge. This "what's good for BMRN is good for SRPT" has finally been debunked, I think, given what the stocks did on Friday. I'd like to go on a bit of a tangent: It really pisses me off when people say (mostly sell side) that "FDA has to approve at least one of these drugs due to activist outcry". No, they don't. And the drugs aren't the same. It's not like FDA is going into this decision trying to see which shitty drug is "less shitty" so they can please the public. These drugs COULD NOT BE MORE DIFFERENT. Drisa: --Well tested in extremely large N, failed study after study. The P1 study is a joke and not a fair comparison to Etep P2. --No evidence of dystrophin --Abysmal safety profile that can cause death Etep: --Efficacy tested in only 12 boys showing remarkable results. The two boys who went non-ambulatory were identical twins. Even including them, 3+ years out given the baselines the results are remarkable. No "large N failed study". Tested in safety now in 100+ boys. According to company, no major AE's. Would be shocked if they lied to us about that. --Dystrophin evidence open to debate but SRPT has done MUCH more in proving that dystrophin is present at meaningful levels. Plus, all boys showed exon skipping. --Safety - excellent as noted above. No painful injection. My point is, it's no comparison. I do not believe, if Etep was similar to Drisa, that it would still be a "FDA must approve 1" type of situation. Let's keep it real here: Only reason Drisa is even getting reviewed right now is because Etep Mom's made such a stink that FDA had to review these drugs. Drisa wouldn't have had a review if Etep didn't exist. Think about that - it's true. So what happens with Etep's docs? I expect them to slam it on efficacy but only so much you can do with 12 boys. There is definitely evidence of efficacy but they will say non-conclusive. With safety, we better hope its clean! Cause in risk/benefit - that risk is damn important when the benefit is unknown. Finally, the dystrophin data is a total wild card. That said, I think it has to read better than Drisa. SRPT did everything the FDA asked. They got 4th biopsy, they re-scored the others. When FDA and SRPT agreed to non placebo confirmatory trial, it's my opinion that was the day #AA was granted - as long as no safety issue popped up in new trial. It's all about RISK/benefit. It was always about this. FDA was right for making SRPT start P3 before allowing NDA to be filled, in some ways. They wanted a large N - not for efficacy but for safety. They now have it. It will be a 3 year delay if FDA rejects Etep. It's is highly likely that the confirmatory trial shows efficacy. I have no idea how much efficacy, but I would be shocked if it didn't show a clear benefit to the control group. Yes, it's not a double blinded study and the control isn't even the same exon. But the FDA blessed this design!!! As they should. The FDA will look so bad rejecting this safe drug if when P3 reads out it is efficacious. I mean super crazy bad. Politically, it's a no brainer giving Etep #AA. But where the bears get it wrong is FDA is not caving to political pressure. Granting this drug #AA is the RIGHT THING TO DO based on the totality of current evidence and the RISK/benefit. Bears seem to think shareholders and advocates want a non-proven, unlikely to work, drug railed through process because they are bullying the FDA into "some" treatment, no matter whether it works or not or whether it's safe or not. This is just wrong. If I was sitting here arguing for Drisa approval, that's exactly what you should think - based on that evidence. By the way, I don't need to remind you that Prosensa and now BioMarin is a public company. If I thought Drisa worked and had a favorable Risk/Benefit, I would have been long Prosensa and I would be long BioMarin now! I have no conflict of interest. I can buy any stock I want to! In conclusion, I put a 95%+ chance of Etep approval and a 90% chance of Drisa rejection. If Drisa gets approved, it will be after a 3 month delay for labelling REMS and include a black box warning. Because of this, I see 100% of market share going to Etep and no pushback from insurance on reimbursement. There is essentially nothing I could learn on Tuesday that would change my mind from these odds. There is no voting question that could put the FDA in a tough position, rejecting a drug where the outside experts voted to approve. Also, BMRN can suck a cock. What an awful company that should be ashamed at itself. |
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Msg # | Subject | Author | Recs | Date Posted |
11288 | Re: My thoughts on SRPT stock going forward | alt225 | 1 | 11/21/2015 8:12:58 PM |
11289 | Re: My thoughts on SRPT stock going forward | bagbest | 0 | 11/22/2015 12:11:57 PM |