Another irrational, meaningless piece by AF - he says there is new risk, because the FDA won't have as much "urgency" to approve Etep if Drisa is approved first. Drisa's PDUFA date was always 2-months ahead, so why is this a "new" risk? He doesn't say the AdCom panel would have less urgency - and practically why would they - despite the rhetoric, their job is not to compare the two drugs, but to decide whether the clinical evidence at hand is sufficient to deem the drug safe and effective. Again, AF doesn't even scratch the surface on the clinical evidence - i.e. the difference between the drugs from a safety, dystrophin production, or even 6MWT results - or even mention that Etep is seeking a conditional approval with their confirmatory trial already substantially enrolled, while Drisa has no commitment to do further studies. AF's whole argument on why the FDA will approve Drisa is that it is "politically expedient" - what a hack!
It is a valid point, since SRPT has followed up with substantial additional data since their official submission date (4th biopsy, additional 6MWT data, including natural history analysis, etc.), while BMRN has admitted that they have no follow-up data to provide since their submission.
It is for a different mutation ("nonsense mutations") and has no bearing on the DMD markets that SRPT is pursuing (i.e. Exons 51, 53, 45, etc.).
No doubt - the Baird write-up captures the true risk facing SRPT - the capricious and irrational behavior of the FDA. They seem to acknowledge that Etep is clearly the better drug based on the data at hand, but depending on how the FDA challenges the AdCom committee, the dystrophin production and safety benefits of Etep may not be brought to the forefront of the decision process. I would have to believe that regardless of how the FDA frames their discussion points, the AdCom members have the ability to challenge the safety and dystrophin production issues of Drisa on their own. An earlier post on this board referenced an Oppenheimer note about potential conflicts that the AdCom members may have, so who knows how that will play into the process. To me, the most interesting aspect of the Drisa AdCom will be the public comments - will there be any parents or doctors coming out to fight for this drug? We know there will be a strong contingent of parents and doctors coming to Etep's AdCom.
Given the price action of the last week, I can't imagine anyone predicting anything with this stock price - we are in bizarro world at this stage. Regardless, your point about Drisa having a "short-term" benefit is not valid - one, because the volatility in 6MWT measures over a short period of time render any short-term changes meaningless (the boy could have been tired or having a bad day at baseline and then have a better day a few months later?) - you need multiple tests over a long period of time for the 6MWT to be valid and even at that it is a flawed test in this patient population; and second, this is a chronic disease, so the idea that they would approve a drug for short-term use does not make sense - neither does the often repeated idea that it might be approved for a subset of the patient population.
"His family is the primary reason serepta is receiving this much notoriety" - what a moron. Sarepta has been receiving "notoriety" and support from several vocal DMD moms who have boys in the Phase II trial for over three years. For you to post an inflammatory message of "Doesn't Work" for a kid that has only been on the drug for 3 months, while parents of boys that have been on the drug for 3+ years and are convinced that the drug works, makes you a complete tool. Move along and stop spreading your misinformed bs!
With evidence from the Phase II trial that dystrophin production does not ramp up until 12-24 weeks, I doubt they would cloud the issue by presenting 24-week 6MWT data from the new trial - you wouldn't expect to see meaningful separation until 36+ weeks.
I seem to recall they were playing around with the dates of the 6MWT to coincide with the 4th biopsy procedures, so the boys wouldn't have to travel two separate times in the same month.
The back-to-back AdCom speculation was overblown - practically, the intent of the scheduling was to minimize the travel/logistics of getting the panel members there. The idea that there would be a "side-by-side" comparison of the drugs is ludicrous - the panel is there to objectively review the evidence of the drug at hand independently from any other filings. It is perfectly feasible that if the FDA was thinking of doing both AdComs in November, the importance of the new data that SRPT has disclosed pushed the timetable back - either way, the PDUFA date would not have changed - i.e. even if the AdCom for Etep was held in Nov and was overwhelmingly positive, the FDA would not have made a decision on approval any earlier than Feb.
The fact that the "market" seems to react so violently to what objectively are non-issues sets us up for another potential gyration when Drisa has their AdCom. Will a negative AdCom for Drisa be good for SRPT's share price or will the "market" interpret it as the FDA not being ready to approve either drug. Unfortunately, if Drisa does get a positive review, it most likely will be a negative for SRPT, even though a potential 2-month head start for an inferior drug should be meaningless in the long run.
It is a non-issue - even in the unlikely event she gets elected, congress will still be controlled (or split to the point of a stalemate) by the Republicans. Frankly, they should go after these companies that take old, off-patent drugs and jack the prices up by 50+ times - we all pay for that through higher taxes/premiums - but that has nothing to do with drugs in the orphan disease space. Congress put the rules in place to provide incentives for companies to do the R&D for rare diseases (instead of creating the next generation cholesterol pill) and they are not going to pull the rug out from under the companies that actually create viable treatments.
I'm trying to decide if the upcoming data in Nov will be significant. The Leerink presentation gave me the impression that the CB-839 data will be from the single agent expansion cohort in solid tumors - they are just initiating the combination studies later this year, so initial data from those won't be available until mid-2016. The single agent data presented earlier was underwhelming - mostly stable disease with one clinical "response" - and the analyst and the CALA CSO seemed to imply that the combo studies were where CB-839 would show more substantive results? Am I reading this right?
The fact that BMRN's CEO would tout the recent decision as and "important win" tells you a lot about how he operates - posture and spin are more important than facts, which is why he is on Cramer's show to begin with. The fact that the analysts following SRPT effectively ignored the decision speaks volumes, as well. Common sense would tell you that Etep and Drisa are so completely different that a Drisa patent would be irrelevant to Etep's IP standing.
I agree that the FDA doesn't need an AdCom to reject Drisa - it would be a very quick meeting when they ask whether they have any Phase III clinical evidence their drug actually produces dystrophin. How can you argue your drug is effective when you can't even prove it works as designed in the production of dystrophin. The only definitive clinical evidence that BMRN has on Drisa is that it has significant safety issues for a drug that is needed to be administered for the life of the patient.
I meant clinical (i.e. in human studies) proof of concept. They have ARC-520 and ARC-AAT already in clinical trials and hopefully ARC-HIF2 (the first w DPC) by late next year.
Given the open-label design of the Monarc trial, any proof of concept data should put the shorts at bay. It is well understood that ARWR doesn't have the capacity to take a HBV treatment full through development/marketing, so a partnership is likely soon after proof of concept is acheived. Unless I am missing something, ARWR seems to be in the clinical lead (with respect to other RNAi HBV players), so who else is big pharma going to target if they are interested in the HBV space? I would hope by Q3 next year the ARC cards start falling into place - even if not, there is enough value in the DPC platform to carry the day.
For someone who purports to "understand corporate finance", you've stepped off the ledge here. Anyone with half a brain knows it is typically better for shareholders for the company to prudently use debt, rather than dilute the shareholders' interests, but the availability of debt to SRPT at this stage of the game is severely limited, or would be extremely expensive, probably including some form of warrant or other dilutive feature. First off, no bank or bond issuer is going to take the regulatory risk that SRPT is facing with respect to getting Etep approved - regardless of how much they believe in the technology, the risk of any FDA delay could put the company's ability service the debt at risk. You never see development stage biotech companies take on significant levels of debt, unless they are secured by real assets - real estate and personal property - which is what SRPT pledged to secure the $50M loan commitment they got earlier this year. Second, that same $50M loan commitment provides strict covenants preventing SRPT from taking on additional debt, so they really did not have an option to borrow.
With respect to the timing of this offering, there are some that would argue they should roll the dice and wait until after the FDA decision in Feb - that would be foolhardy, especially given SRPT's history with the agency and the fact that they are blazing a new trail here with Etep. While I would have liked to see them wait until after the AdCom , which is generally anticipated to be a positive event, the reality is that these investment banking firms want to be able to give their clients a potential short-term pop from an upcoming milestone/event and were probably pushing the company to do the offering before the AdCom. In the long run, another 3.5M shares (less than 10% of the outstanding shares) is not going to be that meaningful in the long run.
The drug works - by next spring the clouds should clear around what the FDA hacks want to see for approval and you'll have approval in the EU. In the mean time, this market could do anything to the share price?
It is hard to imagine three more clueless commentaries. The first guy speaks of a possible "placebo effect" - not sure how your body spontaneously produces dystrophin on a placebo effect and when it comes to the 6MWT, I don't think placebo effects last 3+ years? He also speaks of the FDA probably wanting to see more data - duh, that is why they are filing for accelerated approval and running a confirmatory trial! The other two clowns simply choose BMRN based on their history in the rare disease space - no discussion of the differences in the drugs - not even a mention of safety issues. You would think they would be embarrassed to write about something they obviously know little about and/or have spent little time investigating?
cdavis - Greenspan led the charge to keep the derivatives markets unregulated in the early '90s and didn't even change his tune after the collapse of the hedge fund Long Term Capital Management in 1998 (they used derivatives to leverage $5B of capital into a trillion dollar position on interest rates). I saw an interview of Greenspan after the collapse of the housing market and he admitted he really didn't understand how CDO's worked - he laughed about it, as if his job had nothing to do with it? A complete tool!
His boss/predecessor should have been the first convicted - Greenspan spent twenty years pushing for less and less regulation of banks and financial markets, sat idly by while the sub-prime/housing bubble grew in front of his eyes and then seem dumbfounded when the whole system collapsed. He has caused more structural damage to this country's economy and banking system than anyone in history.