Pasteur: "Has the new Ebola outbreak virus been sequenced?" Yes it has, ironically, by the Pasteur Institute in Lyon, France which confirmed the Ebola strain as Zaire ebolavirus, the same virus that Sarepta's drug treats.
Pasteur: You repeatedly say that Sarepta "can't give its [Ebola] drug away", stating that distribution must be part of a clinical trial. You support your opinion on your experience of "working in the filed of virology for 25 years." Obviously, your virology experience and opinion holds little credibility. Sarepta created its Ebola drug in 4 days and, with permission of the FDA, gave the drug to a clinician who had pricked herself with a needle contaminated with the Ebola virus. Fortunately, she did not contract the virus and the drug was never used - but it would have been used if the infection occurred. The FDA can grant provisional use in the case of an emergency (and what Ebola infection would not be an emergency?). It does not take a clinical trial to release the drug to people that need it. That's a fact.
According to TipRanks, Janney's Sarepta analyst is accurate only 48% of the time. Flipping a coin about the success of a company provides a better prediction.
What?? Krieg came along after the trial ended (though the expansion trial continues). He had nothing to do with the planning of the Ph2b trial. All the rest of your post must be bs, too.
Same with Merrill Lynch: "Sarepta Therapeutics Inc. (NASDAQ: SRPT) was started as Neutral at Merrill Lynch, but the firm issued a $27 price objective (versus a $21.57 close) and it also said that it has significant potential but is very risky."
There are nearly 2000 boys with duchenne in the United States. About 13 percent or those, or roughly 260 boys, may be helped by skipping exon 51 with eteplirsen. Scores of new cases appear every year. If the confirmatory trial enrolls about 70 patients, that leaves nearly 200 boys amenable to treatment outside of the trial. If the annualized cost of eteplirsen treatment is $300,000, that's a potential of $60 million a year, plus an additional $20 million each year for new children with duchenne. That's a good start, and a conservative estimate, just for eteplirsen in the United States. Some won't get the medication, others will come in from Canada and abroad.
I'll bite. The FDA is now more accommodating than ever in advancing this drug, providing encouraging guidance for both an NDA and post-approval confirmation, and by all counts engaging in dialogue with all players like never before seen in the drug approval process. Recent data continues to show statistically relevant evidence of drug benefit. The kids on eteplirsen are doing much better than natural history would expect of duchenne boys of like ages. SRPT will launch immediately upon approval, providing an income stream to fund the confirmatory trial. Clinical development of follow-on exons may not require as much time as for exon 51. The company and the FDA will have loads of data to streamline the process.
I believe SRPT price action today is a result of a decline in the general biotech market. Nothing more. ISIS down more than 5% on no news, Tetraphase down more than 15% on no news, Sarepta down more than 9% on no news. Most of the biotech market is in the red today.
The loss of three boys in the duchenne community this week - Tyler 23, Robert 16 & Steven, 14 - is a sobering reminder of the immediate need for a drug is slow down this deadly disease. The ages of these boys reminds us that disease progression is so variable and, ultimately, final.
Laurie, recent patents are out there; one of Sarepta's morpholino synthesis changes was patented just in 2012. More importantly, modifying and fine-tuning manufacturing of morpholinos is an ongoing process. Some of the refinements are patented, others are not (but remain trade secrets now that morpholinos are in the public domain).
Modulation of exon recognition in pre-mRNA by interfering RNA structure. Van Deutekom. June 24, 2014. Assigned to Academisch Ziekenhuis Leiden (a primary patent licensing source for Prosensa - sort of like University of Australia is for Sarepta).
Sarepta has already come up with a more efficient process. Add patent research to your DD. You're behind in your game. Again.
Yes, Simp. Tred cites the parents of Billy and Max and others about how their boys have been improving in the past couple of months. You cite no parent - not one - who says their child's condition is worsening.
Nonsense. At most it would mean that speculation continues. The market knows no more than we do about the progress of the trial or FDA communications.
Starfe, you should go. Hilary Clinton is speaking. Many many politicos and scientific leaders from the Middle East in attendance. Sarepta is a partner.
Prosci, What is your sense on how well the boys are doing at week 144? Have you heard (or observed) anything to suggest that one or more boys has had a significant increase or decrease in function since the 120 week testing? Thanks.