jlapier, It takes ~ 2 months of patient screening before the first patient is dosed. This was also mentioned in one of the recent articles on the eteplirsen and Essence trials. the official start date for the Essence trial per the clinical trial website is July. Most biotechs PR the start of a trial when the first patient is dosed. In the early days of AVII and Denis Burger he announced the start of the Avicine PhaseIII trial before any patient was dosed. No patient was ever dosed or enrolled in that trial. Old-timers on this board may remember those dark days. We will see what the FDA thinks.
You don't have to believe it. Ed Kaye revealed this factoid during his conversation with the Duchennes advocacy groups. Same discussion where it was revealed that Essence would substitute for P3 eteplirsen.
Baird's Brian Skorney has reported that the FDA is in receipt of the dystrophin data [13 boys] from Phase III trial
The initiation of this trial is scheduled to dose it's first participant in July which means that they began screening boys for this trial in May. Is the FDA awaiting to announce AA on the day Sarepta doses it's first patient? Maybe. The FDA is on record as requiring this trial to "start" before granting AA for eteplirsen. Many in biotech world define the initiation of a clinical trial as the first patient dosed. I contend that once the FDA approves the first clinical site and they begin screening that trial has been initiated. We are about to find out. If Sarepta announces the start of the Essence trial this Tuesday that would be the clearest signal yet that the FDA will grant AA later that afternoon.
Nature Journal recently published animal studies using vivo-PMOs and naked [bare] PMOs in ALS. Bare-PMO's resulted in decreased levels of SOD1 protein levels up to 90% and remarkably 80% in the brain. The researchers referenced their PMO SMA animal studies research and achieved "wild-type" levels of rescued SMA protein.http://www.nature.com/articles/srep21301
Beaver, SRPT has already submitted the Phase III WB dystrophin data without public disclosure. It is similar to when SRPT had FDA Adcom members [April 25] sign nondisclosure agreements upon receipt of non disclosed baseline dystrophin data. A company has some leeway if the data is still considered "non disclosed. I don't agree with this interpretation but obviously Ed kaye believes it is in the best interests of Sarepta to not publicly disclose before the FDA makes their AA decision.
Chaz, I believe Ed Nash is the analyst at SunTrust. Nash has a very long history and relationship with Avi Biopharma/ Sarepta management. I've spoken with him several times over the last 15 years, all regarding AVII/SRPT. His animosity towards the company originates with the original AVII CEO, Denis Burger. He claims that Burger lied to him and since he has been on a mission to denigrate this company. If there is one [maybe two] analyst[s] an investor in SRPT should ignore is Ed Nash [and Jeffries, Wang].
Cops, Kaye presented slide confirming that Essence trial is confirmatory for eteplirsen. Public acknowledgement.
FDA must approve changes in protocol and in this case probably with much back and forth with FDA
Disagree. The exon 45/53 Essence trial is now the confirmatory trial for eteplirsen which means that Sarepta will have close to 3 years of uninterrupted eteplirsen sales with close to 100% market penetration in U.S.. If "shorts" believe that they should stick with their "short" position or re-establish after the meteoric rise to $100 pps on AA approval then they are cognitively delusional.
Brian Skorney is a good biotech analyst who covers Sarepta and has received "background" before from Sarepta. He did not report that he knew what the decision was.