jenn mcnary @jennmcnary 2h2 hours ago
Sareptas 144 week ind data looking in strongly different than Duchenne natural history.
Joe @GantosJ 1m1 minute ago
$SRPT #WMS Poster /Pulmonary Function Is Stable Through Week 144 in DMD Patients
John Hall @johnhallnj 10m10 minutes ago
Have to put things in perspective given mean data previously released for 144. Youngest pt in study appears to drag group at 144 (29.3).
John Hall @johnhallnj 4m4 minutes ago
@johnhallnj I think the indiv $SRPT data is supportive as I view his 144-6MWT as an outlier. PBO delayed, no surprise
Nicholas Skiadas MD @NSkiF1j1 24m24 minutes ago
$SRPT the individual data shows REMARKABLE EFFICACY and safety with eteplirsen. @US_FDA WILL 100% approve. Shorts @TomSilver39 better cover
Nicholas Skiadas MD @NSkiF1j1 28m28 minutes ago
@ValueTraderS $SRPT the "drops" you refer to are minimal from baseline drop sustained before the kids got drug for 1st 24 weeks! "Cmon man"
Keep in mind:
1. eteplirsen is not a cure. drug is clearly slowing down the progression of the disease. no drug has been able to do this
2. eteplirsen is safe so the risk to patients is nil. prosensa had very little treatment affect and there were 80% AE in patients.
3. cancer drugs that show just a 2-3 month slow down of the disease are celebrated and the company is slapped with a multi-billion market cap but srpt's unprecedented results for a deadly disease of children are scoffed at
4. if eteplirsen is given to these children at a young age, before the disease has progressed, it could be turned into a becker's like dmd or even something milder
5. no risk to the fda delaying this drug. even if the drug does not work as well for all patients, the alternative is a fast, painful death. it doesn't make sense to me.
Exactly. Even if RNA gets approval, SRPT will be the drug of choice since it's not only safer (no adverse events for SRPT while 80% for RNA patients) but it works much better. I don't see how eteplirsen won't control the market.
Not necessarily. I don't believe the company is allowed to release this information prior to their presentation. Most likely part of the guidelines which is very normal. Often companies presenting at conferences on the weekend will not release the data until that weekend and often the data is good. I've seen it many times. Seems like WMS is saving the best for last.
You sound very intelligent. I "hear" what you are saying.
Most expect acad to easily get FDA approval and/or be bought out but short interest is higher than srpt. I think a lot of it is smoke and mirrors by big money to scare the little guy
m: CDC head compares Ebola to AIDS, Washington Post says
The world must take action to prevent Ebola from becoming "the next AIDS," Tom Frieden, the director of the Centers for Disease Control and Prevention said today at the world Bank, according to The Washington Post. "In my 30 years in public health, the only thing that has been like (Ebola) is AIDS," Frieden stated, the newspaper said. Drug companies that are working on experimental Ebola treatments include Tekmira (TKMR), Sarepta (SRPT), BioCryst (BCRX), Chimerix (CMRX) and NewLink Genetics (NLNK)
We heard from Dr. Francesco Muntoni from the Dubowitz Neuromuscular Centre about how much dystrophin is expected to be needed to stabilize the muscles and result in features that are like Becker muscular dystrophy (which is the goal of exon skipping drugs). He described a 20% dystrophin production as a reasonable target but also discussed the variability seen in Becker muscular dystrophy, and that some children with Duchenne who get benefit from exon skipping drugs may respond like a mild Becker and some like a more severe Becker.
Dr. Jerry Mendell from Ohio State provided an overview of the eteplirsen clinical program. He reviewed the biopsy data quantifying dystrophin production (dystrophin production was the primary endpoint), the walk data (the 6-minute walk test was the primary clinical endpoint), and the pulmonary data. The pulmonary data was particularly interesting since Dr. Mendell presented the 144 week data in all 12 patients, which showed a plateau of pulmonary function tests from baseline, showing that the children’s pulmonary status has remained stable over the 144 weeks.
There were a few interesting questions from the audience. One was about the ability of eteplirsen to get into the heart or brain. Eteplirsen cannot do that, and for exon skipping drugs to get to the heart or brain would require some changes to the molecule so it could get into those areas. There was also a question about the ability of muscles that already have a lot of fat and fibrosis to have significant treatment effect, with general agreement that the uptake of exon skipping drugs would be expected to be lower in muscles with more fat and fibrosis.
I have been in and out of this one for a while but I now have a core position that I plan holding long going forward. Some have suggested a stock price of $30+ as fair value. My question is that if ACAD hits on other indications, and the market potential is easily over $5+ billion, why couldn't it be valued around where ALXN is valued now? That would give ACAD a market cap of about $300+. Assuming they do hit on the other indications, then the revenues they would bring in would be higher than ALXN. Even if we just assume one indication, I look at stocks like ISIS and ALNY and wonder how they are trading higher than ACAD.
As per Jenn McNary
jenn mcnary @jennmcnary 8m8 minutes ago
This 3-year natural history study serves as a stark contrast to the experience of the boys on eteplirsen.
The 6 minute walk test has been recently chosen as the primary outcome measure in international multicenter clinical trials in Duchenne muscular dystrophy ambulant patients. The aim of the study was to assess the spectrum of changes at 3 years in the individual measures, their correlation with steroid treatment, age and 6 minute walk test values at baseline. Ninety-six patients from 11 centers were assessed at baseline and 12, 24 and 36 months after baseline using the 6 minute walk test and the North Star Ambulatory Assessment. Three boys (3%) lost the ability to perform the 6 minute walk test within 12 months, another 13 between 12 and 24 months (14%) and 11 between 24 and 36 months (12%). The 6 minute walk test showed an average overall decline of −15.8 (SD 77.3) m at 12 months, of −58.9 (SD 125.7) m at 24 months and −104.22 (SD 146.2) m at 36 months. The changes were significantly different in the two baseline age groups and according to the baseline 6 minute walk test values (below and above 350 m) (p
Roth Capital Still Sees 51% Upside For Sarepta’s Stock
October 1, 2014 2:29 PM EDT by Editor Corey Williams in Exclusively Published, Healthcare
In a research report issued yesterday, Roth Capital analyst Debjit Chattopadhyay maintained a Buy rating on Sarepta Therapeutics Inc. (NASDAQ:SRPT) with a $34 price target, which represents a potential upside of 51% from where the stock is currently trading.
Chattopadhyay said, “SRPT’s investigational influenza compound (AVI-7100) has moved to multiple ascending dose testing in healthy volunteers. Importantly, AVI-7100 is based on SRPT’s advanced PMOplus platform, which could lead to lower dose and better COGS compared to the parent PMO chemistry. However, our short-term focus is on the initiation of patient dosing in the DMD pivotal study , hopefully prior to 3Q14 corporate update in November.”
as per a tweet
Skyworks weakness a buying opportunity, says Craig-Hallum
Craig-Hallum views the sell-off in Buy rated Skyworks as overdone and would buy shares aggressively on weakness. The analyst continues to expect Skyworks to report another good quarter and for shares to work higher.