Four-Year Eteplirsen Results Show 10 of 12 Children with Duchenne Muscular Dystrophy Are Still Walking
BY DAN HURLEY
VANCOUVER—Ten of 12 children with Duchenne muscular dystrophy (DMD) were still walking four years after treatment began with the investigational drug eteplirsen, compared with only one of 13 historical controls with DMD, researchers reported here at the AAN Annual Meeting on Tuesday.
Eteplirsen, an antisense oligomer, is designed to skip the mutation in exon 51 of the dystrophin gene in order to allow production of the protein, at least in partly functional form. The oligomer is infused weekly intravenously.
During the first stage of the clinical trial, which lasted for 24 weeks, the 12 children were randomized to eteplirsen 50 mg/kg, eteplirsen 30 mg/kg, or placebo. The primary endpoint was an assessment of dystrophin in skeletal muscle as measured by Western blot and immunofluorescence. The randomized controlled phase was followed by an open-label extension in which all 12 patients received eteplirsen 30 mg/kg or 50mg/kg.
At 36 months, the 12 eteplirsen-treated patients demonstrated a statistically significant advantage by being able to walk 151 meters more than the 13 external controls (p
Pmo-s are third generation antisense ppmo-s are super potent pmo plus is very good also. Sarepta owns all the pmo plus ,all ppmo-s 100 percent could be a 20 billion dollar market .
Replace Janet Woodcock ...if she can't do any better than she has..' I am not impressed at all.
Sarah Roden — March 31, 2016, 9:17 AM EDT
Moving a pipeline drug from inception to commercialization can take years for biotech companies. Analysts weigh in on upcoming catalysts for Sarepta Therapeutics Inc (NASDAQ:SRPT) and Intercept Pharmaceuticals Inc (NASDAQ:ICPT) as both companies try to move their lead candidate drugs one step closer to fruition.
Sarepta Therapeutics Inc
Eteplirsen, Sarepta’s lead pipeline candidate to treat Duchenne Muscular Dystrophy, or DMD, is being deliberated for FDA approval. Stephen Brozak of WBB Securities explains that he is optimistic that the drug will be approved by the FDA in May due its safety profile and the existing need for a DMD treatment.
Brozak highlights the drug’s safety profile, noting, “there has been no concern about the safety of the drug and increasing evidence that eteplirsen can delay the progress of a fatal disease.” With no significant safety concerns in the air, the advisory committee, or AdComm, to the FDA will be focusing on the drug’s efficacy. The analyst explains that the AdComm should evaluate the drug on two standards: “delay of disease progression” and “increased presence” of the disease.
Furthermore, the analyst underscores the involvement of the medical community, explaining that a group of prominent doctors invested in DMD wrote to the FDA, encouraging them to approve the drug. The AdComm to the FDA will meet on April 25, and the analyst is “hopeful” that the body will “render a positive recommendation.” The FDA usually follows the recommendation of the AdComm but does not have to. Consequently, Brozak expects “the FDA will provide conditional approval for eteplirsen on or before May 26, 2016.”
In light of the upcoming AdComm meeting and the analyst’s belief that eteplirsen will receive FDA approval, he reiterates a Strong Buy on Sarepta with a $40 price target.
Sarepta Therapeutic (SRPT) PT Raised to 'Street-High' $60 at Oppenheimer. April 6, 2016 6:29 AM EDT. . Oppenheimer analyst Christopher ...
9:15 Novel Phosphorodiamidate Oligomers (PMOs) for the Treatment of Genetic and Infectious Diseases
Bruce_WentworthBruce Wentworth, Ph.D., Vice President, Biology, Sarepta Therapeutics
PMOs are being tested in advanced clinical trials for the treatment of patients with Duchenne muscular dystrophy (DMD), a rare, X-linked disease that results in progressive muscle loss and premature death. Research has shown that for other disorders, including viral and bacterial infection as well as rare diseases such as Pompe disease, modified PMOs may be more appropriate due to their potential for enhanced delivery and tissue targeting. The PMO-based technology has the potential to be a versatile, modifiable, and widely applicable treatment in any number of disease states.
Watch your kids die slowly and see what you would do. Myself congress needs to be aware of the FDA's games.
Growth from here short term...could be any price in 3 years ,the sky is the limit
You have a lot of dystrophin production a certain way and the FDA was trying to measure it from a different way. you may want to try to understand completely before screaming 1 percent dystrophin because than is really not the only way the to measure at all.
New data that confirms older trial data. will cause massive upside.
Based on the only game in town and exon 51 only for now. The whole world wide open for eteplirsen. Add more exons and really add much larger market cap.
Sarepta owns most of the patents on pmo antisense and more/ just one example here and there is many more that genzyme is getting into.
Genzyme Corporation discusses the exciting new data he and his colleagues presented on antisense therapy that is in development to treat patients with Pompe disease.
Clayton N, et al. Antisense oligonucleotide‐mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease.
Sales for exon 51 world wide is worth a lot but this is the same drug on every exon ,just sequence of the same drug is changed to match the needed exon to skip''same drug '' worth a lot''
They also did not treat this as a rare disease at all....The fda made the rules for the trial ,Sarepta followed and then turned on it all...I believe money gain and loss was the real motive and nothing else...looks like phase 3 trial will maybe clear up more.
Seems to be mounting evidence..........based on recent events......