Public release date: 18-Jun-2013 [ | E-mail | Share ] Contact: NCATS Office of Communications ncatsinfo@mail.nih.gov 301-435-0888 NIH/National Center for Advancing Translational Sciences (NCATS) The National Institutes of Health has awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, including Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia. The collaborative pilot initiative, called Discovering New Therapeutic Uses for Existing Molecules, is led by the National Center for Advancing Translational Sciences (NCATS) and funded by the NIH Common Fund. The process of developing a new therapeutic is long and difficult. The average length of time from target discovery to approval of a new drug is more than 13 years, and the failure rate exceeds 95 percent. This failure rate means, however, that many existing partially developed compounds could be advanced to clinical trials more quickly than starting from scratch. "With thousands of diseases remaining untreatable, there is a sense of urgency to accelerate the pace at which discoveries are transformed into therapies for patients," said Health and Human Services Secretary Kathleen Sebelius. "This program helps forge partnerships between the pharmaceutical industry and the biomedical research … Continue reading →

CAMBRIDGE, MA--(Marketwired - Jun 19, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced updated data from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results at 84 weeks showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes. Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51. After 84 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) showed a statistically significant treatment benefit of 46.4 meters (p0.045) when compared to the placebo/delayed-treatment cohort (n=4). The eteplirsen-treated patients in the mITT population demonstrated less than a 6 percent decline (20.5 meters) from baseline in walking ability. After experiencing a substantial decline earlier in the study, … Continue reading →

Background: Duchenne muscular dystrophy, a progressive muscle disorder that occurs in males, causes a gradual decline in muscle strength. This progressive decline is associated with the development of scoliosis. Previous studies have shown that the use of glucocorticoids slows the progression of scoliosis, but it is unknown if the spine remains straight in the long term. We examined if glucocorticoid treatment has a long-term effect on the prevalence of scoliosis. Fifty-four boys who had been diagnosed with Duchenne muscular dystrophy while they were still walking were enrolled in a non-randomized comparative study of the glucocorticoid deflazacort. The families of thirty boys elected for them to use glucocorticoid treatment and the families of twenty-four boys elected for them not to have this treatment. The boys were matched for important baseline characteristics including age and pulmonary function. Every four to six months, they were examined for the development of scoliosis, and the duration of follow-up for surviving patients was fifteen years. Because surgery was recommended for spinal curves measuring >20 on sitting posteroanterior radiographs, a curve of this magnitude was used as the definition for a patient developing scoliosis. Five boys (21%) in the non-treatment group and one boy (3%) in the … Continue reading →

CAMBRIDGE, Mass. (TheStreet) -- A handful of pre-teen boys suffering with Duchenne muscular dystrophy have now been treated with Sarepta Therapeutics' (SRPT) eteplirsen for 84 weeks. The boys are still walking, defying the debilitating and progressive nature of a disease that would be forcing them into wheelchairs without treatment. As with previous updates from Sarepta's eteplirsen study, no significant safety problems were reported. No serious adverse events, no hospitalizations or treatment discontinuations. Well, there was one disruption in the study but it was actually more evidence of eteplirsen's profound benefit for DMD patients. Here's how Sarepta describes it: One boy in the placebo/delayed-treatment cohort was not able to perform the 6MWT at the Week 84 clinic visit due to a physical injury unrelated to treatment, and therefore had no 6MWT data captured at the Week 84 time point. The boy has recovered from the injury, continues to be ambulatory and is expected to be evaluated on the 6MWT at future clinic visits. Read more from the original source: Sarepta's Eteplirsen Walk-A-Thon Now Stretches For 84 Weeks … Continue reading →

From the first work by Drachman et al. that recognized a benefit from corticosteroids1 in boys with Duchenne muscular dystrophy through the excellent randomized controlled trials (RCTs)2-5 of the 1980s and 1990s, the recognition that daily corticosteroids benefit boys with Duchenne muscular dystrophy is clear. More than forty years of work with hundreds of boys have demonstrated that corticosteroids prolong walking and improve the strength of boys with Duchenne muscular dystrophy. Deflazacort, available in Canada and Europe, is not approved by the U.S. Food and Drug Administration (FDA) for use in the United States. However, there are extensive long-term positive data showing that it is effective in boys with Duchenne muscular dystrophy6,7. Although weight gain with deflazacort is less than that with daily prednisone, other side effects, including loss of height, loss of bone density, and cataracts, do occur. The study by Lebel et al. clearly adds to these data with, to our knowledge, the longest continual follow-up of a treated cohort. However, the known side effects have led to many alternative regimens and drug formulations. Intermittent corticosteroid dosing regimens, including the twice-weekly or alternate-day regimens, show promise in minimizing side effects8-10, but long-term follow-up is not yet available. Despite … Continue reading →

Shares of Sarepta Therapeutics Inc. jumped Wednesday, after the drug developer said its lead candidate, a potential treatment for Duchenne muscular dystrophy, continued to help patients in an extended mid-stage trial. THE SPARK: The Cambridge, Mass., company said patients taking the treatment, eteplirsen, saw less than a 6 percent decline in walking ability through 84 weeks. Some patients have been taking the drug for the study's entire duration, while others took a placebo for the first six months. The patients were evaluated with a six-minute walking test, and the drug appeared to be well-tolerated with no serious side effects. CEO Chris Garabedian said in a statement the company has now demonstrated walking stability for over a year and a half in boys who are 11 years old on average. He noted that many boys with Duchenne muscular dystrophy have lost their ability to walk by then. THE BIG PICTURE: Duchenne muscular dystrophy is a rare and fatal genetic disease that causes increasing muscle weakness and affects one of every 3,500 boys worldwide. Sarepta said death usually occurs before patients reach age 30. Sarepta has no drugs on the market. Eteplirsen is its most advanced product. SHARE ACTION: Up 4 percent, … Continue reading →