Category Archives: Muscular Dystrophy Treatment

MEMPHIS, Tenn.--(BUSINESS WIRE)--GTx, Inc. (Nasdaq: GTXI) today announced the publication in the journal Human Molecular Genetics of results from preclinical studies supporting the potential efficacy of the Companys SARMs, and in particular GTx-026, for Duchenne Muscular Dystrophy (DMD) treatment. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness, affecting approximately 18,000 boys in the United States. Studies examined the treatment of castrated wildtype mice, x-linked muscular dystrophy (mdx) mice, and dystrophin and utrophin double mutant (mdx-dm) mice with GTx-026. Other SARMs in the Companys portfolio, GTx-024 (enobosarm) and GTx-027, showed similar positive effects on muscle mass, function, and histological characteristics. DMD typically afflicts boys around three to five years of age followed by declining physical functions before attaining puberty. Current treatment options for DMD rely on corticosteroids to reduce inflammation, but unfortunately the prolonged use of corticosteroids results in hyperglycemia, osteoporosis, and muscle wasting, which are all counterproductive in this disease, said Ramesh Narayanan, Ph.D., Director, Center for Cancer Drug Discovery and Associate Professor, Department of Medicine, University of Tennessee and a consultant for GTx, Inc. We hypothesize that an androgen receptor agonist may reverse musculoskeletal complications and extend survival in these boys, and in preclinical … Continue reading →

The particular form of muscular dystrophy that afflicts 6-year-old Trayton Backer is the result of busted proteins in the longest gene of his X chromosome. In Montana, it makes Trayton one of kind. Nationwide, only 13 percent of people withmuscular dystrophy have Duchenne's Trayton's diagnosis. And of those, Trayton is the only one in the state. A new treatment, approved late last year by the U.S. Food and Drug Administration, could drastically improve Trayton's life and make his form of muscular dystrophy more manageable. "This treatment really is a breakthrough,"said Trayton's doctor, John Binder, a pediatric neurologist at Billings Clinic. Trayton was diagnosed when he was 4. "I cried," said Emily Oldaugh, Trayton's mom. "The first thing you think of is, 'Oh, my kid's gonna be in a wheelchair.'" Oldaugh had know for a couple of years that something wasn't right. Trayton walked late, his calves were larger than normal and he had trouble crawling up and down stairs. As soon as the doctors were sure it was Duchenne muscular dystrophy, they began to treat it as best they could, she said. But the broken gene at the heart of Duchenne muscular dystrophy has made it frustratingly difficult to treat. … Continue reading →

Clinical-stage biotech company Capricor Therapeutics Inc., has announced positive six-month results from a randomized 12-month Phase 1/2 trial in patients with Duchenne muscular dystrophy (DMD), which was designed to analyze safety and exploratory efficacy. DMD is a rare, genetic disorder that often occurs in boys. It involves progressive muscular weakness and patients often experience frequent falls, have trouble getting up, or performing daily tasks such as eating, as well as learning disabilities, and treatment options are limited. Cardiac disease is the most common cause of death among those with DMD, who often dont survive past their twenties, and early results from the trial showed statistically significant improvements in measures of cardiac and upper limb function in patients treated with the cell-based therapy called CAP-1002. CAP-1002 is comprised of allogeneic cardiosphere-derived cells, or CDCs, which are a type of progenitor cell. The HOPE trial, involved 25 patients, aged 12 years an older, with DMD who had cardiomyopathy, or heart disease secondary to DMD. Thirteen patients were randomized to receive a single dose of CAP-1002, while 12 received usual care. The cell therapy was infused into the three main coronary arties, with a total dose of 75 million cells. MRI assessments showed … Continue reading →

Boston Business Journal Sarepta hikes forecast for Duchenne muscular dystrophy drug sales ... Boston Business Journal Sarepta Therapeutics CEO Ed Kaye said Thursday that he plans to step down from his post at the Cambridge biotech at the end of the year. Kaye, who was ... and more » Continue reading here: Sarepta hikes forecast for Duchenne muscular dystrophy drug sales ... - Boston Business Journal … Continue reading →

Posted April 26, 2017 19:43:27 A US teenager who can walk because of a drug created in WA has joined a campaign for human trials to be run in Australia. Two researchers from Perth's Murdoch University created the drug that has kept 16-year-old Billy Ellsworth, from Pennsylvania, on his feet but the drug is not yet available to Australian families. Billy has Duchenne muscular dystrophy, a rare and fatal genetic disease, which eventually wastes every muscle in the body. It affects one in 3,500 children, mostly boys, and leaves most wheelchair bound by the age of 12. Many people with the condition do not live past 25. "It's good, it feels good to be walking for this long and I'm glad I can," Billy said. The teenager has been one of 12 boys in the United States on a six-year trial of Exondys 51, a drug created by two researchers in WA. The drug targets a specific group of gene mutations which causes the fatal disease rendering it effective in slowing down muscle wasting in about 10 per cent of people with the condition. Terri Ellsworth said what it had done for her son had left her, at times, speechless. … Continue reading →

SOUTH PLAINFIELD, N.J., April 20, 2017 /PRNewswire/ --PTC Therapeutics, Inc. (PTCT) today announced it has completed its acquisition of all rights to Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017. "We are pleased the acquisition was completed ahead of schedule, following early conclusion of the antitrust review period," stated Stuart Peltz, Ph.D., chief executive officer, PTC Therapeutics, Inc. "We've been engaging with key stakeholders in the DMD community to understand their needs and are working to make Emflaza commercially available as soon as possible. PTC is committed to bringing this important therapy to patients with DMD. Our goal is to enable access for eligible patients irrespective of insurance status, and we look forward to discussing commercial launch details on our upcoming quarterly earnings call." Financial terms of the acquisition include a total upfront consideration of $140 million paid to Marathon Pharmaceuticals. Marathon is also entitled to receive payments from PTC based on annual net sales of Emflaza beginning in 2018, which PTC expects will range as a percentage of net sales between the low to mid-20s … Continue reading →

Shares of Capricor Therapeutics (CAPR) were gaining by 11.65% to $3.46 on heavy trading volume Tuesday morning, after the clinical-stage biotechnology company reported positive results from its randomized phase I/II HOPE clinical trial in Duchenne Muscular Dystrophy. The positive top-line results from a safety and exploratory efficacy analysis of six months of data from the randomized 12-month trial shows the CAP-1002 product as an investigational candidate for the treatment of patients with DMD. DMD is a rare, life threatening disease for which there is little treatment, the company said. "In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial. These results clearly support the conduct of a confirmatory clinical trial in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people with this progressive and fatal disease, one that is poorly met by current options," John L. Jefferies, M.D. and the principal investigator of the HOPE trial said. (What will move markets this quarter and how should investors position themselves ahead of time? Jim Cramer sat down with four of TheStreet's top columnists recently to get their views. Click here … Continue reading →

April 24, 2017 The Spinraza group had a 3.9 point improvement at Month 15, while untreated children had a mean decline of 1.0 point Biogen presented Phase 3 study data onSpinraza (nusinersen) that showed a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset spinal muscular atrophy (SMA) vs. untreated children. The findings were presented at the American Academy of Neurology (AAN) annual meeting in Boston, MA. The CHERISH study was a multicenter, randomized, double-blind, sham-procedure controlled study to evaluate the safety and efficacy of Spinraza in children with later-onset SMA (n=126) aged 212 years. In the end-of-study analysis, children who took Spinraza had a highly statistically significant and clinically meaningful improvement in motor function with a treatment difference of 4.9 points in the mean change from baseline to Month 15 in the HFMSE score (P=0.0000001). Compared to baseline, children who took Spinraza had a 3.9 point average improvement at Month 15, while untreated children had a mean decline of 1.0 point. The primary endpoint data at the end-of-study analysis was consistent with the data seen at the interim analysis. Other endpoints, including achieving new motor milestones and upper limb motor function, were also consistently … Continue reading →

Shares of Sarepta Therapeutics (SRPT) were up nearly 2.3% to $33.35 in earlytrading on Monday. A Barron's article over the weekend stated there's a "good chance" that sales of Sarepta's Duchenne muscular dystrophy treatment, Exondys 51, "will ramp up this year and in 2018." Cambridge, Mass.-based Sarepta is scheduled to announce its first quarter numbers on Thursday. Meanwhile, shares of Paratek Pharmaceuticals (PRTK) were up 3.2% to $22.70. The Boston firm unveiled a collaboration with with Zai Lab (Shanghai) to support the development and commercialization of omadacycline for patients in China. Paratek has granted Zai Lab an exclusive license to develop, manufacture and commercialize omadacycline for the greater China territory. The companies will form a joint steering committee to review and oversee development, manufacturing, and commercialization plans. Paratek will receive an upfront payment of $7.5 million and is eligible for additional milestone payments related to development, regulatory, and commercial milestones. Paratek will also be eligible to receive royalty payments on sales of omadacycline in the territory. Among the other biotech movers were shares of PDL BioPharma (PDLI) . The stock was up 2.5% to $2.07 after the Incline Village, Nev.-based company said Monday said it has entered into a settlement … Continue reading →

About Duchenne Muscular Dystrophy DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by an abnormality in the dystrophin complex, a structural element that plays a critical role in muscle fiber integrity, which leads to chronic skeletal and cardiac muscle damage. Patients with DMD typically die in their twenties, most commonly due to heart disease. The incidence of DMD is estimated to be one in every 3,600 live male births, and DMD is believed to afflict approximately 15,000 to 20,000 boys and young men in the U.S. About CAP-1002 CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac progenitor cell. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. CAP-1002 is currently being evaluated in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack and in the Phase I/II HOPE Clinical Trial in boys and young men with DMD. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other medical conditions. Capricor's lead candidate, CAP-1002, is … Continue reading →