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 →

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 →

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 →