By Dow Jones Business News, March 17, 2014, 07:28:00 PM EDT By John Kell Shares of Prosensa Holding NV ( RNA ) jumped Monday after the Dutch biotechnology company reported "encouraging" data from a Phase II study for a Duchenne muscular dystrophy treatment. The compound, drisapersen, is in development to treat DMD, a rare, fatal muscular disorder. DMD is one of the most prevalent rare genetic diseases globally, according to Prosensa, which said the disorder affects as many as 1 in 3,500 boys. The disorder is almost always fatal, and there is no approved disease-modifying therapy. On Monday, Prosensa disclosed data from a 48-week U.S. study in 51 boys with DMD. The results indicated that boys in the higher-dose drisapersen group experienced stabilization and even improvements in their muscle function and physical activity for the 24-week treatment period and maintained that improvement during the 24-week follow-up phase. "The maintenance of the clinically meaningful treatment benefit in the 24-week follow-up phase is very encouraging evidence for the drug's ability to produce prolonged stabilization of disease," said Craig M. McDonald, principal investigator and a professor of pediatrics at the University of California. Investors cheered the news, sending Prosensa's shares up 34% to … Continue reading →

(Nasdaq:RNA), the Dutch biopharmaceutical companyfocusing on RNA-modulating therapeutics for rare diseases with high unmet need,today reported financial results for the full year ending December 31, 2013,and provided an update on the next steps for its exon-skipping platform for thetreatment of Duchenne Muscular Dystrophy (DMD). 'As we have previously stated, we are encouraged by initial findings fromfurther analyses of the aggregate drisapersen data that suggest that treatingearlier in DMD and treating longer shows a delay in the progression of thedisease. These data give us the confidence to engage patient groups, clinicalexperts and regulators to explore a path forward for drisapersen,' said HansSchikan, CEO of Prosensa. Mr. Schikan continued, 'While the past few months have been a very importanttransition period for Prosensa, our primary focus has not changed, which is toimprove the lives and outcomes of boys with DMD. At the start of the year weregained the rights to drisapersen and retained rights to our otherdevelopment-stage compounds for DMD from GlaxoSmithKline (GSK). We arecurrently working closely with GSK to transfer data and other elements coveredin the collaboration as soon as practicable and within 120 days of the January12 effective date of the agreement. Concurrently, we are focused on thecontinued analysis of the … Continue reading →

--------------------------------------------------------------------- Prosensa Committed to Defining a Path Forward for Its Lead Product, Drisapersen LEIDEN, The Netherlands, 2014-03-18 12:00 CET (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (Nasdaq:RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported financial results for the full year ending December 31, 2013, and provided an update on the next steps for its exon-skipping platform for the treatment of Duchenne Muscular Dystrophy (DMD). 'As we have previously stated, we are encouraged by initial findings from further analyses of the aggregate drisapersen data that suggest that treating earlier in DMD and treating longer shows a delay in the progression of the disease. These data give us the confidence to engage patient groups, clinical experts and regulators to explore a path forward for drisapersen,' said Hans Schikan, CEO of Prosensa. Mr. Schikan continued, 'While the past few months have been a very important transition period for Prosensa, our primary focus has not changed, which is to improve the lives and outcomes of boys with DMD. At the start of the year we regained the rights to drisapersen and retained rights to our other development-stage compounds for DMD from GlaxoSmithKline (GSK). We are … Continue reading →

Acceleron Pharma Inc.'s (XLRN: Quote) collaboration partner, Celgene Corp. (CELG: Quote) is scheduled to present interim data from an ongoing phase 2a study of Sotatercept in patients with end stage renal disease on hemodialysis at the National Kidney Foundation 2014 Spring Clinical Meeting. The meeting will take place on April 22-26, 2014. XLRN closed Monday's trading 6.08% lower at $42.50. Alphatec Holdings Inc. (ATEC: Quote) said that its consolidated net revenues for full year 2013 rose to $204.7 million from $196.3 million in 2012. Looking ahead to 2014, the company anticipates annual revenues of $208 million to $215 million, representing approximately 1.6% to 5% growth over 2013. The company also noted that it has settled the OrthoTec vs. Surgiview litigation. Under the terms of the settlement, Alphatec has agreed to pay OrthoTec $49 million over the next 7 years in cash, with an initial cash payment of $17.5 million. ATEC closed Monday's trading 15.11% higher at $1.60. In after-hours, the stock gained another 4.37% to $1.67. Amgen Inc. (AMGN: Quote) announced that a phase III trial evaluating its investigational drug Evolocumab has met its primary endpoint of low-density lipoprotein cholesterol reduction in patients with homozygous familial hypercholesterolemia. In the trial, … Continue reading →