|Healthcare Review: GW Pharmaceuticals, Dicerna Pharmaceuticals, Lifesciences, Rockwell Medical, Amicus Therapeutics|
|By Staff and Wire Reports|
|Monday, 30 June 2014 15:11|
Shares of GW Pharmaceuticals ($GWPH) pop on a 2x surge in volume in early trading. Investors seem to like what they heard during the company's Epidiolex presentation at the Dravet Syndrome conference this past weekend. Several weeks ago, the company reported preliminary results from a clinical trial demonstrating a significant reduction in seizures in the the Epidiolex-treated cohort.Epiolex is designated an Orphan Drug for Dravet and Lennox-Gastaut syndromes.
Demonstrating the upside benefits of being a thinly-traded stock, Dicerna Pharmaceuticals ($DRNA) jumps on turnover of 353K shares, a 2x surge in volume. The upside move is being driven by the news that DCR-PH1, its therapeutic candidate for primary hyperoxaluria type 1 (PH1), inhibits the gene implicated in the pathogenesis of the disease. Investors' enthusiasm might be a bit premature considering that the encouraging results were observed in a preclinical animal model.
The FDA approves Edwards Lifesciences' ($EW) ClearSight noninvasive blood flow and blood volume monitoring system. The device uses a cuff placed on the outside of the finger and custom software to provide automatic real-time data to clinicians for patients unsuitable for arterial lines.
The FDA waives its PDUFA fee for Rockwell Medical's ($RMTI) NDA for its iron replacement therapy Triferic. The Small Business Office of Government Contracting originally denied the waiver on the grounds that the company did not meet the proper size determination criteria. Rockwell appealed the decision and won. The Treasury Department will issue a $2.2M refund check to the firm in the next few weeks.
Amicus Therapeutics ($FOLD) says that top-line data and statistical analysis from the clinical trail (Study 012) evaluating its oral small molecule pharmacological charperone migalastat HCL as a monotherapy for Fabry disease compared to standard-of-care enzyme replacement therapies (ERT) Fabrazyme and Replagal should be available next quarter. The 18-month primary treatment period was completed in Q2.