Factor VIIa-CTP(OPKO Health)

March 9, 2016 By Mark Terry , BioSpace.com Breaking News Staff Although almost everything from the direction the wind blows to the phases of the moon seems to affect stock prices, one thing that can directly affect biotech stocks are U.S. Food and Drug Administration (FDA) decisions. The Motley Fool takes a look at three upcoming decisions that biotech investors might want to keep in mind. Insys Therapeutics Chandler, Ariz.-based Insys Therapeutics, Inc. (INSY) is awaiting a decision by the FDAOn April 1 for oral marinol. The company is focused on a variety of marijuana-derived drugs. Marinol is being evaluated for use in treating anorexia in AIDS patients and as a treatment for nausea and vomiting related to chemotherapy. The company’s marinol is branded Syndros, and is an oral formulation that can be taken as a liquid. Todd Campbell , writing for The Motley Fool, notes that the product has a potential nine-figure annual sales potential and that the market for marinol products is currently $150 million per year and growing. The company’s primary product is Subsys, a fentanyl pain killer for cancer pain. On Feb. 9, the company announced it had started enrollment for its Phase II trial of its pharmaceutical CBD to treat infantile spasms, a catastrophic form of childhood epilepsy. Cannabidiol (CBD) is one of about 60 active cannabinoids found in cannabis (marijuana), and is believed to have more medical applications than tetrahydrocannabinol (THC). Opko Health Miami, Fla.-based OPKO Health (OPK) focuses on diagnostics and therapeutics. At the end of this month, the FDA is considering the company’s Rayaldee, an oral vitamin D prohormone. Rayaldee is being developed to treat people with stage 3 or 4 chronic kidney disease (CKD). The National Kidney Foundation believes 26 million people in the U.S. have CKD and 20 million are in stage 3 or 4. The market is estimated at $12 billion in annual sales. Brian Feroldi , writing for the Motley Fools, says, “There’s at least one big believer that Rayaldee will make its way to market: Opko Health’s billionaire CEO Philip Frost , who has been aggressively adding to his huge position in the company recently. Frost purchased more than 47 million additional shares since December alone.” The company also announced on Feb. 23 that it has dosed the first patient in a Phase IIa study of a long-acting Factor VIIa in hemophilia patients. Factor VIIa is currently only available intravenously. The company’s compound, Factor VIIa-CTP, appears to be usable intravenously and subcutaneously. Jazz Pharmaceuticals Dublin-based Jazz Pharmaceuticals (JAZZ) ’s defibrotide is up for FDA approval on March 31. The company recently announced clinical data of the drug from a Phase III clinical trial in patients with hepatic veno-occlusive (VOD), also called sinusoidal obstruction syndrome (SOS), with multi-organ failure (MOF) post-hematopoietic stem-cell transplantation (HSCT). The drug showed statistically significant improvement in Day +100 survival and in rate of complete response (CR) by Day +100. “Based on the results of this pivotal phase III study, we believe defibrotide provides a promising treatment option for patients with this urgent unmet need,” said Paul Richardson , director of clinical research and clinical program leader at the Jerome Lipper Multiple Myeloma Center , Dana-Farber Cancer Institute and the RJ Corman Professor of Medicine at Harvard Medical School , in a statement. “Although HSCT has improved substantially over the last decade, hepatic VOD/SOS with MOF remains a very real and life-threatening complication post-HSCT, and for which there are no currently approved therapies.” Cheryl Swanson , writing for The Motley Fool, points out that most of Jazz’s revenues come from its narcolepsy drug, Xyrem. The company picked up defibrotide in 2013 when it bought out Gentium for $1 billion. The drug had European approval before that, but the FDA had turned down Gentium’s application, apparently because of questions regarding clinical trial monitoring. “Jazz believes it has now satisfied the FDA’s requests,” writes Swanson. “And with solid data from three clinical studies, and a priority review treatment designation, it seems likely that Jazz’s gamble will soon pay off.”