OPKO Health, Inc. (NYSE:OPK), a multi-national biopharmaceutical and diagnostics company, today reported operating and financial results for its second quarter ended June 30, 2014.
- Rayaldee Meets Primary Endpoints in First Pivotal Phase 3 Trial; NDA Submission on Track for Q4 2014: OPKO announced successful top-line results from the first pivotal Phase 3 trial of Rayaldee. This trial is one of two identical randomized, double-blind, placebo-controlled, multi-site studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. OPKO to announce top line results from the second Phase 3 trial in September 2014 and is on track to submit a New Drug Application (NDA) with the United States (U.S.) Food and Drug Administration (FDA) in the fourth quarter of 2014.
- Submission of IND for Rayaldee as Adjunctive Cancer Therapy: OPKO submitted an Investigational New Drug (IND) Application to the FDA to evaluate Rayaldee as an adjunctive therapy, the first of other possible uses, for the prevention of skeletal-related events (SREs) in patients with bone metastases undergoing anti-resorptive therapy. The clinical trial will commence later in 2014.
- Positive Pediatric Phase 2 Data Reported for hGH-CTP: OPKO announced positive 6 month results from its Phase 2 study evaluating the safety and efficacy of hGH-CTP, a long-acting form of human growth hormone, to treat growth hormone deficiency in children. All three hGH-CTP once-weekly doses resulted in strong catch-up growth during the six month treatment period, with annualized increases in height of more than 12 cm. The results indicated excellent dose dependent pharmacokinetic (PK) and pharmacodynamic (PD) profiles. No serious adverse events were reported.
- Factor VIIa-CTP Received Positive Opinion For Three Orphan Drug Designations in Europe: The European Committee for Orphan Medicinal Products (COMP) gave a positive opinion to recommend approval of orphan drug designations for OPKO's long-acting version of clotting Factor VIIa (Factor VIIa-CTP) to treat bleeding episodes in patients with hemophilia A or B who have inhibitors to Factor VIII, Factor IX or congenital Factor VII deficiency. Factor VII-CTP previously was granted orphan status in the U.S. The European designations may provide OPKO the opportunity to have marketing exclusivity for periods of up to 10 years.
- Adoption of 4Kscore Test is Growing: OPKO completed a validation study and launched the 4Kscore Test in the U.S. through its CLIA accredited OPKO Lab in late March 2014. Since launch, its adoption by Urologists has steadily increased. OPKO expects to launch the 4Kscore Test in Europe through its Spanish subsidiary in September 2014 and elsewhere shortly thereafter. OPKO is working to obtain reimbursement for the 4Kscore Test by payers. The 4Kscore Test makes the prostate biopsy decision process more rational. In the U.S. approximately 1 million biopsies are performed annually with 80% indicating no cancer or only low grade cancer. With the knowledge of the risk (probability) of having high grade cancer provided by the 4Kscore Test, prostate biopsies are more likely to be performed only in men at higher risk. OPKO presented data from its U.S. clinical validation study in a plenary session at the American Urological Association (AUA) in Orlando, FL. The talk, “The 4Kscore Test as a Predictor of High-Grade Prostate Cancer on Biopsy,” was also selected for a plenary presentation at the Spanish National Congress of Urology in June, and will be presented at the 83 rd Annual Meeting of the New England Section of the AUA in October 2014 in Newport, RI.
- Rolapitant Meets all Primary and Secondary Endpoints in Final Phase 3 Trial; On Schedule for a mid-September NDA Filing: OPKO’s partner, TESARO, announced positive results from the third and final Phase 3 trial of Rolapitant, a neurokinin-1 (NK-1) receptor antagonist for the prevention of chemotherapy-induced nausea and vomiting (CINV). The oral formulation of Rolapitant is on track for an NDA filing by mid-September; the intravenous (IV) formulation is expected to be available to patients one year after the oral formulation.
- Inspiro Acquisition Completed: OPKO acquired Inspiro Medical Ltd., an Israeli medical device company with a new platform to deliver inhaled drugs. Inspiro's Inspiromatic™ is a "smart", easy-to-use dry powder inhaler with important advantages over existing devices.
“In the second quarter, we continued to make strides in converting our advanced pipeline of diagnostic and pharmaceutical products into commercial successes,” said Phillip Frost, M.D., Chairman and CEO. “The 4Kscore Test is being increasingly adopted by Urologists because of its utility for identifying patients at the greatest risk of having high-grade prostate cancer and clinical trials of Rayaldee, hGH-CTP and Rolapitant have all provided the anticipated evidence of safety and efficacy. We look forward to making these important products available as rapidly as possible to patients who will benefit from their use.”