2013 Press/Media Releases:

OPKO Completes Patient Recruitment in First Phase 3 Trial of Rayaldee

MIAMI, November 15, 2013 ---- OPKO Health, Inc. (NYSE: OPK), has completed patient recruitment in the first phase 3 trial of Rayaldee™ to treat patients with secondary hyperparathyroidism (SHPT), stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency. This trial is the first 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 SHPT in the targeted population. The endpoints of both studies, which are being conducted in parallel, include vitamin D status and changes in serum calcium, serum phosphorus and plasma intact parathyroid hormone (PTH).

Each of the two pivotal phase 3 trials involves approximately 210 patients recruited at approximately 40 sites in the United States (U.S.). Patients are being stratified by CKD stage and randomized in a 2:1 fashion to receive six months of treatment with either Rayaldee™ or placebo. Top-line data from both trials are expected in mid-2014.

The two pivotal trials are being followed by an open-label extension study in which patients are treated, at their election, for an additional 6 months with Rayaldee™. To date, 129 patients have enrolled in this open-label study.

About Rayaldee™
Rayaldee™ is a first-in-class oral vitamin D prohormone treatment being developed for SHPT in stage 3 and 4 CKD patients with vitamin D insufficiency. It has a proprietary modified-release formulation designed to gradually and reliably raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) while avoiding upregulation of CYP24, a cytochrome P-450 enzyme that reduces the PTH lowering potency of current vitamin D supplements. Activation of calcifediol, the active ingredient in Rayaldee™, by the kidney is tightly regulated, preventing excessive elevation of serum calcium and related side effects which encumber current vitamin D hormone therapies and promote vascular and renal calcification. Once approved, Rayaldee™ is expected to address CKD stage 3 and 4 patients in the U.S. and elsewhere, with SHPT and vitamin D insufficiency.

About Chronic Kidney Disease
CKD is a condition characterized by a progressive decline in kidney function. The kidney is normally responsible for excreting waste and excess water from the body, and for regulating various hormones. CKD is classified in five different stages — mild (stage 1) to severe (stage 5) disease — as measured by the kidney's glomerular filtration rate. According to the National Kidney Foundation, CKD afflicts over 26 million people in the U.S., including more than eight million patients with moderate (stages 3 and 4) and severe (stage 5) forms of CKD. In stage 5 CKD, kidney function is minimal to absent and patients require regular dialysis or a kidney transplant for survival.

About Vitamin D Insufficiency
Vitamin D insufficiency is a condition in which the body has low vitamin D stores, characterized by inadequate blood levels of vitamin D prohormone, known as 25-hydroxyvitamin D. An estimated 70-90% of CKD patients have vitamin D insufficiency, which can lead to SHPT and resultant debilitating bone diseases.

About Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the parathyroid glands secrete excessive amounts of PTH. SHPT arises as a result of vitamin D insufficiency or impaired kidney function that prevents sufficient production of vitamin D hormone to properly regulate calcium and phosphorus metabolism, and PTH secretion. Prolonged elevation of blood PTH causes excessive calcium and phosphorus to be released from bone, leading to elevated serum calcium and phosphorus, softening of the bones (osteomalacia) and calcification of vascular and renal tissues. SHPT affects 40-60% of patients with moderate CKD and approximately 90% of patients with severe CKD.

About OPKO Health, Inc.
OPKO is a multi-national biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large and rapidly growing medical markets by leveraging our discovery, development and commercialization expertise and our novel and proprietary technologies. For more information, visit http://www.opko.com or contact Steven D. Rubin or Juan F. Rodriguez at 305-575-4100.

OPKO Health, Inc.
Steven D. Rubin or Juan F. Rodriguez, 305-575-4100
Source: OPKO Health, Inc.

 
     
     
 
   

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