2016 Press/Media Releases:

OPKO Presents Data on RAYALDEE® at ASN Kidney Week 2016

MIAMI, Nov. 17, 2016 (GLOBE NEWSWIRE) – OPKO Health, Inc. (NYSE:OPK) announced that data on RAYALDEE (calcifediol) extended-release capsules as a treatment for secondary hyperparathyroidism (SHPT) in stage 3 and 4 chronic kidney disease patients with vitamin D insufficiency will be presented later today in a poster presentation at the American Society of Nephrology Kidney Week Meeting, underway in Chicago, IL.

The data showed that RAYALDEE has similar effectiveness and safety in controlling SHPT in both African-American (AA) and non-African-American (nAA) patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency (defined as serum total 25-hydroxyvitamin D levels less than 30 ng/mL).  Serum total 25-hyrdroxyvitamin D levels showed a similar increase in both patient populations beyond the target of 30 ng/mL, a level considered sufficient for CKD patients in published clinical practice guidelines, despite the well established tendency for lower levels in AA patients. Plasma intact parathyroid hormone (iPTH) levels were effectively suppressed by RAYALDEE in AA patients and nAA patients versus placebo treatment.

OPKO's poster presentation entitled "Extended-release Calcifediol is Effective in African-American and Non-African-American Patients with Stage 3-4 CKD, Secondary Hyperparathyroidism and Vitamin D Insufficiency," will be presented by senior author Stuart M. Sprague, DO, Chief, Division of Nephrology and Hypertension, NorthShore University Health System - University of Chicago, Pritzker School of Medicine.

Session details:

Date: Thursday, November 17, 2016
Time: 10:00 a.m. - 12:00 noon Central time
Location: Exhibit Hall, McCormick Place Convention Center
Poster Board: 512
Abstract Link: http://www.abstracts2view.com/asn_2016/view.php?nu=5118&type=abstract

"FDA's approval of RAYALDEE represents an important milestone for OPKO," noted Dr. Phillip Frost, CEO and Chairman of OPKO. "RAYALDEE is the first product to receive FDA approval for this important indication and is one of OPKO's many pharmaceutical products being developed for significant medical problems which will benefit from new treatment options."

Results from two 26 week placebo controlled, double blind phase 3 trials demonstrated that a larger proportion of stage 3 or 4 CKD patients with SHPT and vitamin D insufficiency achieved ≥30% reductions in plasma intact parathyroid hormone (iPTH) when treated with RAYALDEE than with placebo. Vitamin D insufficiency was corrected in more than 80% of the patients receiving RAYALDEE compared with less than 7% of subjects receiving placebo. Mean serum calcium and phosphorus levels increased by 0.1 mg/dL during RAYALDEE treatment compared to placebo treatment, but these changes were deemed clinically irrelevant. No differences in RAYALDEE's efficacy or safety were observed between patients with stage 3 CKD or stage 4 CKD.

"RAYALDEE fills a large void in the current treatment options for SHPT in predialysis patients," commented Dr. Charles W. Bishop, CEO of OPKO's Renal Division. "The current standard of care is high dose vitamin D supplementation, an approach for treating SHPT that is neither FDA approved nor demonstrated to be safe and effective in this population. SHPT is a progressive disease that becomes increasingly debilitating and difficult to treat, necessitating timely and effective treatment."

"RAYALDEE is an important new option for treating SHPT in patients with stage 3 or 4 CKD and vitamin D insufficiency," stated Kevin J. Martin, Director of Research, Division of Nephrology at Saint Louis University School of Medicine. "The great majority of SHPT cases in this patient population are associated with vitamin D insufficiency, a problem that RAYALDEE can correct."

RAYALDEE (calcifediol) extended-release capsules is approved by the U.S. Food and Drug Administration (FDA) for the treatment of SHPT in adult patients with stage 3 or 4 CKD and serum total 25-hydroxyvitamin D levels less than 30 ng/mL.  RAYALDEE is not indicated for the treatment of secondary hyperparathyroidism in patients with stage 5 chronic kidney disease or end-stage renal disease on dialysis.  RAYALDEE has a patented formulation and is designed to raise serum total 25-hydroxyvitamin D (prohormone) concentrations to targeted levels (at least 30 ng/mL) and to reduce elevated iPTH.  OPKO expects to launch RAYALDEE in the U.S. through its dedicated renal sales force in November 2016. The full prescribing information for RAYALDEE is available at www.rayaldee.com.

Potential side effects of RAYALDEE include hypercalcemia (elevated serum calcium), which can also lead to digitalis toxicity, and adynamic bone disease with subsequent increased risk of fractures if intact PTH levels are suppressed by RAYALDEE to abnormally low levels.  Severe hypercalcemia may require emergency attention; symptoms of hypercalcemia may include feeling tired, difficulty thinking clearly, loss of appetite, nausea, vomiting, constipation, increased thirst, increased urination, and weight loss.  Digitalis toxicity can be potentiated by hypercalcemia of any cause.  Excessive administration of RAYALDEE can cause hypercalciuria, hypercalcemia, hyperphosphatemia, or oversuppression of iPTH.  Common symptoms of vitamin D overdosage may include constipation, decreased appetite, dehydration, fatigue, irritability, muscle weakness, or vomiting.  Patients concomitantly taking cytochrome P450 inhibitors, thiazides, cholestyramine, phenobarbital or other anticonvulsants may require dose adjustments and more frequent monitoring.

The most common adverse reactions in clinical trials (≥3% and more frequent than placebo) were anemia, nasopharyngitis, increased blood creatinine, dyspnea, cough, congestive heart failure and constipation.

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 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 20 million patients with moderate (stages 3 or 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. RAYALDEE is only indicated for treating SHPT in patients with stage 3 or stage 4 CKD.

About Secondary Hyperparathyroidism (SHPT)
SHPT is a condition commonly associated with CKD in which the parathyroid glands secrete excessive amounts of iPTH.  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 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.  Vitamin D insufficiency has been associated with increased mortality in CKD.

About OPKO Health, Inc.
OPKO Health is a diversified healthcare company that seeks to establish industry-leading positions in large, rapidly growing markets. Our diagnostics business includes Bio-Reference Laboratories, the nation's third-largest clinical laboratory with a core genetic testing business and a 420-person sales force to drive growth and leverage new products, including the 4Kscore® prostate cancer test and the Claros® 1 in-office immunoassay platform. Our pharmaceutical business features RAYALDEE, an FDA-approved treatment for SHPT in stage 3-4 CKD patients with vitamin D insufficiency, VARUBI™ for chemotherapy-induced nausea and vomiting (oral formulation launched by partner TESARO and IV formulation PDUFA date: January 2017), TT401, a once or twice weekly oxyntomodulin for type 2 diabetes and obesity which is a clinically advanced drug candidate among the new class of GLP-1 glucagon receptor dual agonists, and TT701, an androgen receptor modulator for androgen deficiency indications.  Our biologics business includes hGH-CTP, a once-weekly human growth hormone injection (in phase 3 and partnered with Pfizer), a long-acting Factor VIIa drug for hemophilia (in phase 2a) and a long-acting oxyntomodulin for diabetes and obesity (in phase 1). We also have production and distribution assets worldwide, multiple strategic investments and an active business development strategy.More information is available at www.opko.com.

OPKO Health, Inc.:
Charles W. Bishop
PhD, CEO, Renal Division


Rooney & Associates
Terry Rooney


Marion Janic


Anne Marie Fields


Bruce Voss

Source: OPKO Health Inc.
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