August 15, 2013 12:18pm EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK), has surpassed 50% enrollment in the
second phase 3 trial of Rayaldy ™ to treat patients with
secondary hyperparathyroidism (SHPT), stage 3 or 4 chronic kidney
disease (CKD) and vitamin D insufficiency. This trial is the second of
two identical randomized, double-blind, placebo controlled, multi-site
studies intended to establish the safety and efficacy of Rayaldy ™ 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 phase 3 trials will involve 210 patients recruited at
approximately 40 sites in the U.S. These patients are being stratified
by CKD stage and randomized in a 2:1 fashion to receive six months of
treatment with either Rayaldy ™ or placebo. Dosing with Rayaldy ™ is
titrated, as necessary, to achieve the desired blood concentration and
the targeted reduction in PTH.
“I am pleased to report that these pivotal trials with Rayaldy Capsules
are progressing as planned toward completion in the first half of 2014,”
stated Phillip Frost, M.D., OPKO’s Chairman and Chief Executive Officer.
“Top-line data are expected in mid-2014. We believe Rayaldy ™ will
prove to be superior to high monthly doses of prescription vitamin D 2 ,
a common therapeutic approach which has been shown unreliable in
correcting vitamin D insufficiency in patients with chronic kidney
disease and generally ineffective in controlling elevated levels of
parathyroid hormone. We look forward to advancing the standard of care
for this patient population when Rayaldy ™ is ultimately approved
for marketing.”
About Rayaldy ™
Rayaldy ™ 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
the product 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, Rayaldy ™ will address the
approximately 4 million CKD stage 3 and 4 patients in the U.S. and many
more, 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
prohormones, collectively 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 hormones 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 about 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.
This press release contains "forward-looking statements," as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), regarding product development efforts and other
non-historical facts about our expectations, beliefs or intentions
regarding our business, technologies and products, financial condition,
strategies or prospects. Many factors could cause our actual activities
or results to differ materially from the activities and results
anticipated in forward-looking statements. These factors include those
described in our filings with the Securities and Exchange Commission, as
well as risks inherent in funding, developing and obtaining regulatory
approvals of new, commercially-viable and competitive products and
treatments, including the risks that enrollment of patients for the
Phase 3 clinical trials for Rayaldy ™ may not be completed by the
second half of 2013 or at all, that top-line data may not be available
by mid-2014, that the Phase 3 clinical trials for Rayaldy™ may not be
successful or achieve the expected results or effectiveness, and may not
generate data that would support the approval or marketing of this
product for the indications being studied, that others may develop
products which are superior to Rayaldy ™ , and that Rayaldy ™ may
not have advantages or prove to be superior over presently marketed
products, including the currently used high monthly doses of
prescription vitamin D 2, activated vitamin D
hormone and over-the-counter vitamin D supplements . In addition,
forward-looking statements may also be adversely affected by general
market factors, competitive product development, product availability,
federal and state regulations and legislation, the regulatory process
for new products and indications, manufacturing issues that may arise,
patent positions and litigation, among other factors. The
forward-looking statements contained in this press release speak only as
of the date the statements were made and we do not undertake any
obligation to update forward-looking statements. We intend that all
forward-looking statements be subject to the safe-harbor provisions of
the PSLRA.
OPKO Health, Inc.
Steven D. Rubin or Juan F. Rodriguez, 305-575-4100
Source: OPKO Health, Inc.
Released August 15, 2013
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