May 23, 2013 9:32am EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK), has surpassed 50% enrollment in the first
phase 3 trial of Rayaldy TM 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 Rayaldy TM
as a new treatment for SHPT in the targeted population. The endpoints of
both studies, which will be conducted in parallel, include vitamin D
status and changes in serum calcium, serum phosphorus and plasma intact
parathyroid hormone (PTH).
Each of the 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 TM or placebo.
Dosing with Rayaldy TM is titrated, as
necessary, to achieve the desired blood concentration and the targeted
reduction in PTH.
“ Rayaldy TM is being developed as a much needed
and safer alternative to currently used activated vitamin D hormones,”
commented Dr. David Bushinsky, Chief of Nephrology at the University of
Rochester. “Activated vitamin D hormone therapy is problematic in CKD
patients with secondary hyperparathyroidism arising primarily from
vitamin D insufficiency since it further stimulates catabolism
(breakdown) of the available vitamin D stores and is often associated
with hypercalcemia, a side effect linked to vascular calcification.”
“Secondary hyperparathyroidism develops in CKD patients due to vitamin D
insufficiency or declining kidney function,” explained Joel Z. Melnick,
M.D., Vice President, Clinical Research and Development of OPKO’s Renal
Division. “Most CKD patients have insufficient stores of vitamin D due
to the abnormal upregulation of CYP24, an enzyme which specifically
destroys vitamin D and its metabolites. Many recent studies in CKD
patients have demonstrated that over-the-counter and presently available
prescription vitamin D supplements cannot reliably raise blood vitamin D
prohormone levels or effectively treat SHPT. In contrast, our phase 2b
trial has demonstrated that Rayaldy TM
effectively and safely treats both SHPT and the underlying vitamin D
insufficiency.”
About Rayaldy TM
Rayaldy TM is a first-in-class 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 TM
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 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.
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 or the
results of the Phase 3 clinical trials for Rayaldy TM
may not be completed on a timely basis or at all, 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 TM , and that Rayaldy TM
may not have advantages over presently marketed products, including the
currently used activated vitamin D hormone and over-the-counter and
prescription 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 May 23, 2013
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