May 06, 2014 9:17am EDT
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MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE: OPK), has completed patient enrollment in the
third phase 3 trial of RAYALDEE . This trial is designed to
evaluate the product’s long-term safety and efficacy in treating
secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4
chronic kidney disease (CKD) and vitamin D insufficiency.
This third phase 3 trial is a 6-month open-label extension of two
ongoing and identical randomized, double-blind, placebo-controlled,
multi-site pivotal phase 3 studies for RAYALDEE intended to
support marketing approval in the United States (US). Together, the two
pivotal trials involve approximately 430 patients recruited at
approximately 90 US sites who are receiving six months of treatment with
either RAYALDEE or placebo. Both trials will end in July
2014 and the Company expects to announce top-line data during the third
quarter of 2014. In the open-label extension study, patients either
continue RAYALDEE treatment or switch to RAYALDEE from
placebo treatment. Additional patients will be allowed to enroll in the
open-label extension study through July as they exit the blinded pivotal
trials. The endpoints of all three phase 3 studies include vitamin D
status and changes in plasma intact parathyroid hormone (PTH), serum
calcium and serum phosphorus.
Recently, OPKO also completed a pharmacokinetic study of RAYALDEE
in approximately 50 healthy US volunteers. This study, designed to
evaluate the effect of food on the intestinal absorption of RAYALDEE ,
is the last clinical trial (aside from the above-mentioned phase 3
trials) needed to support the forthcoming New Drug Application (NDA).
OPKO expects that an NDA for RAYALDEE will be filed with the US
Food and Drug Administration in the first quarter of 2015.
“The development of RAYALDEE is proceeding fully as we expected:
on schedule and on budget,” remarked Phillip Frost, M.D., OPKO's CEO and
Chairman. “We believe our proprietary first-in-class vitamin D product
will be a valuable new treatment for chronic kidney disease patients and
we look forward to reporting final data from the ongoing pivotal trials
promptly.”
About RAYALDEE
RAYALDEE is a first-in-class oral vitamin D prohormone treatment
being developed for SHPT in patients with stage 3 or 4 CKD and 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 limit the value of current vitamin D hormone
therapies by promoting vascular and renal calcification. RAYALDEE is
expected to address the approximately 4 million patients in the US, and
many more elsewhere, with stage 3 or 4 CKD, 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 US, including more than eight 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.
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
OPKO is a multinational biopharmaceutical and diagnostics company that
seeks to establish industry leading positions in large, rapidly growing
markets by leveraging its discovery, development and commercialization
expertise and 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, including statements regarding our ability to
successfully launch and commercialize proprietary renal disease
products, expectations about RAYALDEE, its market potential, that
it will address the approximately 4 million patients in the US, and many
more elsewhere, with stage 3 or 4 CKD, SHPT and vitamin D insufficiency,
that RAYALDEE will be a valuable new treatment for CKD patients, the
expected timing for completion of our clinical trials for RAYALDEE,
announcement of top line results, and the submission of an NDA, whether
the trials for RAYALDEE will continue to proceed on schedule and on
budget, and that we will be able to successfully develop, obtain
approval for and launch sales of RAYALDEE. 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 the phase
3 clinical trials for RAYALDEE 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
RAYALDEE, and that RAYALDEE 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 Charles W. Bishop, 305-575-4100
Source: OPKO Health, Inc.
Released May 6, 2014
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