Press Release
NPS Pharmaceuticals Initiates Phase 3 'REPLACE' Study of NPSP558 in Hypoparathyroidism
BEDMINSTER, N.J.--(BUSINESS WIRE)--
"Achieving this important milestone brings us one step closer to filing
a marketing application for NPSP558 as the first therapeutic option for
hypoparathyroidism, one of the few remaining hormone deficiencies
without an approved replacement therapy," said Francois Nader, MD,
president and chief executive officer of
REPLACE Study Design
REPLACE is a double-blind, placebo-controlled trial that will randomize
approximately 110 patients at over a dozen sites in the
The study will consist of a maximum 10-week screening and stabilization period followed by a 24-week treatment period marked by randomization (2:1) to NPSP558 50µg (with the potential for titration up to 75µg and 100µg) or placebo. Following randomization, patients will undergo staged reductions in calcium and vitamin D supplementation, while maintaining a serum total calcium within clinically stable limits. If needed, up-titration of study drug to 75 µg or 100 µg in patients over a six to eight week period will be performed. Patients will continue on their final NPSP558 dose through week 24 and a follow-up period will last from week 24 to week 28.
Efficacy will be demonstrated by achievement or maintenance of a normal or clinically acceptable stable albumin-corrected serum total calcium in parallel with reduced requirements for oral supplementation with calcium and treatment with active vitamin D metabolites or analogs.
The responder status is defined as a patient that demonstrates at least a 50 percent reduction from baseline amounts of oral calcium supplementation and at least a 50 percent reduction from baseline amounts of vitamin D metabolite/analog therapy by week 24 of the study. Patients should have a clinically stable serum calcium level that is established to the satisfaction of the investigator at baseline and is maintained or normalized by week 24 of the study.
The secondary objectives of the study include studying the urinary calcium excretion or hypercalciuria, the proportion of patients with maintenance of a calcium phosphate product in the normal range, and the proportion of patients that achieve independence from supplemental active vitamin D metabolite/analog usage or a calcium supplementation dose of 500 mg/day by Week 24
About NPSP558 (parathyroid hormone 1-84 [rDNA origin] injection)
NPSP558 is a recombinant full-length human parathyroid hormone (PTH
1-84), the main hormone that is needed to maintain the balance of
calcium in the body. Because it mimics the action of natural parathyroid
hormone, NPSP558 has the potential to treat hypoparathyroidism and
return the body to a physiological or "eucalcemic" state. In
In 2007, the FDA granted orphan drug status for NPSP558 for the treatment of hypoparathyroidism. NPS's partner Nycomed markets PTH 1-84 ex-US as Preotact(R) for the treatment of osteoporosis in post-menopausal women at high risk of fractures.
About Hypoparathyroidism
NPS has estimated that approximately 65,000 patients suffer from hypoparathyroidism in the U.S. It is one of the few remaining hormone deficiency states without an approved replacement therapy. Because the primary role of parathyroid hormone is to maintain normal calcium levels in blood, the major consequence of its absence is low circulating calcium levels (hypocalcemia), which can cause tingling of the hands, fingers, and mouth, serious muscle cramping or, in extreme situations, to tetany or convulsions. The goal of currently available treatments for hypoparathyroidism, which include life-long high-dose oral supplementation with calcium and active vitamin D metabolites or analogs, is to reduce the severity of symptoms. However, hypoparathyroidism and the use of oral calcium and vitamin D analogs/metabolites for symptom control can contribute to organ damage and calcification, which is particularly evident in the kidneys.
About
"NPS" and "
Statements made in this press release, which are not historical in
nature, constitute forward-looking statements for purposes of the safe
harbor provided by the Private Securities Litigation Reform Act of 1995.
These statements are based on the company's current expectations and
beliefs and are subject to a number of factors and uncertainties that
could cause actual results to differ materially from those described in
the forward-looking statements. Risks associated to NPS's business
include, but are not limited to, the risk of not successfully executing
its preclinical and clinical studies and not gaining marketing approvals
for GATTEX and NPSP558, the risks associated with the implementation of
a new business strategy, the risks associated with the company's
auction-rate securities, as well as other factors expressed in NPS's
periodic filings with the
CONTACT: Investors:
Susan M.
smesco@npsp.com
or
Media:
edevine@wcpglobal.com
Source:
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