Open-label, dose-escalating study to assess safety and tolerability of NPSP795, which is designed to directly address the underlying molecular defect associated with ADH
BEDMINSTER, NJ, USA I August 4, 2014 I NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, has initiated a Phase 2a study to evaluate the safety and tolerability of NPSP795 in adult patients with Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disorder of calcium homeostasis. ADH is caused by mutations of the calcium-sensing receptor (CaSR) gene that increase the sensitivity of the receptor to serum calcium. NPSP795 is a selective calcium receptor antagonist, which binds to the CaSR and decreases its sensitivity to serum calcium. It’s mechanism of action is believed to restore the normal physiological action of the CaSR and address the underlying molecular defect in ADH to return to normal calcium homeostasis. The company expects to report preliminary top-line data from the study in late 2014 or early 2015.
“This new study is an important step in our exploration of the potential of NPSP795 for the treatment of patients with ADH, who do not have approved treatment options.”
“NPS Pharma is committed to advancing the understanding of Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disease that can lead to serious renal complications,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma. “This new study is an important step in our exploration of the potential of NPSP795 for the treatment of patients with ADH, who do not have approved treatment options.”
About the Phase 2a Study
The non-randomized, open-label, dose-escalating study will evaluate the safety and tolerability of an intravenous (IV) infusion of NPSP795. The study will also test the ability of various doses of NPSP795 to stimulate parathyroid hormone secretion, increase blood calcium and decrease renal calcium excretion. The study is expected to enroll up to 12 patients at the National Institutes of Health’s Clinical Center in Bethesda, MD. Additional information about the study is available here.
About Autosomal Dominant Hypocalcemia (ADH)
Autosomal Dominant Hypocalcemia, or ADH, is an ultra-rare, genetic disease caused by mutations of the calcium-sensing receptor (CaSR) gene that increases the sensitivity of the receptor to serum calcium. The underlying cause of symptoms and complications in patients are two-fold. First, absolute hypocalcemia can cause life-threatening seizures, laryngeal spasms, cardiac arrhythmias, tetany, paresthesias, muscle cramping, and decreased focus. In addition, the body’s perceived hypercalcemia results in inappropriately increased renal calcium excretion, decreased renal phosphate excretion, and hyperphosphatemia, putting patients at risk for renal complications, including renal stones, nephrocalcinosis, and impaired renal function, as well as soft tissue calcifications.
Patients with ADH can present immediately post-birth with severe hypocalcemia that can be life-threatening. Supportive approaches focus on managing the symptoms of hypocalcemia by carefully adjusting oral calcium and active vitamin D, but these efforts can worsen the condition by increasing calcium urine excretion and renal complications. Hypocalcemia may be asymptomatic in some patients with ADH; however, nervous system and kidney complications are often present in these patients.
About NPS Pharma
NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company’s current therapeutic areas of focus are gastrointestinal disease and endocrine disorders. These include Short Bowel Syndrome, a potentially fatal gastrointestinal disorder in which patients may have to rely on parenteral nutrition for their survival; Hypoparathyroidism, a complex endocrine disorder in which the parathyroid glands are either absent or damaged, and the body produces insufficient or no parathyroid hormone; and Autosomal Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium homeostasis caused by mutations of the calcium-sensing receptor gene. NPS Pharma continues to seek in-licensing opportunities to develop new therapies for a broad range of rare diseases, and complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin. NPS Pharma has operations in the U.S., Canada, Europe, Latin America and Japan. Learn more at: www.npsp.com.
SOURCE: NPS Pharmaceuticals
Post Views: 175
Open-label, dose-escalating study to assess safety and tolerability of NPSP795, which is designed to directly address the underlying molecular defect associated with ADH
BEDMINSTER, NJ, USA I August 4, 2014 I NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, has initiated a Phase 2a study to evaluate the safety and tolerability of NPSP795 in adult patients with Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disorder of calcium homeostasis. ADH is caused by mutations of the calcium-sensing receptor (CaSR) gene that increase the sensitivity of the receptor to serum calcium. NPSP795 is a selective calcium receptor antagonist, which binds to the CaSR and decreases its sensitivity to serum calcium. It’s mechanism of action is believed to restore the normal physiological action of the CaSR and address the underlying molecular defect in ADH to return to normal calcium homeostasis. The company expects to report preliminary top-line data from the study in late 2014 or early 2015.
“This new study is an important step in our exploration of the potential of NPSP795 for the treatment of patients with ADH, who do not have approved treatment options.”
“NPS Pharma is committed to advancing the understanding of Autosomal Dominant Hypocalcemia (ADH), an ultra-rare genetic disease that can lead to serious renal complications,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharma. “This new study is an important step in our exploration of the potential of NPSP795 for the treatment of patients with ADH, who do not have approved treatment options.”
About the Phase 2a Study
The non-randomized, open-label, dose-escalating study will evaluate the safety and tolerability of an intravenous (IV) infusion of NPSP795. The study will also test the ability of various doses of NPSP795 to stimulate parathyroid hormone secretion, increase blood calcium and decrease renal calcium excretion. The study is expected to enroll up to 12 patients at the National Institutes of Health’s Clinical Center in Bethesda, MD. Additional information about the study is available here.
About Autosomal Dominant Hypocalcemia (ADH)
Autosomal Dominant Hypocalcemia, or ADH, is an ultra-rare, genetic disease caused by mutations of the calcium-sensing receptor (CaSR) gene that increases the sensitivity of the receptor to serum calcium. The underlying cause of symptoms and complications in patients are two-fold. First, absolute hypocalcemia can cause life-threatening seizures, laryngeal spasms, cardiac arrhythmias, tetany, paresthesias, muscle cramping, and decreased focus. In addition, the body’s perceived hypercalcemia results in inappropriately increased renal calcium excretion, decreased renal phosphate excretion, and hyperphosphatemia, putting patients at risk for renal complications, including renal stones, nephrocalcinosis, and impaired renal function, as well as soft tissue calcifications.
Patients with ADH can present immediately post-birth with severe hypocalcemia that can be life-threatening. Supportive approaches focus on managing the symptoms of hypocalcemia by carefully adjusting oral calcium and active vitamin D, but these efforts can worsen the condition by increasing calcium urine excretion and renal complications. Hypocalcemia may be asymptomatic in some patients with ADH; however, nervous system and kidney complications are often present in these patients.
About NPS Pharma
NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company’s current therapeutic areas of focus are gastrointestinal disease and endocrine disorders. These include Short Bowel Syndrome, a potentially fatal gastrointestinal disorder in which patients may have to rely on parenteral nutrition for their survival; Hypoparathyroidism, a complex endocrine disorder in which the parathyroid glands are either absent or damaged, and the body produces insufficient or no parathyroid hormone; and Autosomal Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium homeostasis caused by mutations of the calcium-sensing receptor gene. NPS Pharma continues to seek in-licensing opportunities to develop new therapies for a broad range of rare diseases, and complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin. NPS Pharma has operations in the U.S., Canada, Europe, Latin America and Japan. Learn more at: www.npsp.com.
SOURCE: NPS Pharmaceuticals
Post Views: 175
