New Findings from REPLACE Study Support Potential of Natpara(TM) to Treat Adult Hypoparathyroidism

BEDMINSTER, N.J.--(BUSINESS WIRE)--

NPS Pharmaceuticals, Inc. (NPSP), a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, announced today that new findings from the double-blind, placebo-controlled Phase 3 REPLACE study of Natpara (recombinant human parathyroid hormone (rhPTH [1-84])) support the drugs therapeutic potential as the first parathyroid hormone replacement therapy for adults with hypoparathyroidism. The findings were presented in oral and poster sessions at the ENDO 2012 annual meeting of The Endocrine Society in Houston, TX. Natpara is a bioengineered replica of human parathyroid hormone that is being developed by NPS Pharmaceuticals as the first replacement therapy for adults with hypoparathyroidism.

These presentations show the potential benefits of replacing the missing parathyroid hormone with Natpara and restoring and maintaining healthy serum calcium levels in patients with this rare and complex metabolic disorder, said Roger Garceau, M.D., senior vice president and chief medical officer of NPS Pharmaceuticals. The consequences of this disorder can be severely disabling, which is why the drugs apparent treatment effects in this study increased serum calcium levels, reductions in calcium and vitamin D supplementation, fewer disease symptoms and increased bone turnover are so impressive. Natpara was also well tolerated, with no significant differences in adverse events compared to placebo. These findings reinforce our belief that Natpara represents a promising and important new treatment option for hypoparathyroidism.

Hypoparathyroidism is characterized by hypocalcemia due to insufficient levels of parathyroid hormone, the bodys principal regulator of calcium and phosphorus. It is the only classic endocrine disorder for which there are no FDA-approved replacement therapies. Current treatment approaches focus on symptom management through high doses of calcium and active vitamin D supplementation, which can lead to serious side effects and long-term consequences.

Treatment with Natpara resulted in significant reductions in calcium and active vitamin D supplements.

In an oral presentation on Saturday, June 23, lead study investigator, John P. Bilezikian, M.D., professor of medicine, Division of Endocrinology, Columbia University College of Physicians and Surgeons, presented an overview of the data from the REPLACE study.

In an intent-to-treat analysis, 53 percent (48/90) of Natpara-treated patients achieved the primary endpoint versus 2 percent (1/44) of placebo-treated patients (p<0.001). The primary efficacy endpoint was defined as a 50 percent or greater reduction in oral calcium supplementation and active vitamin D therapy and a total serum calcium concentration that was normalized or maintained compared to baseline after 24 weeks of treatment.

At week 24, 43 percent (36/84) of patients treated with Natpara were able to achieve independence from active vitamin D therapy and required only a calcium supplementation dose of 500 mg/day or less, as compared to five percent (2/37) of patients treated with placebo (p<0.0001).

Despite the large reductions in supplementation, serum calcium remained at or above baseline levels for the Natpara-treated patients. Treatment with Natpara also resulted in a small decrease in mean 24-hour urinary calcium excretion from baseline and patients experienced fewer hypocalcemic clinical symptoms during the maintenance phase of the study. Natpara was generally well-tolerated and shows promise as an effective replacement therapy for hypoparathyroidism.

Parathyroid hormone has the potential to fill a major therapeutic gap in the effective management of hypoparathyroidism, said lead study investigator, John P. Bilezikian, Professor of Medicine and Pharmacology, Director of the Metabolic Bone Diseases Unit, Division of Endocrinology, Columbia University College of Physicians and Surgeons. Hypoparathyroidism is the only endocrine deficiency disease for which the missing hormone, namely parathyroid hormone, is not an approved therapy. These findings provide hope for patients with hypoparathyroidism who desperately need more options for their care. Many patients rely on the long-term use of high dose calcium and vitamin D to help alleviate symptoms. However, this approach is subject to uneven control in many patients with wide swings in their blood calcium levels and fluctuations in their symptoms. Moreover, long-term high dose calcium and vitamin D can be associated with serious complications, including calcifications in the kidneys, heart, and brain.

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New Findings from REPLACE Study Support Potential of Natpara(TM) to Treat Adult Hypoparathyroidism