NPS Pharmaceuticals (NASDAQ: NPSP), a Bedminster, New Jersey-based orphan drug developer, saw positive results using a hormone replacement therapy drug in a phase 3 clinical trial to treat hypoparathyroidism.
Hypoparathyroidism is a rare endocrine disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus, and for which there is no FDA-approved replacement therapy, according to a release from the company.
Although the symptoms of the disorder are typically managed with large doses of oral calcium supplementation and active vitamin D therapy to reduce the severity of symptoms, the prolonged use of oral calcium supplementation and active vitamin D therapy may result in serious long-term health complications, according to the release.
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Bart Clark, a professor of medicine at the Mayo Clinic and an investigator for the REPLACE study, cheered the results: “Considering hypoparathyroidism is the only endocrine disorder for which we do not have an approved replacement hormone to treat the underlying condition, these data indicate that Natpara may offer a valuable option to achieve a physiological treatment and outcome by delivering the missing hormone.”
Francois Nader MD, CEO of NPS Pharmaceuticals, said: “These positive results from our Phase 3 REPLACE study are an important milestone and bring us one step closer to our goal of providing hypoparathyroidism patients with a much-needed replacement therapy.” He added the company expected to file for FDA approval next year. It’s orphan drug status is expected to fast-track Natpara for approval in 12-18 months.
The company’s shareprice fell 33 percent last week after announcing new data on their drug Gattex for the treatment of short bowel syndrome in which three participants developed cancer and two of them died.
Seeking Alpha analyst Bryce Istvan said at the time that the selloff was premature, pointing out that according to a press release from NPS about the study, each of the participants in question had pre-existing conditions or precursors to cancer.
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Following the announcement about the breakthrough earlier in the day its share price rose about 11 percent to $6.05 before ending the day at $5.42.
