NPS Pharmaceuticals Inc. initiated a pivotal Phase III study for the orphan product teduglutide, a potential treatment for short-bowel syndrome.
Reaching the milestone is key to NPS because it marks the third time in a month that favorable news has flowed from the firm's Salt Lake City office.
Good news began for NPS in early March when Amgen Inc. won FDA approval of Sensipar (cinacalet HCl), a treatment for secondary hyperparathyroidism in patients with chronic kidney disease on dialysis and for hypercalcemia in those with parathyroid carcinoma. NPS licensed the winning candidate to the Thousand Oaks, Calif.-based firm. FDA approval triggered a $10 million milestone to NPS. (See BioWorld Today, March 10, 2004.)
Within weeks, NPS again was in the news - this time for a successful Phase III trial of Preos, a recombinant parathyroid hormone under evaluation for the treatment of osteoporosis. (See BioWorld Today, March 31, 2004.)
Of the recent news, David Clark, NPS's vice president of corporate affairs, told BioWorld Today: "We are at a terrific point in the history of NPS Pharmaceuticals, where we are really making the transition from research and development to commercialization. So we are exploring ways to build out our commercial capabilities and prepare to be an integrated biopharmaceutical company."
NPS's stock (NASDAQ:NPSP) was down 60 cents Tuesday to close at $27.80.
The pivotal trial of teduglutide is a multicenter, double-blind, international study of about 80 patients who will receive daily subcutaneous injections of 0.05 mg or 0.10 mg of teduglutide per kilogram of body weight, or placebo. Dosing will continue for six months, and the primary endpoint is a reduction in the use of an intravenous feeding tube - patients suffering with short-bowel syndrome (SBS) often must rely on a feeding tube to survive. SBS is a condition that results from the surgical removal of significant portions of the bowel following injury or illness.
Clark said the firm has yet to comment on a specific timeline for completing the trial or filing for regulatory approval.
Teduglutide, an orphan product in both the U.S. and Europe, is an analogue of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the growth, proliferation and maintenance of cells lining the gastrointestinal tract. Teduglutide has been shown in animal studies and previous clinical trials to increase the size and number of those cells, thereby increasing the absorptive surface area of the intestines. The drug is not partnered.
Clark said the firm believes teduglutide would increase the absorptive surface area of the remaining section of the bowel so that a patient can derive more benefit from a normal diet and become less reliant on intravenous feeding.
NPS's 21-patient Phase II trial generated favorable results related to increased nutritional uptake, increased energy and positive weight gain, Clark said.