By Randall Osborne
West Coast Editor
United Therapeutics Corp. (UTC) said early data is positive from two Phase III studies of Uniprost, its non-injectable drug for pulmonary hypertension.
"We did two almost-simultaneous pivotal studies," said Fred Hadded, chief financial officer of Silver Spring, Md.-based UTC. "This covers the combined results."
Uniprost was shown effective in preliminary analysis of trial data, improving to a statistically significant degree patients' ability to exercise, which was the primary endpoint.
Formerly known as UT-15, Uniprost is a stable structural analogue of the naturally occurring molecule prostacyclin, levels of which are low in the lung blood vessels of people with pulmonary hypertension.
UTC said patients in the trial also demonstrated statistically significant improvements in "important pulmonary dynamics," as well as meaningful positive response in the signs and symptoms of their disease.
"Analysis is ongoing as we speak," Hadded said. "We anticipate filing a new drug application in the third quarter."
Randomized and double-blinded, the international trial tested 470 patients. Half received Uniprost subcutaneously for 12 weeks, and the other half were given a placebo. All had the option of staying on the drug (or begin taking it) in an open-label study after finishing the pivotal trial. About 500 patients now are being treated, including those who enrolled directly into the open-label study.
Last month, the company began a second Phase III trial of an oral formulation of prostacyclin, called Beraprost, in patients with peripheral vascular disease (PVD). The condition afflicts millions of people in the U.S. and is characterized by pain in the lower leg, cramps, muscle ache or severe fatigue upon exercising, believed to result from inadequate blood flow to the lower limbs. The first Phase III trial of Beraprost, conducted in Europe, yielded significant results. (See BioWorld Today, Feb. 2, 2000, p. 1.)
Hadded said the company is developing Beraprost for early PVD and early pulmonary hypertension. "It's kind of the lighter drug you'd use," he said. "Uniprost has a much more immediate and powerful effect."
Also under way are two Phase II trials, one for UT-77 to treat chronic obstructive pulmonary disease, and one for Ketotop, a transdermal patch that delivers the approved osteoarthritis drug ketoprofen.
UTC raised $62.1 million when it went public last June, and garnered another $80 million in a private placement late last year. (See BioWorld Today, Dec. 27, 1999, p. 1.)
The company's stock (NASDAQ:UTHR) jumped Monday on the news, closing at $82.25, up $12.125.