By Kim Coghill

Washington Editor

United Therapeutics Corp. said Monday it has filed a new drug application for Uniprost, a drug that has been granted orphan drug use in the treatment of pulmonary hypertension.

Company officials anticipate approval in mid-2001 and plan to launch it immediately. Uniprost will be marketed through a network of specialty pharmacy distributors in the United States, as well as in Canada, Europe and Israel, according to Fred Hadeed, chief financial officer of Silver Spring, Md.-based United Therapeutics (UTC).

Formerly known as UT-15, Uniprost is an analogue of prostacyclin designed to be delivered to patients subcutaneously on a continuous basis. The standard measures of the success of a pulmonary hypertension drug are its ability to increase patient walking distance in 6 minutes, and its ability to improve hemodynamics.

"At present, we have about 500 patients and that number is growing daily," Hadeed said. "The only competition is Flolan [from Melville, N.Y.-based Gentiva Health Systems], which is approved for a subset of patients with pulmonary hypertension. The indication we would be approved for would be the broader range."

Kevin Tang, senior biotech analyst with New York-based Deutsche Banc Alex.Brown, said Uniprost is more patient-friendly than Flolan.

"The existing therapy requires a surgically implanted catheter for its delivery and that catheter causes infections in the vast majority of patients," Tang said. "And [Uniprost] is delivered through an infusion device, the same device used to deliver insulin to diabetics. It is delivered into the skin through the belly through a tiny little needle. Patients are mobile and they can do whatever they want; they generally like it."

Although Hadeed would not speculate on the revenue Uniprost will generate, he did say "the only drug at the present time on the market is reimbursed in the neighborhood of about $50,000 to $75,000 [per patient]."

Tang said he anticipates $250 million to $300 million in annual sales.

"We believe the drug is approvable and we believe the data support approval," Tang said. "We think this is a very attractive buy - the stock is down on unfounded concern - but we think it will rebound as it approaches approval in the spring of 2001."

Tang is referring to UTC's 7.6 percent drop in July following a negative report from another analyst. The unfavorable report was based on information that UTC's two Phase III trials of Uniprost failed separately to meet their primary endpoints. (See BioWorld Today, July 17, 2000.)

In response, Hadeed said, "There was a misperception out there about the study, and previous articles were prompted by an analyst who had difficulty understanding the study in its design. Before we commenced our pivotal study, we agreed with the agency on a statistical analysis plan and received input from them. We achieved a p value of .006; the minimum they would accept was .001."

UTC's stock (NASDAQ:UTHR) closed Monday at $72.125, up $8.12, or 12.7 percent.

The two Phase III trials were randomized and double-blind. Half of the 470 patients studied worldwide received Uniprost subcutaneously for 12 weeks and the other half were given a placebo. All had the option of staying on the drug (or begining taking it) in an open-label study after finishing the pivotal trial.

UTC also remains in its second Phase III trial of Beraprost, an oral formulation of prostacyclin used in patients with peripheral vascular disease. The condition 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. (See BioWorld Today, Feb. 2, 2000; and March 28, 2000.)

Hadeed said the last patient in the Beraprost trial is expected to exit in the first half of 2001 and UTC expects to file a new drug application during the second half of 2001. In a prepared statement, Tang said Beraprost is poised to become a new alternative for the treatment of peripheral vascular disease, possessing major safety and efficacy advantages over current treatments.

UTC raised $62.1 million when it went public in June 1999, and garnered another $80 million in a private placement in last December. (See BioWorld Today, Dec. 27, 1999.) In July, UTC had definitive agreements to raise $143 million through a private placement of common stock.

In other news Monday, UTC said its synthesis and manufacturing subsidiary, SynQuest Inc., completed an FDA pre-approval inspection and had the site recommended for approval to manufacturer Uniprost.

No Comments