By Kim Coghill
Following beraprost¿s failure to meet endpoints in peripheral vascular disease patients, United Therapeutics Corp. said it intends to hang its immediate hopes for financial success on sales of Remodulin.
The company¿s stock (NASDAQ:UTHR) responded to the news Monday by falling $6.32, or 38.6 percent, to close at $10.04.
In a conference call Monday, Martine Rothblatt, United Therapeutics¿ chairman and CEO, told reporters and analysts that the company will abandon plans to develop beraprost for intermittent claudication (pain when walking) resulting from peripheral vascular disease (PVD). Preliminary data from a second Phase III trial of beraprost did not confirm the positive results of the European Phase III trial and statistical significance was not achieved in the study¿s endpoints relating to exercise.
On the positive side for the company, in August the FDA¿s Cardiovascular and Renal Drugs Advisory Committee recommended approval of Remodulin (formerly known as UT-15 and Uniprost) as a subcutaneous treatment for pulmonary arterial hypertension (PAH). The company expects approval from the FDA soon. (See BioWorld Today, Aug. 10, 2001.)
But regarding beraprost, ¿It was one of our two lead drugs,¿ Rothblatt said. ¿We are, within the company, very sorry for the patients, the clinicians, the investors and the employees, all of the various stakeholders in United Therapeutics who were counting, as we were in management, on beraprost for peripheral vascular disease. However, this is just one of the assets of United Therapeutics and we all remain extremely committed and confident about our company¿s ability to ultimately deliver success in the pharmaceutical area.¿
Beraprost was studied in about 750 patients in 60 U.S. centers in a trial expected to confirm results of an earlier Phase III trial of 422 patients in Italy and France. (See BioWorld Today, Feb., 2, 2000.)
United Therapeutics licensed beraprost from Toray Industries of Japan in June 2000. UTC was to pay Toray $1 million plus 200,000 shares of common stock, shares that closed at $108.06 the day the deal was signed. In November 2000, UTC entered a five-year deal with Professional Detailing Inc., of Upper Saddle River, N.J., under which PDI would provide prelaunch and commercialization services for beraprost. (See BioWorld Today, June 30, 2000, and Nov. 3, 2000.)
Aside from the PVD indication, beraprost, an oral formulation of prostacyclin, is in Phase III trials to treat pulmonary arterial hypertension. The trial is expected to be complete in February and data should be analyzed by the second quarter of 2002, Roger Jeffs, president of United Therapeutics, told listeners of the conference call. He said the market for that indication should reach about 50,000 patients.
The company also expects a respectable market for Remodulin, an orphan drug.
¿We are prepared to launch Remodulin rapidly and we certainly believe it should have similar success as enjoyed by Flolan [epoprostenol; GlaxoSmithKline plc],¿ Rothblatt said. ¿We believe Flolan is currently doing $200 million annually in sales and we believe Remodulin has a very attractive profile as compared to Flolan in terms of its safety and convenience. And should we be able to achieve the level of sales that Flolan currently earns, the company will be able to produce extremely attractive results for its investors.¿
Flolan is the only product on the market for PAH. However, the day after the FDA panel recommended approval of Remodulin, the same panel recommended approval of Tracleer (bosentan), Actelion Ltd.¿s oral treatment for PAH. And in September, Actelion received an approvable letter from the FDA. (See BioWorld Today, Sept. 19, 2001, and Aug. 12, 2001.)
The difference in the products? Flolan is pumped in constantly, Remodulin is a subcutaneous treatment and Tracleer an oral treatment.
Martine said, ¿We believe we should be able to capture the vast majority, it not all of the [Remodulin] market if we are approved and if the doctors believe Remodulin is appropriate for patients. We would end up with revenues for the company that are substantially greater than our expenses.¿
Rothblatt said United Therapeutics expects Remodulin to be approved in Europe by the end of the year.
For the six months ended June 30, United Therapeutics¿ revenues totaled $2.8 million, up from $856,000. Net losses fell 45 percent to $20 million. Revenues reflect an increase in resales of pumps and supplies to distributors, the company said. Lower losses reflect decreased research and development expenses for acquired products and product rights.
Rothblatt said the company¿s burn rate is about $50 million per year and the company has 20 million outstanding shares. It had about $194 million in cash and equivalents on June 30.
When asked whether United Therapeutics could be profitable on Remodulin alone, Rothblatt said, ¿While it cost us a huge amount of money to develop Remodulin, it is a drug which we believe will be adequate to cover the ongoing fixed costs of the company as well as generate substantial revenue that can enable us to continue developing additional drugs.¿
She added that United Therapeutics¿ burn rate should not change and historically it has remained steady. ¿Certainly, for myself as CEO, I think this makes the company a more stable company.¿