United Therapeutics' inhaled treprostinil was the subject of much chatter at a Feb. 11-14 scientific meeting on pulmonary hypertension. The inhaled version of United's prostacyclin analog Remodulin , already sold in injectable and intravenous forms, could hit a sweet spot in the PAH market. Insiders expect the drug to be approved by first quarter 2009.

During its fourth quarter earnings call Feb. 19, United said higher expenses and the impact of tax-gain differences put a 96 percent dent in the Silver Spring, Md.-based company's profits compared with the same period in 2006. But revenue reached $59.9 million, up 30 percent, thanks mainly to sales of Remodulin for pulmonary arterial hypertension.

United told "The Pink Sheet" DAILY in December that it plans to file an NDA for Inhaled Remodulin - branded Viveta - before June as second-line therapy or an add-on to existing therapies for PAH (¹ Also see "United Therapeutics Drops Ovarian Cancer Compound Following Phase III Failure" - Pink Sheet, 6 December, 2007.).

Viveta, for which United reported strong Phase III data from the TRIUMPH-1 trial in November, would enter a market where Actelion's oral endothelin receptor antagonist (ETRA) Tracleer (bosentan) has long held a strong position (² Also see "Actelion CEO Jean-Paul Clozel: An Interview With “The Pink Sheet” DAILY (Part 1 of 2)" - Pink Sheet, 29 January, 2008.).

Making inroads against Tracleer is Gilead's Letairis (ambrisentan), cleared by FDA last summer as the second oral ETRA, beating Encysive's Thelin (sitaxsentan) to market (³ Also see "Gilead Letairis Will Be Available Through LEAP Restricted Distribution Program" - Pink Sheet, 15 June, 2007.). FDA has hit Thelin with three "approvable" letters, and Encysive plans another Phase III trial (⁴ Also see "Encysive Rules Out FDA Dispute Resolution Path For Thelin NDA" - Pink Sheet, 25 September, 2007.).

Typically, chronic PAH patients get prostacyclin analogs, noted Jennifer Chao, analyst with Deutsche Bank. The class includes Flolan (epoprostenol) from GlaxoSmithKline and Ventavis (iloprost) from Actelion.

"First-line therapy is Revatio [Pfizer's sildenafil], which is basically Viagra , and then they progress onto a Tracleer or Letairis, and after three years of an ETRA, they go to a prostacyclin analog," Chao told "The Pink Sheet" DAILY.

"Flolan sells about $400 million, Ventavis around $60 million to $80 million, so that's a half-billion-dollar market that could eventually quite easily go to inhaled Remodulin," she said.

"This really is the sweet-spot patient population," Chao added. "Because of its sheer ease of dosing and improved compliance, you're likely to see an inhaled Remodulin being used upstream in combination care."

Doctors Voice Enthusiasm For Viveta

At the fourth Symposium on Pulmonary Hypertension in Dana Point, Calif., physicians proved enthusiastic about the vasodilator's new form.

Andrew Fein, analyst with Collins Stewart, noted that a small camp of Viveta skeptics at the meeting worried that the trial's failure to hit its target improvement of 35 meters in the 6-minute walk distance (6MWD) test might hamper approval chances.

"Many of the clinicians acknowledge that though TRIUMPH-1 data was highly statistically meaningful, the question as to whether a 20-meter improvement in 6MWD is clinically meaningful exists," Fein wrote in a Feb. 19 research report. However, he predicted the debate would become "more of a philosophical [one]" when United files the NDA.

United has an oral treprostinil program as well, but Fein found "muted optimism (at best)" for that effort. Oppenheimer analyst Bret Holley called the oral drug a wild card, and wrote in a Feb. 19 research report that positive results from a Phase II/III trial, due in the second half of this year, would be "an upside surprise."

Fourth-quarter earnings per share totaled 8 cents, versus the consensus estimate of 45 cents. A tax benefit of $7.1 million for the quarter in 2007 fell far short of the $47.7 million in the same period the previous year.

"The key metric, however, is Remodulin where fourth-quarter sales of $57 million were just shy of our estimate of $59 million but in line with consensus ($57 million) and flat quarter-over-quarter from record third-quarter sales," wrote JPMorgan analyst Geoffrey Meacham in a research report. "The Street had feared a sequentially down Remodulin number of around $50 million to $55 million."

Meacham called the quarter "solid," and judged a recent pullback in the stock unwarranted. Shares have slumped since just after the start of the year because of insider selling, Chao said.

She called the California meeting "insignificant" in the big picture for United, and cited instead the full data from TRIUMPH-1 due in May at the American Thoracic Society meeting, followed by the NDA filing for Viveta in June.

"Then we're looking at Phase III oral [treprostinil] results, starting in the fourth quarter, and approval of [Viveta] in the first quarter of 2009," she said.

She dubbed United "worthy of consideration for a generalist audience looking for a growth story without mitigated, non-binary regulatory risk. In biotech, those stories are very few and far between."

- Randall Osborne (r.osborne@elsevier.com)

United Therapeutics Reports ‘Solid’ Fourth Quarter As Inhaled Remodulin Gains Fans

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