United Therapeutics Maps 14 Launches, Fibrosis Growth and Xenokidney Ambitions

United Therapeutics NASDAQ: UTHR outlined plans to expand its pulmonary fibrosis, pulmonary hypertension and organ-manufacturing businesses at the Bernstein Healthcare Leaders and Disruptors Conference, with management describing a pipeline of 14 prospective product launches and continued share repurchases.

Founder, Chairperson and Chief Executive Officer Martine Rothblatt said the company is positioned for what she characterized as an “exponential growth trajectory,” driven largely by therapies for idiopathic pulmonary fibrosis (IPF), progressive pulmonary fibrosis (PPF), interstitial lung disease (ILD) and pulmonary hypertension (PH).

Rothblatt said the FDA has accepted United Therapeutics’ new drug application for nebulized Tyvaso in IPF, with a PDUFA action date expected early next year. The company also plans to pursue the use of Tyvaso DPI, an approved dry-powder inhalation product, in IPF and PPF.

She said the company is developing CoughLess Trezmi, a soft-mist inhaled therapy, for ILD, IPF and PPF. United Therapeutics expects to submit an NDA for that product before year-end and anticipates a launch next year, according to Rothblatt.

Another pipeline candidate, Once Daily Ralpi, is being developed for IPF, PPF and ILD. Rothblatt said the molecule has once-daily dosing potential and, in the company’s view, has stronger anti-fibrotic activity than treprostinil.

Rothblatt also discussed the company’s IPF development strategy for nebulized Tyvaso. She said United Therapeutics used secondary endpoints from its prior INCREASE study in PH-ILD to support its request to move into a Phase III IPF study. The FDA required two adequate and well-controlled Phase III trials in IPF, she said.

The company subsequently completed the TETON-1 and TETON-2 studies, which Rothblatt said showed an improvement in forced vital capacity. She said the results supported the company’s expectation that Tyvaso could provide a meaningful clinical benefit for pulmonary fibrosis patients.

United Therapeutics has doubled its sales force in preparation for a potential IPF launch and plans to launch in Europe as well, she said. For PPF, Rothblatt said the FDA allowed the company to conduct a single study. That trial is fully enrolled, with results expected to be unblinded next year.

United Therapeutics has submitted ralinepag, a once-daily oral therapy for pulmonary hypertension, to the FDA. Rothblatt said the...