United Therapeutics Maps 14 Launches, Fibrosis Growth and Xenokidney Ambitions

Key Points

• United Therapeutics is expanding its pulmonary fibrosis and pulmonary hypertension pipeline, with 14 potential launches planned. Key milestones include an FDA decision on nebulized Tyvaso for idiopathic pulmonary fibrosis, a planned CoughLess Trezmi filing, and an expected June 24 PDUFA date for once-daily oral ralinepag.
• The company is actively investing in growth while continuing capital returns, including share repurchases under a $2 billion authorization. Additional programs target progressive pulmonary fibrosis, COPD-associated pulmonary hypertension, and reduced pill burden through combination therapies.
• Xenotransplantation remains a major long-term ambition: United Therapeutics has transplanted four patients in its xenokidney registration trial, aims to enroll 30 by 2028, and targets commercial availability by 2030. It has built three xenograft production facilities and is also developing genetically modified pig hearts and thymus-integrated organs.

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).

Pulmonary fibrosis expansion

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.

Ralinepag and capital allocation

United Therapeutics has submitted ralinepag, a once-daily oral therapy for pulmonary hypertension, to the FDA. Rothblatt said the agency assigned a PDUFA date of June 24 next year. She described ralinepag as the only drug tested in pulmonary hypertension that demonstrated clinical improvement in patients, in addition to slowing disease progression.

The company is also developing a triple-combination version of ralinepag that would include a PDE5 inhibitor and an endothelin receptor antagonist, or ERA, in a single pill. Rothblatt said the program is intended to reduce patients’ pill burden.

Other development programs include TRESMI for COPD-associated pulmonary hypertension, an indication Rothblatt said affects about 600,000 U.S. patients and currently has no approved therapies. The company is also developing ILO Tra, a rescue inhaler for pulmonary hypertension that combines two prostacyclin-type molecules.

Chief Financial Officer James Edgemond said capital allocation remains a major focus as the company invests in its development programs. He said United Therapeutics is actively repurchasing shares under a $2 billion authorization approved by the board earlier this year.

“We think with this explosive growth on the horizon and where we see the value of the stock today is one of the best uses of capital that we are pursuing right now,” Edgemond said.

Organ manufacturing plans

Rothblatt also detailed United Therapeutics’ xenotransplantation programs, which use genetically modified pig organs for human transplantation. The company is conducting a registration trial of its xenokidney program, with four patients already transplanted and two additional procedures planned this year, she said.

After the sixth patient reaches three months following transplantation, United Therapeutics expects to present data to the FDA to seek authorization to continue the trial. Rothblatt said the company expects the trial to enroll 30 patients in total by 2028 and aims for potential commercial availability no later than 2030.

The company has built three commercial xenograft production facilities in Virginia, Rochester, Minnesota, and Houston. Edgemond said United Therapeutics adjusted its manufacturing plans over time to reduce upfront capital requirements and better align future facility investments with commercial demand.

United Therapeutics is also pursuing clinical programs involving xenotransplanted hearts and its UThymoKidney and UHeart concepts. Rothblatt said those programs incorporate thymus tissue intended to train a recipient’s immune system to recognize the transplanted pig organ, potentially reducing or eliminating the need for chronic immunosuppression.

Finally, Rothblatt highlighted the company’s Computational Laboratory for In Silico Molecular Biology, or CLIMB, which she said was developed to create a detailed digital model of the lung for evaluating potential treatments in pulmonary hypertension and pulmonary fibrosis. She said the company has used the model in assessing internal drug candidates and is receiving interest from pharmaceutical companies seeking to test their molecules through the platform.

About United Therapeutics (NASDAQ:UTHR)

United Therapeutics Corporation is a biotechnology company focused on developing and commercializing therapies for serious and life-threatening conditions, with a primary emphasis on pulmonary arterial hypertension (PAH). The company's products are designed to improve pulmonary blood flow and reduce the effects of this progressive disease.

United Therapeutics' PAH portfolio includes Remodulin (treprostinil) injection, Orenitram (treprostinil) extended-release tablets, and Tyvaso (treprostinil), which is available as an inhalation solution and as the Tyvaso DPI dry-powder inhaler.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.