Research
United Therapeutics is advancing a pipeline of research and development projects that includes new indications, formulations, and delivery devices for its existing products. The company's R&D pipeline includes five registration phase studies, primarily in rare lung diseases, and seven preclinical product leads, primarily in organ manufacturing.
In February 2020, United Therapeutics reported that it had successfully completed the INCREASE study of Tyvaso in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) and that the study met its primary endpoint of demonstrating improvement in six-minute walk distance (6MWD).[30] The company submitted the INCREASE study results to the U.S. Food and Drug Administration in support of an efficacy supplement to the Tyvaso New Drug Application and received approval on March 31, 2021.[31]
Ongoing clinical trials of new medicines include ADVANCE OUTCOMES (Phase 3) for ralinepag in pulmonary arterial hypertension; a registration study of the Centralized Lung Evaluation System;[32] and TETON 1 and TETON 2 for idiopathic pulmonary fibrosis (IPF).[33]
United Therapeutics is developing a unique type of biopharmaceutical medicinal product known as a manufactured organ. This type of biologic would be transplanted into the patient as a substitute for a failing organ. The company has regenerative medicine products such as these being developed via xenotransplantation, allogenic recellularization, and autologous bioprinting technologies.[34]
In 2021, researchers at New York University used a ThymoKidney xenokidney in the first transplant of a genetically engineered, nonhuman kidney into a brain-dead human body, observing kidney function over 54 hours.[35] Researchers at The University of Alabama at Birmingham used a UKidney xenokidney in a transplant into a brain-dead recipient, observing kidney function over 77 hours.[36]
In 2021, engineers from the company's Unither Bioelectronics subsidiary conducted the first flight of human lungs for transplant from Toronto Western Hospital to Toronto General Hospital.[37] The lungs were successfully transplanted into Alain Hodak, an engineer from Ottawa, Canada.[38]
In 2022, the UHeart xenoheart was used in the first pig heart to living human transplant.[39] The transplanted pig heart had ten genetic modifications to the pig genome.[40]
In 2022, Martine Rothblatt debuted a 3D printed lung scaffold that was what she called one of the "most complex 3D printed objects ever printed."[41] The printed lung scaffold contained 44 trillion voxels that laid out 4,000 kilometers of pulmonary capillaries and 200 million pulmonary alveoli.
