Key Takeaways:
United Therapeutics Corp. received clearance from the U.S. Food and Drug Administration to begin a human clinical trial of a genetically modified pig heart, a major step in the quest to solve the chronic shortage of donor organs.
"Moving a porcine-derived heart into human clinical trials represents another defining advancement for the field of xenotransplantation," Kristina DeSmet, Senior Director of Product Development at United Therapeutics, said in a statement. "The heart is one of the most complex solid organs to transplant, and proceeding into the clinic reflects years of coordinated scientific progress."
The study, known as EXPRESS, will test the company’s UHeart™ in up to two patients with end-stage heart failure who have no other treatment options. The UHeart comes from a pig with 10 gene edits designed to make the organ more compatible with the human body. Six human genes were added to facilitate immunological acceptance, while four porcine genes were inactivated to reduce the risk of organ rejection and control organ growth. United Therapeutics will submit safety and efficacy data from the first transplant recipient to the FDA before enrolling a second.
This marks the third clinical trial in xenotransplantation for United Therapeutics, a public benefit corporation, broadening its platform from end-stage renal disease to life-threatening heart conditions. The decision comes as nearly 6.7 million adults in the U.S. suffer from heart failure, which accounted for 14.6 percent of all deaths in 2023, according to the CDC. While about 8,000 Americans were on the waitlist for a heart transplant last year, only about 4,000 such procedures were performed.
The EXPRESS study is designed as a seamless Phase 1/2/3 trial to evaluate the safety and efficacy of the UHeart. Key efficacy endpoints include patient and UHeart survival rates, heart function, and quality of life at 24 weeks post-transplant. Participants will be monitored for life for outcomes including survival and potential zoonotic infections.
The clearance from the FDA significantly de-risks the company's ambitious xenotransplantation program and provides a critical validation of its gene-editing approach. Investors will now watch for the initiation of the EXPRESS study and the safety and efficacy data from the first patient, which will be a key catalyst for the UHeart platform and the broader field of xenotransplantation.
This article is for informational purposes only and does not constitute investment advice.
