United Therapeutics Corporation Announces FDA Clearance to Proceed with UHeart Xenotransplantation Clinical Trial

United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance under the company’s Investigational New Drug application to proceed with a clinical study of its investigational UHeart derived from a pig with 10 gene edits. The study, known as EXPRESS, will enroll an initial cohort of up to two participants. United Therapeutics will provide the FDA with safety and efficacy data from the first UHeart xenotransplant recipient in the study before enrolling a second participant. Following FDA review of available safety and efficacy data from the initial two transplants, the study may then be further expanded, with the intent to support a Biologics License Application (BLA) with the FDA.

“Moving a porcine-derived heart into human clinical trials represents another defining advancement for the field of xenotransplantation,” said Kristina DeSmet, Ph.D., DABT, Senior Director, Product Development at United Therapeutics. “The heart is one of the most complex solid organs to transplant, and proceeding into the clinic reflects years of coordinated scientific progress. For United Therapeutics, this milestone represents our third clinical trial in xenotransplantation and underscores the breadth of our platform, spanning end-stage renal disease and now life-threatening heart disease. Together, these programs reinforce our commitment to expanding transplant options for patients who currently have no other alternatives.”

Noah Byrd, Ph.D., RAC, Vice President, Global Regulatory Affairs at United Therapeutics, added: “Patients with end stage heart disease continue to face profound limitations in available treatment options. This FDA clearance to proceed with our EXPRESS clinical trial will allow us to begin evaluating an innovative therapeutic option designed to address this unmet need.”

About EXPRESS

Study Design

The study will start at a single center enrolling up to two participants. If safety and efficacy data from these initial participants are supportive, United Therapeutics intends to expand the study to enroll participants at additional centers to support submission of a BLA. The study is designed as a phase 1/2/3 trial (sometimes referred to as a “phaseless” study) to evaluate safety and efficacy of the UHeart seamlessly without moving through separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals. Participants will receive a UHeart transplant followed by a 24-week post-transplant follow-up period, including the evaluation of all study endpoints and safety assessments. After the 24-week post-transplant follow-up period, participants who received a UHeart will continue to be followed for the rest of their lives, including for survival, UHeart survival, and screening for zoonotic infections.

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