United Therapeutics Corporation Announces First Transplant in EXPAND Clinical Trial of UKidney in Patients With End-Stage Renal Disease

Ongoing EXPAND Study is the First Clinical Trial to Evaluate Xenotransplantation in End-Stage Renal Disease

United Therapeutics Corporation (Nasdaq: UTHR) today announced the first clinical xenotransplantation in its EXPAND study of the UKidney™ in patients with end-stage renal disease (ESRD). The successful transplant operation was performed at NYU Langone Health.

United Therapeutics' UKidney is an investigational xenokidney from a pig with 10 gene edits. Six human genes are added to the pig genome to facilitate immunological acceptance and compatibility of the organ in the human recipient. Four porcine genes are inactivated or "knocked out" to reduce the risk of organ rejection and to moderate organ growth.

"The first transplant in this first-of-its-kind study marks a watershed moment for United Therapeutics' vision to broaden access to transplantable organs," said Leigh Peterson, Ph.D., Executive Vice President, Product Development and Xenotransplantation at United Therapeutics. "This trial moves us closer to offering ESRD patients an alternative to lifelong dialysis, especially those who are unlikely to receive a kidney from a human donor. Our team is grateful for the collaboration that made this possible, and we remain focused on patient safety and scientific progress as the study continues."

"This achievement marks a transformative moment in transplant medicine. The EXPAND study offers new hope to the thousands of Americans currently on the kidney transplant waiting list, many of whom may not survive long enough to receive a human organ. This milestone brings us closer to a future where xenotransplantation may one day address the critical organ shortage crisis," said Robert Montgomery, M.D., D.Phil., the H. Leon Pachter, M.D., Professor of Surgery, Chair of the Department of Surgery at the NYU Grossman School of Medicine, and Director of the NYU Langone Transplant Institute, who led the transplant team that performed the first xenotransplantation in the EXPAND study.

About the Study

Study Design

EXPAND (NCT06878560) is a multicenter, open-label, safety and efficacy study and is intended to support a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA). The study is designed as a combination phase 1/2/3 trial (sometimes referred to as a "phaseless" study) to evaluate safety and efficacy seamlessly without moving through separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals.

Study participants will receive a UKidney 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 UKidney will continue to be followed for the rest of their lives, including monitoring for UKidney function and zoonotic infections.

Efficacy Endpoints

Efficacy endpoints include participant survival rate, UKidney survival rate, change in measured glomerular filtration rateⁱ, and change in quality of life in participantsⁱⁱ at 24 weeks post-transplant. Overall survival time of participants receiving a UKidney and overall survival time of the UKidneys themselves are also efficacy endpoints.

Safety Endpoints

Safety endpoints include the incidence of adverse events and serious adverse events, all-cause mortality, and the incidence of proteinuriaⁱⁱⁱ, zoonotic infections, and opportunistic infections^iv.

Cohort-Based Design

The first cohort will consist of six transplants at two centers. There will be a 12-week waiting period between the first and second transplants. After the initial cohort reaches at least 12 weeks post-transplant, safety and efficacy data will be reviewed by an Independent Data Monitoring Committee to determine if the study should proceed to the next cohort.

After reviewing the data from the first six transplants with the FDA, United Therapeutics plans to amend the study protocol to increase the study sample size to support a BLA and include additional transplant centers.

Additional Key Participation Criteria

Additional key participation criteria include an age of 55 to 70 years old, a diagnosis of ESRD, and at least six months on hemodialysis. Participants will be screened using a crossmatch assay to assess expected immunological compatibility with the UKidney. Participants must not need multiple organ transplants; must not have severe medical co-morbidities, including but not limited to advanced cardiovascular disease, severe peripheral vascular disease, severe neurological disease, chronic pulmonary disease, and uncontrolled diabetes; and must not have a history of medical noncompliance that may preclude adherence to the demands and requirements of xenotransplantation.

Full inclusion and exclusion criteria for this study are available on the clinicaltrials.gov website.

About End-Stage Renal Disease

According to the American Kidney Fund, there are approximately 815,000 patients with kidney failure in the United States and more than 555,000 patients on dialysis, approximately 90,000 of whom are on the U.S. kidney transplant waiting list. Only 22,000 deceased donor kidney transplants occurred in 2024^v. A 2021 study found that three years after starting dialysis, only 12% of patients had been placed on an Organ Procurement and Transplant Network kidney transplant waitlist while more than 40% died^vi.

United Therapeutics: Enabling Inspiration

At United Therapeutics, our vision and mission are one. We use our enthusiasm, creativity, and persistence to innovate for the unmet medical needs of our patients and to benefit our other stakeholders. We are bold and unconventional. We have fun, we do good. We are the first publicly-traded biotech or pharmaceutical company to take the form of a public benefit corporation (PBC). Our public benefit purpose is to provide a brighter future for patients through (a) the development of novel pharmaceutical therapies; and (b) technologies that expand the availability of transplantable organs.

You can learn more about what it means to be a PBC here: unither.com/pbc.

Forward-Looking Statements

Statements included in this press release that are not historical in nature are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, United Therapeutics' vision to broaden access to transplantable organs; the potential to offer ESRD patients an alternative to lifelong dialysis; our plans and aspirations concerning the EXPAND clinical study, including our focus on patient safety and scientific progress as the study continues; the possibility of xenotransplantation to address the critical organ shortage crisis; the potential for expansion of the EXPAND study following future FDA interactions; and our efforts to innovate for the unmet medical needs of our patients, to benefit our other stakeholders, and to pursue our public benefit purpose of developing novel pharmaceutical therapies and technologies that expand the availability of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language, and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of November 3, 2025, and assume no obligation to update or revise the information contained in this press release whether because of new information, future events or any other reason.

View source version on businesswire.com: https://www.businesswire.com/news/home/20251103435431/en/

For Further Information Contact:

Investors

https://ir.unither.com/contact-ir

Media

[email protected]