United Therapeutics Announces Positive Results from Phase 1 Study of miroliverELAP® in Patients with Acute Liver Failure

Key Terms

bioengineered liver medical

A bioengineered liver is a human liver or liver tissue grown or rebuilt in a lab using living cells, scaffolds or 3D-printing techniques to replicate the organ’s structure and function for transplant or therapy. For investors, it represents a potential medical product with large demand and high development cost; success depends on clinical proof, manufacturing scale, regulatory approval and reimbursement—similar to backing a complex new factory that must prove it can reliably make a lifesaving product.

acute-on-chronic liver failure medical

A sudden, severe worsening of an existing long-term liver disease that leads to rapid loss of liver function and often triggers failure of other organs; think of a chronic problem that suddenly collapses into a medical emergency. Investors care because it drives urgent hospital treatments, accelerates demand for drugs, devices and transplants, raises short-term healthcare costs and regulatory urgency for therapies, and can materially affect companies developing liver-related diagnostics or treatments.

decellularized porcine liver scaffold medical

A decellularized porcine liver scaffold is a pig liver that has had its living cells removed, leaving behind the organ’s natural protein framework and blood-vessel channels for use as a template to grow human cells. Investors should care because this material can speed development of lab-grown organs and advanced implants by providing a ready-made structure that may reduce manufacturing time and costs, address transplant shortages, and carry specific regulatory and safety risks.

allogeneic medical

Allogeneic describes a process or material involving different individuals of the same species, such as cells, tissues, or organs donated from one person to another. It is important to investors because products or treatments based on allogeneic sources can enable scalable, off-the-shelf solutions, potentially reducing costs and increasing accessibility in healthcare and biotech industries.

endothelial cells medical

Endothelial cells form the thin inner lining of blood vessels and lymphatic channels, acting like the smooth coating inside a pipe that controls flow, exchanges substances, and responds to injury or inflammation. They matter to investors because many drugs, medical devices, and safety issues target or affect these cells; changes in endothelial function can drive disease, influence clinical trial outcomes, and affect regulatory approval and market potential.

organ procurement organizations (opos) medical

Organ procurement organizations (OPOs) are regional entities that coordinate the identification of potential donors, obtain consent, recover organs and arrange their safe delivery to transplant centers. Think of them as local logistics hubs that manage the supply chain for life-saving organs. Investors care because OPO performance, regulation and funding influence transplant volumes, demand for medical devices, drugs and hospital services, and can affect revenue prospects across the healthcare sector.

investigational new drug regulatory

An investigational new drug is a medication that is still being tested in clinical trials to determine if it is safe and effective for treating a specific condition. For investors, it represents a potential breakthrough that could lead to a new treatment and significant financial gains if successful, but also carries risks since it has not yet been approved for widespread use.

xenotransplantation medical

Transplanting organs, tissues, or cells from one species into a human, most often using genetically modified animals as donors. Investors watch this area because successful xenotransplantation could create large new markets for treating organ failure but also faces steep scientific, regulatory, ethical and reimbursement hurdles—think of fitting a part from a different-model car: if it works it can extend life and value, but compatibility and approval risks are high.

This study was the world’s first FDA-cleared clinical trial using a bioengineered liver. Based on these positive results, United Therapeutics will initiate a phase 2 study

Approximately 30% of acute liver failure patients die because they are ineligible for a liver transplant or a donated liver is not available¹, demonstrating the critical need for organ alternatives

SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- United Therapeutics Corporation (Nasdaq: UTHR) today announced positive results from its phase 1 study of miroliverELAP®, an external liver assist product, in patients with acute forms of liver failure, acute-on-chronic liver failure, and severe acute alcoholic hepatitis, collectively called acute liver failure (ALF).²

Developed by Miromatrix Medical Inc., a wholly owned subsidiary of United Therapeutics, miroliverELAP consists of an external blood circuit and a single-use bioengineered liver sustained outside of the human body to provide temporary liver support to ALF patients. The bioengineered liver is manufactured by seeding a decellularized porcine liver scaffold with allogeneic³ human endothelial⁴ cells and human liver cells isolated from donated human livers. The donated human livers are not suitable for transplantation and are provided by organ procurement organizations (OPOs).

In the study, five ALF patients who were not candidates for a liver transplant were continuously treated with miroliverELAP for at least 44 hours. The single-arm, open label, safety study met the primary endpoint of survival during miroliverELAP treatment and there were no reports of unexpected serious adverse events attributable to the miroliverELAP over a subsequent 32-day follow-up period. Full study results will be presented and published in the second half of 2026.

“This study provides early evidence that miroliverELAP, an innovative bioengineered organ alternative product, has the potential to provide liver support for patients experiencing ALF, giving their native livers more time to recover. Achieving this important milestone allows us to continue advancing miroliverELAP to help save and improve the lives of ALF patients, who face poor outcomes and a desperate need for therapies,” said Jeff Ross, Ph.D., President of Miromatrix.

“United Therapeutics is committed to developing technologies that expand the availability of transplantable organs, and the completion of this clinical trial represents yet another historic achievement for our company. We are thrilled to see positive results from this groundbreaking study of miroliverELAP in ALF patients for whom liver transplantation was not an option. We sincerely thank the patients, physicians, and OPOs who made this trial possible,” said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics.

ALF, a devastating condition that affects thousands of patients each year, is characterized by a rapid loss of liver function in a matter of days or weeks. Approximately 45% of ALF patients will experience spontaneous recovery, while 25% will receive a liver transplant, the only effective ALF treatment. Approximately 30% of ALF patients will die because they are ineligible for a liver transplant or are unable to receive one in time due to the rapid onset of the disease and the drastic shortage of transplantable organs.⁵

United Therapeutics’ organ and organ alternative manufacturing efforts consist of three platforms – xenotransplantation,⁶ allogeneic regenerative medicine, and autologous⁷ regenerative medicine – encompassing four different organs: hearts, kidneys, livers, and lungs. These programs are intended to address the ongoing shortage of transplantable organs for patients with end-stage organ disease.

Miromatrix received clearance from the United States Food and Drug Administration for its Investigational New Drug application for the phase 1 clinical trial of miroliverELAP in 2024. miroliverELAP is investigational and is not approved for any use in any country. In addition to miroliverELAP, Miromatrix is developing mirokidney, a fully transplantable bioengineered kidney, utilizing the same decellularization and seeding technology used in miroliverELAP.

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, statements regarding the future development of our miroliverELAP product, including our expectation that we will commence a phase 2 study; the potential for miroliverELAP to offer a new treatment to improve and save lives of ALF patients; and our goals of innovating for the unmet medical needs of our patients and to benefit our other stakeholders and furthering 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 January 26, 2026, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason.

MIROLIVERELAP is a registered trademark of United Therapeutics Corporation and its subsidiaries.

1 Mendizabal M, Silva MO. Liver transplantation in acute liver failure: A challenging scenario. World J Gastroenterol. 2016 Jan 28;22(4):1523-31. DOI: 10.3748/wjg.v22.i4.1523. PMID: 26819519; PMCID: PMC4721985. https://pmc.ncbi.nlm.nih.gov/articles/PMC4721985/
2 More information on the study is available at https://clinicaltrials.gov/study/NCT06285253.
3 Allogeneic cells come from a donor who is genetically different from the recipient.
4 Endothelial cells form the inner lining of blood vessels.
5 Lee, W. M., Squires, R. H., Jr, Nyberg, S. L., Doo, E., & Hoofnagle, J. H. (2008). Acute liver failure: Summary of a workshop. Hepatology (Baltimore, Md.), 47(4), 1401–1415. https://doi.org/10.1002/hep.22177
6 Xenotransplantation is the process of transplanting living cells, tissues, or organs from one species into another—most commonly from animals into humans. United Therapeutics is conducting research involving transplantation of whole organs from gene-edited pigs into humans.
7 Autologous cells are cells collected from and then used in the same individual.

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20260126667812/en/

For Further Information Contact:

Investor Inquiries

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

Media Inquiries

communications@unither.com

Source: United Therapeutics Corporation