United Therapeutics Reports Positive Phase 1 Results for World's First Bioengineered Liver Device in Acute Liver Failure
Key Insights
United Therapeutics announced positive results from the world's first FDA-cleared clinical trial using a bioengineered liver device, miroliverELAP (search), for treating acute liver failure patients.
The Phase 1 study successfully met its primary safety endpoint in five patients who were not transplant candidates, with continuous treatment for at least 44 hours and no unexpected serious adverse events.
Approximately 30% of acute liver failure patients die due to transplant ineligibility or organ unavailability, highlighting the critical need for alternative treatments like miroliverELAP (search).
United Therapeutics Corporation announced positive results from its groundbreaking Phase 1 study of miroliverELAP (search), marking the world's first FDA-cleared clinical trial using a bioengineered liver device to treat patients with acute liver failure (ALF). The study represents a significant milestone in addressing the critical shortage of transplantable organs and provides new hope for patients facing this devastating condition.
Revolutionary Bioengineered Liver Technology
Developed by Miromatrix Medical Inc., a wholly owned subsidiary of United Therapeutics, miroliverELAP (search) 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 innovative device is manufactured by seeding a decellularized porcine liver scaffold with allogeneic human endothelial cells and human liver cells isolated from donated human livers that are not suitable for transplantation, provided by organ procurement organizations.
Phase 1 Study Results Demonstrate Safety and Efficacy
The single-arm, open-label safety study enrolled five ALF patients who were not candidates for liver transplantation. These patients were continuously treated with miroliverELAP (search) for at least 44 hours. The study successfully met its primary endpoint of survival during miroliverELAP treatment, with no reports of unexpected serious adverse events attributable to the device over a subsequent 32-day follow-up period.
"This study provides early evidence that miroliverELAP (search), 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," said Jeff Ross, Ph.D., President of Miromatrix. "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."
Addressing Critical Unmet Medical Need
ALF is a devastating condition that affects thousands of patients each year, characterized by rapid loss of liver function within days or weeks. Current treatment outcomes reveal the severity of this medical challenge: approximately 45% of ALF patients experience spontaneous recovery, while 25% receive a liver transplant, which remains the only effective treatment. However, approximately 30% of ALF patients 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.
Regulatory Pathway and Future Development
Miromatrix received FDA clearance for its Investigational New Drug application for the Phase 1 clinical trial of miroliverELAP (search) in 2024. Based on the positive Phase 1 results, United Therapeutics plans to initiate a Phase 2 study. Full study results will be presented and published in the second half of 2026. The device remains investigational and is not approved for any use in any country.
Broader Organ Alternative Platform
United Therapeutics' organ and organ alternative manufacturing efforts encompass three platforms – xenotransplantation, allogeneic regenerative medicine, and autologous regenerative medicine – covering four different organs: hearts, kidneys, livers, and lungs. These programs are designed to address the ongoing shortage of transplantable organs for patients with end-stage organ disease. In addition to miroliverELAP (search), Miromatrix is developing mirokidney, a fully transplantable bioengineered kidney, utilizing the same decellularization and seeding technology.
"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," said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics. "We are thrilled to see positive results from this groundbreaking study of miroliverELAP (search) in ALF patients for whom liver transplantation was not an option."