eGenesis and OrganOx Announce U.S. FDA Clearance of IND Application for the Treatment of Patients with Acute-On-Chronic Liver Failure

CAMBRIDGE, Mass. & OXFORD, England–(BUSINESS WIRE)–eGenesis, a biotechnology company developing human-compatible engineered organs to treat patients suffering from organ failure, and OrganOx Ltd., a leading organ medical technology company, today announced the U.S. Food & Drug Administration (FDA) has cleared the investigational new drug (IND) application for EGEN-5784, a human-compatible, genetically engineered porcine liver, used in combination with the OrganOx extracorporeal liver cross-circulation (ELC) system, for patients with acute-on-chronic liver failure (ACLF) suffering from decompensated liver function in the intensive care setting.

In November 2024, OrganOx and eGenesis entered into an exclusive clinical co-development agreement to advance this potentially lifesaving technology for the estimated 35,000 patients in the United States hospitalized each year for acute or acute-on-chronic liver failure^1,2. These patients face limited existing support options and short-term mortality rates as high as 50 percent². eGenesis’ human-compatible, genetically engineered porcine liver, used in combination with OrganOx’s ELC system to support the function of a patient’s decompensated liver, may allow for the potential recovery of the patient’s native liver, or provide sufficient time to receive a liver transplant.

To date, four perfusions with decedent recipients have been completed as part of the PERFUSE-2 study³, demonstrating the feasibility of using EGEN-5784 with the OrganOx ELC system to support patients suffering from liver failure.

As part of the Phase 1 trial, up to twenty (20) patients with ACLF (Grade 2 to Grade 3) and hepatic encephalopathy (≤ Grade 3) and ineligible for transplant will be enrolled across multiple U.S. centers. The trial will evaluate the safety, performance, and clinical activity of the EGEN-5784 liver in combination with the OrganOx ELC system in these patients.

“The FDA’s clearance of EGEN-5784 in combination with the OrganOx ELC system represents a significant advance towards fulfilling our mission to develop safe and effective human-compatible organs that have the potential to transform the treatment of organ failure and extend patients’ lives,” said Michael Curtis, Ph.D., Chief Executive Officer, eGenesis. “In partnership with OrganOx, we are well positioned to evaluate the potential of this breakthrough technology in patients.”

“This collaboration with eGenesis demonstrates a unique application of our platform metra® technology which can potentially save the lives of patients with acute-on-chronic liver failure who have limited treatment options and face high mortality rates,” said Craig Marshall, Chief Executive Officer, OrganOx.

About eGenesis

eGenesis is pioneering a genome engineering-based approach to develop safe, effective transplantable organs to end the global organ shortage. Its platform uniquely addresses cross-species molecular incompatibilities and viral risk via its proprietary genetic engineering platform to improve outcomes for patients in need of a transplant. With demonstrated preclinical success, eGenesis is advancing programs in kidney transplantation, acute liver failure, and heart transplantation. Learn more at www.egenesisbio.com. Follow us @egenesisbio.

About OrganOx

OrganOx is a commercial stage organ technology company, spun out of the University of Oxford in 2008, dedicated to developing technologies to improve outcomes for patients with acute or chronic organ failure. The OrganOx metra® is a normothermic machine perfusion (NMP) platform approved in the U.S., Europe, Canada, and Australia. It has been utilized in over 5,000 liver transplants to date to keep donor livers in a metabolically active state outside the body so that functional assessment of the organ can be performed prior to transplant, leading to an increased number of organs available for transplant. Learn more at www.organox.com.

References

1. Stravitz, R.T. et al. (2023) Future directions in acute liver failure. Hepatology, 78(4), pp. 1266–1289. https://doi.org/10.1097/hep.0000000000000458.
2. Allen, A.M., et al. (2016). Time trends in the health care burden and mortality of acute on chronic liver failure in the United States. Hepatology, 64(6), pp. 2165–2172. https://doi.org/10.1002/hep.28812.
3. OrganOx (2024). Successful Use of a Genetically Engineered Porcine Liver with a Human Donor. January 18th, 2024. Available at: https://www.organox.com/news/view/successful-use-of-a-genetically-engineered-porcine-liver-with-a-human-donor.
4. Jinjuvadia, R. and Liangpunsakul, S. (2014) Trends in alcoholic hepatitis-related hospitalizations, financial burden, and mortality in the United States. Journal of Clinical Gastroenterology, 49(6), pp. 506–511. https://doi.org/10.1097/mcg.0000000000000161.