OrganOx and eGenesis Announce Exclusive Clinical Co-Development Agreement for Treatment of Patients with Liver Failure in the Acute Setting

The companies will focus on the estimated 35,000 patients in the United States hospitalized each year for acute or acute-on-chronic liver failure^1,2. The efficacy of existing liver support options is limited, and patients in liver failure face mortality rates as high as 50 percent². For some patients, utilizing a human compatible, genetically engineered porcine liver to support the function of a patient's decompensated liver may provide time for the recovery of the patient's native liver or time to obtain a liver transplant.

Based on positive clinical data demonstrated in these initial patient populations, the companies may explore the use of this system for the over 300,000 additional patients admitted annually to hospital in the United States in the acute setting with other forms of liver failure³.

Earlier this year, the companies announced a proof of concept demonstration of the approach and have now successfully completed three such experiments as part of the PERFUSE-2 study⁴, evaluating the feasibility of using this perfusion system to support patients suffering from liver failure. The perfusions were performed using the eGenesis liver, EGEN-5784, connected to the OrganOx extracorporeal liver cross-circulation (ELC) system. eGenesis and OrganOx anticipate submitting an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in 2025 to initiate a Phase 1 clinical study.

“Our expanded partnership, and now exclusive co-development agreement with eGenesis, not only strengthens our relationship but also demonstrates our confidence in the technology we're jointly advancing,” said Craig Marshall, CEO of OrganOx. “This co-development further exemplifies our mission to improve treatment options for patients with organ failure.”

The OrganOx ELC system combines automated NMP technology with a patient support accessory device to provide support for a patient's liver function.

“We are excited to partner with OrganOx to address a critical unmet need for patients with liver failure,” said Michael Curtis, Ph.D., CEO of eGenesis. “This collaboration is a crucial step in our mission to develop safe and effective human-compatible organs for patients suffering from organ failure.”

The EGEN-5784 liver carries three classes of edits: (1) knock out of three genes involved in the synthesis of glycan antigens implicated in hyperacute rejection, (2) insertion of seven human transgenes involved in the regulation of several pathways that modulate rejection: inflammation, innate immunity, coagulation, and complement, and (3) inactivation of the endogenous retroviruses in the porcine genome.

About OrganOx
OrganOx is a commercial stage organ technology company, spun out of the University of Oxford in 2008. The company's first product, the OrganOx metra^® normothermic machine perfusion (NMP) system for liver transplantation, has been used to support more than 4,000 liver transplant operations globally, optimizing the use of donated organs by enabling assessment of the quality of livers as well as longer preservation durations. Learn more at www.organox.com.

About eGenesis
eGenesis is pioneering a genome engineering-based approach in the development of safe and effective transplantable organs. The eGenesis Genome Engineering and Production (EGEN™) Platform is the only technology of its kind to comprehensively address cross-species molecular incompatibilities and viral risk via genetic engineering. eGenesis has demonstrated durable preclinical success to date and is advancing development programs for acute liver failure, kidney transplant, and pediatric as well as adult heart transplant. Learn more at www.egenesisbio.com.

Contacts

OrganOx Investor Relations:
Steve Deitsch
Chief Financial Officer, OrganOx
[email protected]

OrganOx Media:
Emma Yang
Health+Commerce
[email protected]

eGenesis Media:
Kimberly Ha
[email protected]

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. 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.
   
4. 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.