United Therapeutics’ bioengineered external liver assist system developed to treat patients with acute forms of chronic liver failure proved promising based on early phase 1 trial results.
Five patients with acute liver failure (ALF) and not eligible for a liver transplant were given continuous treatment with the miroliverFLAP platform for at least 44 hours. All met the primary endpoint of survival, and there were no reports of serious adverse events related to the system over a 32-day follow-up period, the company said in a Jan. 26 press release.
Full study results are expected to be published later this year.
The company’s miroliverELAP platform was developed by its subsidiary Miromatrix that United acquired in 2023 for $91 million.
“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,” Jeff Ross, Miromatrix president, said in a statement. “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.”
The system uses an external blood circuit and single-use bioengineered liver housed outside the human body that provides temporary support for liver functions. The bioengineered liver is produced by seeding a decellularized pig liver scaffold with allogeneic human endothelial cells and human liver cells isolated from donated human livers.
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About 30% of ALF patients will die because they are ineligible for a liver transplant or are unable to receive a transplant in time due to the rapid onset of the disease that can occur in a matter of days or weeks and the paucity of transplantable organs, the company said.
The FDA granted the platform NDA status for phase 1 clinical trials in 2024. It is currently not approved for use in any country. Miromatrix is also working on producing a mirokidney, a fully transplantable bioengineered kidney that uses the same decellularization and seeding technology as the miroliverELAP.