A cell-based therapy developed in Durham for a rare liver disease in newborns has been accepted for regulatory review in Canada.

Health Canada, the country’s health agency, accepted a New Drug Submission for the therapy, called Heparesc, from Promethera Biosciences SA, a Belgian biopharmaceutical company with a recently acquired U.S subsidiary in Durham.

Heparesc is designed to treat neonatal urea cycle disorders (UCDs). UCDs are ultra-rare genetic disorders that cause a toxic buildup of ammonia in the body that can lead to irreversible brain damage, coma and death if not treated.

Liver transplantation is the only curative therapy for the metabolic disorders, and Heparesc could stabilize patients while they await transplantation.

Acceptance of Heparesc for review “is a recognition of our dedication to working with the Canadian regulatory bodies as a start to build a path for innovative cell therapies for patients worldwide and a validation of our approach for severe liver diseases,” Promethera CEO John Tchelingerian said in a news release.

Heparesc was developed by Durham-based Cytonet before Promethera acquired Cytonet in April 2016.

Durham site, formerly Cytonet, now has 20 employees

The Durham site employs 20 people in commercial operations, GMP manufacturing and organ procurement.

“I am very proud of this joint effort, which is a prime example of the successful integration of the Cytonet and Promethera teams,” Tchelingerian said.

The acceptance of the drug submission marks the start of a 300-day review period by Health Canada. A successful review would allow Promethera to commercialize Heparesc in Canada to treat neonatal onset UCD.

The “clean room” at Promethera’s Durham facility.

The drug submission is based on two successfully completed studies of Heparesc.

Heparesc is composed of cryopreserved mature human liver cells, or hepatocytes, obtained from ethically donated livers. The fully functional cells are intended to substitute for missing or deficient urea cycle functions in the diseased livers of newborns.

Phase 2 trial started

Promethera, based in Mont-Saint-Guibert, Belgium, is mainly focused on developing the liver stem cell product HepaStem and the liver progenitor cell product H2Stem to treat acute-on-chronic liver failure, nonalcoholic fatty liver disease and liver fibrosis.

The company has started a Phase 2a trial to establish the safety of HepaStem infusions in patients with acute-on-chronic liver failure.

Last October Promethera raised $11 million in a Series C-extension financing to advance its product pipeline. All major existing investors participated: Vesalius Biocapital, SRIW, Fund+, MGI Global Fund (Mitsui & Co.), Boehringer Ingelheim Venture Fund, and SMS Investments. New investors included Mitsubishi UFJ Capital Co. and Cell Innovation Partners from Japan and LifeLiver Co. from South Korea.