Promethera Announces Review Acceptance of Heparesc New Drug Submission for Neonatal Onset Urea Cycle Disorders by Health Canada

Mont-Saint-Guibert, Belgium, July 3, 2017 – Promethera Biosciences SA, a global innovator in cell-based medicines, today announced that Health Canada has accepted its New Drug Submission (NDS) of Heparesc in neonatal onset Urea Cycle Disorders (UCD) for review. UCDs are ultra-rare genetic disorders causing the accumulation of toxic ammonia levels for which liver transplantation is currently the only curative, but not immediately available, treatment to correct the metabolic defects. Heparesc is composed of cryopreserved mature human liver cells (hepatocytes) obtained from ethically donated livers. The fully functional mature human hepatocytes aim to substitute missing deficient urea cycle functions in neonatal onset patients’ livers. Heparesc could serve as a bridge to transplantation while increasing metabolic stability.

“This achievement 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,” said John Tchelingerian, CEO of Promethera. “While our strategic focus remains on our stem/progenitor cell products such as HepaStem and H2Stem and the larger indications ACLF and NASH, I am very proud of this joint effort which is a prime example of the successful integration of the Cytonet and Promethera teams.”

The acceptance of the NDS completes the validation period and marks the start of a 300-day review period by Health Canada. Upon potential successful outcome of the review, Promethera would be able to commercialize Heparesc in neonatal onset UCD in Canada. The New Drug Submission is based on two successfully completed studies of Heparesc, which is one of the assets Promethera gained access to through the acquisition of Cytonet in April 2016.