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Updated Phase 2a Results For First Stem Cell Trial In ACLF.

MONT-SAINT-GUIBERT, Belgium, April 15, 2019 -- Promethera Biosciences SA, a global innovator in cell-based medicines and liver diseases, announced clinical data from the ongoing phase 2a study of its lead product candidate HepaStem in patients with acute-on-chronic liver failure (ACLF) or acute decompensation (AD) at high risk of developing ACLF.

The data were presented at the International Liver Congress (ILC) on April 13 in Vienna, Austria, by Promethera's principal investigator Frederik Nevens of Katholieke Universiteit Leuven (KULeuven, Belgium).

Liver transplant is the only rescue treatment for patients suffering of ACLF and to a lesser degree for AD patients.

No other therapies can rescue the end-stage-liver failure.

Promethera is developing HepaStem as an alternative to transplant; it comprises liver stem cells obtained from ethically donated healthy human organs and expanded in GMP culture conditions.

The liver stem cells migrate through the blood and reach the liver, where they settle and support the tissue regeneration function.

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In the trial, HepaStem cells have been infused intravenously in single or repeated infusions in multiple ascending doses in patients to assess the tolerability and the safety profile of the treatment.

The trial recruited a total of 19 patients with a mean CLIF-ACLF score of 50 (12 patients) and a mean CLIF-AD score of 54 (7 patients), both parameters used as a disease severity score and predict the 28-day mortality.

With two repeated doses at 0.5 million cells per kilogram of body weight and one infusion of 1 million cells per kilogram of body weight, no adverse events related to HepaStem occurred and no clinically significant changes were shown in platelet count, fibrinogen levels, and coagulation factors following infusion.

In addition to this positive safety profile, the study has been showing trends in efficacy with improvement in three indicators of liver disease severity; Model for End Stage Liver Disease score (MELD), Child-Pugh score and bilirubin levels, at day 28 and up to three months after treatment initiation.

"Pioneering the use of liver stem cells for the treatment of ACLF, we are excited to pass this first major hurdle of establishing a well-defined safety profile, as we continue to advance HepaStem through the clinic," said CMO Etienne Sokal.--

The company said it plans to conduct a larger trial in ACLF focusing on clinical efficacy to restore better liver function, and potentially decrease mortality and need for transplantation.


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Title Annotation:The Latest News...
Publication:Stem Cell Lab World
Date:Apr 29, 2019
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