New Medicines

HepaStem Acute-on-chronic liver failure


New molecular entity
Promethera Biosciences
Promethera Biosciences

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Jun 21Promethera announces a financing agreement for an amount of €20M, which will allow the progress of the clinical development of HepaStem including its PIIb trial [7].
May 11Classified as Advanced Therapy Medicinal product by the EMA Committee for Advanced Therapies [5].


Liver stem cells from ethically donated healthy human organs and expanded in GMP culture
Hepatic failure can occur as a result of many causes. In the UK 990 liver transplants were carried out between April 2017 and March 2018 [2].
Acute-on-chronic liver failure
Intravenous infusion

Trial or other data

Dec 21No updates posted to US trial registry for PIIb HEP102-DHELIVER or PROLNGSTEM trials [6].
Dec 20Data were released by Promethera Biosciences in Nov 2019 from the PIIa HEP101 trial that assessed safety and efficacy of mesenchymal stem cell therapy in patients with acute on chronic liver failure (NCT02946554). The non randomise trial enrolled 24 participants in Belgium, Bulgaria, France and Spain. The primary endpoint is safety of two dose regimens of the therapy in patients with ACLF up to day 28 of the active study period. single and repeated infusions of HepaStem in patients with acute-on-chronic liver failure (ACLF), resulted in decrease in improvement in three indicators of liver disease severity; Model for End Stage Liver Disease score (MELD), Child-Pugh score and bilirubin levels, 28 days and three months after treatment initiation. Decreased in the Child-Pugh score, bilirubin levels by 80%, the Model for End Stage Liver Disease score (MELD) by 50% was reported. The second and third patients (both ACLF) received 1 or 2 infusions of 250 × 10 6 cells (around 3.5 × 10 6 cells/kg/infusion) and recovered from severe epistaxis. One patient of them underwent liver transplantation. No fall in platelets, fibrinogen, or coagulation factors were observed in the trial [4].
Oct 20Long-term, PII PROLONGSTEM safety trial is enrolling by invitation patients aged 5 to 75 years who received HepaStem in the interventional studies conducted by Promethera Biosciences (NCT03343756; P313-2013). The trial is enrolling 22 patients in Belgium, Spain and France [3].
Oct 20PIIb HEP102-DHELIVER trial to evaluate the efficacy and safety of HepaStem in 363 patients with acute on chronic liver failure is recruiting (NCT04229901). Adult patients are being recruited in Bulgaria and Belgium (currently no UK sites). Primary outcome is overall survival 90 days post-first infusion; collection of these data is due to complete Feb 22 [3].
Jan 20PIIb trial investigating safety and efficacy of HepaStem will include 363 patients with ACLF at 110 study sites across 22 countries in Europe. Topline results are expected to be released at a medical conference at the end of 2023 [1].