Mont-Saint-Guibert, Belgium, and Tokyo, Japan, January 8th, 2020 – Promethera® Biosciences SA, a global innovator in cell-based medicines and liver diseases, today announced the initiation of a Phase 2b clinical trial to evaluate the efficacy and safety of HepaStem™, the company’s liver-derived stem cell therapy candidate, in patients with Acute-on-Chronic Liver Failure (ACLF). The trial is open for recruitment and aims to 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.
The DHELIVER study (or HEP102) is a randomized, placebo-controlled, double-blinded, multicenter trial designed to assess the efficacy of HepaStem™ treatment on the overall survival proportion 90 days post-first infusion. Among the secondary trial objectives are additional efficacy assessments such as transplantation-free survival as well as continued evaluation of the treatment’s safety. Patients with Grade 1 or 2 ACLF will be eligible to screen for participation in the trial. The study will target enrolment of approximately 363 patients across two treatment arms: patients receiving two weekly intravenous infusions of HepaStem™ and patients receiving placebo.
“We are developing HepaStem™ as a treatment for ACLF at a fast pace and we are determined to bring it to patients in need as soon as we can. As a potentially pivotal trial, the results obtained here may provide us with sufficient clinical data to file a new drug application,” said Etienne Sokal, MD, PhD, Founder and Group Chief Medical Officer of Promethera®. “Providing a treatment for a severe disease such as ACLF will not only help this patient population, but also greatly inform us in our efforts to develop treatments for other liver diseases, such as NASH.”
“ACLF is a severe, life threatening disease, with no current available treatments. The only option for patients is organ transplant, which is a major procedure and often not accessible. HepaStem™ has the potential to be the first real alternative to liver transplants in such a disease, and help ACLF patients in need,” said John Tchelingerian, PhD, President and Chief Executive Officer of the Promethera® Group. “We are proud and excited to begin working towards the next milestone in the clinical development of HepaStem™ with the Phase 2b trial commencing and are looking forward to achieve the targets we set and bring HepaStem™ one step closer to an approved therapy”
In the previously concluded HEP101 trial, HepaStem™ has proven safe and tolerable in single or repeated injections in a total of 24 patients with Acute-on-Chronic Liver Failure (ACLF) or Acute Decompensation (AD) at high risk of developing ACLF. With one or two repeated doses up to 1.2 million cells per kilogram of body weight, no adverse events related to HepaStem™ occurred in the three-months follow-up period and no clinically significant changes were shown in platelet count, fibrinogen levels, and coagulation factors following HepaStem™ infusion. In addition to the positive safety profile, the study had shown preliminary signs of efficacy with 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.