Stem Cell Therapy for ACLF Progresses to Safety, Efficacy Trial
Promethera Biosciences announced the initiation of a phase 2b clinical trial to evaluate the safety and efficacy of HepaStem, a liver-derived stem cell therapy candidate for patients with acute-on-chronic liver failure, according to a press release.
“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,” Etienne Sokal, MD, PhD, founder and group chief medical officer of Promethera, said in the release. “As a potentially pivotal trial, the results obtained here may provide us with sufficient clinical data to file a new drug application. 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.”
Investigators of the DHELIVER randomized control trial aim to enroll 363 patients with grade 1 or grade 2 ACLF. Trial objectives include transplantation-free survival as well as continued evaluation of safety. Enrolled patients will receive two weekly intravenous infusions of HepaStem or placebo.
In the previous HEP101 trial that included 24 patients with ACLF or high risk for developing ACLF, HepaStem was safe and well-tolerated in single or repeated injections and showed preliminary signs of efficacy with improvement in MELD, Child-Pugh score and bilirubin levels.
“ACLF is a severe, life threatening disease, with no current available treatments,” John Tchelingerian, PhD, president and CEO of the Promethera Group, said. “HepaStem has the potential to be the first real alternative to liver transplants in such a disease, and help ACLF patients in need.”
The company expects to release topline results at the end of 2023.