Promethera Biosciences Presents Updated Phase 2a Results for World’s First Stem Cell Clinical Trial in ACLF at The International Liver Congress™/ EASL 2019

April 15, 2019 Off By BusinessWire

Interim safety results and early efficacy signals with HepaStem bode
well for subsequent confirmatory trials in ACLF and provide valuable
insights for imminent start of clinical evaluation in NASH

MONT-SAINT-GUIBERT, Belgium–(BUSINESS WIRE)–Promethera Biosciences SA, a global innovator in cell-based medicines
and liver diseases, today 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 was presented in an oral
presentation during the late breaker session at The International Liver
Congress^™ (ILC) 2019 on April 13, 2019, in Vienna, Austria by
Promethera’s principal investigator Prof. F. Nevens, KULeuven, BE.

Currently, liver transplant is the only rescue treatment for patients
suffering of ACLF and to a lesser degree for AD patients. There are no
other existing therapies that can rescue the end-stage-liver failure.
Promethera is developing HepaStem as an alternative to transplant; it
consists of liver stem cells that are obtained from ethically donated
healthy human organs and expanded in GMP culture conditions. These liver
stem cells migrate through the blood and reach the liver, where they
settle and support the tissue regeneration function.

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 HepaStem 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 Etienne Sokal, M.D., Ph.D., Promethera’s Chief
Scientific & Medical Officer. “HepaStem has a broad therapeutic
potential, being a possible first alternative to organ transplant for an
ever-growing patient population in dire need, and we are relentless in
our drive to bring it to patients in the safest and fastest way
possible. The next stages of our development plan will be to conduct a
larger trial in ACLF focusing on clinical efficacy to restore better
liver function, and potentially decrease mortality and need for
transplantation.”

Prof. Nevens added, “We are happy to report we have developed a safe
dosing protocol with HepaStem for patients with Acute-on-Chronic Liver
Failure (ACLF) or acute decompensation (AD) at risk of developing ACLF.
While these are early results, our preliminary data point to liver
function improvement. We can only hope this treatment will not only
prove to be efficacious in the future, but also help the numerous
patients with ACLF and AD and bring them to a stage where organ
transplantation can again be considered, or behavioral changes can start
helping again.”

About Promethera Biosciences

Promethera Biosciences is a global innovator in liver therapeutics whose
mission is to bring life-saving treatments to reduce the need for liver
transplantation. Our lead clinical program, derived from our patented
cell technology platform HepaStem, is designed to benefit from its
immune-modulatory and anti-fibrotic properties. In addition to our
cell-based pipeline we develop antibody technologies, such as the
antiTNF-R1 antibody Atrosimab, to complement and diversify our
therapeutic options. We are a team of international experts operating
out of facilities in Mont-Saint-Guibert, Belgium, Durham, NC, USA,
Tokyo, Japan and Basel, Switzerland.

Promethera®, HepaStem®, H2stem®, are all registered trademarks of the
PROMETHERA group.