- The Phase 2a program will include two separate studies
-
Phase 2a efficacy and safety study for PXL770 is underway with data
results anticipated 1H 2020 -
Pharmacokinetic (PK)/pharmacodynamic (PD) study for PXL770 is
expected to initiate 2Q 2019 with results anticipated 2H 2019
LYON, France–(BUSINESS WIRE)–POXEL
SA (Euronext: POXEL – FR0012432516), a biopharmaceutical company
focused on the development of innovative treatments for metabolic
disorders, including type 2 diabetes and non-alcoholic steatohepatitis
(NASH), today announced initiation of the Phase 2a program for PXL770, a
direct adenosine monophosphate-activated protein kinase activator
(AMPK), for the treatment of NASH.
The Phase 2a twelve-week, multicenter, randomized, double-blind,
placebo-controlled, parallel group study, which will assess efficacy and
safety, has been initiated. In this study, three doses of PXL770 versus
placebo will be administered. Approximately 100 nonalcoholic fatty liver
disease (NAFLD) patients who likely have NASH are expected to be
included in this study across clinical sites in the US. The primary
endpoint of the study will measure the change in liver fat mass based on
magnetic resonance imaging-estimated proton density fat fraction
(MRI-PDFF), a novel imaging-based biomarker that allows fat mapping of
the entire liver. The study will also assess the effects of PXL770 on
other metabolic and non-metabolic biomarkers as well as safety and
tolerability. In addition, the effect of PXL770 on hepatic de novo
lipogenesis (DNL) and glucose tolerance, will be investigated. Data
results from the Phase 2a study are anticipated in the first half of
2020.
In addition to the Phase 2a study, a separate four-week PK/PD study is
expected to be initiated during the second quarter of 2019. During this
study, PXL770 will be administered to approximately 32 patients. This
study will assess the PK profile of PXL770 in NAFLD patients and its
effects on hepatic and metabolic parameters in the target population.
Data results from this study are expected during the second half of 2019.
“AMPK is a major regulator of energy metabolism and its activation is
expected to show beneficial effects in metabolic diseases, such as
NASH,” said Pascale Fouqueray, MD, PhD, EVP, Translational Medicine and
Early Clinical Development at Poxel. “Supported by positive preclinical
mechanistic and efficacy results in a DIO-NASH model, we believe that
PXL770 is uniquely positioned to treat the underlying root causes of
fatty liver diseases as well as to specifically target each step of the
pathophysiology of the disease, including liver steatosis, inflammation,
ballooning and fibrosis. PXL770 may also provide benefits for
co-morbidities, including those related to cardiovascular disease.”
“By targeting the master regulator of cellular energy, PXL770 has a
unique and differentiated profile compared to other drug candidates in
development for the treatment of NASH,” said Thomas Kuhn, CEO of Poxel.
“With the acquisition of PXL065, a mitochondrial pyruvate carrier
inhibitor, we have expanded our presence in NASH, and we are one of only
a few biotechnology companies with two clinical programs in development
for this disease. The underlying pathophysiological mechanisms that
contribute to the development and progression of NAFLD and NASH are
highly complex and support the need for the development of novel
therapies that act on different targets. Both of our programs have the
potential to be developed as monotherapy or in combination together or
with other agents.”
Poxel previously announced data results from a Phase 1b multiple
ascending dose trial and a drug-drug interaction study of PXL770 in a press
release titled, “Poxel Announces Favorable Results for PXL770 Phase
1b Multiple Ascending Dose Trial and Drug-Drug Interaction Study.”
About NASH
Non-alcoholic steatohepatitis (NASH) is a metabolic disease with no
clear disease origin that is quickly becoming a worldwide epidemic. It
is characterized by the accumulation of fat in the liver causing
inflammation and fibrosis. The disease can be silent for a long period
of time, but once it accelerates, severe damage and liver cirrhosis can
occur, which can significantly impact liver function or can even result
in liver failure or liver cancer. Typical risk factors for NASH include
obesity, elevated levels of blood lipids (such as cholesterol and
triglycerides) and diabetes. Currently no curative or specific therapies
are available.
About PXL770
PXL770 is a first-in-class direct adenosine monophosphate-activated
protein kinase (AMPK) activator. AMPK is a central regulator of multiple
metabolic pathways leading to the control of lipid metabolism, glucose
homeostasis and inflammation. Based on its central metabolic role,
targeting AMPK offers the opportunity to pursue a wide range of
indications to treat chronic metabolic diseases, including diseases that
affect the liver, such as non-alcoholic steatohepatitis (NASH).
About Poxel SA
Poxel uses its development expertise in metabolism to advance a pipeline
of drug candidates focused on the treatment of metabolic disorders,
including type 2 diabetes and non-alcoholic steatohepatitis (NASH). We
have successfully completed the Phase 2 clinical program for our
first-in-class lead product, Imeglimin, which targets mitochondrial
dysfunction, in the U.S., Europe and Japan. Together, with our partner
Sumitomo Dainippon Pharma, we are conducting the Phase 3 Trials of
IMeglimin for Efficacy and Safety (TIMES) program for the treatment of
type 2 diabetes in Japan. Our partner Roivant Sciences is responsible
for Imeglimin’s development and commercialization in countries outside
of Poxel’s partnership with Sumitomo Dainippon Pharma, including the
U.S. and Europe. PXL770, a first in class direct adenosine
monophosphate-activated protein kinase (AMPK) activator, is advancing
into a Phase 2a proof-of-concept program for the treatment of NASH.
PXL770 could also have the potential to treat additional metabolic
diseases. PXL065 (deuterium-stabilized R-pioglitazone), a mitochondrial
pyruvate carrier (MPC), is in Phase 1 and being developed for the
treatment of NASH. Poxel also has additional earlier-stage programs,
including deuterated drug candidates for metabolic, specialty and rare
diseases. We intend to generate further growth through strategic
partnerships and pipeline development. (Euronext: POXEL, www.poxelpharma.com)
Contacts
Poxel SA
Jonae R. Barnes
Senior Vice President,
Investor Relations and Public Relations
jonae.barnes@poxelpharma.com
+1 (617) 818-2985
Investor relations / Media – EU/US
Trophic Communications
Stephanie
May or Joanne Tudorica
may@trophic.eu
+49
89 238 877 34 or +49 171 185 56 82
Investor relations / Media – France
NewCap
Alexia
Faure/Nicolas Merigeau
poxel@newcap.eu
+33
1 44 71 98 55