POXEL SA (Euronext – POXEL - FR0012432516), a clinical stage
biopharmaceutical company developing innovative treatments for
chronic diseases with metabolic pathophysiology, including
non-alcoholic steatohepatitis (NASH) and rare disorders, today
announced the formation of its Scientific Advisory Board (SAB) for
rare metabolic diseases. The new SAB will initially focus on
supporting Poxel's X-linked adrenoleukodystrophy (ALD) program.
“I am delighted to welcome this distinguished group of
scientific thought leaders to Poxel’s SAB for rare metabolic
diseases. These therapeutic indications, including ALD, are areas
of unsurpassed unmet medical need where treatments are not
available or very limited. We are grateful for their informed
counsel and deep knowledge and experience that will help shape
Poxel’s discovery and clinical-stage programs and further advance
our mission to develop therapies for rare metabolic diseases. We
look forward to continuing to collaborate with this accomplished
group of advisors as we expand our clinical programs, and initiate
Phase 2a studies for ALD in early 2022, with both PXL065, a novel
deuterium modified thiazolidinedione and PXL770, a first-in-class
direct AMPK activator,” said David E. Moller, Poxel's Chief
Scientific Officer.
Poxel recently hosted its first SAB meeting on rare diseases,
which led to highly productive discussions regarding the potential
of its compounds and the future of its programs in ALD, including
the design of its two proof-of-concept (POC) Phase 2a studies which
the Company intends to initiate early 2022.
The members of the Rare Metabolic Diseases Scientific Advisory
Board are the following:
Prof. Florian Eichler, MD, is an expert in inherited
diseases with metabolic pathophysiology that affect the nervous
system. Dr. Eichler received training in pediatric neurology and
neurogenetics at Harvard and Johns Hopkins. He is currently the
director of the Center for Rare Neurologic Disorders and the
director of the Leukodystrophy Service at Massachusetts General
Hospital (MGH), Harvard Medical School, US. His research focus is
on the genetics of peroxisomal disorders, lipid metabolism, and
gene therapy for neurodegenerative diseases. Dr. Eichler is also
President of ALD Connect.
Prof. Marc Engelen, MD, PhD, is a clinical researcher, a
specialist in pediatric neurology, gastroenterology and
endocrinology, and an expert in peroxisomal disorders and
leukodystrophies at Amsterdam University Medical Centers,
Netherlands. Dr. Engelen received his MD and PhD degrees at the
University of Amsterdam. Along with Stephan Kemp and other members
of his team, Dr. Engelen's research efforts have unveiled novel
approaches to detecting, diagnosing and monitoring neurometabolic
diseases including ALD.
Prof. S. Ali Fatemi, MD, is a physician-scientist leader
in pediatric neurology and neurometabolic diseases. Dr. Fatemi was
trained at the Medical University of Vienna, Austria and at Kennedy
Krieger Institute, Johns Hopkins, US. He founded the Moser Center
for Leukodystrophies and is now Chief Medical Officer at the
Kennedy Krieger Institute, Baltimore, US. His research is focused
on basic and translational studies pertaining to the
pathophysiology of ALD.
Prof. Stephan Kemp, PhD, is a translational research
expert in genetic neurometabolic diseases. He was trained at Johns
Hopkins University, Kennedy Krieger Institute, and at the
University of Amsterdam, where he is a longstanding faculty member.
His research focuses on lipid metabolism and neurotoxicity and he
has published many seminal papers on the pathobiology of ALD. He
also leads efforts focused on newborn screening for rare metabolic
disorders in the Netherlands.
Prof. Fanny Mochel, MD, PhD, is an expert in
inborn errors of metabolism. She received training in genetics and
neuroscience at University Pierre and Marie Curie in Paris and she
is a faculty member in genetics at this university. Dr. Mochel also
leads the French reference Center on Neurometabolic diseases and is
co-chair of the French society for inborn of errors of metabolism
and a council member of the Society for the Study of Inborn Errors
of Metabolism. Her research efforts include characterization and
treatment of brain energy deficiencies in neurometabolic disease,
the identification of novel biomarkers, metabolomics and in vivo
metabolic imaging, as well as therapeutic approaches targeting the
Krebs cycle.
Dr. Jaspreet Singh, PhD, is a neuroscientist researcher
focusing on ALD pathophysiology, neuroinflammation, biomarker
development and testing novel therapeutic options. He was trained
in India and at the Medical University of South Carolina, US. He is
currently a faculty member in the Department of Neurology at the
Henry Ford Health System in Detroit, US.
Prof. Keith Van Haren, MD, is a pediatric neurologist and
an expert in ALD. He received medical and specialty training at the
University of Rochester Medical School, Harvard Medical School, and
Stanford University, US. Dr. Van Haren is a faculty member in
Neurology and Neurological Sciences at Stanford. He cares for
patients including many with ALD and also leads a laboratory
focusing on the study of single-gene mutations and attendant
molecular mechanisms leading to neuroinflammation in humans.
About ALD X-linked adrenoleukodystrophy (ALD) is an
orphan neurometabolic disease caused by mutations in the ABCD1 gene
which encodes for a key protein that is required for metabolism of
very long chain fatty acids (VLCFA) by peroxisomes (cellular
organelles). ALD is the most common leukodystrophy with a
prevalence similar to hemophilia – up to 1/10,000 individuals in
the general population have ALD [https://rarediseases.org]. Forms
of this disease include cerebral ALD (C-ALD) and
adrenomyeloneuropathy (AMN) which is the most common form –
typically occurring in adolescence through adulthood. AMN is
characterized by chronic and progressive distal axonopathy
involving the long tracts of the spinal cord and to a lesser extent
the peripheral nerves resulting in progressive stiffness and
weakness in the legs, impaired gait and balance, incontinence, and
loss of sensation. All men are affected, and many women also
present with features of AMN with a later onset. C-ALD is
characterized by inflammatory demyelination of cells in the brain
and typically afflicts children, but many men with AMN may also
develop cerebral disease; these white matter brain lesions lead to
severe neurologic deficits and death. There are no approved
medicines for ALD (other than glucocorticoid supplements for
associated adrenal insufficiency). C-ALD when first detected in
early childhood, can be treated with hematopoietic stem cell
transplantation. HSCT is currently limited to early stage of C-ALD
and this procedure is at risk of severe adverse reactions.
About Poxel SA
Poxel is a clinical stage biopharmaceutical company
developing innovative treatments for chronic serious diseases
with metabolic pathophysiology, including non-alcoholic
steatohepatitis (NASH) and rare disorders. Poxel has clinical
and earlier-stage programs from its adenosine
monophosphate-activated protein kinase (AMPK) activator and
deuterated TZD platforms targeting chronic and rare metabolic
diseases. For the treatment of NASH, PXL065
(deuterium-stabilized R-pioglitazone) is in a streamlined Phase 2
trial (DESTINY-1). PXL770, a first-in-class direct AMPK
activator, has successfully completed a Phase 2a proof-of-concept
trial for the treatment of NASH, which met its objectives. For the
rare inherited metabolic disorder, adrenoleukodystrophy (ALD), the
company intends to initiate Phase 2a proof of concept studies with
PXL065 and PXL770 in patients with adrenomyeloneuropathy (AMN).
TWYMEEG® (Imeglimin), Poxel’s first-in-class lead product
that targets mitochondrial dysfunction, has been approved and
launched for the treatment of type 2 diabetes in Japan. Poxel
expects to receive sales-based payments and royalties from Sumitomo
Dainippon Pharma. Poxel has a strategic partnership with Sumitomo
Dainippon Pharma for Imeglimin in Japan, China, South Korea, Taiwan
and nine other Southeast Asian countries. The Company intends to
generate further growth through strategic partnerships and pipeline
development. Listed on Euronext Paris, Poxel is headquartered in
Lyon, France, and has subsidiaries in Boston, MA, and Tokyo,
Japan.
For more information, please visit: www.poxelpharma.com
All statements other than statements of historical fact included
in this press release about future events are subject to (i) change
without notice and (ii) factors beyond the Company’s control. These
statements may include, without limitation, any statements preceded
by, followed by or including words such as “target,” “believe,”
“expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,”
“project,” “will,” “can have,” “likely,” “should,” “would,” “could”
and other words and terms of similar meaning or the negative
thereof. Forward-looking statements are subject to inherent risks
and uncertainties beyond the Company’s control that could cause the
Company’s actual results or performance to be materially different
from the expected results or performance expressed or implied by
such forward-looking statements.
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Poxel SA Catherine David Investor Relations &
Communication Manager catherine.david@poxelpharma.com +33 7 64 57
61 78 Aurélie Bozza Investor Relations & Communication Director
aurelie.bozza@poxelpharma.com +33 6 99 81 08 36 Elizabeth Woo
Senior Vice President, Investor Relations & Communication
elizabeth.woo@poxelpharma.com Investor relations / Media
NewCap Emmanuel Huynh or Arthur Rouillé poxel@newcap.eu +33 1 44 71
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