Poxel Announces its Participation at Upcoming Scientific Conferences Related to Adrenoleukodystrophy (ALD)
27 Settembre 2021 - 8:30AM
Business Wire
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 that the Poxel team will participate at several upcoming
scientific conferences related to X-linked adrenoleukodystrophy
(ALD), a severe orphan neurometabolic disease with no approved
therapies.
Poxel's scientific team will present data and plans pertaining
to ALD that align with the recently announced new strategic
direction of increasing Poxel’s focus on rare metabolic
diseases.
Phase 2a clinical Proof of Concept (POC) biomarker studies,
examining PXL065 and PXL770 in patients with adrenomyeloneuropathy
(AMN), the most common subtype of ALD which affects the nervous
system and adrenal glands, are planned to initiate in early 2022
with data readouts anticipated by year end 2022.
Poxel is committed to focus its pipeline on high value, rare
metabolic indications and NASH, with the goal of creating pipeline
synergies, maximizing resources, and driving shareholder value.
Upcoming Scientific Conferences
- 11th International Meeting on AMPK – Evian-les-Bains,
France (in person) Date: September 26-30, 2021 Poxel
will deliver the following oral presentations:
- on Monday, September
27, 3:00pm CET, “Characterization of a first-in-class direct
AMPK activator, PXL770, for NASH and other metabolic disorders:
From Preclinical to Clinical” by Sophie Bozec, PhD, Senior Vice
President, R&D Pharmacology and Scientific Communication -
on Tuesday, September 28, 9:30am CET,
“Potential therapeutic utility of direct AMPK activators for
X-linked adrenoleukodystrophy” by Pierre-Axel Monternier, Senior
Manager, Pharmacology
- World Congress of Neurology (WCN) (virtual)
Date: October 3-7, 2021 Poxel will deliver a poster
presentation entitled: “Validation of Direct AMP Kinase Activation
for Treatment of X-linked Adrenoleukodystrophy”
- National Organization for Rare Disorders (NORD) Summit
(virtual) Date: October 18-19, 2021 Poxel will deliver
poster presentations entitled: “(R)-pioglitazone – PXL065 – for
Treatment of X-Linked Adrenoleukodystrophy (ALD)” and “Validation
of Direct AMP Kinase (AMPK) Activation for Treatment of X-Linked
Adrenoleukodystrophy (ALD)”
- ALD Connect Annual Meeting (virtual) Date: November
12-13, 2021 Members of Poxel’s scientific team will participate
and present at this conference.
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 thiazolidinedione (D-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, X-linked
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 - EU/US Trophic Communications
Stephanie May or Valeria Fisher poxel@trophic.eu +49 171 185 56 82
or +49 175 804 1816
Investor relations / Media - France NewCap Emmanuel Huynh
or Arthur Rouillé poxel@newcap.eu +33 1 44 71 94 94
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