Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy
17 Maggio 2022 - 05:45PM
Business Wire
POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage
biopharmaceutical company developing innovative treatments for
serious chronic diseases with metabolic pathophysiology, including
non-alcoholic steatohepatitis (NASH) and rare metabolic disorders,
is pleased to announce that the U.S. Food and Drug Administration
(FDA) has granted Orphan Drug Designation (ODD) to PXL065 and
PXL770 for the treatment of patients with adrenomyeloneuropathy
(AMN), the most common form of X-linked adrenoleukodystrophy (ALD).
PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer
of pioglitazone. PXL770 is a novel, first-in-class direct adenosine
monophosphate-activated protein kinase (AMPK) activator. Both
compounds are preparing to enter into Phase 2a clinical
Proof-of-Concept (POC) biomarker studies as soon as possible,
subject to financing.
Poxel CEO, Thomas Kuhn, commented: “The award of Orphan Drug
Designation to both PXL065 and PXL770 by the FDA is an additional
regulatory milestone for the development of these potential
medicines in X-linked adrenoleukodystrophy, for which there is
currently no approved therapy. This status could give either
molecule a seven-year window of exclusive marketing rights
following FDA approval, and, along with its recent Fast Track
Designation, gives us confidence in the strategic shift into rare
diseases we initiated last year. We remain excited to pursue
treatments for ALD as this represents an area with very high unmet
medical need. We look forward to the next steps as we are preparing
for the initiation of our Phase 2a clinical studies as soon as
possible. To enable this effort, we continue to pursue various
financing options that will also extend our cash runway,
prioritizing non-dilutive sources.”
Orphan Drug Designation (ODD)
ODD is granted by the FDA to novel therapeutics for diseases or
conditions that affect fewer than 200,000 individuals in the U.S.
Orphan Drug Designation1 gives a company a potential seven-year
window of exclusive marketing rights following FDA approval, along
with a reduction in certain application fees, and tax credits for
expenses related to qualified clinical trials conducted after
orphan designation is received.
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. Nearly all men with a
diagnosis of ALD will develop AMN, 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 royalties and sales-based payments from Sumitomo
Pharma. Poxel has a strategic partnership with Sumitomo 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
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1 For more information on Orphan Drug Designation, see :
https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products
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Investor relations / Media
Aurélie Bozza Investor Relations & Communication Senior
Director aurelie.bozza@poxelpharma.com +33 6 99 81 08 36
Elizabeth Woo Senior Vice President, Investor Relations &
Communication elizabeth.woo@poxelpharma.com
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