Poxel Announces PXL065 Granted FDA Fast Track Designation for X-linked Adrenoleukodystrophy
16 Febbraio 2022 - 05:45PM
Business Wire
- Phase 2a clinical Proof-of-Concept biomarker study for
PXL065 in adrenoleukodystrophy (ALD) now anticipated to start
midyear, with results to follow in early 2023
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 Fast Track Designation (FTD) to PXL065 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 that is preparing to enter into a Phase 2a clinical
Proof-of-Concept (POC) biomarker study midyear.
Poxel CEO, Thomas Kuhn, commented: "By awarding Fast Track
Designation to PXL065, the FDA recognizes the drug's potential to
address a significant unmet medical need for patients with ALD,
where no approved therapies currently exist. This is a very
powerful acknowledgement and good news for patients waiting to be
treated. Fast Track Designation confers multiple benefits that are
of great value and have the potential to substantially accelerate
the approval timeline for PXL065 in ALD. We look forward to working
closely with the FDA as we prepare to embark on our Phase 2a
clinical study for PXL065 now planned to begin midyear, followed by
results in early 2023.”
Fast Track Designation (FTD)
- FTD is designed to expedite development of pharmaceutical
products which demonstrate the potential to address unmet medical
needs in serious or life-threatening conditions.
- FTD provides Poxel with substantially enhanced access to FDA,
including opportunities for face-to-face meetings and written
consultations throughout the remaining development of PXL065.
- Drugs with FTD are eligible to apply for Accelerated Approval
and Priority Review at the time of a New Drug Application (NDA)
submission, which may result in faster product approval.
- FTD also allows for 'rolling review', whereby Poxel may submit
completed sections of the NDA as they become available, rather than
at the end of development.
Next Steps
The Phase 2a clinical POC biomarker study for PXL065 in X-linked
ALD is anticipated to begin midyear, followed by results in
early 2023.
Fast Track Designation
Introduced under the FDA Modernization Act (1997), Fast Track
Designation (FTD) may be awarded by the FDA to investigational
drugs which treat a serious or life-threatening condition, and
which fill an unmet medical need. Filling an unmet medical need is
defined as providing a therapy where none exists or providing a
therapy which may be potentially better than available therapy. The
FDA notes that "the purpose of the Fast Track program is to get
important new drugs to the patient earlier1”. FTD must be requested
by the sponsor company and must be accompanied by a detailed review
of both preclinical and clinical data.
The key benefits of FTD comprise enhanced access to the FDA,
with regular and more frequent opportunities for consultation and
discussion. In addition, drugs with FTD may be eligible for
Accelerated Approval, in which a new medicine is approved prior to
the availability of definitive data, and Priority Review, in which
the standard 10-month review process is reduced to six months.
Drugs with FTD may also enter a 'rolling review' of their NDA
submission, in which sections are submitted and reviewed as they
become available, substantially expediting the approval
process.
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
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.
1
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track
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