- Following the recent approval of TWYMEEG® (Imeglimin)
in Japan and associated potential future revenues, Poxel to
accelerate and expand rare metabolic disease programs leveraging
existing platforms and proven capabilities
- Poxel to advance its deuterated thiazolidinediones (dTZD)
and direct adenosine monophosphate-activated protein kinase (AMPK)
activator platforms in rare metabolic diseases with initiation of
PXL065 and PXL770 Phase 2a clinical Proof-of-Concept (POC) studies
in X-linked adrenoleukodystrophy (ALD) in early 2022; data expected
in Q4 2022
- Continued commitment to non-alcoholic steatohepatitis (NASH)
through PXL065 DESTINY Phase 2 trial with results expected in Q3
2022; reassessment of PXL770 future development in NASH pending
results from PXL065 Phase 2 trial and both Phase 2a POC studies in
ALD
- Webcast and conference call on Monday, July 12, at 6:00pm
CEST (in French), 1:15pm ET (in English)
POXEL SA (Euronext – POXEL - FR0012432516), a clinical stage
biopharmaceutical company developing innovative treatments for
chronic serious diseases with metabolic pathophysiology, including
non-alcoholic steatohepatitis (NASH) and rare disorders, today
announced a new strategic direction 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.
"Based on the recent approval of TWYMEEG® (Imeglimin) in Japan
with the associated infusion of cash and potential for future
royalty and sales-based payments, we completed a strategic pipeline
evaluation focused on determining the optimal value-creating
opportunities for our technologies,” said Thomas Kuhn, CEO of
Poxel. “Following our recent achievements and a thorough review of
the Company’s programs, we are excited to announce a new strategic
direction for Poxel, with an increasing focus on rare metabolic
indications that represents the intersection of high unmet medical
needs, promising pre-clinical and clinical data, opinion leader
enthusiasm, significant commercial opportunity, and attractive time
horizons. Moreover, we believe that by leveraging our existing
platforms and proven capabilities to develop products in rare
metabolic diseases in addition to NASH, we can be more efficient
with our resources and more expediently deliver novel medicines to
patients with an even stronger potential to create significant
value for the benefit of our shareholders. We also anticipate
expanding our rare disease pipeline with additional internal and
external clinical opportunities.”
“Our first rare disease development program targets ALD, a
serious monogenic disorder with no approved pharmaceutical therapy,
where both direct AMPK activation and non-genomic pathways
modulated by TZDs have been implicated as therapeutic
opportunities,” commented David E. Moller, MD, Chief Scientific
Officer of Poxel. “Our recent preclinical data demonstrates that
both approaches have substantial efficacy potential. This includes
the reversal of pathology in patient-derived cells and improvements
in the phenotype of the classical rodent animal model. Moreover,
since there are strong preclinical biomarker signals, we believe
that our pending Phase 2a studies will provide meaningful near-term
results that could then lead to a pivotal trial.”
Based on results from the ongoing PXL065 DESTINY Phase 2 NASH
trial and the planned Phase 2a POC biomarker studies for PXL065 and
PXL770 in ALD, the Company intends to select one program, either
PXL065 or PXL770, to advance in NASH and one program to advance in
ALD. In parallel with the Company’s efforts in ALD, another
important goal is to launch an additional rare disease development
program in 2022. The Company believes that this strategy will
expand the addressable market opportunity for its development
programs and offers stakeholders a more diversified clinical
pipeline.
As a result of the review process and portfolio prioritization,
the Company is announcing the following clinical development
program updates:
- In ALD, Phase 2a clinical POC biomarker studies of PXL065 and
PXL770 are planned to initiate in early 2022, with data expected by
year end 2022. The initial focus will be on patients with
adrenomyeloneuropathy (AMN), the largest subtype of ALD. Two
identical studies will enroll adult male AMN patients and assess
the effect of PXL065 and PXL770 over 12 weeks of treatment on
pharmacokinetics, safety, and efficacy using relevant biomarkers,
including the impact on elevated very long-chain fatty acids
(VLCFA), the hallmark plasma marker of disease.
- In NASH, PXL065, deuterium-stabilized R-pioglitazone, is in a
streamlined Phase 2 trial (DESTINY). Patient screening is finished
and enrollment is now expected to complete in Q3 2021, with topline
data anticipated approximately one year later. This Phase 2 36-week
trial in noncirrhotic biopsy-proven NASH patients will assess three
doses of PXL065 compared to placebo in at least 120 patients. The
results of this trial will be used to help identify the dose or
doses for a Phase 3 registration trial.
- Initiation of the NASH Phase 2b trial for PXL770, a
first-in-class, oral direct AMPK activator, will be postponed,
pending results from the ongoing PXL065 Phase 2 trial in NASH and
both Phase 2a POC biomarker studies in AMN. In the STAMP-NAFLD
Phase 2a trial, completed at the end of 2020, PXL770 was observed
to produce significant improvements in liver fat content and liver
enzymes with a greater response in patients with co-existing Type 2
diabetes mellitus (T2DM); in these patients, additional
improvements in glycemia were observed. PXL770 was observed to be
safe and well tolerated.
Corporate Update
- To support this new strategic direction and following the
recent approval of TWYMEEG in Japan, which completes a significant
chapter in Poxel’s development, the composition of Poxel’s Board of
Directors has evolved with the appointment of Dr. John Kozarich as
a director during the June 23, 2021 general assembly meeting, who
also becomes the chair of the scientific committee of the Board,
and the departure of Bpifrance Participations as Board observer,
effective July 9, 2021.
- In addition, the approval of TWYMEEG in Japan enabled Poxel to
draw down the third and final tranche of EUR 13.5 million from the
IPF loan, which was received on June 30, 2021.
The Poxel executive management team will host a conference call
to present the new strategic plan. The call will be led by Thomas
Kuhn, Chief Executive Officer of Poxel, who will be joined by
external experts in ALD and by members of the executive management
team to answer questions.
The conference calls will be held on July 12th:
- In French, at 12:00 pm ET (New York) / 6:00 pm
CEST (Paris time).
To register for the webcast:
https://us02web.zoom.us/webinar/register/WN_I5qo1FHkSkm9WNOBjfGHOg
- In English, at 1:15 pm ET (New York) / 7:15 pm
CEST (Paris time).
To register for the webcast:
https://us02web.zoom.us/webinar/register/WN_VVZYJ6JIQgqEdlQcT8HTIw
A presentation will be available in the Investors section of the
Poxel website. The replay of the video conference will be available
on Poxel’s website:
https://www.poxelpharma.com/en_us/investors/company-information/corporate-presentations
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, insulin resistance,
elevated levels of blood lipids (such as cholesterol and
triglycerides) and type 2 diabetes. Type 2 diabetes is also a
frequent co-morbid condition (estimated to be present in up to 50%
of NASH patients). Currently no curative or specific therapies are
available.
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 CALD
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). 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. In 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 for the
treatment of type 2 diabetes in Japan. With this approval, Poxel is
entitled to receive milestones, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210711005019/en/
Poxel SA Catherine David Investor Relations &
Communication Manager catherine.david@poxelpharma.com +33 7 64 57
61 78
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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