Poxel Announces Publication in Kidney International of PXL770 Preclinical Results in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
01 Marzo 2023 - 7:45AM
Business Wire
- Results support the development of PXL770 in a Phase 2
clinical program for ADPKD
Regulatory News:
POXEL SA (Paris:POXEL) (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, announced today the publication of
preclinical results in autosomal dominant polycystic kidney disease
(ADPKD) for PXL770, a novel, first-in-class direct adenosine
monophosphate-activated protein kinase (AMPK) activator.
To access the publication online in the life sciences journal,
Kidney International, please use the following link: A novel direct
adenosine monophosphate kinase activator ameliorates disease
progression in preclinical models of Autosomal Dominant Polycystic
Kidney Disease. (kidney-international.org)
“ADPKD remains a major cause of end stage renal disease and is
associated with substantial additional unmet medical needs. Since
the only approved medicine for ADPKD, tolvaptan, has moderate
efficacy and known liabilities, new therapeutic approaches are
urgently needed,” said David E. Moller, MD, Executive Vice
President and Chief Scientific Officer of Poxel, “AMPK has been
strongly implicated as a target for ADPKD where pathophysiology
involves metabolic perturbations and is closely related to key
biochemical pathways that are modulated by AMPK activation. Here,
for the first time, we showed beneficial effects of a direct and
selective AMPK activator in preclinical ADPKD models from three
species mouse, human, and dog. These results confirm the potential
utility of AMPK activation for this disease and support the
development of PXL770 in a Phase 2 clinical program for ADPKD.”
About ADPKD
Autosomal dominant polycystic kidney disease, or ADPKD, is a
form of chronic kidney disease which is caused by mutations in the
PKD1 or PKD2 genes. This causes multiple cysts, or pouches filled
with fluid, to form in the kidneys. Autosomal dominant (AD) relates
to how the disease is passed down from the parent to child. With
ADPKD, cysts develop and grow in the kidneys over time. These cysts
continuously grow, causing the kidneys to increase in size and
volume. Over time, the growing cysts make it harder for the kidneys
to function and eventually lead to kidney failure. Most people with
ADPKD have pain, high blood pressure, and kidney failure at some
point in their lives.
ADPKD is the fourth leading cause of chronic kidney disease
(CKD), affecting 1 in every 400 to 1,000 people (approximately
140,000 patients in the US) and is the most common kidney disorder
passed down through family members. More than 50% of ADPKD patients
develop renal failure by age 50, followed by dialysis and/or kidney
transplantation. Only one drug, tolvaptan, is approved to attenuate
progression and is associated with severe liver adverse events and
poor tolerability (polyuria).
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. For the treatment of
NASH, PXL065 (deuterium-stabilized R-pioglitazone) met its
primary endpoint in a streamlined Phase 2 trial (DESTINY-1). In
rare diseases, development of PXL770, a first-in-class
direct adenosine monophosphate-activated protein kinase (AMPK)
activator, is focused on the treatment of adrenoleukodystrophy
(ALD) and autosomal dominant polycystic kidney disease (ADPKD).
TWYMEEG® (Imeglimin), Poxel’s first-in-class product that
targets mitochondrial dysfunction, is marketed for the treatment of
type 2 diabetes in Japan by Sumitomo Pharma and Poxel expects to
receive royalties and sales-based payments. Poxel has a strategic
partnership with Sumitomo Pharma for Imeglimin in Japan, China, and
eleven other Asian countries. 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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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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