LYON, France, September 27, 2021– (COMMERCIAL THREAD) –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 diseases, today announced that the Poxel team will attend several upcoming scientific conferences related to X-linked adrenoleukodystrophy (ALD), a severe orphan neurometabolic disease without approved therapies.
The Poxel science team will present ALD data and plans 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 that affects the nervous system and glands adrenal glands, are expected to start in early 2022 with data readings expected by the end of 2022.
Poxel is committed to focusing its pipeline on rare and high value-added metabolic indications and NASH, with the aim of creating pipeline synergies, maximizing resources and generating shareholder value.
Upcoming scientific conferences
11e International meeting on AMPK – Evian-les-Bains, France (in person)
Date: September 26-30, 2021
Poxel will make the following oral presentations:
– Monday September 27 at 3:00 p.m. CET, “Characterization of a first-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
– Tuesday September 28 at 9:30 am CET, “Potential therapeutic utility of direct AMPK activators for X-linked adrenoleukodystrophy” by Pierre-Axel Monternier, Senior Director, Pharmacology
World Congress of Neurology (WCN) (virtual)
Date: October 3-7, 2021
Poxel will present a poster presentation titled: “Validation of direct activation of AMP kinase for the treatment of X-linked adrenoleukodystrophy”
Summit of the National Organization for Rare Diseases (NORD) (virtual)
Date: October 18-19, 2021
Poxel will present poster presentations entitled: “(R) -pioglitazone – PXL065 – for the treatment of X-linked adrenoleukodystrophy (ALD)” and “Validation of direct activation of AMP Kinase (AMPK) for the treatment of X-linked adrenoleukodystrophy (ALD)”
ALD Connect Annual Meeting (virtual)
Date: November 12-13, 2021
Members of the Poxel science team will participate and present at this conference.
X-linked adrenoleukodystrophy (ALD) is an orphan neurometabolic disease caused by mutations in the ABCD1 gene that encodes a key protein required for the metabolism of very long chain fatty acids (VLCFA) by peroxisomes (cell organelles) . ALD is the most common leukodystrophy with a prevalence similar to hemophilia – up to 1 / 10,000 people 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 – usually occurring from adolescence to adulthood. AMN is characterized by a chronic and progressive distal axonopathy involving the long bundles of the spinal cord and, to a lesser extent, the peripheral nerves, resulting in progressive stiffness and weakness of the legs, impaired gait and movement. balance, incontinence and loss of sensation. All males are affected and many females also exhibit features of AMN with a later onset. C-ALD is characterized by inflammatory demyelination of brain cells and usually affects children, but many men with AMN can also develop brain disease; these white matter brain damage results in severe neurological deficits and death. There are no approved drugs 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 the early stage of C-ALD and this procedure carries a risk of serious side effects.
About Poxel SA
Poxel is a clinical-stage biopharmaceutical company developing innovative treatments for severe chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare diseases. Poxel has clinical and early-stage programs of its adenosine monophosphate activated protein kinase (AMPK) 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, successfully completed a Phase 2a proof of concept trial for the treatment of NASH, which met its goals. 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) . TWYMEEGÂ® (Imeglimin), Poxel’s first flagship product that targets mitochondrial dysfunction, approved and launched for the treatment of type 2 diabetes in Japan. Poxel expects to receive payments based on sales 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 countries in Southeast Asia. The Company intends to generate additional 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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