• This program rewards companies innovating on strategic themes for French sovereignty
• Additional evidence of the technological value of Poxel's assets

Regulatory News:

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 metabolic disorders, announces today to be the winner of the 2023 edition of the I-nov contest for its program in adrenoleukodystrophy (ALD), a rare neurometabolic disease, for which there is currently no treatment. This distinction is a recognition of the potential of the Company's drug candidates in development for this indication, PXL770 and PXL065.

The prize for this contest, financed by the French State via the France 2030 plan, operated by Bpifrance in collaboration with ADEME and awarded during a ceremony organized on July 4, 2023 in Paris, includes a grant which will contribute in part¹ in the financing of the two phase IIa proof-of-concept clinical studies for PXL770 and PXL065. These studies are ready to be launched in adult male patients with adrenomyeloneuropathy (AMN), the most common form of ALD, subject to additional funding, which the Company is currently working on.

Thomas Kuhn, Chief Executive Officer of Poxel, states: “This award recognizes all the efforts that our teams deploy on a daily basis to develop innovative treatments against rare metabolic diseases such as adrenoleukodystrophy. We are particularly proud of this recognition of our programs and, with the support of the France Relance program, we look forward to being able to initiate, once we have secured additional funding, these two Phase IIa clinical studies for this high stake’s indication”.

The I-nov contest

The "i-Nov Innovation Competition", funded by the France 2030 plan, aims to support innovative projects carried out by start-ups and SMEs, and to promote the accelerated emergence of leading companies in their field, which can claim global reach.

It selects, within the framework of a procedure encouraging competition, innovative projects with particularly high potential for the French economy. It includes co-financing for research, development and innovative projects, with a total costs ranging between €1 million and €5 million, and which contributes to accelerating the development and marketing of innovative solutions and technologies.

This competition is organized by the General Secretariat for Investment, the Ministry of Economy, Finance and Industrial and Digital Sovereignty, the Ministry of Ecological Transition and Territorial Cohesion, the Ministry of Higher Education and Research, the Ministry of Energy Transition, the Ministry of Health and Prevention, and the Ministry of Culture.

With France 2030 plan, the State made it a priority to make France a leader in the establishment and development of innovative and sovereign companies whose projects promote ecological, energy, digital or food transitions, as well as the decarbonization of our economy and our way of life.

The i-Nov competition, funded by the French State via France 2030 plan, is operated by Bpifrance in collaboration with ADEME.

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. 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

¹ Up to 35% to 45% of the estimated eligible costs.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230705050254/en/