Rare Daily Staff

France-based Poxel said it will shift strategic direction to focus its pipeline on high value, rare metabolic indications and NASH, following its partner Sumitomo Dainippon Pharma winning approval for Twymeeg for the treatment of type 2 diabetes in Japan.

The approval at the end of June triggered a $15.9 milestone payment to Poxel from Sumitomo. Following that, the company completed a strategic pipeline evaluation focused on determining the best way to create value.

“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,” said Thomas Kuhn, CEO of Poxel. “We also anticipate expanding our rare disease pipeline with additional internal and external clinical opportunities.”

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.

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.

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.

In parallel with the company’s efforts in ALD, it said it will work to launch an additional rare disease development program in 2022. The company believes that this will allow it to expand the addressable market opportunity for its development programs and offers stakeholders a more diversified clinical pipeline.

Photo: Thomas Kuhn, CEO of Poxel

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