Poxel Obtains Orphan Drug Designation (ODD) from the FDA for PXL065 and PXL770

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(Boursier.com) — poxels a clinical-stage biopharmaceutical company developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to PXL065 and PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD).
PXL065 is a novel R-stereoisomer of pioglitazone stabilized by deuterium substitution. PXL770 is a novel, first-in-class, direct adenosine monophosphate-activated protein kinase (AMPK) activator. A phase IIa proof-of-concept (POC) clinical study will be launched as soon as possible for each of these two drug candidates, subject to obtaining additional funding.

Poxel Chief Executive Officer Thomas Kuhn said, “Receiving Orphan Drug Designation from the FDA is an additional regulatory step for the development of PXL065 and PXL770 in X-linked adrenoleukodystrophy, an indication that currently has no approved treatment.This status could provide PXL065 and PXL770 with potential market exclusivity for seven years after FDA approval and, with the recent achievement of Fast Track status, confirms our desire to pursue the strategic reorientation towards rare diseases initiated last year. We remain enthusiastic about the idea of ​​developing treatments in ALD, an area where the unmet medical needs are very high. We are preparing actively launch, as soon as possible, our phase IIa clinical studies.To this end, we continue to explore various financing options, prioritizing non-dilutive sources, in order to extend our funding horizon”.

Orphan Drug Designation (ODD)

The ODD is granted by the FDA to new treatments for diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status can provide a company with potential market exclusivity for seven years after FDA approval, as well as discounts on certain submission fees, and tax credits for expenses. related to qualified clinical trials conducted after obtaining orphan designation.

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