Poxel Announces Positive Update Following FDA Meeting for PXL065 for Treatment of NASH
November 08, 2019
• FDA feedback supports plan to advance PXL065 using the 505(b)(2) regulatory pathway for NASH
• 505(b)(2) process offers opportunity for a streamlined and efficient development plan
• Poxel provides update on PXL065 Phase 2 trial designed to identify optimal dose or doses for Phase 3 registration trial; initiation expected 2Q 2020
• LYON, France--(BUSINESS WIRE)-- POXEL S.A. (Euronext – POXEL - FR0012432516), a biopharmaceutical company focused on the development of innovative treatments for metabolic disorders, including type 2 diabetes and non-alcoholic steatohepatitis (NASH), announced today an update for PXL065 following a positive meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter 2019. Based on feedback from the FDA, Poxel plans to advance PXL065 using a 505(b)(2) regulatory pathway, which will in part reference and rely on the Actos® (pioglitazone) product label and relevant published literature. NASH is a serious disease and PXL065 has the potential to address an unmet medical need, so the Company intends to seek Fast Track Designation. PXL065, the deuterium-stabilized R-stereoisomer of pioglitazone, is a mitochondrial pyruvate carrier (MPC) inhibitor being developed for the treatment of NASH.
• “To my knowledge, PXL065 is in the unique position of being the first NASH clinical candidate being developed using a 505(b)(2) regulatory pathway with the potential to bridge to the vast amount of safety and efficacy data from pioglitazone studies,” said Stephen A. Harrison, MD, Visiting Professor of Hepatology, Radcliffe Department of Medicine, University of Oxford, UK. “Based on preclinical and clinical results, I am excited about the potential for an improved therapeutic profile for PXL065 compared to pioglitazone.”
• A 505(b)(2) NDA contains full safety and effectiveness reports but has some of the information required for NDA approval, such as safety and efficacy information on the active ingredient, from studies not conducted by or for the NDA applicant. Utilizing this regulatory pathway has the potential to result in a less expensive and faster route to approval compared with a traditional development path.
• “We are very pleased with the FDA feedback for the PXL065 development program. Using the 505(b)(2) regulatory process should enable reliance on relevant Actos® data, including the product label and published literature, which is anticipated to be used as part of the data package for a future new drug application (NDA) for PXL065, pending successful completion of the Phase 2 and Phase 3 trials. The 505(b)(2) process in this context should provide the opportunity for an efficient and streamlined development plan with reduced financial and development risk,” said Thomas Kuhn, CEO of Poxel. “We plan to initiate a Phase 2 trial for PXL065 for the treatment of NASH during the second quarter of 2020 in biopsy-proven NASH patients. The primary objective of this trial is to determine the optimal dose or doses to be tested in a Phase 3 registration trial.”