Pharnext Announces PXT3003 for CMT-1A

Pharnext, a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on big genomics data and artificial intelligence, today announced that the United Kingdom's Medicine and Healthcare products Regulatory Agency (MHRA) has granted Promising Innovative Medicine (PIM) designation to its lead drug candidate, PXT3003, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in patients 16 years and older. A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS) in the treatment, diagnosis or prevention of life-threatening or seriously debilitating conditions with unmet need. "We are delighted by the MHRA's decision to award the PIM designation to PXT3003 as it further validates the potential of our lead drug candidate as an innovative treatment approach to address the high unmet medical need in patients with CMT1A," said Daniel Cohen, M.D., Ph.D., co-founder and Chief Executive Officer of Pharnext. "All existing data indicate that PXT3003 is a safe and well tolerated drug combination. We look forward to continuing our discussions with U.S. and European regulatory authorities to advance the clinical development of PXT3003 and initiate as quickly as possible an additional pivotal Phase 3 trial in the U.S. and Europe." PXT3003 has also been granted Orphan Drug status in the U.S. and Europe and has received Fast Track designation from the U.S. Food and Drug Administration (FDA). About PXT3003 Pharnext's first-in-class PLEODRUG(TM) PXT3003, developed using Pharnext's R&D platform, PLEOTHERAPY(TM), is a novel oral fixed-dose combination of baclofen, naltrexone and sorbitol, with Orphan Drug Designation in the U.S. and E.U. PXT3003, Pharnext's lead PLEODRUG(TM), has shown positive results both in non-clinical pharmacology and clinical studies for the treatment of CMT1A. In non-clinical pharmacology studies, PXT3003 inhibited the overexpression of the PMP22 gene, improved myelination of peripheral nerves and motor / sensory impairments. In a Phase 2 clinical study in 80 adult patients with CMT1A, PXT3003 was safe and well tolerated. In addition, PXT3003 showed trends in multiple efficacy endpoints beyond stabilization, particularly the ONLS scale. These results were published in the Orphanet Journal of Rare Diseases (OJRD) in December 2014. In October 2018, PXT3003 completed an international Phase 3 trial in 323 patients 16 years and older for the treatment of CMT1A, confirming the excellent safety profile of the combination and demonstrating an encouraging efficacy profile. The Phase 3 extension study is currently ongoing.