• PXT3003 is in Ph III in the US and Europe
• Granted orphan status in US and Europe
• CMT is one of the most common inherited neurological disorders

Pharnext SA (Paris:ALPHA) announced on 6/14/2018 that PXT3003 was granted priority review for Charcot-Marie-Tooth Type 1A disease (CMT1A) by the China Food and Drug Administration (CFDA).
Pharnext’s first-in-class PLEODRUG PXT3003 is currently in a Phase 3 clinical trial in Europe and the United States, with results expected before the end of 2018. PXT3003, developed using Pharnext’s R&D platform PLEOTHERAPY, is a novel oral fixed-low dose combination of baclofen, naltrexone and sorbitol, with EMA and U.S. FDA Orphan Drug Designation.

Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 - Jean-Martin Charcot and Pierre Marie in Paris, France, and Howard Henry Tooth in Cambridge, England. CMT, also known as hereditary motor and sensory neuropathy (HMSN) or peroneal muscular atrophy, comprises a group of disorders that affect peripheral nerves. The peripheral nerves lie outside the brain and spinal cord and supply the muscles and sensory organs in the limbs. Disorders that affect the peripheral nerves are called peripheral neuropathies.

GeneNet Co, Ltd, a joint venture between Tasly and Pharnext, owns the commercialization rights for PXT3003 for CMT1A in Greater China (Mainland China, Hong Kong, Taiwan, and Macau), as well as exclusive license rights to all PXT3003 patents applied for and authorized in Greater China.

Pharnext's PLEOTHERAPY platform is intended to systematize the identification and development of new combinations of existing drugs. First an inventory of all possible therapeutic targets is created for a particular disease. The repositories of known drugs used in unrelated indications are screened to identify synergistic combinations.