Pharnext, Developer of CMT1A Treatment Candidate PXT3003, Raises 16 Million Euros

Pharnext SA — the French biopharmaceutical company developing PXT3003 as a therapeutic candidate to treat Charcot-Marie-Tooth disease type 1A (CMT1A) — announced that it has successfully raised 16 million euros, or $19.7 million, through a private placement.

The financial term refers to the sale of stocks, bonds, or securities directly to a private investor, as opposed to a public offering. Proceeds from this private placement will be used by Pharnext to fund its current strategy and continue to grow ahead of anticipated results from the phase 3 trial of PXT3003 as a potential treatment for CMT1A.

“We are thrilled by the major strategic and clinical advances we have achieved in our programmes, and look forward to reporting the results of the Phase 3 trial of our first-in-class product PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A in the second half of 2018,” Daniel Cohen, M.D., Ph.D., co-founder and chief executive officer of Pharnext, said in a press release.

Pharnext develops novel therapeutics through a new drug discovery pipeline – called PLEOTHERAPY™ – which is based on large-scale genomic data and artificial intelligence.  Pharnext uses PLEOTHERAPY™ to identify and determine the most efficient combinations of drugs called PLEODRUG™.

Through PLEOTHERAPY™, Pharnext developed PXT3003 – a combination drug that consists of baclofen (a muscle relaxant), naltrexone (an opioid antagonist), and sorbitol (a laxative). PXT3003 has been shown to help patients with CMT1A manage their symptoms, particularly by reducing levels of the PMP22 protein, which is found at high levels in CMT patients and causes nerve damage. PXT3003 has received orphan drug status in both Europe and the United States.

An ongoing phase 3 clinical trial, called PLEO-CMT (NCT02579759), is investigating the use of PXT3003 in 323 patients with mild-to-moderate CMT1A across Europe and North America. As part of the study, patients are randomized to receive one of two PXT3003 doses or a placebo two times a day for up to 15 months. Intermediate analysis of data from clinical trials shows promising results. Additionally, PXT3003 has passed two different safety evaluations, indicating that it is both safe and efficacious.