The objective of the program is to discover and to develop a PleodrugTM for CMT1A disease, in response to a clear unmet medical need. The future product will have a socio-economic impact, by facilitating the management of the disease and by improving the quality of life of patients since there is no treatment today.

• The team reconstructed the complex biological network associated with the disease from the 30 human genes known to be involved in the disease
• Therapeutic targets were identified and different combinations of off patent drugs registered for other indications were selected for screening
• Several candidates were screened in relevant in vitro and in vivo models
• A Pleolead PXT3003 was selected (combination of 3 approved drugs at low dose)
• A clinical formulation was developed
• Preclinical safety and pharmacokinetic studies were conducted
• The program entered into Phase II clinical development in December 2010 (only 3 years after initiation of the R&D program instead of the usual 8 years) - Press release EN or FR. Additional information is available in the following websites :
  • The Pharnext CMT1A Clinical Trial at AFSSAPS Web Site ►
  • The Pharnext CMT1A Clinical Trial at clinicaltrials.gov Web Site ►
  • The Pharnext CMT1A Clinical Trial at CMT France Web Site ►
  • The Pharnext CMT1A Clinical Trial at AFM (Téléthon) Web Site ►
• In parallel, biomarker candidates are under screening.
• PHARNEXT has completed patient recruitment for its Phase II clinical trial of PXT3003 (the company’s first Pleodrug™) in Charcot-Marie-Tooth disease. Press Release (EN or FR)

Institutional partners: OSEO / Industrial partners: IPSEN which signed an option for license agreement in June 2009 (Read More ►).