Editorial note: This post refers to Pharnext and its experimental CMT1A therapy PXT3003. Pharnext is no longer operating, and PXT3003 is not an approved or available treatment. We have kept this post as part of our historical record.

The First Potential Treatment for CMT1A

HNF and Pharnext have maintained a collaborative relationship since 2013, working together toward potential treatments for CMT1A. Key milestones from that partnership include:

  • HNF identified its Centers of Excellence as clinical sites for the adult Phase III trial of PXT3003, and played an essential role in patient recruitment.
  • HNF created awareness and expanded knowledge of CMT across medical professional communities through conferences, educational materials, and media outreach.
  • On October 16, 2018, Pharnext announced positive topline results from the pivotal Phase 3 trial of PXT3003 for CMT1A.
  • HNF is helping establish pediatric clinical trial sites for PXT3003, with launch anticipated in mid-2019.
  • HNF’s Externally-led Patient-Focused Drug Development (PFDD) meeting for the FDA helped raise critical awareness of the impact CMT has on patients’ quality of life.
  • HNF supports the CMT&ME app (sponsored by Pharnext), an international observational real-world data study exploring the impact of CMT on patients.
“Thank you to the Hereditary Neuropathy Foundation and their Centers of Excellence for assisting Pharnext with patient recruitment for PXT3003. We are thrilled with the outcome of the trial and with the clearly demonstrated efficacy of PXT3003 in addressing the debilitating disease progression of CMT1A.”, Professor Daniel Cohen, MD, PhD, Pharnext Co-Founder and Chief Executive Officer