US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III study

PARIS, France, 7:00 a.m., June 10, 2020 (CET) – Pharnext SA (FR0011191287 – ALPHA), a biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence, today provided an update on the regulatory and clinical status of PXT3003, its lead program in Charcot-Marie-Tooth Type 1A (“CMT1A”). 

Regulatory Update 

PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration (“FDA”). In Pharnext’s previous interactions with FDA, the agency provided guidance that an additional Phase III study would be required. Based on our most recent interactions with FDA through a Type C meeting, FDA has now provided a very clear path to NDA submission for approval of our lead clinical program. 

The FDA has agreed with the key elements of Pharnext’s approach for the developmental pathway to approval for PXT3003. Specifically, the FDA has indicated that a single pivotal Phase III study in CMT1A delivering robust results could be sufficient for approval of PXT3003. This study design will be similar to the earlier Phase III study of PXT3003 that yielded encouraging top line results in October 2018. The FDA has agreed that the primary endpoint will again be Overall Neuropathy Limitations Scale (ONLS). Notably, the FDA has also agreed that the factorial study requirement for combination drugs can be carried out in a preclinical CMT1A disease animal model, and not in a human Phase III clinical trial as typically required. The animal factorial study, a requirement for NDA filing, will be done under GLP or GLP-like conditions and will have a similar study design and use the same CMT1A model as our previously successful preclinical factorial study. 

Phase III Trial Design Update 

For the upcoming Phase III pivotal study, as recommended by FDA, Pharnext will use ONLS as the primary endpoint, as was used in the previous Phase IIIstudy. We will have two arms in the study which will compare the high dose vs placebo. The high dose showed encouraging results in the earlier Phase IIIstudy. In addition, we have resolved the past manufacturing issue with our high dose oral solution and will now use the earlier successful low dose oral solution formulation in a higher volume to deliver the high dose in our upcoming Phase III trial. We will also be utilizing a more convenient new unit dose package format, stick packs (liquid sachets), that will assure optimal patient compliance and more accurate dosing. 
Pharnext plans to initiate the final pivotal Phase III clinical study before the end of Q1 2021.

Dr David Horn Solomon, Chief Executive Officer commented, “We are grateful that the FDA has provided strong and specific guidance to complete pivotal studies towards NDA submission and approval for PXT3003 in CMT1A. Both the Phase III clinical trial as well as the preclinical animal study will be informed in their design by the earlier Phase III clinical trial that provided encouraging top-line results, and the earlier animal study that was successful, respectively. We have addressed earlier manufacturing issues and look forward to PHARNEXT | CREATING NEW SOLUTIONS Page 2 initiating the Phase III clinical trial before the end Q1 2021. Our goal at Pharnext is to provide CMT1A patients and their caregivers a new therapeutic to treat this disease where no therapy currently exists.”