A clinical-stage biopharmaceutical company that has developed a novel molecule to treat the newly discovered type 2 CMT gene which causes SORD deficiency. Experts believe that SORD deficiency could very well become the most common subtype of CMT 2. HNF has been instrumental in helping to facilitate and accelerate the approval of their clinical lead product AT-007, a Aldose Reductase Inhibitor (ARI) currently in clinical trials.

“Initiatives such as the HNF registry bring together critical data from patients, caregivers and clinicians, including genetic tests and electronic health records. This information can help inform clinical trials of therapies for the 150,000 people in the US who are living with Charcot-Marie-Tooth disease and have no treatment options available,” said Shoshana Shendelman, CEO & Founder of Applied Therapeutics.

A late stage clinical biopharmaceutical company has developed a novel synergistic drug combination to treat the most common type of CMT, CMT1A. The drug Syngility is a fixed-dose in combination of baclofen, naltrexone and sorbitol to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves which is the primary pathophysiology problem for CMT1A. HNF has been in partnership with Pharnext since 2012 and is a key contributor to supporting the development and commercialization of Syngility. Expected results of their pivotal phase III is expected by Q4, 2023.

“GRIN data has been essential in clinical trial protocol design, site selection, and efficient patient recruitment for the pivotal Phase III clinical study of our lead asset PXT3003, the PREMIER trial, in development for the most common type of CMT, CMT1A. In addition, the robust data collected in GRIN, together with the ongoing CMT&Me digital survey, will enable a better understanding of the burden of disease, comorbidities, and critical patient demographics as we move forward to a potential commercialization of PXT3003 if approved by the FDA. Topline data of the PREMIER trial are expected in the last quarter of 2023.” Xavier Paoli M.Sc. Pharnext, Chief Operating Officer

We believe this clinical trial has the potential to be a crucial turning point in the effort to finally provide an efficacious treatment for patients suffering from CMT1A,” said Daniel Cohen, M.D., Ph.D., co-founder and CEO of Pharnext. “Today’s therapeutic options are very limited and mostly palliative in nature. Our PLEODRUG® PXT3003 has already demonstrated safety, tolerability and improvements in CMT1A patient disability in a Phase 2 trial. Given this positive safety assessment by the DSMB, we are hopeful to bring this much needed potential therapy to patients suffering from this debilitating condition upon completion of this Phase 3 trial.”