Pharnext is developing new therapies for both rare and common neurological disorders using its proprietary Pleotherapy platform. Lead programme PXT3003 for CMT1A has entered pivotal Phase III trials. PXT864 for Alzheimer’s disease has completed Phase IIa but has been deprioritised and will be advanced through partnerships.
Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for neurodegenerative diseases that currently lack curative or disease-modifying treatments. Lead programme PXT3003 is a synergistic fixed-dose combination of baclofen, naltrexone and sorbitol formulated as an oral solution, identified with the Pleotherapy R&D approach. It is in Pivotal Phase III clinical development for CMT1A. Top line data are expected in Q423. Data from the open label PLEO-CMT-FU extension study of the first Phase III programme suggests continuous treatment benefit after total trial time of five years. In October, Pharnext finalised a strategic financing agreement worth €20.7m with Neovacs by issuing bonds and associated warrants, possibly giving Neovacs access to one-third of Pharnext’s capital, if fully converted (vests in January 2024).
PXT3003 could potentially be the first approved treatment for CMT1A. This disease is a debilitating rare (prevalence of 1/5,000) peripheral neuropathy with high unmet medical need where patients suffer from pain, progressive muscle atrophy and cramps in the limbs. The CMT1A development pipeline is early stage with PXT3003 the most clinically advanced asset (Phase III) for this indication.