NEURONAX, the biopharmaceutical company specialized in neuronal regeneration, has been authorized to start its Phase I clinical trial by the Healthcare Authorities (French National Agency for Medicines and Health Products Safety (ANSM) for France and The Federal Agency for Medicines and Health Products (FAMHP) for Belgium).
This first clinical trial in humans with the NX210 product is titled ” A Phase I Study to Investigate the Safety and Potential Activity of Single Intrathecal Administration of NX210 in Patients with Spinal Cord Injury “. The primary endpoint of this study is to evaluate the safety of NX210 in patients with acute spinal cord injury. Secondary and exploratory endpoints include biological effectiveness of the experimental drug and functional recovery.
The dose escalation study, will take place in at least 5 French and Belgian hospitals and will recruit up to 36 patients. The coordinating investigators are: Prof. Chazal and Dr. Sakka of the CHU of Clermont-Ferrand for France, and Prof. Raftopoulos of the Université Catholique de Louvain (UCL) for Belgium.
Research on embryogenesis has made it possible to discover a protein called SCO-spondin. This protein is very involved in the formation of the central nervous system. In humans, the organ producing SCO-spondin disappears after birth thus strongly reducing the chances of “regrowth” of neurites. The NEURONAX research team extracted and synthesized the peptide of interest (NX210) from SCO-Spondin and then characterized its different properties. With a unique mechanism of action, NX210 has demonstrated neuroprotective and neurodegenerative properties suggesting applications in the treatment of pathologies of the nervous system, especially in cases of spinal cord injury but also with a high potential in neurodegenerative diseases.
Preclinical studies have demonstrated the good safety profile of the product as well as its efficacy on the protection of nerve cells, the regrowth of neurites and especially the recovery of functionality, including motricity in the injured animal.
NX210 has been granted the status of “Orphan Drug Designation” by the European Medicines Agency (EMA). Several patents, some of which have been recently filed by NEURONAX, provide an enhanced protection to the product and cover in particular: manufacturing of the peptide, its application in neurodegenerative diseases, its use in combination with stem cells and its anti-apoptotic activity for the neurons.
“With NX210, we can protect neurons and re-create a promoting environment in which neurites naturally grow and build nerve connections. Beyond this first clinical trial in spinal cord trauma, we have the ambition to try NX210 in other therapeutic indications”, confirms Stéphane Gobron, founder and Chief Scientific Office of the company.
“The unique properties of this peptide allow us to have the ambition to play a major role in neuronal pathologies. Alliances will enable us, on the one hand, to strengthen our ability to develop a franchise in the trauma of the spinal cord, and on the other hand to demonstrate its real interest in neurodegenerative diseases. We are currently looking for € 4 to 6 millions to accelerate our development plans”, underlines Yann Godfrin, Chief Executive Officer of NEURONAX.