Amphix Bio, a pre-clinical stage company developing a new therapeutic modality for regenerative medicine, announced on Wednesday that it has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead candidate, AMFX-200, for treatment of acute spinal cord injury (SCI).

AMFX-200 is based on a technology platform called Supramolecular Therapeutic Peptides (STPs), where peptides function both as a drug to activate cell receptors and as a physical nanofibre scaffold to support tissue regeneration. This technology was developed in the research group of Professor Samuel Stupp at Northwestern University.

Leveraging the STP platform, Amphix Bio says that it is advancing regenerative therapeutics for neurological and musculoskeletal conditions. Starting with established biology and known high-impact targets for cellular regeneration, the company bridges the gap between scientific discovery and clinically viable therapeutics. In preclinical models of acute spinal cord injury, a single injection of AMFX-200 into the spinal cord enables motor neurons from the brain to regrow past the injury site, re-establish severed connections, and restore motor function -- effectively reversing paralysis.

Amphix Bio received preliminary feedback from the FDA on the drug development program for acute SCI late last year, and says that it is now focused on completing safety studies required for regulatory approval to begin a first-in-human clinical trial in SCI patients.