Amphix Bio Achieves Orphan Drug Status for Breakthrough Spinal Cord Injury Treatment

Amphix Bio's New Milestone in the Treatment of Acute Spinal Cord Injury

Amphix Bio, a pre-clinical company that is building a new therapeutic modality for regenerative medicine, has received orphan drug designation for AMFX-200, a treatment for acute spinal cord injury (SCI). SCI is an extreme condition that might lead to permanent paralysis, with around 18,000 new cases registered each year in the United States.

About AMFX-200

The AMFX-200 is related to a technology platform named ‘supramolecular therapeutic peptides (STPs), in which peptides operate as a physical nanofiber scaffold and as a medicine to activate cell receptors to help tissue regeneration. The technology was first discovered in the research community of Professor Samuel Stupp at Northwestern University.

The northwestern group achieved a breakthrough in the year 2021 by learning the capability and potential of controlling the number of motions of molecules within the nanofibers. This was by far a remarkable strategy that improved biological potential and bolstered unmet neural regeneration. By accelerating the STP platform, Amphix is developing regenerative therapeutics for musculoskeletal and neurological conditions. With the well-established biology and related targets for cellular regeneration, the company is filling the gap to enable scientific discovery to reach clinical therapeutics.

The preclinical models of acute spinal cord injury include a single injection of amfx-200 for spinal cords that provide motor neurons from the brain to recover from the past injury, restore motor function, and re-develop extreme connections. This ensures the reversal of paralysis.

The FDA gave preliminary feedback to Amphix on its drug development program regarding SCI last year. The company is now focusing on the completion of the needed safety studies for regulatory approval, so that the company will start with first-in-human clinical trial in patients of SCI.

Views and Statements

Chief scientific officer of Amphix Bio, Professor Samuel Stupp (at Northwestern University), said, “From the time we published the results in 2021, we also validated the potential of these molecules to recreate functional neural tissue in our additional preclinical models, in other neural injuries, in types of sci and neurodegenerative disease. It demonstrates a paradigm shift in the neural regeneration therapeutic development.”

A neurosurgeon at the University of Miami and the Miami project to cure paralysis, Dr. James Guest said, “I’ve witnessed the destruction and disturbance of a SCI patient and their families. The past attempts for treatments had several limitations, but amphix has taken a new approach to neural regeneration.”