Amphix Bio receives FDA Orphan Drug Designation for neural regeneration therapeutic to treat acute spinal cord injury

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• USA - English
• USA - English

CHICAGO, July 16, 2025 /PRNewswire/ -- Amphix Bio, a pre-clinical stage company developing a new therapeutic modality for regenerative medicine, received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its lead candidate AMFX-200 for treatment of acute spinal cord injury (SCI). SCI is a devastating condition often leading to permanent paralysis, with approximately 18,000 new cases each year in the United States.

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 nanofiber scaffold to support tissue regeneration. This technology was pioneered in the research group of Professor Samuel Stupp at Northwestern University. The Northwestern team made a key breakthrough in 2021 by discovering the ability to control the collective motion of molecules within the nanofibers, a strategy that enhanced biological potency and stimulated unprecedented neural regeneration.

Leveraging the STP platform, Amphix Bio 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.

"Since we first published these results in 2021, we have further validated the ability of these molecules to regenerate functional neural tissue in additional preclinical models, different types of spinal cord injury, and in other neural injuries and neurodegenerative diseases," said Stupp, who also serves as Chief Scientific Officer of Amphix Bio. "This represents a paradigm shift in the development of neural regeneration therapeutics."

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

"Experiencing a spinal cord injury is devastating for patients and their families, as it leads to a lifetime of lost mobility, high healthcare costs, and a heavy reliance on caregivers," said Dr. James Guest, a neurosurgeon at the University of Miami and the Miami Project to Cure Paralysis. "Previous attempts at treatments have faced many limitations but Amphix Bio is taking a completely new approach to neural regeneration, with the potential for a breakthrough in the field."

About Orphan Drugs

The FDA's Orphan Drug program is designed to encourage and support the development of treatments for rare diseases or conditions. Benefits of Orphan Drug Designation include financial incentives such as tax credits for clinical trials, exemption from user fees, and up to seven years of market exclusivity after approval. Companies are also eligible to apply for clinical trial grants through the Office of Orphan Product Development to offset the cost of efficacy and/or safety studies.

About Amphix Bio

Amphix Bio spun out from Northwestern University in Chicago, IL in 2021. The company is developing a new platform technology, Supramolecular Therapeutic Peptides (STPs), that instruct cells to initiate regenerative processes and restore function lost from injury, disease, or aging. These therapies trigger targeted signaling pathways while forming scaffolds to support tissue growth. Amphix Bio's goal is to use regenerative medicine to increase human healthspan — the length of time that people can live productive, fulfilling lives.

Learn more at www.amphixbio.com, or contact us at [email protected].

SOURCE Amphix Bio