# Toxicology and Efficacy Studies of Intrathecal VersaMab-101 for spinal cord injury treatment

> **NIH NIH R44** · VERSAPEUTICS INC · 2023 · $2,380,552

## Abstract

PROJECT SUMMARY/ABSTRACT
 Spinal cord injury (SCI) is estimated to affect between 249,000 and 363,000 Americans, with about 17,730
new injuries occurring each year. There are around 42000 SCI patients that are veterans in the United States.
Nearly half of all SCIs occur in patients between the ages of 16 and 36, which results in individuals living with
SCI for decades. The lifetime costs of living with spinal cord injury can average up to $5.1M per patient for
individuals with high tetraplegia. So far, there are no FDA approved drug therapeutics for SCI, which highlights
a huge unmet medical need for those patients. Most spinal cord injuries are anatomically incomplete, which means
by reestablishing neural circuits in the spinal cord, those patients would have functional recovery potential. To
initiate the recovery process, injured axons from the remaining neurons above or near the injury level need to
regenerative growth to bypass the lesion site, reconnect to the neurons below the injury level, and then reestablish
the neural circuits. The major obstruction that prevents axon regrowth is the chemical and physical barriers that
accumulated at the lesion site quickly after injury, which blocks the axonal regenerative growth. For instance,
when axons regrowth to the lesion site, the Wnts protein reinduced there will interact with Ryk receptor that
reinduced on the axons, and then stop axon regeneration. To remove this obstruction, the interactions between
Ryk receptor and Wnts protein must be blocked.
 Advanced from the first-in-class research conducted in a world-famous research group in the University
of California, San Diego (UCSD), we developed a novel humanized monoclonal antibody drug candidate,
VersaMab-101, which could block the interaction between Wnt and the Ryk receptor. After injection into rats
with spinal cord injury, this antibody promoted axons regenerative growth and bypass the lesion site by stopping
the toxic interaction between Ryk and Wnts. The re-established neural circuits would then promote functional
recovery in rats.
 This novel therapeutic will benefits the patients with acute spinal cord injury by promoting axon
regeneration and improving their behavior recovery. This therapeutic will also benefit the whole community by
reducing the cost of long-term care. According to FDA recommendations in our pre-IND meeting, we propose to
conduct GLP-complaint toxicity study in rats in this grant application to evaluate the VersaMab-101 safety
profiles. We will also investigate the minimal required efficacy dose for rats with spinal cord contusion injuries.

## Key facts

- **NIH application ID:** 10697262
- **Project number:** 2R44NS120397-02
- **Recipient organization:** VERSAPEUTICS INC
- **Principal Investigator:** Miao Sun
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $2,380,552
- **Award type:** 2
- **Project period:** 2023-09-18 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10697262

## Citation

> US National Institutes of Health, RePORTER application 10697262, Toxicology and Efficacy Studies of Intrathecal VersaMab-101 for spinal cord injury treatment (2R44NS120397-02). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10697262. Licensed CC0.

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