# Development of KCC2 neuromodulators to treat paralysis after spinal cord injury.

> **NIH NIH R44** · AXONIS THERAPEUTICS, INC. · 2022 · $773,769

## Abstract

Development of KCC2 neuromodulators to treat spinal cord injury.
Abstract:
There are no approved therapies for treating paralysis after spinal cord injury (SCI), despite roughly 17,000
people suffering SCI per year and about 300,000 people living with chronic SCI in US. For the vast majority of
patients, injury leaves them incapable of walking or, if they have suffered a cervical SCI, entirely dependent
upon others for assistance in all of their activities of daily living from feeding to personal care. The cost to these
individuals, and to society as a whole, are significant as SCI often occurs in adults during their peak earning
years. Neuromodulation after SCI has the potential to restore functions, as shown even in chronic SCI patients
using epidural electrical stimulation in clinical case studies. We are developing a non-invasive, oral
neuromodulating drug that can widely treat the majority of SCI patients who have spared, but dysfunctional,
spinal cord tissue. After SCI there is an excitation/inhibition imbalance in the spared spinal cord tissue that
leads to paralysis, spasticity and neuropathic pain. This imbalance is caused by a decrease in the CNS-
specific chloride transporter called KCC2. Indeed, restoring KCC2 by both pharmacological and genetic
methods in spared spinal cord neurons leads severely paralyzed rodents to regain stepping ability.
Furthermore, KCC2 enhancer drugs reduce neuropathic pain and spasticity in rodent models. In this
application, we propose to carry out non-GLP and GLP IND-enabling preclinical research on drug efficacy and
safety of our novel KCC2 enhancer drug, AXN-006. After completion of the proposed aims, AXONIS goal is to
be ready to commission GMP drug product manufacturing, file an IND and move rapidly into a first-in-human
Phase 1 clinical trial in healthy volunteers. The overarching goal is the commercialization of a first-in-class oral
KCC2 enhancer drug to treat paralysis, pain and spasticity in SCI, as well as other relevant neurological
disorder indications.

## Key facts

- **NIH application ID:** 10483094
- **Project number:** 2R44NS120762-02
- **Recipient organization:** AXONIS THERAPEUTICS, INC.
- **Principal Investigator:** Joanna Stanicka
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $773,769
- **Award type:** 2
- **Project period:** 2022-03-01 → 2024-02-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10483094

## Citation

> US National Institutes of Health, RePORTER application 10483094, Development of KCC2 neuromodulators to treat paralysis after spinal cord injury. (2R44NS120762-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10483094. Licensed CC0.

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