# MITOFUSIN AGONISTS TO TREAT NEURODEGENERATIVE DISEASE

> **NIH NIH R41** · MITOCHONDRIA IN MOTION, INC. · 2020 · $224,701

## Abstract

Mitofusin agonists for the treatment of neurodegenerative diseases
Gerald W Dorn II, MD
Mitochondria in Motion, Inc.
Washington University in St Louis School of Medicine
Abstract: There are a number of rare neurodegenerative diseases, including
Amyotrophic Lateral Sclerosis (ALS) and Huntington’s Disease (HD), for which there is
no available or effective therapy and which lead to significant morbidity and mortality in
affected populations. Mitochondria in Motion, Inc. will develop and produce
investigational first-in-class small molecule mitofusin agonists, under an FDA approved
IND, to treat these conditions. Mitofusin agonists enhance mitochondrial fitness,
metabolism, and trafficking within cells, thus improving homeostatic functioning and
injury-responses of cells adversely impacted by genetic mitochondrial dysfunction. Our
published disease focus for mitofusin agonists was Charcot-Marie-Tooth disease type
2A, caused by mutations in our drug’s protein target, Mitofusin 2. Here, we
hypothesized that intervention with a mitofusin agonist would have beneficial effects on
other genetic peripheral neuropathies with a mitochondrial component, which is
supported by our preclinical data in ALS and HD patient-derived cells. Thus, we will fill
an unmet healthcare need and build a commercial enterprise to serve the ~20,000
Americans with ALS and the ~150,000 Americans suffering from or at genetic risk for
developing HD, their caregivers and families. In this Phase I STTR we propose to
optimize the pharmacokinetic properties of mitofusin agonists for systemic
administration and blood-brain-barrier penetration (Aim 1), and complete in vivo
feasibility and validation studies of mitofusin agonists to delay disease progression in
the well characterized SOD1G93A mouse model of ALS. Our deliverable in Phase I will
be a mitofusin agonist(s) ready for STTR Phase II IND-enabling studies and validation
in an expanded number of orphan diseases, to prepare for future first-in-human trials.

## Key facts

- **NIH application ID:** 10020801
- **Project number:** 5R41NS113642-02
- **Recipient organization:** MITOCHONDRIA IN MOTION, INC.
- **Principal Investigator:** Gerald W. Dorn
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $224,701
- **Award type:** 5
- **Project period:** 2019-09-30 → 2021-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10020801

## Citation

> US National Institutes of Health, RePORTER application 10020801, MITOFUSIN AGONISTS TO TREAT NEURODEGENERATIVE DISEASE (5R41NS113642-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10020801. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*