# MITOFUSIN AGONISTS TO TREAT NEURODEGENERATIVE DISEASE

> **NIH NIH R41** · MITOCHONDRIA IN MOTION, INC. · 2021 · $78,500

## 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:** 10290982
- **Project number:** 3R41NS113642-02S1
- **Recipient organization:** MITOCHONDRIA IN MOTION, INC.
- **Principal Investigator:** Gerald W. Dorn
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $78,500
- **Award type:** 3
- **Project period:** 2021-03-15 → 2021-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10290982

## Citation

> US National Institutes of Health, RePORTER application 10290982, MITOFUSIN AGONISTS TO TREAT NEURODEGENERATIVE DISEASE (3R41NS113642-02S1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10290982. Licensed CC0.

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