Mighty Therapeutics Partners with the POLG Foundation to Develop Novel Preclinical Model for POLG Mitochondrial Disorders and Parkinson's Disease

Trailblazing approach to leverage learnings from rare POLG disorders to inform development for Parkinson's and other age-related neurological diseases

NEEDHAM, Mass., Sept. 15, 2026 /PRNewswire/ -- Mighty Therapeutics (the "Company" or "Mighty"), a commercial-stage biotechnology company pioneering a new class of medicines that directly target mitochondrial dysfunction in rare and age-related diseases, is celebrating World Mitochondrial Disease Awareness Week by announcing today its initiation of a research collaboration with The POLG Foundation; Dr. Carolyn Sue, Kinghorn Chair of Neurodegeneration at Neuroscience Research Australia (NeuRA); and The Jackson Laboratory (JAX) to develop a novel preclinical mouse model for Parkinson's disease, mitochondrial diseases driven by polymerase gamma (POLG) mutations, and other mitochondrial diseases characterized by neurodegeneration.

"Mighty Therapeutics is at the forefront of translating recent findings on the link between mitochondrial dysfunction and neurodegeneration into innovative therapies with the potential to address some of medicine's most intractable diseases," said David A. Brown, PhD, Chief Scientific Officer at Mighty. "We are honored to work with leaders in academia, advocacy, and biomedical research to create a new tool that could unlock advances in POLG disorders, Parkinson's disease, and beyond."

With support from The POLG Foundation, the JAX Rare Disease Translational Center generated a novel mouse model carrying a pathogenic mutation observed in humans with POLG-related mitochondrial disease. POLG mutations disrupt the efficient replication of mitochondrial DNA and are one of the genetic predictors of Parkinson's disease. In this new collaboration, JAX Associate Professor A. Phillip West, PhD, will generate double mutant models harboring both POLG mutations and other mutations in proteins associated with Parkinson's disease.

"There is a critical connection between POLG mutations, mitochondrial dysfunction, and Parkinson's disease in the aging population," said Carolyn Sue, PhD, Kinghorn Chair of Neurodegeneration at NeuRA, Professor of Neurology at the University of New South Wales (UNSW), and Director of Neurosciences at Prince of Wales Hospital (POWH). "Current preclinical models are unable to recapitulate clinical aspects of Parkinson's disease or POLG disorders, presenting a significant barrier for new therapeutic development in either indication. This study brings together preclinical models of these two related diseases into a single model. If successful, the model could provide a new way to test for therapeutic efficacy, potentially paving the way for new mechanism-based treatments."

"Families affected by POLG disorders live with the heartbreaking possibility that their loved ones may lose the ability to walk, eat, hear, or see—and may even die prematurely. Furthermore, there is a severe lack of treatments and limited understanding of these conditions," said Brian Tseng, M.D., Ph.D., Chief Executive Officer of The POLG Foundation. "Today's news represents an important collaboration by premier experts and organizations to advance research with far-reaching impacts. Because POLG disorders involve dysfunction of mitochondria—the powerhouses of our cells—studying them does more than help rare disease patients; it illuminates the mechanics of aging and more common conditions like Parkinsonism, neurodegenerative diseases, cancers, and liver, GI, and immune dysfunction."

Mighty is progressing elamipretide, a first-in-class mitochondria-targeted investigational therapeutic, in primary mitochondrial disease due to POLG mutations, based on encouraging evidence from its MMPOWER-3 and 3 NuPOWER studies showing a genotype-dependent response in the POLG1 subgroup. The company was awarded a research grant in 2023 from the Michael J. Fox Foundation to evaluate bevemipretide, a next-generation mitochondria-targeted investigational therapeutic, in Parkinson's disease, for which it continues to evaluate dosing to inform potential clinical development.

About The POLG Foundation
The POLG Foundation is a 501(c)(3) non-profit research advocacy organization established in 2022 by a POLG family. The POLG Foundation raises awareness/resources to fuel research into POLG-related mitochondrial diseases. With the goal of developing effective treatments and a cure, The POLG Foundation connects researchers, clinicians, patients and families, key patient organizations and biopharma. For more information, please visit https://polgfoundation.org/ 

About The Jackson Laboratory
The Jackson Laboratory (JAX) is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center. JAX leverages a unique combination of research, education, and resources to achieve its bold mission: to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. Established in Bar Harbor, Maine, in 1929, JAX is a global organization with nearly 3,000 employees worldwide and campuses and facilities in Maine, Connecticut, California, Florida, New York, and Japan. For more information, please visit www.jax.org.

About Mighty Therapeutics
Mighty Therapeutics, together with its wholly owned operating subsidiary, Stealth BioTherapeutics, Inc., is advancing novel therapies for people living with diseases involving mitochondrial dysfunction. Grounded in rigorous science and inspired by meaningful patient partnerships, the company is building a proprietary pipeline to directly address bioenergetic deficits at the source.

In September 2025, Mighty marked a historic milestone with the U.S. Food and Drug Administration (FDA) approval of its first commercial therapy, establishing both the first FDA-approved treatment for Barth syndrome and the first FDA-approved therapy to directly target mitochondria.

Today, Mighty's development portfolio encompasses rare and age-related diseases. Mighty continues to develop elamipretide in Barth syndrome and POLG-related mitochondrial disease. Mighty's Phase 3 ReNEW clinical trial of elamipretide in dry age-related macular degeneration is fully enrolled, with data expected late 2027, and Mighty is progressing its next-generation clinical candidate, bevemipretide, into a Phase 2/3 clinical trial in dry age-related macular degeneration by year-end. Mighty is also developing systemic bevemipretide for Parkinson's disease and continues to develop preclinical assets MYTX-255 and MYTX-589 for rare mitochondrial disorders. For more information, visit www.mightytx.com.

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