The United Mitochondrial Disease Foundation’s venture philanthropy arm, The Mito Fund, has invested in Pretzel Therapeutics, a Massachusetts-based company with research facilities in Mölndal, Sweden. Pretzel is focused on using cellular energetics to improve survival and quality of life for various conditions.
The lead program, PX578, recently entered Phase 1 clinical development in the U.S. And it aims to restore function to mutant mitochondrial DNA polymerases, enabling for the repopulation of depleted mitochondrial DNA (mtDNA). This strategy shows promise for treating POLG disorders and other severe conditions linked to mtDNA depletion.
POLG-related mitochondrial diseases affect multiple organs and are one of the most common inherited mitochondrial disorders, impacting about 1 in 10,000 patients. Currently, there is no FDA-approved therapy for these diseases.
The Mito Fund conducts a thorough review process for investments, overseen by a committee of experts in mitochondrial disease and pharmaceutical development, and approved by UMDF’s Board of Trustees. Launched in summer 2023, the Mito Fund seeks to advance the development of treatments for mitochondrial diseases, providing financial support while leveraging UMDF’s expertise to minimize risks for investors and researchers.
Other companies in the Mito Fund portfolio include:
— Napigen Therapeutics – A company that aims to harness the potential of mitochondria to impact human healthcare with its unique organelle genome editing tools.
— Pierrepont Therapeutics – A company focused on advancing to the clinic a proprietary intracellular enzyme replacement therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).
— Khondrion – A clinical-stage biopharmaceutical company focused on discovering and developing therapies targeting primary mitochondrial disease (PMD). Khondrion’s lead asset sonlicromanol is in clinical development to improve fatigue (lack of energy), muscle weakness, and other symptoms in people living with m.3243A>G Primary Mitochondrial Disease, including classic MELAS, MIDD, and Multi-system Phenotypes.
KEY QUOTES:
“The collective disease types targeted by PX578 represent some of the most common and relentlessly progressive forms of mitochondrial disease. There still remains much for Pretzel to do, but ultimately an approval would meet a huge unmet need in our community.”
“We received tremendous feedback from our committee when it comes to the potential of PX578. But in our conversations with Pretzel, the thing that also excited us was the company’s commitment to partnering with patient advocacy groups like UMDF to advance broad POLG-related research. If we’re ever going to get to an approved therapy, that’s a critical component, and we look forward to deepening the relationship with Pretzel.”
UMDF’s Science and Alliance Officer Dr. Philip Yeske
“We are honored to receive this investment from The Mito Fund, whose commitment to improving the lives of patients living with mitochondrial disease aligns deeply with our mission. This funding not only validates the promise of our scientific approach, but also supports development of PX578, the lead therapeutic in our energetics restoration franchise, with disease modifying potential across rare mtDNA depletion syndromes and broader degenerative diseases.”
Gabriel Martinez, Ph.D., Chief Scientific Officer and Co-Founder of Pretzel Therapeutics
