UMDF & Khondrion Announce The Mito Fund’s Investment in Khondrion to Support Development of One of the Most Advanced Drug Candidates for Primary Mitochondrial Disease

The United Mitochondrial Disease Foundation’s (UMDF) venture philanthropy arm, The Mito Fund, has invested in Khondrion, a Netherlands-based clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease (PMD).

Khondrion is currently preparing to initiate a Phase 3 study investigating the potential of its lead proprietary drug candidate, sonlicromanol, to treat adult patients with PMD due to the m.3243A>G mutation, the most common genetic defect causing multi-systemic PMD, which has an estimated prevalence of 4.4:100,000 impacting hundreds of thousands of patients worldwide.

One of the most advanced drug candidates in development for PMD, sonlicromanol is a disease-modifying, potentially first-in-class, brain-penetrant redox-modulator with anti-inflammatory properties, that targets key metabolic and inflammatory pathways underlying PMD. Its ongoing development is supported by compelling clinical data from Khondrion’s Phase 2b program, published earlier this month in the peer-reviewed scientific journal, Brain, showing strong patient benefits from sonlicromanol in multiple outcome measures of global health, quality of life, mood, fatigue, pain and balance control.

“We believe sonlicromanol holds incredible potential for treating primary mitochondrial disease which impacts hundreds of thousands of people worldwide,” said Dr. Philip Yeske, UMDF’s Interim Managing Director and Science and Alliance Officer. “But it’s not just about numbers. The diseases associated with this mutation are devastating and remarkably progressive. The approval of this therapy would meet an urgent and unmet therapeutic need.”

Said Khondrion’s Chief Executive Officer, Prof. Dr. Jan Smeitink, added “Patients with primary mitochondrial disease currently have no treatment options available to them. Through our clinical trials to date, we see the great potential sonlicromanol may have on these patients, particularly on their most burdensome symptoms of chronic fatigue and muscle weakness. With the support and investment by the UMDF via The Mito Fund, we look forward to further progressing our clinical work on sonlicromanol at pace in our upcoming Phase III trial in adult patients with PMD.”

Like all investments made by The Mito Fund, the Khondrion consideration went through a rigorous review process by UMDF’s Venture Philanthropy Investment Committee, an advisory group consisting of world-class experts in the areas of mitochondrial disease, pharmaceutical development, and private equity investments. After receiving the committee’s recommendation, the investment was then approved by UMDF’s full Board of Trustees.

“From an investment perspective, there were several really attractive elements to the investment. First, sonlicromanol represents an innovative treatment for an underserved population. It’s also a mature clinical stage asset, which shortens the runway to approval,” said Dr. Yeske. “UMDF looks forward to partnering with Khondrion in advancing this investigational therapeutic in a patient-centric manner.”

Launched in the summer of 2023, The Mito Fund is UMDF’s most ambitious effort to date to ramp up the development of mitochondrial disease treatments and cures. The fund’s venture philanthropy model is designed not only to financially support the development of the next generation of mitochondrial disease treatments, but also to leverage UMDF’s experience and network to accelerate the development process and lower the risk associated with entry into the field for a wide range of investors and researchers.

This is the second official investment for The Mito Fund. In April 2024, the fund announced an investment in Pierrepont Therapeutics, Inc., which is working on a preclinical proprietary intracellular enzyme replacement therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).

Companies exploring the mitochondrial science space are encouraged to start a conversation about investment opportunities at www.themitofund.org. Proposals are accepted on a rolling basis.

For more than 25 years, The United Mitochondrial Disease Foundation (UMDF) has built a global network of patients, researchers, clinicians, institutions, and industry partners dedicated to fighting mitochondrial disease. Together with the mito community, UMDF is committed to a mission of funding the best science no matter where it is found in the world while at the same time providing critical education, advocacy, and support-focused programs and services to patient families.

In the last year …

• Nearly 1,500 affected patients and families depended upon UMDF for support.
• More than 400 clinicians listened to UMDF’s Bench-to-Bedside seminars.
• UMDF advocates had more than 1,000 touchpoints with their congressional offices.
• UMDF provided hundreds of patients with no-cost genetic testing to help shorten the diagnostic journey.

Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with primary mitochondrial disease (PMD). The company’s lead asset, sonlicromanol, is a potentially first-in-class, brain-penetrant redox-modulator with anti-inflammatory properties, that targets key metabolic and inflammatory pathways underlying PMD.

One of the most advanced, potentially disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has been tested in three clinical trials in patients with m.3243A>G PMD, as well as in the first wave of a 6-month Phase 2 study in children with genetically confirmed PMD and who suffer from motor symptoms.

Sonlicromanol has been granted orphan drug designations for the treatment of MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a rare pediatric disease designation in the US for the treatment of MELAS.

Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment. For more information visit www.khondrion.com.