The United Mitochondrial Disease Foundation's (UMDF) venture philanthropy arm, The Mito Fund, has invested in Khondrion, a Netherlands-based biopharmaceutical company, to advance the clinical development of sonlicromanol, a promising therapy for primary mitochondrial disease (PMD). Khondrion is preparing to initiate a Phase III study to evaluate sonlicromanol in adult patients with PMD caused by the m.3243A>G mutation, the most common genetic defect associated with multi-systemic PMD.
Sonlicromanol: A Potential First-in-Class Therapy
Sonlicromanol is a disease-modifying, potentially first-in-class, brain-penetrant redox-modulator with anti-inflammatory properties. It targets key metabolic and inflammatory pathways implicated in PMD. Data from a Phase IIb trial, published in Brain, demonstrated significant patient benefits across multiple outcome measures, including global health, quality of life, mood, fatigue, pain, and balance control.
Addressing Unmet Needs in PMD Treatment
"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. He emphasized the urgent need for effective therapies, noting that the diseases associated with the m.3243A>G mutation are "devastating and remarkably progressive."
Prof. Dr. Jan Smeitink, CEO of Khondrion, stated that currently, "patients with primary mitochondrial disease currently have no treatment options available to them." He expressed optimism about sonlicromanol's potential to alleviate burdensome symptoms like chronic fatigue and muscle weakness, based on observations from previous clinical trials.
The Mito Fund's Investment Strategy
The Mito Fund's investment in Khondrion underwent a rigorous review process by UMDF's Venture Philanthropy Investment Committee, comprising experts in mitochondrial disease, pharmaceutical development, and private equity investments. The investment was subsequently approved by UMDF's Board of Trustees.
Dr. Yeske highlighted the attractive elements of the investment, noting that sonlicromanol represents an innovative treatment for an underserved population and is a mature clinical-stage asset, which shortens the runway to approval.
About The Mito Fund and UMDF
Launched in 2023, The Mito Fund aims to accelerate the development of mitochondrial disease treatments and cures. It employs a venture philanthropy model to support the development of next-generation therapies and leverage UMDF's expertise and network.
The United Mitochondrial Disease Foundation (UMDF) has been dedicated to fighting mitochondrial disease for over 25 years, building a global network of patients, researchers, clinicians, institutions, and industry partners. UMDF focuses on funding research and providing education, advocacy, and support to patient families.
Khondrion's Focus on Mitochondrial Disease
Khondrion is a clinical-stage biopharmaceutical company focused on developing therapies for PMD. Sonlicromanol has been tested in multiple clinical trials in patients with m.3243A>G PMD and has received orphan drug designations in Europe and the US for various mitochondrial disorders, including MELAS syndrome, Leigh disease, and MIDD.
