Mitochondrial diseases currently have no cure, but a new oral drug candidate could potentially improve clinical outcomes for patients with the most common genetic cause of the disease.
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In a Phase 2b trial, Khondrion’s sonlicromanol led to significant health benefits for patients with mitochondrial disease linked to a common mutation.
When a mutation occurs in mitochondrial DNA, it can disrupt the organelle’s ability to produce energy. If the balance of mutant to healthy mitochondria in a cell — known as heteroplasmy — shifts toward more mutant than healthy ones, it can lead to mitochondrial disease, a rare and debilitating group of disorders with no known cure. Because heteroplasmy can vary across cells, organs, and even among individuals within a family, mitochondrial diseases often…