Khondrion Secures €5M to Advance Small Molecule Therapy Toward Phase 3 Trial

Khondrion, a clinical-stage biotech company focused on primary mitochondrial disease (PMD), has secured up to €5M in Innovation Credit from the Netherlands Enterprise Agency, alongside additional funding from its existing private investors. This combined support is intended to fund the first phase (Wave 1) of the company’s planned Phase 3 clinical trial of sonlicromanol, a small-molecule drug candidate targeting the m.3243A>G mutation, the most common genetic form of mitochondrial disease.

See also: The Explosion of Therapeutic Modalities: Small Molecules, Biologics, and Everything in Between

The trial, expected to begin in the second half of 2025, represents a potentially important step in Khondrion’s longer-term aim to develop a first approved therapy for this complex and often debilitating group of disorders. The company sees the Innovation Credit as a vote of confidence in both the company’s scientific approach and the broader need for therapies that address the underlying causes of PMD.

Khondrion's team; Source: Khondrion

PMD refers to a set of progressive diseases linked to dysfunctional cellular energy production. These conditions can affect multiple organs and currently have no approved treatments that directly target their genetic root. Sonlicromanol has previously been tested in four clinical studies, including a Phase 2b study in 2022 where participants reported sustained improvement in several symptoms. Even though the primary study endpoints were not met, these positive trends resulted in the second  Phase IIb extension study which lasted 52 weeks till 2025 and operated a larger test pool.

Sonlicromanol was reported to be safe and well tolerated, with no serious adverse effects. The data suggest potential benefits across several clinical domains, including cognition, mood, pain, fatigue, and balance, with more noticeable effects observed in the longer-term.

Topics: Clinical Trials