Khondrion, a clinical-stage biopharmaceutical company focused on developing therapies for primary mitochondrial disease (PMD), has been awarded an Innovation Credit of up to €5 million from the Netherlands Enterprise Agency. This government funding, combined with significant investment from private backers, will finance the initial phase of the company's pivotal Phase 3 clinical trial for sonlicromanol, a potential first-in-class treatment for m.3243A>G PMD.
The Phase 3 trial is expected to commence in the second half of 2025, marking a critical step toward delivering the first approved therapy for this rare genetic disorder. The m.3243A>G mutation represents the most common genetic form of mitochondrial disease, a condition for which no approved treatments currently exist.
Addressing a Critical Unmet Need
Primary mitochondrial diseases affect approximately 250,000 people worldwide. These rare, progressive disorders disrupt cellular energy production, leading to severe multisystem symptoms affecting the brain, muscles, and digestive tract. The lack of approved therapies that directly address the underlying causes has left patients with limited treatment options.
Jan Smeitink, CEO of Khondrion, emphasized the significance of this funding: "Receiving this support from the Dutch government, together with the continued commitment of our trusted investors, represents a powerful endorsement of Khondrion's work and the urgent need to bring innovative treatments to the PMD community."
He added, "With sonlicromanol, we are advancing a potentially transformative therapy designed not only to alleviate symptoms but also to modify the course of this devastating disease, offering new hope to patients and families who have long been waiting for effective solutions."
Promising Clinical Evidence
Sonlicromanol has already demonstrated encouraging results in earlier clinical investigations. The drug candidate has been studied in four clinical trials to date, with adult patients in the Phase 2 program reporting continued and progressive improvements in key symptoms, as documented in a recent publication by Smeitink et al. in the journal Brain (2025).
Notably, patients who elected to participate in a named patient program following completion of the Phase 2b studies have been receiving sonlicromanol for more than two and a half years, providing additional evidence of the drug's favorable safety profile over extended periods.
Mechanism and Potential Impact
As a first-in-class small molecule therapy, sonlicromanol targets the cellular dysfunction caused by mitochondrial disease. The drug is designed to address the energy production deficits that characterize PMD, potentially offering both symptomatic relief and disease-modifying effects.
The advancement to Phase 3 represents one of the most significant developments in the mitochondrial disease treatment landscape. If successful, sonlicromanol could become the first approved therapy specifically targeting m.3243A>G PMD, addressing a substantial unmet medical need.
Strategic Implications
The funding package, combining government support with private investment, not only enables Khondrion to proceed with the critical Phase 3 trial but also positions the company as a leader in mitochondrial disease therapeutics. The company has indicated that this milestone creates meaningful opportunities for potential partners interested in joining their mission.
For patients and families affected by mitochondrial disease, the progression of sonlicromanol to late-stage clinical development offers renewed hope for a condition that has historically had limited therapeutic options. The Phase 3 trial will be closely watched by the rare disease community as it could potentially change the treatment paradigm for this devastating disorder.
