Khondrion Secures up to €5 Million Innovation Credit from Dutch Government plus Additional Funding from Current Investors to Advance Phase 3 Clinical Trial in m.3243A>G Primary Mitochondrial Disease

Government support, alongside private investments, funds first wave of pivotal Phase 3 trial of sonlicromanol for m.3243A>G Primary Mitochondrial Disease (PMD)

NIJMEGEN, Netherlands, May 6, 2025 /PRNewswire/ -- Khondrion, a clinical-stage biopharmaceutical company pioneering therapies for primary mitochondrial disease (PMD), today announced it has been awarded an Innovation Credit of up to €5 million from the Netherlands Enterprise Agency, a government agency that supports entrepreneurs.

This vital government funding, matched by a significant investment from Khondrion's committed private backers, will fund Wave 1 of the company's pivotal Phase 3 clinical trial of sonlicromanol. Sonlicromanol is a first-in-class small-molecule candidate therapy for m.3243A>G PMD, the most common genetic form of mitochondrial disease. The Phase 3 trial is expected to launch in H2-2025, marking a crucial milestone in Khondrion's mission to deliver the first approved treatment for these devastating, multisystem disorders.

A Major Step Forward for the Mitochondrial Disease Community

Primary mitochondrial diseases are a group of rare, progressive, and life-altering disorders that disrupt the body's ability to generate energy. This leads to a wide range of severe symptoms across multiple organ systems, including the brain, muscles, and digestive tract. For the over 250.000 of people living with PMD worldwide, there are currently no approved therapies that directly address the causes underlying the disease.

"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," said Jan Smeitink, CEO of Khondrion. "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."

With the Phase 3 trial on the horizon, Khondrion is taking a bold step towards changing the lives of patients suffering from mitochondrial disease — and creating meaningful opportunities for partners ready to join this journey.

Sonlicromanol: one of the most advanced drug candidates in development for PMD

Sonlicromanol has been investigated in four clinical trials. Adult patients participating in our extensive Phase 2 programme have reported continued and progressive improvements on key symptoms (see Smeitink et al., Brain 2025; https://doi.org/10.1093/brain/awae277). Those patients that elected to participate in a named patient program following completion of the Phase 2b programme, have been receiving sonlicromanol for more than two and a half years now, thus confirming the drug's favorable safety profile.

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SOURCE Khondrion