Government support, alongside private investments, funds first wave of pivotal Phase 3 trial of sonlicromanol for m.3243A>G Primary Mitochondrial Disease (PMD)
NIJMEGEN, the Netherlands – 6 May 2025 – 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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Notes to editors
About Khondrion
Khondrion is a clinical-stage biopharmaceutical company leading the development of innovative therapies targeting primary mitochondrial diseases (PMDs). With a focus on first-in-class treatments that address the devastating effects of mitochondrial dysfunction, Khondrion aims to transform the lives of patients living with these highly impactful multisystem disorders.
One of the most advanced, disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has been tested in three clinical trials in patients with m.3243A>G PMD, as well as in the first wave of a 6-month Phase 2 study in children with genetically confirmed PMD and who suffer from motor symptoms.
Sonlicromanol has been granted orphan drug designations for the treatment of MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the U.S. It has also been granted a rare pediatric disease designation in the US for the treatment of MELAS.
Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment. For more information visit www.khondrion.com.
About Primary Mitochondrial Disease
Mitochondrial disease occurs when mitochondria, found within all cells of the human body except erythrocytes, and responsible for producing the energy necessary for cells to function, are defective. This can result in a wide range of serious and debilitating illnesses occurring shortly after birth or later in life. Signs and symptoms of these can include cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth.
Originally referred to as MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and strokelike episodes), primary mitochondrial disease associated with the m.3243A>G variant in the mitochondrial genome is now considered to include a spectrum of phenotypes including classic MELAS, MIDD syndrome (maternally inherited diabetes mellitus and deafness), MP (mixed phenotypes) and CPEO (chronic progressive external ophthalmoplegia). Sonlicromanol has been developed as a disease-modifying intervention for all primary mitochondrial disease, including all of the syndromes related to the m.3243A>G variant. Learn more: https://www.khondrion.com/melassyndrome/.
Forward-looking statements
This press release may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the company’s clinical trials and presentation of data from clinical trials for sonlicromanol. Although the company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the company’s control. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “goal,” “intend,” “may,” “anticipate,” “foreseen,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “potential,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof.
Forward-looking statements are subject to inherent risks and uncertainties beyond the company’s control that could cause the company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
Contacts:
Khondrion BV
Prof. Dr. Jan Smeitink, CEO
E-mail: info@khondrion.com
Tel: +31-24-7635000
www.khondrion.com
