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Khondrion shares progress of sonlicromanol development programme at Mitochondrial Medicine 2020 virtual conference

NIJMEGEN, the Netherlands – Monday, November 30, 2020: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the Company will provide a development overview of its Phase 2B clinical-stage drug sonlicromanol in a poster lightning talk session at the Mitochondrial Medicine 2020 virtual conference, being held from November 30 to December 2, 2020.

Sonlicromanol is Khondrion’s wholly-owned, potentially first-in-class oral small molecule and one of the most clinically-advanced disease-modifying drug treatments for mitochondrial disease in development.

An ongoing Phase 2B clinical trial in patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is investigating the effect of sonlicromanol on cognitive functioning. Cognitive impairment is becoming increasingly recognised in mitochondrial disease patients and can have a significant and debilitating impact on many aspects of their lives. Sonlicromanol’s potential to positively affect cognitive decline is supported by preclinical research showing improvements in brain parameters related to cognition. Results from a completed Phase IIa study showed a significant improvement in attention and mood related outcomes among patients treated with sonlicromanol compared to placebo.

Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: "The annual Mitochondrial Medicine conference is always an important global scientific meeting for all those working to achieve a better future for patients with mitochondrial disease.”

“The impact on everyday life from cognitive impairment caused by mitochondrial disease can be significant. It can affect everything we do involving thought or memory. Changes in thinking and memory can be among the most confusing and scary symptoms because of their broad impact across patients’ lives. They are also some of the least talked about. We believe sonlicromanol has the potential to be an important disease-modifying treatment option for these patients and we look forward to sharing our progress with colleagues.”

Sonlicromanol has been granted Orphan Drug Designation for MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe and for all inherited mitochondrial respiratory chain disorders in the USA. It has also been granted a Rare Pediatric Disease designation in the US for the treatment of MELAS.  

Khondrion’s poster, “Sonlicromanol – a Phase 2B clinical stage drug for primary mitochondrial disease: Mode of action and development programme”, will be presented in a lightning talk session on Tuesday December 1, at 2pm GMT during Session 3: Clinical Trials. For more information on the Mitochondrial Medicine 2020 virtual conference, visit

About Khondrion

Khondrion is a privately held clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Prof. Jan Smeitink, a world-leader in mitochondrial medicine, the company is advancing its proprietary science through a wholly-owned clinical and preclinical small molecule pipeline of potential medicines.  

The company’s in-house discovery engine is using unique live-cell imaging technologies, patient-derived cell lines and predictive cell-based disease models to build a portfolio of promising compounds. Active discovery programmes are underway developing new therapies, biomarkers and new read-out technologies in the field of mitochondrial diseases.

To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with a global clinical and academic network and patient organisations internationally. Khondrion is headquartered in Nijmegen, The Netherlands. For more information visit


About mitochondrial disease

Mitochondrial disease occurs when mitochondria, found within all cells of the human body 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. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, MIDD (maternally inherited diabetes and deafness), LHON (Leber's hereditary optic neuropathy) and other respiratory chain/ oxidative phosphorylation disorders, are all examples of mitochondrial disease.

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Last Updated: 30-Nov-2020