Khondrion receives EMA agreement on its Paediatric Investigation Plan for sonlicromanol NIJMEGEN, the Netherlands – 18 January, 2021: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Medicines Agency (EMA) has accepted its Paediatric Investigation Plan (PIP) for the development of its wholly-owned lead asset, sonlicromanol, for children. Sonlicromanol is Khondrion’s 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 IIb clinical trial in adult patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is investigating the effect of sonlicromanol on cognitive functioning. Sonlicromanol has been granted Orphan Drug Designations for the treatment of MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a Rare Paediatric Disease (RPD) designation in the US for the treatment of MELAS. A PIP outlines a company’s strategy for the investigation of new medicinal products in children and an approved PIP is a prerequisite for filing a Marketing Authorisation Application (MAA) for a new medicinal product in the European Union. With the acceptance of the PIP, Khondrion plans to initiate a double-blind, randomised, placebo-controlled paediatric trial across various sites in Europe to evaluate the safety and efficacy of sonlicromanol in children from birth to less than 18 years of age with genetically confirmed mitochondrial disease affecting oxidative phosphorylation and symptoms of motor impairment. The trial is expected to begin in H1 2021. Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: "The acceptance of this PIP by the EMA is an important milestone in our efforts to bring sonlicromanol to all individuals living with mitochondrial disease who might benefit from this disease-modifying potential treatment. Developing sonlicromanol for a paediatric population is a key part of our clinical research programme here at Khondrion.” Dr. Rob van Maanen, Chief Medical Officer at Khondrion, said: "Mitochondrial diseases present a serious unmet need in children, with those diagnosed early in life experiencing a progression of symptoms that can have a significant impact on their quality of life. We look forward to progressing our research plans to further understand the potential of sonlicromanol to treat these diseases in early life.” – ENDS – Contacts: Khondrion BVProf. Dr. Jan Smeitink, CEOE-mail: email@example.com Tel: +31-24-3617505 www.khondrion.com Consilium Strategic Communications Mary-Jane Elliott, David Daley, Melissa Gardiner E-mail: firstname.lastname@example.org Tel: +44 20 3709 5700 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 www.khondrion.com 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 including 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.