7 October 2020
Khondrion to present at Mitocon’s 10th Italian Meeting on Mitochondrial Diseases
NIJMEGEN, the Netherlands – 7 October, 2020: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, will present at Mitocon’s 10th Italian Meeting on Mitochondrial diseases, taking place virtually on October 9-10, 2020.
The annual meeting organized by Mitocon, Italy’s leading mitochondrial disease patient association, brings together physicians, researchers and patients from around the world, providing a unique opportunity to discuss the latest research in mitochondrial disease and the current understanding around potential pathways towards diagnosis and treatment. Since the first event in 2011, it has helped to strengthen international partnerships and collaborations between the medical community and patients, enabling knowledge sharing and the progress of research in the fight against mitochondrial diseases.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: "Mitochondrial diseases are complex disorders and despite the significant progress that has been made in recent years, there is still a lot of work to do in bringing treatments to patients suffering from these debilitating diseases. The entire mitochondrial disease community, including patient groups, academia and the industry, need to work together if we are to succeed in our goal of combatting mitochondrial diseases.”
Prof. Smeitink will provide a progress update on sonlicromanol, Khondrion’s lead asset, on Friday October 9, 2020, in the session “Current trials and industry”, which begins at 14.30 (CEST). Sonlicromanol is currently in Phase IIb clinical development for MELAS spectrum disorders. The United States (US) Food and Drug Administration (FDA) recently granted sonlicromanol a rare pediatric disease (RPD) designation for the treatment of patients with MELAS syndrome. Sonlicromanol has already been granted Orphan Drug Designations for MELAS syndrome, MIDD and Leigh disease in Europe, and for all inherited mitochondrial respiratory chain disorders in the US.
For more information on, or to register for Mitocon’s 10th Italian Meeting on Mitochondrial Diseases visit www.mitocon.it
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Prof. Dr. Jan Smeitink, CEO
Consilium Strategic Communications
Mary-Jane Elliott, David Daley, Melissa Gardiner
Tel: +44 20 3709 5700
Khondrion is a 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.
Khondrion’s lead asset, sonlicromanol, is a potentially first-in-class oral small molecule in Phase IIb clinical development to treat MELAS spectrum disorders (including MELAS syndrome (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes), maternally inherited diabetes and deafness (MIDD) and mixed phenotypes). It has been granted rare pediatric disease (RPD) designation by the United States Food and Drug Administration (FDA) for MELAS syndrome and orphan drug designation (ODD) for all inherited mitochondrial respiratory chain disorders. In Europe, sonlicromanol has been granted ODD for MELAS syndrome, MIDD and Leigh disease.
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 occuring 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 syndrome (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes), 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.