NIJMEGEN, the Netherlands – 14 September 2022: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announces that Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, will participate in the mitoNice international congress taking place in Nice, France, from tomorrow until Saturday 17 September.
Hosted by the French patient association, AFM-Téléthon, mitoNice is an international forum dedicated to mitochondrial medicine, bringing together more than 200 experts in the field who will share the latest research and progress in the development of innovative therapies to treat mitochondrial diseases.
Dr. Smeitink will join the plenary session, “New treatments for mitochondrial diseases: update on recent and current clinical trials”, on Saturday 17 September from 08:30 to 10:00am (CEST). His presentation, “Sonlicromanol in primary mitochondrial disease MELAS spectrum disorders”, will provide a development overview on sonlicromanol, Khondrion’s lead clinical asset.
Sonlicromanol is being developed to treat a range of mitochondrial diseases in children and adults. A first-in-class, oral small molecule, it targets key underlying mechanisms of mitochondrial disease based on its unique triple mode of action: redox modulation to help restore the cell’s metabolism, radical trapping preventing ferroptotic cell death, and mPGES-1 inhibition resulting in anti-inflammatory effects. Headline results from the Company’s Phase IIb programme, in adult patients with MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, are expected later this year.
Prof. Dr. Jan Smeitink, Chief Executive Officer of Khondrion, commented: “This conference brings together physicians, researchers and patients from around the world and provides us with an important opportunity to discuss the latest research in mitochondrial disease and the current understanding around potential pathways towards diagnosis and treatment. This international exchange and collaboration between physicians, the scientific community and patients is key to developing the much-needed new treatment approaches to tackle these severe and debilitating diseases.”
For more information on, or to register for mitoNice, visit www.mitonice.org
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Prof. Dr. Jan Smeitink, CEO
Tel: +31 24 7635000
Consilium Strategic Communications
Mary-Jane Elliott, David Daley, Melissa Gardiner
Tel: +44 20 3709 5700
Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with inherited mitochondrial diseases. Based on proprietary science and a deep biological understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate sonlicromanol, a first-in-class, oral small molecule targeting key underlying mechanisms of mitochondrial disease based on the drug’s unique triple mode of action. Sonlicromanol is a reductive and oxidative distress modulator with anti-inflammatory properties.
One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol is currently being tested in a Phase IIb trial and a 12-month open-label extension study in adult patients with MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, as well as in a 6-month Phase II study in children with genetically confirmed primary mitochondrial diseases and who suffer from motor symptoms. The compound 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 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 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.