Khondrion to participate in the 16th Annual Dutch Inborn Error Meeting and the United Mitochondrial Disease Foundation (UMDF) Bench to Bedside seminar
NIJMEGEN, the Netherlands – 24 October 2023: Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announces that Prof. Dr. Jan Smeitink, Chief Executive Officer will participate in the Annual Dutch Inborn Error Meeting on 16 November 2023 and the Bench to Bedside seminar organised by the US based UMDF on 11 December 2023. During both events, Dr. Smeitink will present an update on sonlicromanol’s Phase 2b programme in Primary Mitochondrial Disease m.3243A>G MELAS, MIDD and Mixed Phenotype Patients.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “Opportunities to share knowledge at these kind of events, are instrumental to progress research and development of mitochondria-targeted therapies. The ongoing 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.”
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Notes to editors
About the Phase 2b programme in adult m.3243A>G spectrum disorders
KHENERGYZE – A 28-day double-blind, randomised, placebo-controlled, multi-centre, three-way cross-over Phase 2b study examining cognitive function in adult patients with a specific genetically confirmed DNA mutation in the mitochondrial transfer RNALeu(UUR) (MT-TL1m.3243A>G). This mutation is responsible for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, including classical MELAS and MIDD (maternally inherited diabetes and deafness) syndromes, and mixed phenotypes.
Further details are available on clinicaltrials.gov (NCT04165239) and eudract.ema.europe.eu (2019-000599-40).
KHENEREXT – An open-label extension study, enabling continued sonlicromanol treatment for patients who had completed the KHENERGYZE Phase 2b study and the collection of longer-term data. Alongside safety, the study also gathers a broad set of primary and secondary clinical outcome measures, including fatigue and motor-function related parameters.
Further details are available on clinicaltrials.gov (NCT04604548) and eudract.ema.europe.eu (2020-000832-23).
About Khondrion
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. Sonlicromanol is a first-in-class, oral small molecule targeting key underlying mechanisms of primary mitochondrial disease based on its uniquely differentiated triple mode of action: reductive distress modulation to help restore the cell’s metabolism, oxidative distress modulation preventing ferroptotic cell death, and selective mPGES-1 inhibition resulting in anti-inflammatory effects. Based on the favourable benefit-risk profile shown accross the clinical studies performed so far, Khondrion is finalizing the design of a global Phase 3 study which is currently foreseen to start in H2 2024.
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.
Ongoing pre-clinical research of sonlicromanol shows potential broader applications of the drug in other rare diseases and more common disorders.
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), and other respiratory chain / oxidative phosphorylation disorders, are all examples of mitochondrial disease. MELAS spectrum disorders are some of the most frequently observed primary mitochondrial diseases, in which all patients are characterised by an underlying point mutation (m.3243A>G) in the maternally inherited MT-TL1 gene.
Contacts:
Khondrion BV
Prof. Dr. Jan Smeitink, CEO
E-mail: info@khondrion.com
Tel: +31-24-7635000