NIJMEGEN, the Netherlands – 9 October, 2024: 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 14th Mitochondrial Disease Conference, taking place October 25th-27th, 2024, in Padova, Italy.
The annual meeting organized by Mitocon, Italy’s leading mitochondrial disease patient association, brings together physicians, researchers, patients and industry 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 2010, 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: “Primary Mitochondrial Disease is an umbrella term encompassing hunderds of gene defects affecting the mitochondrial oxidative phosphorylation system. Many of these gene defects are ultra-rare while others are more common like the m.3243A>G mutation in the mitochondrial DNA transfer RNA of Leucine. The latter mutation is associated with classic MELAS syndrome (approximately 5-10% of patients with this particular mutation), MIDD syndrome (approximately 40-50%) and mixed phenotypes1”.
Prof. Smeitink will provide a progress update on sonlicromanol, Khondrion’s lead asset, on Saturday October 26, 2024, in the session “Roundtable with Pharmaceutical Industries and Regulatory Authorities”, which begins at 11.30 (CET). The Khondrion Team, which recently completed the Phase 2b Program with sonlicromanol in adults patients with the m.3243A>G mutation, is preparing for a Phase 3 registrational trail in Europe and the US. The United States (US) Food and Drug Administration (FDA) has 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 14th Mitochondrial Disease Conference visit https://www.mitocon.it/14-convegno-sulle-malattie-mitocondriali-2024/ 1 Six-year prospective follow-up study in 151 carriers of the mitochondrial DNA 3243 A>G variant. de Laat P, Rodenburg RR, Roeleveld N, Koene S, Smeitink JA, Janssen MC.J Med Genet. 2021 Jan;58(1):48-55. doi: 10.1136/jmedgenet-2019-106800.
Contacts:
Khondrion BV
Prof. Dr. Jan Smeitink, CEO
E-mail: info@khondrion.com
Tel: +31-24-7635000