About SME instrument and the Khon2btreat project
The SME Instrument is part of the European Innovation Council (EIC) pilot that supports top-class innovators, entrepreneurs, small companies and scientists with funding opportunities and business acceleration services. Khondrion’s project KHON2bTreat “Late Clinical Development of KH176: an innovative orphan drug to reach mitochondrial disease patients & market” is one of the 65 granted projects selected out 1664 proposals.
Jan Smeitink, Khondrion’s CEO, said “This H2020 SME instrument grant is a great opportunity for Khondrion enabling us to accelerate the clinical development of KH176 for patients suffering from mitochondrial disease who have an unmet medical need for clinical relevant treatments. We are extremely proud to be awarded with the most competitive grant for SME’s in Europe and are eager to start and bring this innovative project to a success.”
KH176 is an orally bio-available small molecule in development by Khondrion for the treatment of mitochondrial (-related) diseases. The compound is a member of a new class of potential Khondrion drugs essential for the control of oxidative and redox alterations. Khondrion reported earlier that KH176 was well tolerated, appeared safe and provided first evidence of efficacy in the KHENERGY study, a Phase II exploratory trial with oral KH176 in the m.3243A>G multisystem mitochondrial MELAS and MIDD syndromes and mixed phenotypes.
About mitochondrial diseases
Mitochondria, the cell’s powerhouses, produce the energy necessary for life. Mitochondrial failure, due to either mutations in the mitochondrial genome or the nuclear DNA, is associated with a broad range of diseases, including orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS, MIDD and LHON syndromes as well as diseases like Parkinson’s Disease. Cellular consequences like abnormal mitochondrial architecture, reactive oxygen species production and alterations in the cellular redox-state are common findings in these diseases. Khondrion’s drug development strategy is based on counteracting these cellular consequences to stop disease progression and to restore normal cellular function.
Khondrion is an innovative clinical-stage pharmaceutical company focusing on developing small molecule therapeutics for mitochondrial (-related) diseases. The potential of several lead compounds to serve as new treatment modalities for mitochondrial disease is currently being explored. Khondrion’s KH176 has been granted Orphan Drug Designation (ODD) for Leigh disease and MELAS syndrome in Europe and for all inherited mitochondrial respiratory chain disorders in the USA. Thanks to its strategic partnership with the Radboud Center for Mitochondrial Medicine of the Radboud University Medical Center, Nijmegen, The Netherlands, Khondrion has access to all mitochondrial tools, technologies and expertise. Besides, Khondrion has established collaborations with patient organizations, patient advocacy groups and university research groups around the world as well as with small, medium and large enterprises. Khondrion is among others supported by the Dutch Foundations Energy4All, Join4Energy, Road4Energy, Ride4Kids, Tim Foundation, Zeldzame Ziekten Fonds, and National and European Governments. Khondrion has established a strong intellectual property position protecting its emerging product portfolio through the filing of multiple broad patent applications. For more information, please visit www.khondrion.com