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May  26,  2015

NIJMEGEN – Khondrion, the Dutch biopharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the initiation of a Phase 1 Clinical Trial with its frontrunner compound KH176. The Trial will be a randomized, double blind, placebo-­‐ controlled, dose-­‐escalating study.

 

Mitochondrial diseases are progressive, often early fatal diseases caused by a defect in the cell’s powerhouses, the mitochondria.  “The initiation of the KH176 Phase 1 Trial is a pivotal milestone in the mitochondrial disease drug development program of our company “ said Jan Smeitink, Khondrion’s CEO and Professor of Mitochondrial Medicine at the Radboud University Medical Center, Nijmegen, The Netherlands. “Our Team is very enthusiastic about the fact that this significant milestone has been reached much earlier than expected".

About Mitochondrial Disease

Mitochondrial diseases are devastating, early fatal, multi-­‐system disorders that affect 1:5000 live births. These rare diseases are caused by mutations in two different genomes, the nuclear DNA and the mitochondrial DNA, and most often give rise to a biochemical defect involving the mitochondrial oxidative phosphorylation system. Mutations of the mitochondrial DNA are maternally transmitted and consequently every child born in the family (100% recurrence risk) can be affected.  As mitochondria, the cells powerhouses, are present in virtually every cell of the body all organs and tissues can be affected leading to symptoms like mental retardation, epilepsy, deafness, blindness, heart-­‐ and liver-­‐ failure, muscle weakness and severe exercise intolerance.

About KH176

KH176 is an orally bio-­‐available small molecule developed by Khondrion for the treatment of mitochondrial diseases. KH176 is a member of a class of drugs essential for the control of oxidative and redox pathology. KH176 successfully passed extensive safety evaluations, supporting further clinical  development.

About Khondrion

Khondrion is an innovative Dutch biopharmaceutical mitochondrial medicine company focusing on developing small molecule therapeutics for mitochondrial diseases.  The potential of several lead compounds to serve as new treatment modalities for mitochondrial disease is currently being explored.  KH176 is Khondrion’s first compound to enter the clinical trial phase. Thanks to its  strategic partnership with the Nijmegen Center for Mitochondrial Disorders 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 a privately held biopharmaceutical company, among others supported by the Dutch Foundations Energy4All, Join4Energy, Road4Energy, Tim Foundation, Zeldzame Ziekten Fonds, Prinses Beatrix  Fonds, and National and European Governments. For more information, please visit www.khondrion.com.

Cautionary Note Regarding Forward-­‐Looking Statements

This press release contains "forward-­‐looking statements" as defined in the Private Securities  Litigation Reform Act of 1995, including regarding product development and production.  The reader is cautioned not to rely on these forward-­‐looking statements.  These statements are based on   current expectations of future events.  If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Khondrion.  Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product development, including the uncertainties of clinical success and the timeline for the availability of KH176. None of the Khondrion Companies undertake to update any forward-­‐looking statements as a result of new information or future events or developments.

For more information please contact:

Jan Smeitink
Chief Executive Officer

phone: +31-­‐6-­‐22933832
email:  This email address is being protected from spambots. You need JavaScript enabled to view it.

Philips van Leydenlaan 15
6525 EX Nijmegen
The Netherlands

www.khondrion.com 

 

© 2019 Int. Mito Patients