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December 4, 2015 -­‐ FOR IMMEDIATE RELEASE

NIJMEGEN – Khondrion, the Dutch biopharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the successful completion of its Phase 1 Clinical trials with its lead compound KH176. KH176 is an orally bio-­‐available small molecule developed by Khondrion for the treatment of mitochondrial (-­‐related) diseases. The compound is a member of a new class of Khondrion drugs essential for the control of oxidative and redox pathologies. The Phase 1 Clinical trials, performed in healthy male volunteers, consisted of single ascending and multiple dosing arms both randomized, placebo controlled and double-­‐blinded. The study deemed that KH176 is well tolerated and has an excellent pharmacokinetic profile.

Jan Smeitink, Khondrion’s CEO and Professor of Mitochondrial Medicine at the Radboud University Medical Center, Nijmegen, The Netherlands, said “With the successful outcome of the KH176 Phase I Clinical trials we have set an important step to accomplish our mission – making a substantial contribution to the development of drugs for patients suffering from mitochondrial diseases".


Khondrion expects to initiate randomized, placebo-­‐controlled, double-­‐blinded Phase II Clinical trials in 2016.

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 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.

About Khondrion

Khondrion is an innovative Dutch biopharmaceutical 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 lead compound 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 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, 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.

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Cautionary Note Regarding Forward-­‐Looking Statements

This press release contains forward-­‐looking statements. All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, future      financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-­‐looking statements. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions are intended to identify forward-­‐looking statements, although not all forward-­‐looking statements contain these identifying words. Any forward-­‐looking statements represent our views only as of today and should not be  relied upon as representing our views as of any subsequent date. 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. While we may elect to update these forward-­‐looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.


Khondrion BV
Prof. dr. Jan Smeitink, CEO
E-mail:  This email address is being protected from spambots. You need JavaScript enabled to view it.
Tel: +31-24-3610639

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