EFRO Grant to support the KHENERGYC study

12 September 2016

Nijmegen, September 12, 2016


THE KHENERGYC STUDY: European Union EFRO grant to boost the development of Khondrion’s KH176 for children with mitochondrial disease

NIJMEGEN, the Netherlands – Khondrion, the clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases has received almost 1 mEuro from the EFRO (Europees Fonds voor Regionale Ontwikkeling) of the European Union. The grant will be used to start the preparations and execution, of the KHENERGYC STUDY, a phase 2 clinical trial with KH176 in children with mitochondrial disease.

Khondrion’s 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 redox pathologies. In December 2015 Khondrion reported that KH176 demonstrated a favorable pharmacokinetic profile and an acceptable safety profile in a randomized, placebo-controlled, double blind phase 1 clinical trial, performed in healthy male volunteers.

Last Thursday Khondrion announced the initiation of the KHENERGY study, a phase 2 clinical trial of KH176 in adult patients with mitochondrial diseases.

Jan Smeitink, Khondrion’s CEO, said “This European Union EFRO grant enables us, in parallel with the initiated KHENERGY study, to begin with all regulatory preparations aiming at a final execution of a phase 2 clinical trial in children with mitochondrial disease who have an unmet medical need for clinically relevant treatments. I am proud that already in the fourth year since its operational start Khondrion’s team members have reached this important new milestone”.

This project will be executed under the supervision of Khondrion in partnership with the Radboud University Nijmegen Medical Center and third parties.

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.

About Khondrion
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 a privately held pharmaceutical 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. For more information, please visit www.khondrion.com
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.