Khondrion announces completion of patient recruitment and first-in-patient dosing in its KHENERGY study, a phase 2 clinical trial with KH176 in MELAS/MIDD and mixed mitochondrial syndromes

NIJMEGEN, The Netherlands – Khondrion, a clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the completion of patient recruitment and first-in-patient dosing in its KHENERGY study. The study is a Phase 2 clinical trial with KH176 in patients harboring the m.3243A>G mutation in the mitochondrial genome.

“We are satisfied that patient recruitment and first-in-patient dosing in our KHENERGY study was completed ahead of our original planning”, said Jan Smeitink, MD, PhD, MAE, and founding CEO of Khondrion. “This is one of the two phase 2 trials we are conducting in patients with mitochondrial disease, and it is designed to assess the safety and efficacy of our lead compound KH176 in adults suffering from mitochondrial disease”.

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 Khondrion drugs essential for the control of oxidative and redox alterations. In December 2015 Khondrion reported that KH176 demonstrated a favorable pharmacokinetic profile and an acceptable safety profile in randomized, placebo-controlled, double blind Phase 1 clinical trials, performed in healthy male volunteers.

The KHENERGY STUDY is a single-center, double-blinded, randomized, placebo-controlled 2-way cross over phase 2 trial involving 20 patients. Patients are required to meet major screening criteria before starting the treatment phase of the trial. The screening period from recruitment to initiating treatment is approximately 4 weeks. The phase 2 study is supervised by dr. Mirian Janssen (MD, PhD), Department of Internal Medicine-Metabolic Diseases of the Radboud Center for Mitochondrial Medicine at the Radboudumc, Nijmegen, The Netherlands.

Patients will receive KH176 in a 100 mg twice-daily oral dosing schedule. The primary efficacy endpoint will be objective, quantitative, and clinically relevant gait assessments. The study will also explore changes in other measures of clinical relevance and biomarkers associated with mitochondrial functioning.

Based on anticipated learning’s from the KHENERGY study the company will ask protocol assistance from regulatory authorities to develop a multi-center pivotal trial to better characterize the potential benefits of KH176 in patients with mitochondrial disease.

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 a leading 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 expert centers and 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

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.

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