Khondrion initiaties phase 2 trial

8/9/16Khondrion initiates the KHENERGY study, a phase 2 clinical trial of KH176 in MELAS/MIDD and mixed mitochondrial syndromes

NIJMEGEN, The Netherlands – Khondrion, the clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the initiation of the KHENERGY study, a Phase 2 clinical trial with KH176 in patients harboring the m.3243A>G mutation in the mitochondrial genome. This mitochondrial DNA mutation is associated with Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS syndrome), Mitochondrial Diabetes and Deafness (MIDD syndrome) and mixed phenotypes.

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 oxidative and redox pathologies. 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.

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 and now the initiation of the KHENERGY STUDY in adult patients we have set an important step to accomplish our mission – making a substantial contribution to the development of drugs for patients suffering from mitochondrial (-related) diseases. Based on anticipated learning’s from the KHENERGY study we will be initiating discussions with the EMA to develop pivotal trials to better characterize the potential benefits of KH176 in patients with mitochondrial disease”. Khondrion expects to have enrolled all 20 patients in the fourth quarter of this year.

The KHENERGY STUDY will be a single-center, double-blinded, randomized, placebo-controlled 2-way cross over Phase 2 trial. Patients will receive KH176 orally in a twice a day dosing schedule. The primary efficacy endpoint will be objective, quantitative, clinical relevant gait assessments. The study will also explore changes in other measures of clinical relevance and biomarkers associated with mitochondrial functioning.

Read the full press release or visit the Khondrion website.


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