Khondrion clinical trial on target

Smeitink Jan

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

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. Khondrion expects to report the topline results of the study in the middle of 2017, as previously planned.


“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, theme Mitochondrial diseases, 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 study should encounter its full complement of treated patients to meet the previously stated goal of topline data readout in the middle of 2017. The phase 2 study is supervised by Mirian Janssen, Dept of Internal Medicine - Metabolic Diseases.

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.


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