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LS@W alumnus Khondrion published the results of their phase 2 KHENERGY study on safety and efficacy of KH176 in patients with mitochondrial disease

KHENERGY study provides first data on safety and efficacy of KH176 in patients with mitochondrial disease

Results from phase IIa study of innovative reduction-oxidation modulator published in Clinical Pharmacology and Therapeutics 

NIJMEGEN, the Netherlands – September 2018: Khondrion, the leading clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, today announces that data from its KHENERGY phase IIa study, examining the safety and efficacy of KH176 in patients with mitochondrial disease, have been published in Clinical Pharmacology and Therapeutics.

KH176 is a new innovative cellular redox modulator that, based on extensive investigations in preclinical studies, has the potential to positively change the clinical burden of MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and other mitochondrial diseases.

The study, in 18 patients with an m.3243A>G mutation in their mitochondrial genome, showed that KH176 was well tolerated and appeared safe at the 100mg twice-daily oral dosing regimen. No significant improvements in gait parameters were obtained but positive effects in mood related outcomes and alertness were observed and will be further investigated for clinical significance in future studies, given the prevalence of depressive symptoms among patients with mitochondrial disease.

Commenting on the study results, Prof. Dr. Jan Smeitink, Chief Executive Officer of Khondrion, said: “We are very pleased with the results from KHENERGY which reaffirm the positive profile of KH176 seen in earlier studies. While the study was exploratory in nature, we have seen early indications of the potential impact this medicine could have on the clinical symptoms of mitochondrial disease. Plans are now underway to progress towards pivotal studies to confirm a true treatment effect on mitochondrial disease-related symptoms.”

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.

Patients with an m.3243A>G mutation in their mitochondrial genome show chronically progressive, often early fatal, multisystem disorders for which no clinically beneficial treatment is available.

Cellular consequences like abnormal mitochondrial architecture, reactive oxygen species production and alterations in the cellular redox-state are common findings in mitochondrial diseases. Khondrion’s drug development strategy is based on counteracting these cellular consequences to stop disease progression and to restore normal cellular function.