Khondrion Reports Positive Preliminary Data on Mid-Stage Trial in MELAS and MIDD Syndromes

Rare Daily Staff

Khondrion presented promising results from a mid-stage exploratory study of KH176, its experimental therapy to treat MELAS and MIDD syndromes, two rare genetic mitochondrial disorders.

MELAS is an acronym for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes, a condition that is multiple systems within the body including the brain, nervous system and muscles. MIDD is an acronym for maternally inherited diabetes-deafness syndrome. Both conditions are caused by genetic mutations that affect mitochondria, which are known as the powerhouse of cells.

Jan Smeitink, Khondrion’s CEO, presented the preliminary data from the study at the Dutch Life Sciences Conference. The final report from the study is expected during the first quarter of 2018.

The study is a single-center, double blind, randomized, placebo-controlled two-way crossover trial involving 20 patients. Patients received KH176 in a 100 mg, twice-daily oral dosing schedule for one month. Efficacy endpoints included quantitative assessments, as well as questionnaires evaluating the mood and quality of life of patients. The study also explored biomarkers associated with mitochondrial functioning.

Two outcome measures of alertness showed positive trends. All other aspects failed to show a statistically significant improvement. The study showed statistically significant improvements in the total Beck Depression Inventory score and its affective sub-domain. Positive trends were observed in the HADS depression subsection and the RAND-36 SF affective symptoms. Self-reported outcomes revealed a relieve of migraine in three of three affected subjects.

The preliminary findings of this study showed a promising safety profile and the pharmacokinetic analysis of KH176 showed that the candidate drug’s maximum blood concentrations remained below the pre-defined safety threshold obtained in evaluations from an earlier study.

Smeitink said based on the outcome of the study the company will immediately continue with all necessary steps enabling the next phases of its KH176 development program, including preparations for a late-stage clinical trial.

November 22, 2017

Photo: Jan Smeitink, CEO of Khondrion