Netherlands-based pharmaceutical company Khondrion has reported favourable data from preliminary analysis of the Phase II KHENERGY clinical trial evaluating KH176 for mitochondrial disorders, MELAS, and MIDD syndromes and mixed phenotypes.
KH176 is an orally bioavailable small molecule therapeutic with orphan drug designation in Europe to treat Leigh disease and MELAS syndrome, and in the US for all inherited mitochondrial respiratory chain disorders.
The preliminary data is reported to have indicated a promising safety profile, while the pharmacokinetic analysis revealed KH176’s maximum blood concentrations lower than the pre-defined safety threshold determined in Phase I trial.
Furthermore, the firm reported statistically significant improvement in the total Beck Depression Inventory score and affective sub-domain.
Khondrion CEO Jan Smeitink said: "Based on the outcome of the Phase II study, we have decided to immediately continue with all necessary steps, enabling the next phases of our KH176 development programme, including all Phase III preparations."
The single-centre, double-blind, randomised, placebo-controlled, two-way crossover KHENERGY trial evaluated oral, twice-daily 100mg dose of KH176 over one month in 20 subjects.
Efficacy endpoints of the trial were objective, quantitative assessments, and questionnaires to assess the mood and quality of life of the participants.
Results from the Phase II trial also revealed positive trends in two aspects of alertness, HADS depression subsection and the RAND-36 SF affective symptoms.
The firm expects to complete final reporting from the trial in the first quarter of 2018.