KH176 by Khondrion had a favorable safety profile and improved depression and migraine in some forms of mitochondrial disease, according to preliminary results from an ongoing Phase 2 trial.
The study included carriers of a gene defect called m.3243A>G who have MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) and MIDD (maternally-inherited diabetes and deafness) syndromes. The final results are expected in the first quarter of 2018.
Preliminary results were presented at the Dutch Life Sciences Conference by Jan Smeitink, Khondrion’s CEO.
The KHENERGY study (NCT02909400) is a Phase 2, placebo-controlled trial involving 20 patients. They were treated with KH176 for one month. Endpoints included objective assessments of disease severity and questionnaires that evaluated the mood and quality of life of patients. Potential biomarkers as indicators of mitochondrial functioning were also studied.
The investigation was supervised by Mirian Janssen, MD, PhD, of the Radboud Center for Mitochondrial Medicine at the Radboudumc University in Nijmegen, The Netherlands.
KH176 was well-tolerated by the participants in the trial. Improvements and positive trends were seen in depression according to three different questionnaires. Self-reported improvements were seen in migraines in three out of three affected patients.
KH176 is a member of a new class of potential Khondrion drugs essential for the control of oxidative and redox alterations. The Phase I trial (NCT02544217) showed that KH176 was well-tolerated in healthy men. The results of this study were recently published in the article titled, “KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers,” in the Orphanet Journal of Rare Diseases.
“Given the relatively short duration of this study, these findings are encouraging,” Janssen said.
Khondrion‘s KH176 has been granted orphan drug designation for Leigh disease and MELAS syndrome in Europe and for all inherited mitochondrial respiratory chain disorders in the U.S.