Rare Disease Report

KH176 Shows Potential in Phase 2 KHENERGY Study

NOVEMBER 22, 2017
Mathew Shanley
New results from Khondrion’s Phase 2 KHENERGY study were presented at the Dutch Life Sciences Conference in the Netherlands today.

While the final reporting of the study isn’t scheduled to be released until the first quarter of 2018, the results were so encouraging that Khondrion decided to share the preliminary data regarding efficacy and safety in front of an interested group.

Data from the exploratory trial of KH176 in the m.3243A>G multisystem mitochondrial encephalopathy lactic acidosis (MELAS) and maternally inherited diabetes-deafness (MIDD) syndromes, and mixed phenotypes were presented by Professor Jan Smeitink, the company’s CEO.

Both conditions are caused by genetic mutations in the mitochondria. MELAS affects several of the body’s systems, particularly the brain and nervous systems, as well as the muscles. It can result in a buildup of lactic acid, called lactic acidosis, and the increased acidity in the blood can cause symptoms like vomiting, abdominal pain, fatigue, muscle weakness, and difficulty breathing. MIDD is a form of diabetes caused by a shortage of insulin and characterized by hyperglycemia, or high blood sugar levels.

KH176 is essential for the control of oxidative and redox alterations.

The study is a single-center, double-blind, randomized, placebo-controlled 2-way crossover trial that enrolled 20 patients, each of whom receive KH176 in a 100 mg twice-daily oral dosing schedule for one month. The study had efficacy endpoints including objective, quantitative assessments and questionnaires evaluating the mood and patients’ quality of life (QOL).

Additionally, the study investigated biomarkers connected to mitochondrial functioning.

"The preliminary findings of this study related to adverse events showed a promising safety profile. Also, the pharmacokinetic analysis of KH176 showed that the candidate drug's maximum blood concentrations remained below the pre-defined safety threshold obtained in Phase I evaluations," said Dr. Edwin Spaans, Khondrion's Chief Medical Officer in a press release.

While the duration of the study was short, the findings show potential. Two aspects in the 4-weeks treatment arm exhibited positive trends and statistically significant improvements were observed in the total Beck Depression Inventory score and its affective sub-domain. Positive trends were also seen in the HADS depression subsection and the RAND-36 SF affective symptoms.

Results of the Phase 1 trial of this study were recently published in the Orphanet Journal of Rare Diseases, and based on the new data, Khondrion intends to continue with the KH176 development program, including the steps necessary to prepare for a Phase 3.

For more information on potential breakthrough therapies in the rare disease community, follow Rare Disease Report on Facebook and Twitter.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.