Khondrion Presents Phase II KHENERGY Trial Data

- Data supporting Phase III development of KH176 in mitochondrial disease Khondrion, a leading clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, today announced results from its KHENERGY study, a Phase II exploratory trial with oral KH176 in the m.3243A>G multisystem mitochondrial MELAS and MIDD syndromes and mixed phenotypes. The results of the trial were presented by Prof. Jan Smeitink, Khondrion's CEO, at the Dutch Life Sciences Conference. "The final reporting of the KHENERGY study is planned for Q1 2018, but encouraged by the results, we wanted to share these preliminary data regarding safety and efficacy now," said Jan Smeitink. The KHENERGY study is a Phase II, single-center, double blind, randomized, placebo-controlled 2-way crossover trial involving 20 patients. Patients received KH176 in a 100 mg twice-daily oral dosing schedule for one month. Efficacy endpoints included objective, quantitative assessments as well as questionnaires evaluating the mood and quality of life of patients. The study also explored biomarkers associated with 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. Of the functional outcomes measures, two aspects of alertness showed positive trends. All others, did not show a positive signal in the four weeks treatment arm. With regard to clinical outcomes, statistically significant improvements were observed 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 an amelioration of migraine in three out of three affected subjects. "Given the relatively short duration of this study, these findings are encouraging," according to dr. Mirian Janssen (MD, PhD), Principal Investigator. "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 program, including all Phase III preparations," said Jan Smeitink. About KH176  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 results of this study were recently published in the Orphanet Journal of Rare Diseases. About Khondrion  Khondrion is a privately held leading clinical-stage pharmaceutical company. The potential of several lead compounds to serve as new treatment modalities for mitochondrial disease is currently being explored. Khondrion's KH176 has been granted Orphan Drug Designation (ODD) for Leigh disease and MELAS syndrome in Europe and for all inherited mitochondrial respiratory chain disorders in the USA. Khondrion has established collaborations with patient organizations, patient advocacy groups, university expert centers and research groups around the world as well as with small, medium and large enterprises. The company is supported by the Dutch Foundations Energy4All, Join4Energy, Road4Energy, Ride4Kids, Tim Foundation, Zeldzame Ziekten Fonds, and National and European Governments. Khondrion has a strong intellectual property position protecting its emerging product portfolio via granted and multiple, broad patent applications. Based on the outcome of the KHENERGY study the company continues preparing for a pivotal program to confirm the potential benefits of KH176 in patients with mitochondrial disease. For more information and a cautionary note regarding forward-looking statement concerning this press release, please visit http://www.khondrion.com