NIJMEGEN, The Netherlands, November 16, 2017 /PRNewswire/ --
Khondrion, a clinical-stage pharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today that its CEO Prof. Dr. Jan Smeitink will present first results of Khondrion's KHENERGY study at the Dutch Life Sciences Conference in Oss, The Netherlands, on November 22 at 10 am.
The KHENERGY study is a single-center, double-blinded, randomized, placebo-controlled, explorative 2-way cross over Phase 2 trial involving 20 patients with MELAS syndrome, MIDD syndrome or mixed phenotypes harboring the m.3243A>G mutation in the mitochondrial genome. The study was supervised by Dr. Mirian Janssen (MD, PhD), of the Radboud Center for Mitochondrial Medicine at the Radboudumc, Nijmegen, The Netherlands.
Patients received KH176 in a 100 mg twice-daily oral dosing schedule. The efficacy endpoints were objective, quantitative, and clinically relevant assessments. The study also explored changes in other measures of clinical relevance and biomarkers associated with mitochondrial functioning.
KH176 is an orally bio-available small molecule in development by Khondrion for the treatment of mitochondrial (-related) diseases. The compound is a member of a new class of Khondrion candidate drugs essential for the control of oxidative and redox alterations. Khondrion reported earlier that KH176 demonstrated favorable pharmacokinetics and a favorable safety profile in randomized, placebo-controlled, double blind Phase 1 clinical trials, performed in healthy male volunteers. Results of these studies were recently published in the Orphanet Journal of Rare Diseases.
Khondrion is a privately held leading clinical-stage pharmaceutical company focusing on developing small molecule therapeutics for mitochondrial (-related) diseases. 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. Khondrion has a strong intellectual property position protecting its emerging product portfolio via granted and multiple, broad patent applications. For more information, please visit http://www.khondrion.com.