The Randomized Controlled Phase 2b KHENERGYZE Trial of Sonlicromanol in Primary Mitochondrial Disease MELAS Spectrum Disorders: Topline Results

 

Dr Jan SmeitinkDr. Jan Smeitink, from Khondrion B.V., Netherlands, will join us this year to present his most recent findings on "The randomized controlled phase 2b KHENERGYZE trial of sonlicromanol in primary mitochondrial disease MELAS spectrum disorders: topline results" 

MELAS spectrum disorders caused by the 3243A>G mutation in the MT-TL1 gene lead to hampered oxidative phosphorylation with, as immediate cellular consequences, both reductive and oxidative stress.The clinical spectrum is heterogeneous with classical mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) and maternally inherited diabetes mellitus and deafness (MIDD) as most frequently encountered phenotypes. Sonlicromanol modulates key metabolic and inflammatory pathways in the pathogenesis of MELAS-SD models.

Here, Dr. Smeitnik reports the results of a 3-way cross over RCT which, together with the already completed phase 1 and phase 2a trials and the ongoing phase 2b open label extension study, will guide the design of the pivotal KHENERFIN trial. 

Targeting Mitochondria 2022 Congress
October 26-28, 2022 - Berlin, Germany 
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