The first patient has been dosed in the pivotal Phase 3 KHENERFIN study evaluating sonlicromanol for the treatment of m.3243A>G primary mitochondrial disease. This milestone marks an important step in Khondrion’s lead program as it advances into the registrational phase of development.

Khondrion is a clinical-stage biopharmaceutical company dedicated to developing therapies for patients with primary mitochondrial diseases, conditions associated with significant unmet medical need. Building on proprietary science and deep expertise in mitochondrial biology, the company is advancing sonlicromanol with the aim of addressing the underlying mechanisms of mitochondrial dysfunction.

The KHENERFIN study is a randomized, placebo-controlled Phase 3 clinical trial in the EU, UK and US, designed to assess the efficacy and safety of sonlicromanol in adult patients affected by the most common genetic form of primary mitochondrial disease (the m.3243A>G mutation). The initiation of patient dosing is a significant achievement for Khondrion and a hopeful advance for the mitochondrial disease community, as sonlicromanol progresses into late-stage clinical testing for a disorder with no approved disease-modifying treatments.

3D‑PharmXchange is proud to support Khondrion as a strategic partner in this Phase 3 journey and beyond, contributing CMC and QA expertise that underpins the readiness of the program for late‑stage clinical execution. We congratulate the Khondrion team on reaching this milestone and are pleased to continue our collaboration as the KHENERFIN trial moves forward.

Read the full announcement from Khondrion at this link.