Key points from article :
Khondrion, a clinical-stage biopharmaceutical company focused on mitochondrial diseases, has encountered a setback in its phase 2b clinical trial for its lead drug candidate, sonlicromanol. While the trial did not meet its primary endpoint, the company remains optimistic about the drug's potential and plans to move forward with a phase 3 study.
Sonlicromanol is designed to target the underlying mechanisms of mitochondrial disease by addressing oxidative stress and mitochondrial dysfunction. The phase 2b trial aimed to assess the efficacy of sonlicromanol in improving cognitive function in patients with a specific mitochondrial DNA mutation.
In phase 2 clinical trial, 27 people who had a specific confirmed mitochondrial DNA mutation and with clinical signs of mitochondrial disease received one of two doses of sonlicromanol or placebo orally twice a day. After 28 days of treatment, participants in the sonlicromanol arm showed no significant improvement in their attention domain scores compared to the placebo group. This outcome follows an earlier phase 2 trial that also failed to demonstrate significant improvements in gait parameters. Given these results, Khondrion has shifted its focus to other aspects of mitochondrial disease in its ongoing research.
Although the primary endpoint was not met, Khondrion highlighted positive trends in post-hoc analyses of other cognitive and mood-related endpoints. Additionally, the company pointed to encouraging data from a 52-week open-label follow-up study, which showed potential improvements in mitochondrial disease symptoms.
Despite the mixed results, Khondrion remains confident in the future of sonlicromanol. The company plans to engage with regulatory authorities to initiate a phase 3 clinical trial in late 2025. This decision is based on the belief that the totality of the data, including the post-hoc analyses and long-term follow-up data, supports further development of sonlicromanol.
While the scientific community awaits further data, it remains to be seen whether sonlicromanol will ultimately prove to be a viable treatment for mitochondrial diseases.
