Khondrion gears up for pivotal mitochondrial disease trial after taking an early look at midphase data

Final data from the one-month phase 2 trial are due in the first quarter of next year.

Khondrion is preparing to advance mitochondrial disease program KH176 into phase 3. The vote of confidence in the drug follows an early look at phase 2 data on the intracellular redox-modulating agent.

Nijmegen, the Netherlands-based Khondrion is testing KH176 against placebo in 20 patients with mitochondrial DNA tRNALeu(UUR) m.3243A>G mutations. Final data from the one-month phase 2 trial are due in the first quarter of next year. But CEO Jan Smeitink shared a look at the findings to date at the Dutch Life Sciences Conference.

Khondrion is assessing KH176 in terms of its effect on motor abilities and movement—the primary endpoint—and a long list of secondary objectives. At this early stage, the results are a mixed bag. 


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KH176 chalked up a statistically significant improvement against a depression score—a secondary endpoint—and positive trends against other secondary clinical outcomes. 

In terms of functional outcomes, Khondrion saw positive trends against two aspects of alertness. However, the trial is yet to pick up a positive signal on all the other functional outcomes. The preliminary nature of the data means there is time for that to change. 

Other aspects of the data have offered encouragement to the company.  

“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 predefined safety threshold obtained in phase 1 evaluations," Khondrion CMO Edwin Spaans said in a statement.

Taken together, the data were strong enough for Khondrion to continue preparing for a phase 3 trial of KH176 and take other steps to support its ongoing development. Attention now turns to next year’s readout.

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