A phase 2 double-blinded, placebo-controlled trial of KPAX002 as a treatment for ME/CFS (n=128)  

This phase 2 double-blinded, placebo-controlled trial adds to the findings of a previously published phase 1 trial exploring the safety and efficacy of KPAX002 as a potential treatment for ME/CFS. The results of all planned efficacy analyses showed a trend in favor of the KPAX002 treatment group when compared to placebo. The treatment and placebo groups showed no significant difference with regard to rates of treatment-emergent adverse events (Grade 2 or greater), which is notable since side effects such as anxiety, palpitations, dizziness, and insomnia are frequently reported with methylphenidate administration.

During 12 weeks of treatment, KPAX002 treated subjects demonstrated improvement in multiple outcome measures, including the CIS total score and the VAS for fatigue and concentration disturbances. Two key subgroups with the greatest treatment response were identified: 1) subjects with more severe ME/CFS symptoms at baseline and 2) subjects with both fatigue and pain. It is reasonable to assume that sicker (more symptomatic) ME/CFS subjects are more likely to have significant mitochondrial pathology and would therefore demonstrate an enhanced response to a mitochondrial support treatment.

- 36% of the patients taking the KPAX002 treatment had a >20% reduction in overall ME/CFS symptoms after 28 days.

- More symptomatic patients at baseline experienced double the improvement as patients beginning the trial with less severe symptoms.

- Treatment with KPAX002 was demonstrated to be safe and well tolerated in patients with ME/CFS.