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Among the 18 patients enrolled at the MTD, there were 3 (16.6%) patients with a complete response (CR), 9 with a partial response (PR), 2 with stable disease and 4 with progressive disease. The PR + CR rate was 67% with a 95% Clopper-Pearson (exact) confidence interval of 41% to 87%.

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In all, 18 patients were treated at this CPI-613 dose without dose-limiting toxicity, although grade 3–4 adverse events were common, report Angela Alistar, from Wake Forest University School of Medicine in Winston-Salem, North Carolina, USA, and co-authors.

These included hyperglycaemia (55%), hypokalaemia (33%), diarrhoea (28%), peripheral sensory neuropathy (28%), lymphopenia (28%), abdominal pain (22%) and anaemia (22%).

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