APO866, an injectable molecule that induces cell death by inhibiting the biosynthesis of NAD+ (oxidized nicotinamide adenine dinucleotide), failed to demonstrate efficacy in the treatment of patients with relapsed/refractory cutaneous T-cell lymphoma, according to a research letter published in JAMA Dermatology.1

Because previous studies have shown in vitro and in in vivo that lymphocytes and hematologic cancer cells are very sensitive to APO866, researchers sought to evaluate the agent in a phase 2 clinical trial.

For the multicenter, open-label, single-arm study, researchers enrolled 14 patients with relapsed/refractory cutaneous T-cell lymphoma. Patients were to receive APO866 at a dose of 0.126 mg/m2/hour over 96 hours via continuous IV infusion every 28 days for 3 cycles; however, only 5 patients completed the 3 cycles.

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Results showed that at week 16, 1 of 12 evaluable patients achieved a partial response, 6 had stable disease, and 5 had progressive disease. No patient achieved a complete response.

In regard to safety, a total of 18 serious adverse events were reported, of which 7 were related to APO866. These included pyrexia, lymphopenia, spondylitis, staphylococcal sepsis, rhabdomyolysis, and thrombocytopenia. Eighty-six percent of the 14 patients experienced mild to moderate adverse events.

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Ultimately, although APO866 demonstrated a reasonable safety profile in cutaneous T-cell lymphoma, it was not powerful enough in this treatment setting. Therefore, researchers see no role for APO866 in cutaneous T-cell lymphoma but potentially for other conditions.


  1. Goldinger SM, Bischof SG, Fink-Puches R, et al. Efficacy and safety of APO866 in patients with refractory or relapsed cutaneous T-cell lymphoma: a phase 2 clinical trial [published online ahead of print March 23, 2016]. JAMA Dermatol. doi: 10.1001/jamadermatol.2016.0401.