APO866 Not Effective for Cutaneous T-Cell Lymphoma

The injectable drug APO866, designed to induce apoptosis, did not have efficacy against cutaneous T-cell lymphoma, according to the results of small study published as a Research Letter in JAMA Dermatology.

“In the present study, APO866 showed a reasonable toxic effect in [cutaneous T-cell lymphoma],” wrote researcher Simone M. Goldinger, MD, from the department of dermatology at University Hospital of Zurich, Switzerland, and colleagues. “However, the drug was not powerful enough, and the study was stopped early after the prespecified interim analysis owing to lack of drug efficacy.”

According to the study, APO866 works by inhibiting NAD (oxidized nicotinamide adenine dinucleotide), thereby inducing cell death. Previous studies of the drug had shown that lymphocytes were very sensitive to APO866.

This study included 14 patients with confirmed relapsed or refractory cutaneous T-cell lymphoma. Patients were given APO866 every 28 days for 3 cycles. The drug was administered through continuous infusion via pump at 0.126 mg/m² per hour for 96 hours. The primary endpoint was the proportion of patients who responded at 16 weeks.

No patient in the trial achieved a complete response and only one achieved a partial response. Six of the 12 patients had stable disease. A per protocol analysis included only the five patients who completed all 3 cycles of the drug. In the per-protocol patients, one patient had a partial response and four of five had stable disease.

Among patients who had to withdraw, two withdrew because of adverse events. Eighty-six percent of patients had mild to moderate adverse events. Eighteen serious adverse events occurred but only seven were related to the drug.

Based on these results, the researchers concluded that there was no reason to continue studying this drug in cutaneous T-cell lymphoma.

“However, owing to its mode of action with immunosuppression and insulin-mimicking effects, APO866 might play a role in the treatment of other conditions,” they wrote.

This study was sponsored by Apoxis SA and later by TopoTarget A/S.