Notable Labs reports PPMP predicted fosciclopirox trial outcome

Notable Labs and CicloMed announced initial safety and efficacy data of fosciclopirox and the performance of PPMP, Notable’s Predictive Precision Medicines Platform, from the Phase 2a trial in adult patients with relapsed/refractory acute myeloid leukemia, R/R AML. Fosciclopirox is sponsored by CicloMed and co-developed by both companies. In the Phase 2a trial, PPMP-predicted patient responses were confirmed by the actual responses to treatment. Fosciclopirox, a patented, parentally administered prodrug of a commercially available topical antifungal agent, ciclopirox, is being developed for the treatment of bladder cancer and AML. This Phase 2a study was designed to characterize the safety and efficacy of fosciclopirox in R/R AML and, in parallel, to test PPMP’s ability to predict patient responses to treatment. Patients were not selected for enrollment based on PPMP results and, to avoid potential bias, PPMP analysis was blinded to the actual patient outcomes during the trial. Clinical efficacy in this heavily pretreated group of patients was defined by standard criteria in AML medical research and practice. Eighteen heavily pre-treated patients were enrolled in the trial with nine patients evaluable for response assessment per-protocol. Fosciclopirox, administered at the Recommended Phase 2 Dose, was well-tolerated. However, none of the nine evaluable patients achieved a complete response. Stable disease, over four months, was observed in two evaluable patients. Importantly, PPMP predicted that all patients enrolled into this trial would be non-responsive to fosciclopirox. The accuracy of this prediction was confirmed by the actual patient responses to treatment. These PPMP results indicate that the enrolled patient population was biased towards non-responsiveness to fosciclopirox and suggest that the negative clinical outcome of this Phase 2a trial may have been avoided if PPMP had initially been used to selectively enroll only predicted responders. Further analyses are ongoing.