- PPMP Accurately Predicted Patient Outcomes in Fosciclopirox Phase 2a Leukemia Trial -
- Predictive Results Further Advance PPMP and Corroborate Outcomes from 4 PPMP Validation Trials -
- Furthers Opportunity to Accelerate PPMP Use in Front-End Drug Development -
“The successful outcome prediction means that Notable’s PPMP platform accomplished its goal in this trial,” stated
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.
“Obviously, we would have preferred observing therapeutic responses in this Phase 2a trial in heavily pretreated AML patients,” shared
“Once our PPMP team merged the blinded PPMP and clinical data, we observed that the patients’ PPMP-predicted non-responsiveness to fosciclopirox was accurate and confirmed by the actual patient responses,” said
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* Blood Adv, 2020 Jun 23;4(12):2768-2778; ASH Poster (2021) https://notablelabs.com/images/notable/Publications/ASH-MDA-Notable.pdf); Cancers (
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