On December 18, 2023 Notable Labs, Ltd. ("Notable") (Nasdaq: NTBL), a clinical-stage therapeutic platform company developing predictive precision medicines for cancer patients, and CicloMed, LLC ("CicloMed"), a developmental-stage pharmaceutical company focusing on unmet medical needs in oncology, reported 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 (NCT04956042) (Press release, Notable Labs, DEC 18, 2023, View Source [SID1234638654]). 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.
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"The successful outcome prediction means that Notable’s PPMP platform accomplished its goal in this trial," stated Thomas Bock, M.D., Chief Executive Officer of Notable. "These data further corroborate and extend our four independent PPMP validation trials in collaboration with leading medical centers*."
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 Tammy Ham, Chief Executive Officer of CicloMed. "But based on the PPMP results, we will assess the merits of conducting a prospective trial in which we use PPMP to identify and selectively enroll patients predicted to respond to fosciclopirox treatment."
"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 Joseph Wagner, PhD, Chief Scientific Officer of Notable. "This additional validation gives us further confidence that PPMP will play a significant role in drug development and suggests PPMP should be advanced to the front-end of clinical development programs to maximize success and avoid from the start, the risk and cost of enrolling treatment non-responsive patients."