Volasertib is a potent Polo-like kinase 1 (PLK-1) inhibitor. PLK-1 is a key regulator of the cell cycle, is over-expressed in many cancers, and has been associated with poor survival. Volasertib has been studied in numerous clinical trials alone and in combination with other approved therapies in a number of cancers including patients with acute myeloid leukemia, AML, who have been ineligible for aggressive front-line treatments and bone marrow transplant.
In randomized clinical trials, volasertib demonstrated meaningful clinical responses in a subset of studied patients in both a phase 2 trial and a phase 3, with a response rate of 31% and 25% respectively. Unfortunately, there was another subset of patients that did not benefit from volasertib but still experienced toxicity. Although the phase 2 trial reached statistical significance, without a predictive biomarker to identify and select the patients who benefitted, the phase 3 trial failed to show statistically significant results.
In an upcoming and redesigned phase 2 trial, Notable will leverage its high-fidelity Predictive Precision Medicines Platform to identify and select volasertib-responsive patients prior to their treatment, enroll precisely the predicted clinical responders, and fast-track volasertib’s clinical development in this patient population.
Global development and commercialization rights to volasertib in adult disease indications were acquired by Notable from Oncoheroes Biosciences in 2021. Volasertib received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the United States Food and Drug Administration (FDA) in 2020 for use in treating pediatric rhabdomyosarcoma.
Fosciclopirox (CPX-POM), a pro-drug of ciclopirox, was invented by scientists at The University of Kansas Cancer Center, a National Cancer Institute designated cancer center and the Institute for Advancing Medical Innovation (IAMI), University of Kansas Medical Center’s product development enterprise. Notable and CicloMed have initiated a Phase 1B/2A clinical trial of fosciclopirox in patients with refractory acute myelogenous leukemia (AML) under the terms of a co-development agreement. In the ongoing open-label, Phase 1B/2A trial currently underway at The University of Kansas Cancer Center, Notable is applying its high-fidelity Predictive Precision Medicines Platform to assess patient sensitivity to fosciclopirox.
CicloMed holds the primary responsibility for executing clinical trial operations while Notable is primarily focused on optimizing Notable’s Predictive Precision Medicine Platform. Both parties will be responsible for research and commercialization costs associated with an AML clinical development program.