CNS Pharmaceuticals Doses First Group of Patients with Berubicin, an FDA-Designated Fast Track Drug Candidate, in the Potentially Pivotal Study for the Treatment of Glioblastoma Multiforme
September 30, 2021 at 08:05 am
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CNS Pharmaceuticals, Inc. announced the dosing of the first patients in its Berubicin clinical development program for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer. Further patient enrollment, randomization and dosing is currently underway as well as a robust lineup of clinical sites located globally which are advancing toward activation and enrollment. Berubicin is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier currently being evaluated in a global potentially pivotal study evaluating its efficacy and safety. The potentially pivotal trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. Approximately 243 patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive Berubicin or lomustine for the evaluation of Overall Survival, the primary endpoint of the study. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm. In the Phase 1 clinical trial previously conducted evaluating Berubicin in patients with previously treated disease, 44% of the patients showed clinical benefit (49% of the Avastin-naïve patient population), with the demonstration that it was effective based on this patient population with a dismal median survival rate of only 14.6 months from diagnosis. One patient in the Phase 1 study had a durable Complete Response (CR, a demonstrated lack of detectable cancer cells) that has continued for 14 years, and another patient had a durable partial response, with others showing substantial stabilization of disease. Additionally, the novel anthracycline agent Berubicin appears to have a toxicity profile consistent with that of other anthracyclines and demonstrates activity as monotherapy for recurrent glioblastoma multiforme. Berubicin has side effects that are able to be effectively treated and managed.
CNS Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system (CNS). The Company's lead drug candidate, Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is in development for the treatment of a number of serious brain and CNS oncology indications, including glioblastoma multiforme (GBM), an aggressive and incurable form of brain cancer. In addition, the Company is advancing the development of its WP1244/WP1874 drug technology, which utilizes anthracycline and distamycin-based scaffolds to create small molecule agents. The Company is also evaluating the use of WP1244/WP1874 in the treatment of other primary brain and central nervous system cancers, as well as cancers metastatic to the brain including pancreatic, ovarian, and lymphomas.
CNS Pharmaceuticals Doses First Group of Patients with Berubicin, an FDA-Designated Fast Track Drug Candidate, in the Potentially Pivotal Study for the Treatment of Glioblastoma Multiforme