On September 30, 2021 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported 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 (Press release, CNS Pharmaceuticals, SEP 30, 2021, View Source [SID1234590605]). 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.
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"As one of the most aggressive, deadly and treatment-resistant cancers that forms in the brain where current standard of care remains ineffective in ~60% of patients, the treatment landscape for GBM is in desperate need of a better treatment option. We continue to be encouraged by the data seen from Berubicin to date and are excited to now have patient dosing underway in this potentially pivotal trial. Our team is committed to expeditiously advancing the study and look forward to further understanding the full potential of Berubicin to offer improvement and overall survival in this devastating disease," stated Erin Dunbar, M.D., founding physician of the Brain Tumor Center and Director of Neuro-Oncology at Piedmont Atlanta Hospital, and Principal Investigator for the study.
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.
"I am extremely pleased with the progress made to-date in this potentially pivotal trial. Our team has been working intensely to open sites in the U.S. and in Europe, understanding where we can best advance this important study. With hundreds of potentially competing GBM trials currently enrolling patients, the fact that we’ve been able to bring these initial sites on-line and get patients enrolled and dosed not only supports our strategic evaluation and selection, but also allows our data demonstrating Berubicin’s potential effectiveness to continue to convince the medical community that we have a new drug with impressive potential. With the de-risked profile of Berubicin, its mechanism of action, history of development, encouraging Phase 1 data, and safety in study design, I am personally more optimistic about our work than at any time since joining the Company," commented John Climaco, CEO of CNS Pharmaceuticals.
"The investigator dedication to advance the science and development of Berubicin we’re seeing here is meaningful to these patients that are out of options. Berubicin provides an innovative option for treatment in GBM as a safe and potentially effective therapy. We look forward to providing data at the interim analysis of the study," added Sandra L. Silberman, M.D., Ph.D. Chief Medical Officer of CNS Pharmaceuticals.
A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.
The FDA recently granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA. Taken together the Company believes these important designations can be seen as a recognition of the significance of not only the unmet clinical need in GBM, but of our Berubicin program.
For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.
About Berubicin
Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc. Berubicin, was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.