On December 18, 2023 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 recommendation of the independent Data Safety Monitoring Board (DSMB) that the Company’s ongoing global, potentially pivotal trial of the investigational agent, Berubicin for the treatment of glioblastoma multiforme (GBM) continue without any modification (Press release, CNS Pharmaceuticals, DEC 18, 2023, View Source [SID1234638643]). The recommendation follows the DSMB’s pre-specified futility analysis of unblinded (to the DSMB only) efficacy and safety data in the Company’s trial of Berubicin versus Lomustine, a standard of care in patients with recurrent GBM. More specifically, the DSMB reviewed the primary endpoint of overall survival (OS) and secondary efficacy measures progression-free survival (PFF) and overall response rate (ORR), as well as safety data in evaluable patients. In order to support continuing the trial, Berubicin’s efficacy had to be at least comparable to Lomustine’s on the primary endpoint (OS).

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"Having successfully reached this milestone, we believe that this recommendation reflects Berubicin’s acceptable efficacy and safety profile, as defined in the trial protocol, as of the interim analysis. Building on the foundation of strong enrollment laid by our team, our investigators and their patients, the independent findings of the DSMB add to the Phase 1 trial data, where 44% of treated patients received a clinical benefit of stable disease or better," said John Climaco, CEO of CNS Pharma. "Finding an effective treatment for GBM remains one of the great challenges in oncology, and more meaningful options for the many patients who fail first-line therapy are still desperately needed after decades of research. With this recommendation to continue the study, our long-held belief that Berubicin will ultimately address the unmet clinical need of GBM patients now moves closer to becoming reality."

The potentially pivotal study of Berubicin is a multicenter, open-label, randomized controlled study in adult patients with recurrent GBM (WHO Grade IV) after failure of standard first-line therapy and compared to Lomustine. Overall Survival is a rigorous endpoint that the FDA has recognized as the basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.

For more information about this trial, visit clinicaltrials.gov and reference identifier NCT04762069.

The FDA has granted CNS Pharmaceuticals Fast Track Designation for Berubicin, which enables more frequent interactions with the agency for guidance on expediting the development and review process. Additionally, the Company has received Orphan Drug Designation from the FDA for using Berubicin to treat malignant glioma, which may provide seven years of marketing exclusivity upon approval of a New Drug Application (NDA).

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.