CNS Pharmaceuticals, Inc. announced completion of planned enrollment in its global potentially pivotal study evaluating Berubicin, the Company's novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier, for the treatment of GBM, an aggressive and incurable form of brain cancer. 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. The study has enrolled 247 patients across 46 clinical trial sites in the U.S., Italy, France, Spain, and Switzerland.

The primary endpoint of the study is Overall Survival (OS), a rigorous endpoint 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. As announced on December 18, 2023, the interim analysis of efficacy and safety data in the potentially pivotal study of BerubICin was successfully completed. The recommendation of the independent Data Safety Monitoring Board (DSMB) was that the Company's ongoing global, potentially pivotal trial of the investigational agent Berubicin for the treatment of GBM continue without any modification.

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. In order to support continuing the trial, Berubicin's efficacy had to be at least comparable to Lomustine's on the primary endpoint (OS). 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).