CNS Pharmaceuticals, Inc. announced that it has received approval from the U.S. Food and Drug Administration (FDA) for its ongoing potentially pivotal global study evaluating the efficacy and safety of Berubicin compared with Lomustine (Gleostine) administered after first line therapy for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer. 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.

The primary endpoint of the study is Overall Survival (OS), which is a rigorous endpoint that the 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. Results from the trial will compare Berubicin to a current standard of care (Lomustine), with a 2 to 1 randomization of patients to receive either Berubicin or Lomustine. The amended protocol expands eligibility for the study to patients who have received additional treatments as part of the first line therapy for their disease considering advancements in this area.

This change was made due to the complexity of new agents introduced as a component of first line therapy, which allows an additional group of patients that can enroll on the study after what may constitute multiple procedures as their initial treatment. A pre-planned, non-binding futility analysis will be performed after 30 to 50% of all planned patients have completed 6 months on therapy. This evaluation will include safety as well as secondary efficacy endpoints.

Enrollment will not be paused during this interim analysis. The FDA has granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with them to provide guidance on expediting the development and review process. Additionally, the Company has also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA.