FDA Approves IND Application for Brain Cancer Drug Candidate

CNS Pharmaceuticals announced the Investigational New Drug (IND) application for its lead product candidate, Berubicin, for the treatment of Glioblastoma Multiforme (GBM) is now approved and in effect as filed with the US Food and Drug Administration (FDA). The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy. Recent correspondence between the Company and the FDA resulted in modifications to the previously disclosed trial design, including designating overall survival (OS) as the primary endpoint of the study. OS 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.

"Since becoming a public company, our clear focus has been on advancing the clinical development of Berubicin. We will now rapidly move to initiate our Phase 2 trial of Berubicin for adults with GBM and expect to begin enrolling patients in the first quarter of next year," said John Climaco, CEO of CNS Pharmaceuticals. "The Company will transform within the next several months as Berubicin becomes the subject of up to three active clinical trials, which include our randomized, controlled Phase 2 trial in the U.S., and 2 trials planned by our sublicensee WPD in Poland. We are entering an area with significant unmet medical need since the current treatment paradigm for GBM remains bleak, as this aggressive and currently incurable form of brain cancer continues to claim high mortality rates. We have a tremendous opportunity ahead of us as we continue our mission to improve patient outcomes for GBM and build on the promising results demonstrated by Berubicin in its Phase 1 clinical trial."

As previously announced, the planned trial will evaluate the efficacy of Berubicin in patients with GBM who have failed primary treatment for their disease. Results will compare Berubicin to the current standard of care, with a 2 to 1 randomization of patients to either receive Berubicin or Lomustine. The trial's adaptive design is intended to allow an interim analysis of the data to demonstrate meaningful differences in efficacy between treatments and then to allow an adjustment to the size of the patient population in the trial for maximum efficiency in terms of time in development. Based on this, the trial has the potential to provide data to the FDA that may allow an expedited pathway for development. However, there can be no assurance that the FDA will support any potential request for an expedited pathway to approval or further development.