Updated results from on-going potentially pivotal study of Berubicin in adult patients with recurrent glioblastoma multiforme, after failure of standard first line therapy

"Berubicin has demonstrated its capability to be an innovative treatment in GBM that is safe and well tolerated, which has the potential to be a novel and effective therapy for this disease. Based on the results we've seen preclinically and in the clinic thus far, including these updated results from our on-going potentially pivotal study, we remain optimistic that Berubicin may provide a much needed clinical benefit for GBM patients. We look forward to upcoming interim results that will evaluate Berubicin compared to the current standard of care (Lomustine) for second line therapy in this disease," commented Dr. Silberman.

As of the data cutoff for the poster, 151 patients had been enrolled, 105 on Berubicin and 46 on Lomustine. Enrolled patients received 7.5 mg/m2/day of intravenous (IV) Berubicin over 2 hours for 3 consecutive days (one cycle) every 21 days. Previous results from this study were presented in November 2022 at the Society for Neuro-Oncology (SNO) 27th Annual Meeting.

Summary of Updated Results from the Potentially Pivotal Study: i. All patients enrolled show comparable demographics for each arm, including age, gender, race, BSA, and KPS. In addition, patients have been stratified by MGMT methylation, allowing a reasonable comparison of efficacy based on this prognostic feature between the two groups. The unmethylated MGMT population comprises approximately 38% of all enrolled patients. ii. Approximately 50% of patients on both arms have completed the study, i.e. have participated in an end of treatment (EOT) visit, and withdrawal prior to EOT is relatively small, although almost twice the percent of patients in the Lomustine group compared to Berubicin have withdrawn. Primary reasons for withdrawal across both arms include Adverse Events, Physician Decision, Withdrawal by Patient, and Death as well as Other nonspecified reasons. iii. All grades of adverse events occurring in more than 10% of patients, as well as Grade 3-5 events, are presented in the poster, with the overall percentages similar in the Berubicin and Lomustine arms. In terms of myelosuppression (complete blood counts), neutrophil, and red blood cell count [anemia] reductions are also similar however thrombocytopenia seems to be higher in the Lomustine arm.

The primary endpoint of the study is Overall Survival (OS), 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.

CNS Pharmaceuticals recently announced it has now reached the criteria required by the study protocol to conduct a pre-planned, non-binding futility analysis, which an independent Data Safety Monitoring Board (DSMB) will review to determine whether to recommend continuing the study as planned or modifying the study based on Berubicin showing potential value as a second-line treatment for patients with glioblastoma. CNS Pharmaceuticals previously reported that the Company would conduct this analysis after at least 50% of the patients in the population to be analyzed for the interim analysis (30-50% of the total number of patients for this trial) had reached the primary efficacy endpoint, as provided for in the study protocol. The DSMB will review the number of deaths in each arm to ensure that the overall survival of patients receiving Berubicin shows at least a statistically significant comparability to those receiving Lomustine. Additional analyses will include comparisons of secondary endpoints, including progression-free survival (PFS), response rates, and safety assessments. The Company is confident it will announce the results from the interim analysis publicly before the year end. Enrollment will continue during the interim analysis.

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, which may provide seven years of marketing exclusivity upon approval of an NDA.