CNS Pharmaceuticals (NASDAQ:CNSP) has announced updated results from its ongoing pivotal trial evaluating Berubicin in adult patients with recurrent glioblastoma multiforme (GBM) after failure of standard first-line therapy. The data, presented at the 29th Annual Meeting of the Society for Neuro-Oncology (SNO), highlight improved patient retention and comparable safety profiles compared to lomustine, a standard of care. The study enrolled 239 patients, with primary analysis data expected in the first half of 2025.
Trial Design and Patient Demographics
The potentially pivotal study, identified as CNS-201, is a randomized, controlled trial comparing Berubicin to Lomustine. Key inclusion criteria involve adult patients with recurrent GBM (WHO Grade IV) following the failure of initial standard therapy. According to the company, the patient demographics, including age, gender, race, BSA, and KPS, are comparable across both treatment arms. Approximately 40% of patients in each arm presented with unmethylated MGMT, ensuring a balanced comparison of efficacy regardless of methylation status.
Patient Disposition and Study Completion
An interim analysis of the data indicates a higher percentage of patients in the Berubicin arm completed the study (84.7%) compared to the Lomustine arm (78.9%). Conversely, a greater percentage of patients in the Lomustine arm withdrew from the study (19.7%) compared to the Berubicin arm (11.0%). The most common reasons for withdrawal were patient preference (4.9% vs. 9.2%) and death (1.2% vs. 5.3%).
Safety Profile
The safety profiles of Berubicin and Lomustine were relatively similar, with most adverse events showing comparable incidence rates. A slightly greater percentage of patients receiving Berubicin experienced all grades or Grades 3-5 anemia, headache, and decreased neutrophil counts. In contrast, a significantly greater percentage of patients receiving Lomustine experienced decreased platelet counts and thrombocytopenia.
Regulatory Designations and Future Expectations
Berubicin has been granted Fast Track Designation by the FDA, facilitating more frequent interactions with the agency to expedite development and review. Additionally, it has received Orphan Drug Designation for treating malignant glioma, potentially providing seven years of marketing exclusivity upon NDA approval. CNS Pharmaceuticals anticipates reporting primary analysis data from the study in the first half of 2025. The primary endpoint is Overall Survival (OS).
Berubicin: A Novel Anthracycline
Berubicin is a novel anthracycline and the first to cross the blood-brain barrier. Anthracyclines are a class of anticancer agents known for their effectiveness against various cancers. Berubicin induces DNA damage in targeted cancer cells by interfering with topoisomerase II, an enzyme critical for cell proliferation. A Phase 1 trial showed positive responses, including a durable complete response, in brain cancer patients.
