CNS Pharmaceuticals has reported the primary analysis of its clinical trial evaluating Berubicin in adults with recurrent or progressive Glioblastoma Multiforme (GBM), an aggressive brain cancer.

The study compared Berubicin to Lomustine, a standard second-line therapy, across multiple endpoints. While the trial did not demonstrate a statistically significant difference in overall survival, Berubicin showed clinically relevant outcomes comparable to Lomustine, with a continued favourable safety profile, including the absence of anthracycline-related cardiotoxicity.

The trial, a multicenter, open-label, randomized controlled study, included 252 patients across North America and Europe, with Berubicin administered at a 2:1 ratio versus Lomustine. Patients continue to be followed for overall survival, and ongoing analyses include advanced imaging review, pharmacokinetics and additional clinical endpoints. The data appear comparable to Lomustine in clinically important endpoints, including overall survival and progression-free survival, across all patients, including those with high-risk tumor markers.

Sandra Silberman, md, chief medical officer at CNS Pharmaceuticals, said: “Clinical results in a prior Phase 1 trial indicated Berubicin’s potential to improve outcomes for patients with previously treated Glioblastoma. In this analysis overall survival is comparable between Berubicin and Lomustine. We are awaiting long-term follow-up of patients still alive as well as those still on trial, and will evaluate our substantial clinical dataset to obtain additional insights. Given the persistent unmet clinical need in GBM and other brain cancers, the results we’ve seen with Berubicin in this study warrant further investigation of this drug.”

The study incorporated a comprehensive patient phenotype assessment using well-recognized biostatistical techniques, including a pre-specified futility analysis. Further clinical analyses are ongoing to generate informed hypotheses for future trials. Erin Dunbar, md, principal investigator and director of the Piedmont Brain Tumor Center in Atlanta, Georgia, noted: “As the longest accruing Investigator, I have witnessed rapid imaging responses and excellent clinical tolerability throughout the trial. I hope to see continued investigation of Berubicin to further evaluate its potential as an important tool to treat primary and/or metastatic cancer patients of all ages who urgently need additional options.”

Berubicin is a novel anthracycline designed to cross the blood-brain barrier and avoid cardiotoxicity, a limitation of other anthracyclines. According to Silberman, “Because of the cardiotoxicity associated with other anthracyclines, the fact that this study showed no cardiotoxicity with Berubicin, even in those receiving the drug for over a year, suggests Berubicin could be a valuable recourse for patients with recurrent or progressive cancers. Berubicin also did not exhibit the pulmonary toxicity or the thrombocytopenia associated with Lomustine, suggesting it could be a way for patients to avoid those side effects during treatment.”

CNS continues development of TPI 287, a novel taxane, which has published clinical efficacy data in GBM and has demonstrated a favourable safety profile in hundreds of patients. John Climaco, jd, chief executive officer at CNS, added: “We are grateful for the commitment and dedication of patients, caregivers, and investigators who supported this program. We are exploring further Berubicin development as well as evaluating opportunities for applying the methodology and strategy from this program to other drugs for CNS malignancies, including our proprietary drug TPI 287.”