CNS Pharmaceuticals Announces Primary Analysis of Berubicin in Second Line Treatment of Glioblastoma Multiforme

Berubicin showed clinically relevant outcomes comparable to Lomustine across multiple endpoints, but not a statistically significant difference in overall survival, the primary endpoint.

The safety profile continues to be favorable, including the absence of anthracycline related cardiotoxicity.

Analysis of outcomes are ongoing, including advanced imaging review, PK, and clinical endpoints.

The company also continues development of a novel taxane, TPI 287, with published clinical efficacy data in Glioblastoma Multiforme.

HOUSTON, TX / ACCESS Newswire / March 25, 2025 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, today announced the primary analysis of its clinical trial (NCT04762069) evaluating Berubicin, the first anthracycline demonstrated to extensively cross the blood-brain barrier (BBB), for the treatment of recurrent or progressive Glioblastoma Multiforme (GBM), an aggressive and usually fatal brain cancer. This analysis did not demonstrate statistically significant superiority in overall survival, the primary endpoint. However, although the trial was not powered to determine non-inferiority, the data appear comparable in clinically important endpoints in all patients, including those with the most unfavorable tumor markers. Additionally, patients experienced no cardiotoxicity, a risk that curtails the use of other anthracyclines, and the safety profile continues to be favorable in this patient population.

The trial compared Berubicin to Lomustine, a current standard of care in recurrent or progressive GBM. The protocol design incorporated the importance of patient phenotype, utilizing well recognized biostatistical techniques in both a pre-specified futility analysis and this analysis. The data appear comparable to Lomustine in important clinically relevant endpoints, including overall survival (OS) and progression free survival (PFS) across all patients treated with Berubicin. Further, the aggregate data facilitate multiple additional clinical analyses for informed hypothesis generation.

"Clinical results in a prior Phase 1 trial indicated Berubicin's potential to improve outcomes for patients with previously treated Glioblastoma," said Sandra Silberman, MD, PhD, Chief Medical Officer of CNS Pharmaceuticals. "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."

Erin Dunbar, MD, a Principal Investigator and the 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, the first to readily penetrate the blood brain barrier and show no cardiac toxicity, thus overcoming two limitations to the use of one of the most effective classes of drugs for the treatment of cancer. "Because of the cardiotoxicity associated with other anthracyclines," Dr. Silberman said, "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." She added, "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. Furthermore, its primary mechanism of action, topoisomerase II inhibition, is agnostic to specific tumor histology, making it a potentially more generalizable therapy."

CNS Chief Executive Officer John Climaco, JD further 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. TPI 287 is a taxane-derivative with published clinical and radiologic data showing activity in Glioblastoma Multiforme, as well as a favorable safety profile in hundreds of patients to date."

About the Berubicin Trial
This ongoing study of Berubicin is a multicenter, open-label, randomized controlled study in adult patients with recurrent GBM (WHO Grade IV - IDH Wild-Type), comparing Berubicin to Lomustine, an accepted treatment for second line therapy in this disease. The study has included 252 patients across North America and Europe, randomizing Berubicin to Lomustine 2:1. Continuation of patients on treatment and their follow-up for overall survival is proceeding. Final data will be included in a future analysis.

For more information about this trial, visit clinicaltrials.gov and reference identifier NCT04762069.

About Berubicin
Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of primary and metastatic cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc. Berubicin was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.

About CNS Pharmaceuticals, Inc.
CNS Pharmaceuticals is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system.

The Company's lead drug candidate, Berubicin, is the first anthracycline to appear to cross the blood-brain barrier. Berubicin is the subject of the Company's late-stage, fully-enrolled, global clinical trial for the treatment of glioblastoma multiforme (GBM), an aggressive and currently incurable form of brain cancer.

The Company's second drug candidate, TPI 287, is an abeotaxane which stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. Similar to Berubicin, TPI 287 has shown the potential to cross the blood-brain barrier and treat CNS tumors. TPI 287 has been well tolerated in over 350 patients to date, including in clinical trials as a monotherapy and in combination with bevacizumab for the treatment of recurrent neuroblastoma and medulloblastoma, as well as refractory prostate cancer and melanoma, and in tauopathy disease, which can result in dementia.

For more information, please visit www.CNSPharma.com, and connect with the Company on X and LinkedIn.

Forward-Looking Statements
Some of the statements in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this release include, without limitation, the results of any future investigation of Berubicin and the release of the final data and analysis from the clinical trial. These statements relate to future events, future expectations, plans and prospects. Although CNS believes the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. CNS has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, including market and other conditions and those discussed under Item 1A. "Risk Factors" in CNS's most recently filed Form 10-K filed with the Securities and Exchange Commission ("SEC") and updated from time to time in its Form 10-Q filings and in its other public filings with the SEC. Any forward-looking statements contained in this press release speak only as of its date. CNS undertakes no obligation to update any forward-looking statements contained in this press release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events, except as required by law.

CONTACTS:
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SOURCE: CNS Pharmaceuticals, Inc.