CNS Pharmaceuticals Achieves Completion of Planned Enrollment in Potentially Pivotal Study of Berubicin for the Treatment of Glioblastoma Multiforme (GBM)

Rhea-AI Summary

CNS Pharmaceuticals, Inc. announces the completion of planned enrollment in its global potentially pivotal study evaluating Berubicin for the treatment of GBM, an aggressive and incurable form of brain cancer. The study has enrolled 247 patients across 46 clinical trial sites in the U.S., Italy, France, Spain, and Switzerland. The interim analysis of efficacy and safety data in the study was successfully completed, with a positive recommendation from the independent Data Safety Monitoring Board (DSMB) to continue the trial without modification.

Insights

Oncology Doctor

The completion of enrollment in a potentially pivotal study for a novel oncology drug such as Berubicin represents a significant step in the drug development process. Berubicin's unique ability to cross the blood-brain barrier could be transformative for treating glioblastoma multiforme (GBM), a notably aggressive brain cancer with limited treatment options. The primary endpoint of Overall Survival (OS) is a critical measure of effectiveness, directly correlating to a patient's lifespan post-treatment. The positive recommendation from the Data Safety Monitoring Board (DSMB) to continue the trial without modification indicates that Berubicin has shown enough potential in terms of efficacy and safety to warrant further investigation.

Financial Analyst

For investors, the progression of CNS Pharmaceuticals' trial into its final stages is a noteworthy event, as it brings the company closer to a possible New Drug Application (NDA) submission if the results are favorable. The market for GBM treatments is substantial, given the high unmet need for effective therapies. The stock market often reacts positively to such milestones, particularly when the interim analysis suggests potential efficacy. However, it's important to note that the actual impact on CNS Pharmaceuticals' financial position will depend on the final study results, regulatory approval and the drug's commercial potential in a competitive oncology market.

Market Research Analyst

Analyzing the broader market implications, the successful development of Berubicin may influence the competitive landscape of brain cancer treatments. With GBM being a difficult-to-treat condition, a new effective therapy could quickly gain market share and set a new standard of care. This could disrupt existing treatment regimens and impact the revenues of companies providing current standard treatments. Additionally, the focus on a high-profile condition like GBM raises the profile of CNS Pharmaceuticals within the biopharmaceutical sector, potentially attracting partnership opportunities or acquisition interest.

HOUSTON, TX / ACCESSWIRE / January 17, 2024 / 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 completion of planned enrollment in its global potentially pivotal study evaluating Berubicin, the Company's novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier, for the treatment of GBM, an aggressive and incurable form of brain cancer.

The potentially pivotal study of Berubicin is a multicenter, open-label, randomized controlled study in adult patients with recurrent GBM (WHO Grade IV) after failure of standard first-line therapy and compared to Lomustine. The study has enrolled 247 patients across 46 clinical trial sites in the U.S., Italy, France, Spain, and Switzerland. The primary endpoint of the study is Overall Survival (OS), a rigorous endpoint 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.

John Climaco, CEO of CNS Pharmaceuticals commented, "The completion of planned enrollment is yet another important milestone for the Company. With the DSMB's recent positive recommendation based on the interim analysis and the completion of planned enrollment, we are now focused on bringing this study across the finish line. We remain hopeful in our effort to address this devastating disease and potentially offering an effective treatment in GBM that is safe and well tolerated."

As announced on December 18, 2023, the interim analysis of efficacy and safety data in the potentially pivotal study of Berubicin was successfully completed. The recommendation of the independent Data Safety Monitoring Board (DSMB) was that the Company's ongoing global, potentially pivotal trial of the investigational agent Berubicin for the treatment of GBM continue without any modification. The recommendation follows the DSMB's pre-specified futility analysis of unblinded (to the DSMB only) efficacy and safety data in the Company's trial of Berubicin versus Lomustine, a standard of care in patients with recurrent GBM. More specifically, the DSMB reviewed the primary endpoint of overall survival (OS) and secondary efficacy measures progression-free survival (PFF) and overall response rate (ORR), as well as safety data in evaluable patients. In order to support continuing the trial, Berubicin's efficacy had to be at least comparable to Lomustine's on the primary endpoint (OS).

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

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 for using Berubicin to treat malignant glioma, which may provide seven years of marketing exclusivity upon approval of a New Drug Application (NDA).

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 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 a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently in development for the treatment of a number of serious brain and CNS oncology indications including glioblastoma multiforme (GBM), an aggressive and incurable form of brain cancer.

For more information, please visit www.CNSPharma.com, and connect with the Company on Twitter, Facebook, 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 press release include, without limitation, the timing of the topline results and whether the FDA will recognize the Company's primary endpoint as a basis for approval. 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 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.

CONTACTS:
Investor Relations Contact
JTC Team, LLC
Jenene Thomas
833-475-8247
CNSP@jtcir.com

SOURCE: CNS Pharmaceuticals, Inc.

View the original press release on accesswire.com

FAQ

What is the primary endpoint of the study?

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

How many patients were enrolled in the study?

The study has enrolled 247 patients across 46 clinical trial sites in the U.S., Italy, France, Spain, and Switzerland.

What is the interim analysis of efficacy and safety data?

The interim analysis of efficacy and safety data in the study was successfully completed, with a positive recommendation from the independent Data Safety Monitoring Board (DSMB) to continue the trial without modification.

What is the investigational agent being studied?

The investigational agent being studied is Berubicin, the Company's novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier, for the treatment of GBM.