CNS Pharmaceuticals Berubicin Trial is On Schedule to Commence in March 2021

Potentially pivotal study evaluating efficacy of Berubicin in the treatment of adult Glioblastoma Multiforme (GBM), one of the most aggressive types of brain cancer

HOUSTON, Feb. 25, 2021 /PRNewswire/ -- CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, today indicated that the clinical program for Berubicin is on track to start enrolling patients in March 2021. CNS' lead product candidate, Berubicin, is a novel anthracycline and the first anthracycline to cross the blood-brain barrier. It is in development for the treatment of a number of serious brain and CNS oncology indications.

Preparations are proceeding on schedule for the Company's study evaluating the efficacy of Berubicin in the treatment of GBM. The Company intends to enroll subjects across approximately 35 clinical sites in the U.S., with 21 sites currently confirmed and in a start-up process.  The Company also plans to expand the trial into western Europe.

"I am very pleased with our progress and the team's execution towards the start of our program. We have made significant advancements and are now finalizing clinical site selection and preparing to begin patient screening, which we expect to commence next month," commented John Climaco, CEO of CNS Pharmaceuticals. "Berubicin's promising results demonstrated in the Phase 1 clinical trial build on sixty years of clinical experience with anthracyclines.  Berubicin is an entirely novel molecule that represents an opportunity to recognize the powerful benefits of this tried-and-true class of drugs for neuro-oncology in general and in the fight against GBM in particular. We are deeply committed to driving this program forward as expeditiously as possible with the prime focus on our mission to improve patient outcomes for GBM."

The potentially pivotal trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. The primary endpoint of the study is Overall Survival. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm. Results from the trial will compare Berubicin to the current standard of care, with a 2 to 1 randomization of patients to receive either Berubicin or Lomustine. For more information about the trial, visit (identifier NCT04762069).

"With a dismal survival rate of only 14.6 months from diagnosis, and no currently approved second-line therapies for GBM, there remains an urgent, critical need in the treatment landscape for glioblastoma, one of the most aggressive, deadly and resistant cancers that form in the brain. GBM patients are in need of hope for a treatment that can offer improvement in progression free as well as overall survival. Given our current understanding of the promise of Berubicin, our trial design could be the best opportunity to provide the FDA with data demonstrating an effective treatment option. We look forward to beginning patient screening in the coming weeks and learning more about Berubicin in the treatment of GBM," added Dr. Sandra Silberman, CMO of CNS Pharmaceuticals.

Anticipated Upcoming Milestones:

  • Patient screening in the Company's clinical trial for the treatment of glioblastoma will start in March 2021 with the first patient dosed shortly thereafter;
  • Our sublicensee partner in Poland, WPD Pharmaceuticals, will initiate a Phase 2 multicenter clinical trial of Berubicin in GBM in the first half of 2021;
  • The WPD trial in adults with GBM will include an interim analysis of the first 18 patients by Q4 2021 for efficacy as well as an extensive pharmacokinetic profile in these patients;
  • WPD will commence a multicenter Phase 1 pediatric trial for malignant CNS tumors in 2021;
  • CNS will conduct pre-clinical evaluation of Berubicin for additional CNS cancers and cancers metastatic to the brain, including development of potential combination therapies for these indications; and
  • CNS will expand our pipeline in the evaluation of other drugs for brain cancers.

The FDA has granted CNS Pharmaceuticals Orphan Drug designation for Berubicin, which provides seven years of marketing exclusivity upon approval of an NDA.  CNS Pharmaceuticals intends to file for additional patents relating to Berubicin to further secure intellectual property protections.

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 developing novel treatments for primary and metastatic cancers of the brain and central nervous system. Its lead drug candidate, Berubicin, is proposed for the treatment of glioblastoma multiforme (GBM), an aggressive and incurable form of brain cancer. CNS holds a worldwide exclusive license to the Berubicin chemical compound and has acquired all data and know-how from Reata Pharmaceuticals, Inc. related to a completed Phase 1 clinical trial with Berubicin in malignant brain tumors, which Reata conducted in 2006. In this trial the overall response rate of stable disease or better was 44%. This 44% disease control rate was based on 11 patients (out of 25 evaluable patients) with stable disease, plus responders. One patient experienced a durable complete response and remains cancer-free as of Feb. 20, 2020. These Phase 1 results represent a limited patient sample size and, while promising, are not a guarantee that similar results will be achieved in subsequent trials. During 2021, CNS expects to commence a Phase 2 clinical trial of Berubicin for the treatment of GBM in the U.S., while a sub-licensee partner undertakes a Phase 2 trial (with extensive PK) in adults and a first-ever Phase 1 trial in pediatric GBM patients in Poland. Its second drug candidate, WP1244, is a novel DNA binding agent that has shown in preclinical studies that it is 500 times more potent than the chemotherapeutic agent daunorubicin in inhibiting tumor cell proliferation.

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