About CNS Pharmaceuticals

CNS Pharmaceuticals, Inc. operates as a clinical stage pharmaceutical company. The company focuses on the development of anti-cancer drug candidates for the treatment of brain and central nervous system tumors, based on intellectual property that it licenses under license agreements with Houston Pharmaceuticals, Inc. (HPI) and The University of Texas M.D. Anderson Cancer Center (UTMDACC) and owns pursuant to a collaboration and asset purchase agreement with Reata Pharmaceuticals, Inc. (Reata). The company's lead drug candidate, Berubicin, may be a significant development in the treatment of Glioblastoma and other CNS malignancies, and if approved by the U.S. Food and Drug Administration (FDA), could give Glioblastoma patients an important new therapeutic alternative to the standard of care. Berubicin is an anthracycline, which is a class of drugs that are among the most powerful and extensively used chemotherapy drugs known. While the company's focus is on the development of Berubicin, it is also in the process of attempting to secure intellectual property rights to additional compounds that it plans to develop into drugs to treat CNS and other cancers. Berubicin was discovered at UTMDACC by Dr. Waldemar Priebe, the founder of the company. Through a series of transactions, Berubicin was initially licensed to Reata. Reata initiated several Phase I clinical trials with Berubicin for CNS malignancies, one of which was for malignant gliomas, but subsequently allowed their Investigational New Drug (IND) with the U.S. Food and Drug Administration (FDA) to lapse for strategic reasons. This required the company to obtain a new IND for Berubicin before beginning further clinical trials. On December 17, 2020, the company announced that its IND application with the FDA for Berubicin for the treatment of Glioblastoma Multiforme was in effect. It initiated this trial for patient enrollment during the second quarter of 2021 with the first patient dosed during the third quarter of 2021 to investigate the efficacy of Berubicin in adults with Glioblastoma Multiforme who have failed first-line therapy. The first patient on the trial was treated during the third quarter of 2021. Correspondence between the company and the FDA resulted in modifications to its initial trial design, including designating overall survival (OS) as the primary endpoint of the study. On December 28, 2017, the company obtained the rights to a worldwide, exclusive royalty-bearing, license to the chemical compound commonly known as Berubicin from HPI in an agreement it refers to as the HPI License. The patents it licensed from HPI expired in March 2020. On June 10, 2020, the FDA granted Orphan Drug Designation (ODD) for Berubicin for the treatment of malignant gliomas. ODD from the FDA is available for drugs targeting diseases with less than 200,000 cases per year. ODD may enable market exclusivity of 7 years from the date of approval of an New Drug Application (NDA) in the United States. The ODD now constitutes the company's primary intellectual property protections although it is exploring if there are other patents that could be filed related to Berubicin to extend additional protections. With the Reata Agreement and the HPI License, the company has obtained all rights and intellectual property necessary to develop Berubicin. As stated earlier, it is the company's plan to obtain additional intellectual property covering other compounds which, subject to the receipt of additional financing, may be developed into drugs for brain and other cancers. On January 10, 2020, the company entered into a Patent and Technology License Agreement (the WP1244 Agreement) with The Board of Regents of The University of Texas System, an agency of the State of Texas, on behalf of the UTMDACC. Pursuant to the WP1244 Agreement, the company obtained a royalty-bearing, worldwide, exclusive license to certain intellectual property rights, including patent rights, related to its portfolio of WP1244 drug technology. On May 7, 2020, pursuant to the WP1244 portfolio license agreement, the company entered into a Sponsored Research Agreement with UTMDACC to perform research relating to novel anticancer agents targeting CNS malignancies. The company's first product under development is Berubicin, a development stage anthracycline intended to treat glioblastoma. Berubicin is an anthracycline, a class of drugs that are among the most powerful chemotherapy drugs known. Berubicin intercalates into deoxyribonucleic acid (DNA) and interrupts topoisomerase II activity, resulting in the inhibition of DNA replication and repair, and ultimately RNA and protein synthesis. Based on evidence developed from animal models and limited clinical data derived from a Phase 1 human clinical trial, Berubicin appears to cross the blood brain barrier and target cancer cells, specifically glioblastoma, more effectively and efficiently than any other known anthracyclines. Berubicin hydrochloride (HCl) is a novel synthetic anthracycline with a chemical structure similar to doxorubicin HCl, a cytotoxic anthracycline topoisomerase II inhibitor isolated from cultures of Streptomyces peucetius var. caesius. Doxorubicin HCl Injection and Doxorubicin HCl for Injection, drugs related in chemical structure and mechanism of action to Berubicin, are approved by the FDA for the treatment of various cancers, including acute lymphoblastic leukemia, acute myeloblastic leukemia, Hodgkin lymphoma, Non-Hodgkin lymphoma, metastatic breast cancer, metastatic Wilms' tumor, metastatic neuroblastoma, metastatic soft tissue sarcoma, metastatic bone sarcomas, metastatic ovarian carcinoma, metastatic transitional cell bladder carcinoma, metastatic thyroid carcinoma, metastatic gastric carcinoma, and metastatic bronchogenic carcinoma, as well as part of a multiagent adjuvant chemotherapy for the treatment of women with axillary lymph node involvement after resection of primary breast cancer. A liposomal formulation of doxorubicin HCl is also approved for the treatment of ovarian cancer, AIDS-related Kaposi's sarcoma, and multiple myeloma. Reata licensed in berubicin HCl with the intent of developing it for commercialization. On December 28, 2004, Reata filed an initial IND (IND 68,279; Serial No. 000) for an injection formulation of berubicin HCl (RTA 744 Injection) for the treatment of anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic mixed oligo-astrocytoma, glioblastoma, and gliosarcoma. Three clinical trials were initiated under IND 68,279, two phase 1 trials and one phase 2 trial. The initial phase 1 trial (Study RTA 744-C-0401) was completed, and the maximum tolerated dose determined. A 44% disease control response rate was observed. On May 24, 2019, the company's sublicensee, WPD, signed the Granting Agreement with the Polish National Center for Research and Development for co-funding of research and development work in the amount of 22,033,066 PLN (approximately U.S. $5,798,875) for new drug development as a part of the project New approach to glioblastoma treatment addressing the critical unmet medical need, undertaken pursuant to the WPD Sublicense. Intellectual Property Under the HPI License the company obtained the exclusive right to develop certain chemical compounds for use in the treatment of cancer anywhere in the world. It has licensed the right to certain intellectual property covering products comprised of anthracycline antibiotic compound, methods for manufacture and use for the treatment of cancer. The licensed intellectual property originally included certain material patents in the United States and their foreign counterparts throughout the world. The U.S. patents have expired, and as such, the company may be subject to increased competition. On June 10, 2020, the FDA granted Orphan Drug Designation (ODD) for Berubicin for the treatment of malignant gliomas. ODD from the FDA is available for drugs targeting diseases with less than 200,000 cases per year. On July 24, 2021, the company received Fast Track Designation from the FDA for Berubicin. Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The company is exploring the possibility to file additional patent applications that potentially might allow for further increase of the exclusive market protection for use of Berubicin. License Agreements On November 21, 2017, the company entered into a Collaboration and Asset Purchase Agreement with Reata (the Reata Agreement). Pursuant to the Reata Agreement the company purchased all of Reata's intellectual property and development data regarding Berubicin, including all trade secrets, knowhow, confidential information and other intellectual property rights. On December 28, 2017, the company entered into a with Houston Pharmaceuticals, Inc. (HPI). HPI is affiliated with Dr. Waldemar Priebe, its founder. Pursuant to this agreement, the company obtained a worldwide exclusive license to the chemical compound commonly known as WP744. On August 30, 2018, the company entered into a sublicense agreement with WPD Pharmaceuticals, Inc. (WPD), pursuant to which it granted WPD an exclusive sublicense, even as to its, for the patent rights it licensed pursuant to the HPI License within the following countries: Poland, Estonia, Latvia, Lithuania, Belarus, Ukraine, Moldova, Romania, Bulgaria, Serbia, Macedonia, Albania, Armenia, Azerbaijan, Georgia, Montenegro, Bosnia, Croatia, Slovenia, Slovakia, Czech Republic, Hungary, Chechnya, Uzbekistan, Kazakhstan, Kyrgyzstan, Tajikistan, Turkmenistan, Greece, Austria, and Russia. On January 10, 2020, the company entered into a Patent and Technology License Agreement (the WP1244 Agreement) with The Board of Regents of The University of Texas System, an agency of the State of Texas, on behalf of UTMDACC. Pursuant to the WP1244 Agreement, the company obtained a royalty-bearing, worldwide, exclusive license to certain intellectual property rights, including patent rights, related to the WP1244 drug technology. On March 20, 2020, the company entered into a Development Agreement with WPD Pharmaceuticals (WPD) (the Development Agreement), a company founded by Dr. Priebe. Pursuant to the Development Agreement, WPD agreed to use its commercially reasonable efforts in good faith to develop and commercialize certain products that WPD had previously sublicensed, solely in the field of pharmaceutical drug products for the treatment of any viral infection in humans, with a goal of eventual approval of in certain territories consisting of: Poland, Estonia, Latvia, Lithuania, Belarus, Ukraine, Romania, Armenia, Azerbaijan, Georgia, Slovakia, Czech Republic, Hungary, Uzbekistan, Kazakhstan, Greece, Austria, Russia, Netherlands, Turkey, Belgium, Switzerland, Sweden, Portugal, Norway, Denmark, Ireland, Finland, Luxembourg, Iceland. The term of the Development Agreement will expire on the expiration of the sublicense pursuant to which WPD has originally sublicensed the products, which will occur upon the expiration of the patents subject to the sublicense agreement, the earliest of which expires in 2024. On February 19, 2021, CNS entered into an Investigational Medicinal Product Supply Agreement with WPD, a related party. CNS agreed to sell the Berubicin drug product to WPD at historical cost of manufacturing without markup so that WPD may conduct the clinical trials contemplated by the sublicense agreement. On October 24, 2022, the company received confirmation from its third party depot service provider that the vials had been transferred into its inventory. As such, this matter is now fully resolved. On November 21, 2022, CNS entered into an Investigational Medicinal Product Supply Agreement with Pomeranian Medical University (PUM) in Szczecin, Poland. CNS agreed to sell berubicin hydrochloride drug product (and related reference standards) to PUM at a discount to the historical cost of manufacturing so that PUM may conduct an investigator-initiated clinical trial of Berubicin in CNS lymphomas. As of March 29, 2023, the first batch of berubicin drug product vials have been ordered but not yet delivered. Research and Development Expense The company's research and development expense included $9,300,055 for the year ended December 31, 2022. Governmental Regulation The pharmaceutical drug product candidates that the company develops must be approved by the FDA before they may be marketed and distributed. Any pharmaceutical products for which the company receives FDA approvals are subject to continuing regulation by the FDA, including among other things, current good manufacturing practices (cGMP) compliance, record-keeping requirements, reporting of adverse experiences with the product, providing the FDA with updated safety and efficacy information, product sampling and distribution requirements, complying with certain electronic records and signature requirements and complying with FDA promotion and advertising requirements, which include, among others, standards for direct-to-consumer advertising, prohibitions on promoting pharmaceutical products for uses or in patient populations that are not described in the pharmaceutical product's approved labeling (known as 'off-label use'), industry-sponsored scientific and educational activities and promotional activities involving the internet. History CNS Pharmaceuticals, Inc. was founded in 2017. The company was incorporated as a Nevada corporation in 2017.