CNS Pharmaceuticals secures orphan drug status for cancer drug

HOUSTON - CNS Pharmaceuticals, Inc. (NASDAQ:CNSP), a micro-cap biopharmaceutical company with a market capitalization of $3.56 million, has announced the successful acquisition of Orphan Drug Designation for its investigational drug TPI 287 from Cortice Biosciences, Inc. According to InvestingPro analysis, the company appears undervalued despite its shares falling nearly 100% over the past year. The U.S. Food and Drug Administration (FDA) had previously granted the designation for TPI 287 for the treatment of gliomas, pediatric neuroblastoma, and progressive supranuclear palsy.

TPI 287, a novel abeotaxane, shares a mechanism of action with other taxanes like paclitaxel and docetaxel, which involves the stabilization of microtubules to inhibit cell division, leading to apoptosis and cell death. What sets TPI 287 apart is its potential to cross the blood-brain barrier, a significant trait for treating central nervous system (CNS) tumors. Clinical data, including a Phase 1 trial for glioblastoma multiforme (GBM) in combination with bevacizumab, have shown promising results with 3 Complete Responses and 9 Partial Responses out of 23 patients.

John Climaco, CEO of CNS Pharmaceuticals, highlighted the operational efficiency of the company and its dedication to developing therapies for neuro-oncology. He noted the significance of the Orphan Drug Designation, which may offer seven years of market exclusivity post-approval. Climaco also emphasized the company’s commitment to addressing the critical need for effective GBM treatments.

The Orphan Drug Designation by the FDA Office of Orphan Products Development is aimed at encouraging the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation offers several benefits, including tax credits, fee exemptions, and the potential for market exclusivity.

CNS Pharmaceuticals plans to initiate patient enrollment for a Phase 2 study of TPI 287 by the end of 2025. While the drug candidate has been tested in over 350 patients across various clinical trials and has shown a favorable safety profile and high tolerability, InvestingPro data reveals the company maintains a healthy current ratio of 3.43, indicating strong short-term liquidity. Investors should note that the company’s next earnings report is scheduled for May 14, 2025. InvestingPro subscribers have access to 15 additional key insights about CNSP’s financial health and market performance.

This announcement is based on a press release statement from CNS Pharmaceuticals, Inc.

In other recent news, CNS Pharmaceuticals announced that its clinical trial for Berubicin, a treatment for aggressive brain cancer, did not demonstrate a significant difference in overall survival compared to Lomustine. Despite this, Berubicin showed a favorable safety profile, prompting the company to continue analyzing the trial’s outcomes. In a strategic move to secure funding, CNS Pharmaceuticals expanded its stock sale agreement with A.G.P./Alliance Global Partners, increasing the potential aggregate sales price from $30.2 million to $43.5 million. This agreement is part of a larger shelf registration statement, allowing the company to sell additional shares to support ongoing research and development.

Additionally, CNS Pharmaceuticals confirmed that its cash reserves are expected to fund operations into the first quarter of 2026, maintaining its NASDAQ Capital Market listing. The company executed a 1-for-50 reverse stock split to comply with NASDAQ’s minimum price requirement, consolidating shares to increase the share price. CEO John Climaco mentioned plans to release primary data analysis from its global clinical trial for Berubicin in the first half of 2025, while also initiating the clinical program for another drug candidate, TPI 287, by the end of the year. These developments reflect CNS Pharmaceuticals’ efforts to manage financial stability and advance its drug development initiatives.

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