CNS Pharmaceuticals' Chief Medical Officer Sandra Silberman, M.D., Ph.D., presented the company's lead drug candidate TPI 287 at the 2025 Brain Tumor Biotech Summit held at Lenox Hill Hospital in New York on June 5, 2025. The presentation, titled "The Future and Promise of TPI 287- A Brain Penetrating Taxane with Documented Evidence of Efficacy Against Glioblastoma," highlighted the potential of this novel abeotaxane for treating brain tumors.
Mechanism of Action and Blood-Brain Barrier Penetration
TPI 287 operates through the same mechanism as other taxanes, including paclitaxel (Taxol®) and docetaxel, by stabilizing microtubules and inhibiting cell division, ultimately causing apoptosis and cell death. However, TPI 287 distinguishes itself from conventional taxanes through its ability to potentially cross the blood-brain barrier (BBB). While most taxanes are substrates for multi-drug resistant transporters that maintain the BBB, TPI 287's clinical data suggest it can overcome this limitation to treat central nervous system tumors.
Clinical Efficacy Data
The most compelling evidence for TPI 287's therapeutic potential comes from a Phase 1 trial treating glioblastoma patients. In this study, TPI 287 was administered in combination with bevacizumab (Avastin®), resulting in 3 complete responses and 9 partial responses out of 23 evaluable patients. This represents a notable response rate for glioblastoma multiforme (GBM), one of the most aggressive and treatment-resistant brain cancers.
Regulatory Recognition and Broad Clinical Testing
TPI 287 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of gliomas, pediatric neuroblastoma, and progressive supranuclear palsy. This designation recognizes the drug's potential to address unmet medical needs in rare diseases.
The compound has undergone extensive clinical testing, with over 350 patients enrolled across multiple trials. TPI 287 has been evaluated both as a monotherapy and in combination with bevacizumab for treating various conditions, including recurrent glioblastoma, recurrent neuroblastoma and medulloblastoma, advanced malignancies, progressive neoplastic disease, advanced unresectable pancreatic cancer, metastatic melanoma, and breast cancer metastatic to the brain.
Safety Profile
According to CNS Pharmaceuticals, TPI 287 has demonstrated an excellent safety profile and high tolerability among patients throughout its clinical development program. This favorable safety profile is particularly significant given the challenges associated with treating brain tumors and the vulnerability of patients with central nervous system cancers.
Company Focus
CNS Pharmaceuticals is a clinical-stage biopharmaceutical company specializing in developing novel treatments for primary and metastatic cancers of the brain and central nervous system. The company's focus on this challenging therapeutic area addresses a significant unmet medical need, as brain tumors often have limited treatment options due to the blood-brain barrier's protective function that prevents many drugs from reaching tumor sites.
The 2025 Brain Tumor Biotech Summit serves as a platform for fostering collaboration between neuroscience innovators, oncology researchers, and biotechnology investors to accelerate the development of life-saving therapies for patients with brain tumors and other central nervous system diseases.
