CNS Pharmaceuticals (CNSP) Investor Update summary

Pipeline expansion and strategic focus

• Exclusive in-licensing of TPI 287, a late-stage, blood-brain barrier permeable microtubule inhibitor, for brain malignancies, complementing the Berubicin program, with upfront and milestone-based payments.

• TPI 287 is orphan-designated, providing 7 years of marketing exclusivity, and has been studied in over 350 patients, showing strong efficacy and safety as monotherapy and in combination with bevacizumab.

• TPI 287 demonstrated a 60% overall response rate, 96% disease control rate, and 13.4-month median overall survival in recurrent glioblastoma patients.

• The company spent over four years evaluating 200+ compounds before selecting TPI 287 for its unique mechanism and robust efficacy.

• A new corporate brand and website were launched to reflect the commitment to GBM treatment innovation.

Clinical and regulatory development plans

• Plans are underway to initiate a phase 3/registration program for TPI 287 in recurrent GBM, especially in MGMT unmethylated patients, with study initiation targeted for 2025.

• The pivotal trial may compare TPI 287 plus bevacizumab to bevacizumab alone, with overall survival as the primary endpoint.

• The company will leverage its established clinical network and infrastructure, built for Berubicin, to accelerate TPI 287 development and reduce time and cost, with estimated upfront savings of $10–$15 million.

• Berubicin has completed enrollment in a pivotal global trial for recurrent GBM, with topline results expected in the first half of 2025.

• Interim futility analysis for Berubicin was positive, with the Data Safety Monitoring Board recommending continuation without modification.

Market potential and differentiation

• TPI 287 and Berubicin are complementary, targeting different patient subgroups and disease stages, with potential for combination therapy and no expected commercial or clinical trial cannibalization.

• TPI 287’s ability to penetrate the brain opens opportunities for treating other brain metastases, such as from breast cancer.

• The company’s approach aims to establish a foundational cytotoxic chemotherapy backbone for GBM, addressing a gap in current treatment paradigms.

• There is potential therapeutic synergy between Berubicin and TPI 287, offering new combination strategies for brain malignancies.

• Investor confidence is expected to grow as the company demonstrates rigorous, science-driven development and leverages its established network.