CNS Pharmaceuticals (CNSP) Investor Update summary

Company overview and clinical focus

• Dedicated to neuro-oncology with two main programs: berubicin and TPI 287, both designed to cross the blood-brain barrier and address unmet needs in brain tumors.

• Berubicin is a novel anthracycline with no observed cardiotoxicity, developed at MD Anderson, and is in a fully enrolled pivotal trial with data expected in the first half of 2025.

• TPI 287 is a blood-brain barrier permeable taxane, recently in-licensed, with plans for clinical trials in 2025.

• The company has built a robust clinical network and efficient team, enabling rapid global trial enrollment and future scalability.

Glioblastoma (GBM) landscape and unmet need

• GBM is a highly lethal disease with limited effective treatments; chemotherapy benefits only about a third of patients.

• Standard therapies include surgery, alkylating agents, radiotherapy, and Tumor Treating Fields, but survival rates remain poor.

• Clinical trials are crucial due to the scarcity of effective options, making new therapies highly anticipated.

Blood-brain barrier and drug innovation

• The blood-brain barrier restricts most chemotherapies, necessitating drugs that can penetrate it for brain tumor treatment.

• Berubicin was engineered to cross the barrier and has shown superior potency and activity in preclinical and early clinical studies.

• Early human trials demonstrated significant tumor responses and long-term survival in some patients, with a favorable safety profile.