CNS Pharmaceuticals (CNSP) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Key program updates

• Lead program Berubicin is a novel anthracycline, first of its kind to cross the blood-brain barrier, with a pivotal study fully enrolled and top-line data expected in H1 2025.

• No evidence of cardiotoxicity for Berubicin, differentiating it from other anthracyclines; developed at MD Anderson Cancer Center.

• TPI-287, a late-stage abeotaxane, recently in-licensed, also penetrates the blood-brain barrier and has been studied in over 350 patients.

• Both drugs have orphan designation and target glioblastoma, an aggressive, currently incurable brain cancer with high unmet need.

• Berubicin's pivotal trial is one of the largest for glioblastoma, with overall survival as the primary endpoint; results expected within 6–12 months.

Clinical results and pipeline potential

• Phase I Berubicin study showed 44% of patients had stable disease or better, with two partial responders and one durable complete response lasting 17 years.

• TPI-287 demonstrated a 60% overall response rate, 96% disease control, and 13.4-month median survival, exceeding typical outcomes for glioblastoma.

• Both drugs are designed to address the challenge of the blood-brain barrier, a major obstacle in treating brain cancers.

• Berubicin may have broader applications in metastatic cancers with brain involvement, such as breast cancer, due to its mechanism.

• Intellectual property protections are robust, with orphan drug status and ongoing patent efforts, including plans for European filings.

Strategic execution and outlook

• Enrollment for Berubicin completed in January 2024; all investigators and drug supply are in place for the pivotal trial.

• TPI-287 will enter clinical studies for glioblastoma in 2025, building on the established global clinical network.

• The company is well-capitalized to complete the Berubicin study and deliver results.

• Leadership team includes experienced drug developers with track records in blockbuster oncology drugs.

• Focus remains on expanding the neuro-oncology pipeline and addressing large unmet needs in brain and metastatic cancers.