Celcuity (CELC) Jefferies 2024 Global Healthcare Conference summary

Clinical development updates

• Lead candidate gedatolisib targets the PAM/PI3K/AKT/mTOR pathway, focusing on HR-positive, HER2-negative breast cancer and metastatic castration-resistant prostate cancer.

• Phase III breast cancer study is enrolling women post-CDK4/6 and letrozole, randomizing by PIK3CA status, with separate controls and hierarchical endpoints.

• Enrollment for the wild type cohort reached 50% in Q1, with data expected by the end of H2 2025.

• Over 200 sites in 20+ countries are managed via CROs, with frequent monitoring and strict adherence to protocol.

• Patient population is consistent with prior studies, excluding prior chemo and including bone-only patients with measurable lesions.

Safety and pharmacokinetics

• Gedatolisib shows a favorable safety profile, with low discontinuation due to adverse events, attributed to IV administration and balanced PK properties.

• IV route avoids first-pass liver exposure, reducing hyperglycemia risk compared to oral PAM inhibitors.

• Weekly dosing maintains plasma concentrations above 80% pathway inhibition throughout the dosing window.

• Preclinical and clinical data indicate higher potency and antitumor control versus single-node inhibitors.

Market opportunity and competitive landscape

• U.S. addressable market for second-line HR-positive, HER2-negative breast cancer is estimated at over $5 billion, with ex-U.S. markets more than doubling the patient pool.

• Gedatolisib’s efficacy and safety profile may offer differentiation from competitors, with potential for multi-billion dollar peak revenue.

• Full-dose regimens and simultaneous pathway inhibition are expected to yield superior outcomes compared to substandard dosing in comparator studies.