Celcuity (CELC) Stifel 2024 Healthcare Conference summary

Pipeline and clinical development

• Lead asset gedatolisib targets the PI3K/AKT/mTOR pathway, with three main programs: two in breast cancer and one in prostate cancer.

• Phase 3 breast cancer trial includes biomarker-positive (PIK3CA mutant) and negative (wild type) cohorts, with initial wild type data expected late Q1 or Q2 2025 and mutant data in the second half of 2025.

• Frontline HR+, HER2- breast cancer study is set to enroll its first patients in Q2 2025; a prostate cancer study with darolutamide is ongoing.

• Phase 1 data showed a 63% objective response rate and median PFS of 12.9 months, with similar efficacy in both wild type and mutant cohorts.

• Nonclinical data suggest gedatolisib is equally potent in wild type and mutant tumors, unlike other pathway inhibitors.

Study design and regulatory strategy

• Trials are designed with separate biomarker cohorts to meet FDA requirements and reflect different standards of care.

• Enrollment split was 60% wild type and 40% mutant, slightly different from initial assumptions but not affecting statistical powering.

• Control arm for wild type is fulvestrant, with a median PFS benchmark of about three months; study is well powered for efficacy.

• Event-driven endpoints have led to a timeline extension due to enrollment dynamics, but full enrollment has now been achieved.

• Breakthrough therapy designation may enable accelerated NDA review if data are favorable, with preparations for a potential Real-Time Oncology or Priority Review.

Commercial opportunity and market strategy

• Addressable market for second-line post-CDK4/6 breast cancer is estimated at $5 billion, with a potential 40% penetration and $2 billion peak revenue.

• Frontline endocrine-resistant breast cancer market is estimated at $3 billion; early study initiation could add $1 billion in net present value.

• Sales force ramp-up is planned to begin 18 months before launch, with field force training starting a quarter before launch.