Gedatolisib: A Novel PAM Inhibitor - Gedatolisib, a pan-PI3K/mTOR inhibitor, shows potential as a highly potent and cytotoxic agent with a differentiated mechanism of action and pharmacokinetic profile[7, 23] - Gedatolisib has demonstrated compelling preliminary results in HR+/HER2- advanced breast cancer (ABC) patients, with a median progression-free survival (mPFS) of 48 months in 1st line and 12.9 months in 2nd line settings[7, 23] - Gedatolisib is more potent against each node than other PAM inhibitors, with >300X higher potency and 1.5x – 2.8x higher cytotoxicity than other PAM inhibitors in vitro[30] - Gedatolisib has a lower rate of Grade 3/4 hyperglycemia (>95% lower) and treatment-related discontinuations (>80% lower) compared to approved PI3K inhibitors[31] Clinical Development and Market Opportunity - Celcuity is conducting a Phase 3 study in 2nd line HR+/HER2- ABC patients and expects to begin enrolling a Phase 3 study in 1st line patients in Q2 2025[7, 158] - The PAM pathway is the most frequently altered pathway in solid tumors (38%), yet drug revenues from PAM inhibitors are a small fraction of other targeted therapy classes[10, 18] - The potential patient population for PAM inhibitors in breast and prostate cancers is estimated to be >500,000 in the US, EU5, and Japan[10] - US market opportunity for Gedatolisib in HR+/HER2- Breast Cancer and Advanced Prostate Cancer is estimated at ~$5-$6 Billion for 2L ABC Post-CDKi + AI, ~$10B+ for 1L ABC ET Sensitive, ~$3B for 1L ABC ET Resistant, $6-$8B for High Risk EBC Adjuvant, $8B+ for 1L/2L mCRPC Post-ARi, $10B+ for 1L mCRPC, $6-$8B for nmCRPC, $10B+ for mHSPC[52] Financial Position - Celcuity has cash, cash equivalents, and short-term investments of $205 million as of Q1 2025, expected to fund operations through 2026[8, 159]
Celcuity (CELC) Earnings Call Presentation