Summary of the Oncology Innovation Summit - CellCuity Fireside Chat Company Overview - **Company**: CellCuity - **Focus**: Development of a platform to quantify signaling pathway activity in live tumor cells, specifically targeting the PI3K AKT mTOR pathway, referred to as the PAM pathway, which is a significant oncogenic pathway and represents a large untapped drug development opportunity in solid tumors [4][5] Key Assets and Studies - **Lead Asset**: Gadotelisib, a pan-PI3K inhibitor - **Ongoing Studies**: - Phase 3 study in second-line patients with ER-positive, HER2-negative advanced breast cancer, data expected in Q3 for wild type cohort and Q4 2025 for PIK3CA mutant cohort [5][6] - Phase 3 study for first-line HR-positive, HER2-negative advanced breast cancer, enrollment expected to begin this quarter [6] - Phase 1b study in prostate cancer for metastatic castration-resistant prostate cancer, top-line data expected later this month [6] Differentiation in the Market - **Mechanism of Action**: Unlike other inhibitors that target single components of the PAM pathway, CellCuity's approach addresses multiple targets, which is crucial due to feedback loops that can lead to adaptive resistance [9][10] - **Potency**: Gadotelisib is reported to be 300 times more potent than other approved single-target PAM inhibitors and is the only drug effective in both tumor cells with and without PIK3CA mutations [11][12] Clinical Expectations and Comparisons - **Hazard Ratio (HR)**: A hazard ratio of 0.5 or below is considered compelling for the upcoming phase 3 wild type data [20] - **Comparative Studies**: Previous studies have shown HRs ranging from 0.59 to 0.68, with no study reporting an HR below 0.5 in similar patient populations [20][21] - **Phase 1b Data**: In the phase 1b study, 60% of patients had an objective response, with 49% progression-free at 12 months, indicating favorable results compared to existing therapies [38][39] Safety Profile - **Adverse Events**: Gadotelisib has shown a lower incidence of hypoglycemia compared to other drugs in the class, with a discontinuation rate of 4% in early studies versus over 26% for other drugs [48][49] - **Patient Tolerance**: Preliminary data suggests good tolerance among patients, with no discontinuations due to adverse events related to hypoglycemia [48][49] Market Opportunity and Administration - **Efficacy vs. Safety**: The primary factors influencing physician decisions will be efficacy and safety, with convenience being a secondary consideration [51] - **Infused vs. Oral Drugs**: Infused drugs are more common in oncology, and while oral drugs offer convenience, they can lead to compliance issues due to side effects [54][55] - **Financial Considerations**: Infused drugs may have less cumbersome reimbursement processes compared to oral drugs, which can expose patients to high co-pays [54][55] Conclusion - The upcoming data in Q3 and Q4 is highly anticipated, with expectations for significant insights into the efficacy and safety of CellCuity's lead asset, gadotelisib, in treating advanced breast cancer and prostate cancer [56]

Financial Data and Key Metrics Changes - The company's net loss for Q1 2025 was $37 million, or $0.86 per share, compared to a net loss of $21.6 million, or $0.64 per share for Q1 2024 [20] - Non-GAAP adjusted net loss was $34.7 million, or $0.81 per share for Q1 2025, compared to a non-GAAP adjusted net loss of $19.9 million, or $0.59 per share for Q1 2024 [20] - Research and development expenses increased to $32.2 million for Q1 2025 from $20.6 million in Q1 2024, with a significant portion attributed to clinical trial activities [20][21] - General and administrative expenses rose to $3.9 million for Q1 2025 from $1.8 million in Q1 2024 [21] - Net cash used in operating activities was $35.9 million for Q1 2025, compared to $17.1 million for Q1 2024 [22] - The company ended the quarter with approximately $205.7 million in cash, cash equivalents, and short-term investments [22] Business Line Data and Key Metrics Changes - The company is focused on three clinical programs, with significant revenue potential if regulatory approvals are obtained [7][8] - The Phase III VICTORIA-one trial is designed to evaluate gadotelisib in combination with fulvestrant for advanced breast cancer patients [9] - The VICTORIA-two trial is a global Phase III study evaluating gadotelisib as a first-line treatment for HR positive, HER2 negative advanced breast cancer [15][16] - The Phase 1B2 trial is assessing gadotelisib in combination with darolutamide for metastatic castration-resistant prostate cancer [17] Market Data and Key Metrics Changes - The company estimates that nearly 200,000 late-stage cancer patients globally would be eligible for treatment with gadotelisib if approved [8] - The peak revenue potential for the second-line indication of gadotelisib could exceed $2 billion with just 40% market penetration [15] Company Strategy and Development Direction - The company aims to transition to a commercial stage company following potential FDA approvals for its clinical programs [10] - The focus is on developing effective therapies for advanced breast cancer patients resistant to endocrine therapy, addressing a significant unmet need in the market [16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming clinical data readouts, with top-line data expected from the VICTORIA-one trial in Q3 2025 [7][10] - The company recognizes the importance of demonstrating clinically meaningful results in terms of progression-free survival (PFS) to gain market acceptance [12][14] Other Important Information - The company is collaborating with Dana Farber Cancer Institute and Massachusetts General Hospital to evaluate gadotelisib in endometrial cancer [18] - The company expects its current cash reserves to fund clinical development activities through 2026 [22] Q&A Session Summary Question: Timing for VICTORIA-one data readout - Management expects to lock the database in June and report results in Q3 2025, with no anticipated delays [27][28][30] Question: Impact of SERNA6 on second-line setting - Management believes SERNA6 will not affect their patient population as it pertains to first-line CDK4/6 patients [36][37] Question: Minimum HR for wild type update - Management indicated that an incremental three months in PFS would be considered clinically meaningful, but specifics will not be disclosed until data is available [39][40] Question: Change in timing for wild type readout - Management clarified that variability in event distribution among trial arms influenced the timing, but they remain confident in the Q4 timeline for the PIK3CA mutant population [43][44] Question: Prior proof of concept data for prostate cancer - Management discussed the encouraging non-clinical data supporting the efficacy of gadotelisib compared to single-node inhibitors [47][49]