Core Insights - Celcuity Inc. has been granted U.S. Patent No. 12,350,276 for the clinical dosing regimen of its lead drug candidate, gedatolisib, in ER+/HER2- breast cancer patients, extending patent exclusivity until 2042 [1][2] - The company has a robust patent portfolio for gedatolisib, including 13 granted patents in the U.S. and 290 patents in foreign jurisdictions [2] - Celcuity plans to announce topline data for the PIK3CA wild-type cohort of the VIKTORIA-1 clinical trial in Q3 2025 and for the PIK3CA mutant cohort in Q4 2025 [3] Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for various solid tumor indications, with gedatolisib as its lead candidate [4] - Gedatolisib is a potent pan-PI3K and mTORC1/2 inhibitor that targets the PI3K/AKT/mTOR pathway, differentiating it from other therapies [4] - The company is currently conducting multiple clinical trials, including the Phase 3 VIKTORIA-1 trial for HR+/HER2- advanced breast cancer and the Phase 1/2 CELC-G-201 trial for metastatic castration-resistant prostate cancer [4]

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]

Core Insights - Celcuity Inc. announced preliminary clinical data for gedatolisib in two early phase clinical trials, demonstrating promising efficacy and safety profiles in treating metastatic cancers [1][4][5] Phase 1 Clinical Trial in mCRPC - In the Phase 1 trial evaluating gedatolisib plus darolutamide in men with metastatic castration resistant prostate cancer (mCRPC), the six-month radiographic progression-free survival (rPFS) rate was reported at 66% [1][4] - The trial involved 38 patients, with two arms receiving different doses of gedatolisib (120 mg and 180 mg) alongside darolutamide [2] - No patients discontinued treatment due to treatment-related adverse events (AEs), and less than 3% experienced Grade 3 stomatitis [4][5] Phase 2 Clinical Trial in HER2+ Metastatic Breast Cancer - In the Phase 2 trial for HER2+ metastatic breast cancer (mBC), the objective response rate (ORR) was 43% among 44 patients treated with gedatolisib plus trastuzumab-pkrb [1][6] - The median number of prior anti-HER2 therapies for enrolled patients was four or more, with 86% having received at least three prior treatments [6] - No patients discontinued treatment due to treatment-related AEs, and no Grade 3 hyperglycemia was reported [5][6] Future Directions - The company plans to amend the clinical trial protocol to explore additional dosing options for gedatolisib in the ongoing Phase 1/1b trial [4] - Up to six patients will be enrolled in each of three arms to determine the recommended Phase 2 dose (RP2D), with a total of approximately 30 subjects expected to be treated with the RP2D in the Phase 2 dose expansion study [4][5] - Celcuity is also conducting other clinical trials, including a Phase 3 trial evaluating gedatolisib in combination with fulvestrant for HR+/HER2- advanced breast cancer [7]

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]

Company Overview - Celcuity Inc. is a clinical-stage biotechnology company focused on developing targeted therapies for oncology, specifically for multiple solid tumor indications [2] - The company's lead therapeutic candidate, gedatolisib, is a potent pan-PI3K and mTORC1/2 inhibitor that blocks the PI3K/AKT/mTOR pathway comprehensively [2] - Celcuity is headquartered in Minneapolis and provides further information on its website [2] Clinical Trials - A Phase 3 clinical trial, VIKTORIA-1, is currently enrolling patients to evaluate gedatolisib in combination with fulvestrant with or without palbociclib for HR+/HER2- advanced breast cancer [2] - A Phase 1b/2 clinical trial, CELC-G-201, is ongoing to assess gedatolisib in combination with darolutamide for metastatic castration-resistant prostate cancer [2] - Another Phase 3 clinical trial, VIKTORIA-2, is recruiting patients to evaluate gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for HR+/HER2- advanced breast cancer [2] Investor Engagement - Brian Sullivan, CEO and Co-founder of Celcuity, will present at investor conferences and be available for one-on-one meetings [1] - Live webcasts of the presentations will be accessible on the company's website, with replays available shortly after the events [1] - Upcoming events include a fireside chat at the TD Cowen: 6th Annual Oncology Innovation Summit on May 28, 2025, and at the Jefferies Global Healthcare Conference on June 5, 2025 [3]

Clinical Trials and Development - Gedatolisib is currently being evaluated in multiple clinical trials, including the Phase 3 VIKTORIA-1 trial for HR+/HER2- advanced breast cancer, with the first patient dosed in December 2022[69]. - The VIKTORIA-1 trial aims to enroll 351 subjects with PIK3CA WT tumors, with topline data expected in Q3 2025, while enrollment for PIK3CA MT tumors is ongoing[70]. - The company has initiated a Phase 1b/2 trial (CELC-G-201) for gedatolisib in combination with darolutamide for patients with metastatic castration-resistant prostate cancer, with the first patient dosed in February 2024[71]. - Site activation for the VIKTORIA-2 Phase 3 trial is underway, with the first patient expected to be dosed in Q2 2025[76]. - The company initiated the VIKTORIA-1 Phase 3 clinical trial in 2022 and plans to report topline data in the fourth quarter of 2025[81]. - The company has initiated a second Phase 3 clinical trial, VIKTORIA-2, with the first patient dosing planned for the second quarter of 2025[82]. Financial Performance - For the three months ended March 31, 2025, the company reported a net loss of approximately $37.0 million, compared to a net loss of $21.6 million for the same period in 2024, representing a 71% increase in losses[89]. - Research and development expenses for the three months ended March 31, 2025, were approximately $32.2 million, an increase of $11.6 million or 56% compared to the same period in 2024[90]. - General and administrative expenses for the three months ended March 31, 2025, were approximately $3.9 million, reflecting an increase of $2.1 million or 112% compared to the same period in 2024[92]. - Interest expense for the three months ended March 31, 2025, was $3.2 million, an increase of $1.8 million or 127% compared to the same period in 2024[94]. - Net cash used in operating activities was approximately $35.9 million for the three months ended March 31, 2025, compared to $17.1 million for the same period in 2024[110][111]. - Net cash provided by investing activities was approximately $24.3 million for the three months ended March 31, 2025, compared to $3.5 million for the same period in 2024[112][113]. - Net cash provided by financing activities was approximately $5.6 million for the three months ended March 31, 2025, primarily from the exercise of 695,650 common stock warrants at an exercise price of $8.05[114]. - The company raised an aggregate of approximately $374.6 million of net proceeds through sales of its securities from inception through March 31, 2025[96]. - The company has an accumulated deficit of approximately $308.9 million as of March 31, 2025[79]. Revenue Potential and Market Position - The company estimates peak revenue potential for gedatolisib in the second-line indication could exceed $2 billion with 40% market penetration if FDA approval is obtained for both PIK3CA WT and MT populations[75]. - Gedatolisib's unique mechanism of action allows for comprehensive inhibition of the PI3K/AKT/mTOR pathway, potentially overcoming resistance seen with isoform-specific inhibitors[60]. - The company has exclusive global development and commercialization rights to gedatolisib under a license agreement with Pfizer, enhancing its competitive position in the market[59]. Safety and Efficacy - Gedatolisib has shown a high overall response rate (ORR) of 85% in treatment-naïve patients and 63% in all evaluable patients in the Phase 1b clinical trial[66]. - The median progression-free survival (PFS) for treatment-naïve patients receiving gedatolisib combined with palbociclib plus letrozole was 48.6 months[67]. - Gedatolisib's safety profile is favorable, with only 7% of patients experiencing Grade 3 or 4 hyperglycemia in clinical trials, compared to 39% for an FDA-approved oral PI3K inhibitor[63]. Future Expectations and Risks - The company expects research and development expenses to increase as it continues to develop gedatolisib and conduct multiple clinical trials[106]. - The company believes its current cash, cash equivalents, and short-term investments will be sufficient to finance clinical development activities through 2026[107]. - The company may seek to raise additional capital through various means, which could result in dilution to existing shareholders[108]. - The company anticipates increased costs associated with being a public company and plans to raise capital to fund its operations and expansion[120]. - The company faces known and unknown risks that may materially affect its results, including challenges in developing and commercializing gedatolisib[121]. - There are uncertainties and costs associated with clinical studies and the complexity of demonstrating safety and efficacy for regulatory approval[121]. - The company may encounter difficulties in managing growth, such as hiring and retaining qualified personnel[121]. - Changes in government regulations and tightening credit markets could impact the company's access to capital[121]. - The company has no obligation to update forward-looking statements unless required by law[122]. - As a smaller reporting company, the company is not required to provide disclosures about market risk[123].

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]

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 [21] - 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 [21] - Research and development expenses increased to $32.2 million in Q1 2025 from $20.6 million in Q1 2024, with a significant portion attributed to clinical trial activities [21][22] Business Line Data and Key Metrics Changes - The company is focused on three clinical programs, with expectations for significant revenue generation if regulatory approvals are achieved [6][7] - The VICTORIA-one trial is designed to evaluate gadotelisib in combination with fulvestrant, targeting patients with hormone receptor positive HER2 negative advanced breast cancer [8][10] - The VICTORIA-two trial is a global phase three study evaluating gadotelisib as a first-line treatment for similar patient populations [15][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 [7] - The peak revenue potential for the second-line indication 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 [9][10] - There is a focus on addressing the significant unmet need for effective therapies in advanced breast cancer, particularly for patients resistant to endocrine therapy [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving key clinical milestones, including data readouts from ongoing trials in 2025 [6][9] - The company recognizes the importance of demonstrating clinically meaningful results in terms of progression-free survival (PFS) to gain acceptance among oncologists [12][14] Other Important Information - The company ended the quarter with approximately $205.7 million in cash, cash equivalents, and short-term investments, expected to fund clinical development activities through 2026 [22][23] - The company is collaborating with Dana Farber Cancer Institute and Massachusetts General Hospital to evaluate gadotelisib in endometrial cancer [19] Q&A Session Summary Question: Timing for data readout from VICTORIA-one trial - Management indicated that the primary completion date is expected in June, with data available in Q3 2025, and typically, the time from locking the database to reporting data is no more than three months [27][30] Question: Impact of SERNA6 on second-line treatment - Management believes SERNA6 will not affect their trial as it pertains to first-line CDK4/6 patients, and they expect physicians to continue treatment with gadotelisib if data is favorable [36][38] Question: Minimum hazard ratio for wild type update - Management stated that an incremental three months in PFS would be considered clinically meaningful, but they are not providing specific projections at this time [39][40] Question: Change in timing for wild type readout - Management clarified that the timing change is due to the complexity of the three-arm trial, but they are confident about the Q4 timeline for the PIK3CA mutant population [44][46] Question: Prior proof of concept data for prostate cancer - Management discussed the encouraging data for gadotelisib in prostate cancer and indicated that the update will focus on primary analysis and safety data, with further data to be reported at a medical meeting [48][52]

Financial Performance - Net loss for Q1 2025 was $37.0 million, or $0.86 loss per share, compared to a net loss of $21.6 million, or $0.64 loss per share in Q1 2024[11]. - Non-GAAP adjusted net loss for Q1 2025 was $34.7 million, or $0.81 loss per share, compared to $19.9 million, or $0.59 loss per share in Q1 2024[11][23]. - GAAP net loss for Q1 2025 was $36.997 million, compared to a loss of $21.612 million in Q1 2024, representing a 71.2% increase in losses year-over-year[25]. - Non-GAAP adjusted net loss for Q1 2025 was $34.699 million, up from $19.904 million in Q1 2024, indicating a 74.5% increase in adjusted losses[25]. - GAAP net loss per share for Q1 2025 was $0.86, compared to $0.64 per share in Q1 2024, reflecting a 34.4% increase in loss per share[25]. - Non-GAAP adjusted net loss per share for Q1 2025 was $0.81, compared to $0.59 per share in Q1 2024, showing a 37.3% increase in adjusted loss per share[25]. Operating Expenses - Total operating expenses for Q1 2025 were $36.1 million, up from $22.5 million in Q1 2024, representing a 60% increase[8]. - Research and development expenses increased to $32.2 million in Q1 2025 from $20.6 million in Q1 2024, a rise of approximately 56%[9]. - Net cash used in operating activities for Q1 2025 was $35.9 million, compared to $17.1 million in Q1 2024[13]. Assets and Cash Position - Cash, cash equivalents, and investments totaled approximately $206 million as of March 31, 2025, expected to fund clinical development through 2026[5][13]. - Total current assets decreased to $217.6 million as of March 31, 2025, from $244.6 million at the end of 2024[19]. Clinical Trials - The topline data for the PIK3CA wild-type cohort of the VIKTORIA-1 trial is anticipated in Q3 2025, with the primary completion date projected for June 2025[5][6]. - Enrollment is ongoing in the PIK3CA mutant cohort of the VIKTORIA-1 trial, with topline data expected in Q4 2025[5][6]. - The VIKTORIA-2 Phase 3 trial is on track to dose its first patient in Q2 2025[5][6]. Shareholder Information - Weighted average common shares outstanding increased to 43,052,757 in Q1 2025 from 33,612,054 in Q1 2024, a growth of 28.0%[25].

Core Insights - Celcuity Inc. reported financial results for Q1 2025, highlighting progress in its clinical pipeline and upcoming data readouts [1][2] Business Highlights - The company anticipates topline data for the PIK3CA wild-type cohort of the VIKTORIA-1 trial in Q3 2025 and for the PIK3CA mutant cohort in Q4 2025 [2][5] - A clinical trial collaboration is being initiated with Dana Farber Cancer Institute and Massachusetts General Hospital to evaluate gedatolisib in combination with other therapies for endometrial cancer [5][6] Financial Results - Total operating expenses for Q1 2025 were $36.1 million, up from $22.5 million in Q1 2024 [7] - Research and development expenses increased to $32.2 million from $20.6 million year-over-year, with a significant portion attributed to employee and consulting costs [8] - General and administrative expenses rose to $3.9 million from $1.8 million, driven by increased employee and consulting expenses [9] Net Loss - The net loss for Q1 2025 was $37.0 million, or $0.86 loss per share, compared to a net loss of $21.6 million, or $0.64 loss per share, in Q1 2024 [10] - Non-GAAP adjusted net loss for Q1 2025 was $34.7 million, or $0.81 loss per share, compared to $19.9 million, or $0.59 loss per share, in the prior year [10][21] Cash Position - As of March 31, 2025, the company reported approximately $206 million in cash, cash equivalents, and investments, expected to fund clinical development activities through 2026 [11]