Celcuity Inc. · NASDAQ:CELC
Celcuity — Investor Presentation (August 2025)
50 slides from Celcuity's investor presentation, August 2025
Slide 1: Unlocking the Potential of Treating Cancers That Involve the PI3K/AKT/mTOR Pathway
Unlocking the Potential of Treating Cancers That Involve the PI3K/AKT/mTOR Pathway August 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains statements that constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to Celcuity’s business, operations, and financial condition, and include but are not limit
Slide 3: Topline VIKTORIA-1 PIK3CA Wild-Type Cohort Data
Topline VIKTORIA-1 PIK3CA Wild-Type Cohort Data In patients with HR+/HER2-/PIK3CA wild-type (WT) Gedatolisib Triplet advanced breast cancer (ABC), mPFS was 9.3 months vs. 2.0 months for fulvestrant gedatolisib plus fulvestrant and palbociclib (triplet) 7.3-month incremental improvement in mPFS
Slide 4: The Celcuity Opportunity
The Celcuity Opportunity Effectively treating PAM pathway driven tumors is one the largest opportunities in oncology 1 • Gedatolisib’s differentiated MOA and PK profile result in a highly potent, cytotoxic, and well tolerated PAM inhibitor 2 • Phase 3 VIKTORIA-1 WT cohort efficacy results showed unp
Slide 5: Unlocking the Potential of Treating Cancers That Involve the PI3K/AKT/mTOR Pathway
Unlocking the Potential of Treating Cancers That Involve the PI3K/AKT/mTOR Pathway Largest untapped drug One of the most important Most highly altered of all development opportunity oncogenic pathways signaling pathways1 in solid tumors PI3K/AKT/mTOR (PAM) Proportion of alterations Breast and prosta
Slide 6: Difficult to Safely and Comprehensively Inhibit the PAM Pathway
Difficult to Safely and Comprehensively Inhibit the PAM Pathway Optimal efficacy may require inhibition of all Class I PI3K isoforms and mTORC1 and mTORC2 Multiple pathway targets Therapeutic window for oral provide functional redundancy PI3K/mTOR inhibitors is narrow If only a single target is inhi
Slide 7: Gedatolisib Has a Highly Differentiated Mechanism of Action and Potency
Gedatolisib Has a Highly Differentiated Mechanism of Action and Potency Potential First-in-Class PAM (PI3K, AKT, mTOR) Inhibitor with superior cytotoxicity vs. single node PAM inhibitors Cell-Free Biochemical Dose Response Analysis Live Cell Proliferation Rate Dose Response Analysis7 IC50 (nM)1 Aver
Slide 8: Gedatolisib PK Properties and IV Administration Optimize Safety Profile
Gedatolisib PK Properties and IV Administration Optimize Safety Profile Lower toxicity vs. approved PI3K inhibitors Gedatolisib1 Alpelisib2,3 Copanlisib3 Duvelisib3 Idelalisib3 Gedatolisib vs. PI3K-α and pan-PI3K drugs Pan-PI3K (single-agents) Target(s) PI3K-α Pan-PI3K PI3K-δ PI3K-δ mTOR o >95% lowe
Slide 9: Gedatolisib Single Agent Safety Profile
Gedatolisib Single Agent Safety Profile Phase 1 Trial: gedatolisib at maximum tolerated dose (MTD) - 154 mg weekly (IV)1 MTD Arm (n=42) • Limited incidence of Grade 3 adverse events Related TEAE’s > 20% • The most frequent AE, stomatitis, is manageable Grade 1 Grade 2 Grade 3/4 with prophylactic ste
Slide 10: Safety Data for Gedatolisib vs. Single Node PAM Inhibitors
Safety Data for Gedatolisib vs. Single Node PAM Inhibitors Fewer patients reported AE when treated with gedatolisib compared to other PAM inhibitors gedatolisib alpelisib everolimus capivasertib Grade 1-2 (solid); Grade 3+ (dots) Percent of Subjects with AE 78 79 69 17 9 58 39 55 9 7 46 43 40 40 39
Slide 11: Clinical Development Programs
Clinical Development Programs Current 2nd Line HR+/HER2- Advanced 1st Line HR+/HER2- Advanced 2nd Line Metastatic Castration Breast Cancer Breast Cancer Resistant Prostate Cancer Phase 3 clinical trial for gedatolisib with Phase 3 clinical trial for gedatolisib + Phase 1b/2 clinical trial for gedato
Slide 12: The PAM Pathway is the Most Underdeveloped Target in Solid Tumors
The PAM Pathway is the Most Underdeveloped Target in Solid Tumors PAM is the most frequently altered pathway in PAM revenue potential comparable to solid tumors CDK4/6 & AR therapies $10.1B 38% $8.8B % Tumors 2022 Global w/Altered Revenues2 Pathways1 8% 5% 4% $0.5B Target PAM HER2 EGFR ALK Target PA
Slide 13: Multiple potential blockbuster indications in both tumor types
Multiple potential blockbuster indications in both tumor types HR+/HER2- Breast Cancer Advanced Prostate Cancer 156,880 US EU5+J 117,000 102,000 82,880 Addressable 91,000 93,000 Patient 72,000 62,000 Population 54,000 48,000 49,000 53,000 38,000 32,000 28,000 74,000 17,000 55,000 44,000 48,000 34,00
Slide 14: Key Gedatolisib Patents
Key Gedatolisib Patents Loss of exclusivity now expected to occur in 2042; expect new formulations to extend this period Subject Matter Patent Expiration Date Note Composition of Matter (API) Includes 209 days of patent term adjustment (PTA), and Dec 2034 (generic and species) expected 5 years of
Slide 15: Gedatolisib for Advanced Breast Cancer (ABC)
Gedatolisib for Advanced Breast Cancer (ABC) 15
Slide 16: ER, CDK4/6, & PI3K/mTOR are Interdependent Drivers of HR+/HER2- ABC
ER, CDK4/6, & PI3K/mTOR are Interdependent Drivers of HR+/HER2- ABC Dysregulation of these pathways promotes excessive cell proliferation and resistance to apoptosis ER and PI3K/mTOR CDK4/6, ER and PI3K/mTOR6-10 • Activation of the PAM pathway induces • Estrogen promotes cyclin D1 transcription estr
Slide 17: HR+/HER2- Breast Cancer Treatment Landscape
HR+/HER2- Breast Cancer Treatment Landscape1 ~30,000 women in US and ~33,000 women in 5EU and Japan die from breast cancer annually2 Localized and Regional Advanced and Metastatic Stage I-III Stage III (inoperable) or Stage IV Disease Low Recurrent Higher 1st 2nd 3rd 4th ~25% Recur Risk Recurrent Ri
Slide 18: Phase 3 VIKTORIA-1 2nd Line HR+, HER2- ABC
Phase 3 VIKTORIA-1 2nd Line HR+, HER2- ABC 18
Slide 19: Clinical Strategy: Simultaneous Blockade of PAM, ER, & CDK4/6 Pathways
Clinical Strategy: Simultaneous Blockade of PAM, ER, & CDK4/6 Pathways Clinical Hypothesis • Blockade of interdependent ER, PI3K, mTOR & CDK signaling pathways is required to optimize anti-tumor control • PAM inhibition:1-4 • Blockades PAM pathway and limits cross- activation when ER or CDK4/6 is in
Slide 20: VIKTORIA-1: Phase 3 Trial Design Overview for 2L HR+/HER2- ABC
VIKTORIA-1: Phase 3 Trial Design Overview for 2L HR+/HER2- ABC Arm A Gedatolisib + Palbociclib + Fulvestrant N = 117 PIK3CA Wild-type Arm B Primary Endpoints (WT) R Gedatolisib + Fulvestrant Topline PFS 1:1:1 N = 117 Reported A vs. C N = 392 B vs. C Arm C1 Fulvestrant Patients with N = 117 HR+/HER2-
Slide 21: VIKTORIA-1: Topline Efficacy Data
VIKTORIA-1: Topline Efficacy Data Both primary endpoints met; HR and incremental mPFS established new milestones in HR+/HER- ABC Topline Efficacy Milestones Achieved Gedatolisib Triplet Most favorable hazard ratio ever reported by any Phase 3 trial in mPFS was 9.3 months vs. 2.0 months for fulve
Slide 22: VIKTORIA-1: Additional Findings and Next Steps
VIKTORIA-1: Additional Findings and Next Steps Discontinuation rates and safety profile better than observed in Phase 1b study Additional Findings Next Steps The treatment discontinuation rates due to a TRAE for the gedatolisib triplet and doublet were lower than observed in Arm D of the Phase 1b
Slide 23: How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape?
How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape? Gedatolisib and approved regimens with Phase 3 data for 2L HR+/HER2-/PIK3CA WT post-CDK4/6i Patient Median Progression-Free Survival Population 2L Gedatolisib + Fulvestrant + Palbociclib 9.3 months 2L Gedatolisib + Fulvestrant 7.4
Slide 24: How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape?
How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape? Gedatolisib regimens showed highest incremental mPFS improvement versus endocrine therapy Patient Incremental mPFS improvement relative to endocrine therapy Population 2L Gedatolisib + Fulvestrant + Palbociclib Δ 7.3 months 2L Ged
Slide 25: How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape?
How Does the Gedatolisib Regimen Potentially Fit in the 2L Landscape? Hazard ratios for gedatolisib regimens are unprecedented Patient Hazard Ratio Population (lower is better) Geda + Fulv+ 2L 0.24 Palbo 2L Gedatolisib + Fulvestrant 0.33 2L Elacestrant 1 0.55 ESR1 MT 1L/2L Imlunestrant + Abemaciclib
Slide 26: Phase 1B Data: Geda-Triplet & SOC for 2L HR+/HER2-/PIK3CA MT ABC Post-CDKi
Phase 1B Data: Geda-Triplet & SOC for 2L HR+/HER2-/PIK3CA MT ABC Post-CDKi Data from PIK3CA MT Cohort of VIKTORIA-1 expected in late 2025 Patient Population 2nd Line ER+/HER2-/PIK3CA Mutant ABC 2L mPFS 12.9 months PIK3CA Gedatolisib + Fulvestrant + Palbociclib1 ORR 63% WT/MT Alpelisib + Fulvestrant
Slide 27: Phase 3 VIKTORIA-2 1st Line HR+, HER2- ABC
Phase 3 VIKTORIA-2 1st Line HR+, HER2- ABC 27
Slide 28: VIKTORIA-2: Phase 3 Study Features for 1L HR+/HER2- ABC
VIKTORIA-2: Phase 3 Study Features for 1L HR+/HER2- ABC Global open-label randomized study (~200 sites) Key eligibility criteria: • ER+/HER2- advanced or metastatic breast cancer Key Considerations • No prior treatment for advanced or metastatic breast cancer 1L endocrine treatment resistant pat
Slide 29: VIKTORIA-2: Phase 3 Trial Design Overview for 1L HR+/HER2- ABC
VIKTORIA-2: Phase 3 Trial Design Overview for 1L HR+/HER2- ABC Will conduct small safety run-in with gedatolisib plus ribociclib plus fulvestrant prior to Phase 3 Arm 1 Gedatolisib + CDK4/6i + Fulvestrant Patients with N = 319 Primary Endpoints HR+/HER2- ABC who Patients randomized 1:1 are treatment
Slide 30: Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)
Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)1 mPFS of 48.6 months, mDOR of 46.9 months, and ORR of 79% Treatment-Naïve Patients who are Endocrine Treatment Sensitive (ETS) (N=41) Escalation Arm A Expansion Arm A Total Treatment Naïve Progression-Free Survival n = 11 n =
Slide 31: Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)
Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)1 mPFS and ORR for treatment-naïve ETS patients compares favorably to published data for SOC palbociclib + letrozole2 Median Progression Free Survival Tumor Size Change 48.6 Months ORR = 79% (26/33) 100 90 % Change from Baseli
Slide 32: Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)
Phase 1B: Gedatolisib + Palbociclib + Letrozole in 1L HR+/HER2- ABC (N=41)1 mOS data for treatment-naïve patients ETS compares favorably to published data for current SOC Median Overall Survival 77.3 Months Overall survival (%) Relevant OS data in 1L setting Palbociclib + letrozole: 53.8 months2
Slide 33: Gedatolisib Combo vs. SOC for 1L HR+ / HER2- ABC (Endocrine Sensitive)
Gedatolisib Combo vs. SOC for 1L HR+ / HER2- ABC (Endocrine Sensitive) Gedatolisib Combo Offers Potential for Superior mPFS Compared to 1L SOC 1st Line HR+/HER2- ABC mPFS 48.6 months Gedatolisib + Palbociclib + Letrozole 1 ORR 79% mPFS 27.6 Months Palbociclib + Letrozole 2 ORR 55% Letrozole 2 mPFS 1
Slide 34: Relevant Comparisons to VIKTORIA-2 Control
Relevant Comparisons to VIKTORIA-2 Control B2151009 study results for 1L patients compares favorably to published data for 1L ETS patients Gedatolisib + Palbociclib + Palbociclib + Palbociclib + Letrozole Letrozole Fulvestrant N=41 1 N=441 2 N=164 3 WT / MT MT PIK3CA Status NR (76% / 22%) (100%) End
Slide 35: B2151009 Arm D: Safety Summary for Phase 3 Dosing
B2151009 Arm D: Safety Summary for Phase 3 Dosing G + P + F was well tolerated overall; < 4% discontinuation rate Arm D (n=27) • Discontinuation of gedatolisib due to AE - <4% Gedatolisib + Palbociclib + Fulvestrant • Alpelisib – 26% discontinued 1 (180 mg IV, 3 weeks on, one week off) • Everolimus
Slide 36: Gedatolisib for Prostate Cancer
Gedatolisib for Prostate Cancer 36
Slide 37: Androgen Signaling is the Key Driver of Prostate Cancer
Androgen Signaling is the Key Driver of Prostate Cancer The PI3K/AKT/mTOR (PAM) pathway helps promote excessive cell proliferation and resistance to apoptosis The AR Pathway is the The PAM Pathway Plays a Key Primary Therapeutic Target Role in mCRPC • The androgen receptor (AR) drives the • AR and P
Slide 38: Prostate Cancer Disease and Treatment Landscape
Prostate Cancer Disease and Treatment Landscape1,2 34,700 men in US and 62,400 men in 5EU and Japan die from prostate cancer annually3,4 nmCRPC non-metastatic castration resistant Localized Systemic Rising PSA mCRPC mCRPC Disease Prostate or Tumor Biochemical 1L 2L Stage Cancer recurrence Progressio
Slide 39: Limited Benefit for 2L HRR- mCRPC Patients After Treatment with AR Inhibitor
Limited Benefit for 2L HRR- mCRPC Patients After Treatment with AR Inhibitor Significant need for better therapeutic options st 1 Line Treatment Outcomes 2nd Line Treatment Outcomes (post AR inhibitor treatment) 20.0 mPFS 7.3 months 16.5 ORR 17% mPFS (mo) 6.7 5.6 5.6 5.5 Drugs 3.8 months Xtandi 1 Zy
Slide 40: Combining a PAM Inhibitor with an AR Inhibitor has Strong Scientific Rationale
Combining a PAM Inhibitor with an AR Inhibitor has Strong Scientific Rationale Biological parallels between mCRPC and HR+ ABC – PAM and hormonal pathway drive progression 1 PI3K/mTOR + AR Inhibition Treatment Rationale • Favorable clinical data in mCRPC with PAM inhibitors provides “proof-of-concept
Slide 41: CELC-G-201: Phase 1b/2 Trial Design Overview
CELC-G-201: Phase 1b/2 Trial Design Overview Evaluating gedatolisib plus darolutamide to determine preliminary safety and efficacyRP2D Dose Escalation Dose Expansion Determine RP2D, assess safety and tolerability Primary endpoint: rPFS6 Arm 1 Cohort A Gedatolisib 120 mg + Gedatolisib + Darolutamide
Slide 42: CELC-G-201: Preliminary Topline Data for Gedatolisib + Darolutamide
CELC-G-201: Preliminary Topline Data for Gedatolisib + Darolutamide rPFS6 for G + ARi (darolutamide) compares favorably to historical data for ARi monotherapy Treatment CELC-G-201 6-month rPFS Probability Regimen (trial) Arms 1 & 2 (N=38) rPFS6 66% Gedatolisib + ARi 66% (CELC-G-201) Discontinuation
Slide 43: Additional Early Phase Clinical Data
Additional Early Phase Clinical Data 43
Slide 44: Gedatolisib + Trastuzumab Biosimilar in 3L+ HER2+ ABC Patients (N=44)
Gedatolisib + Trastuzumab Biosimilar in 3L+ HER2+ ABC Patients (N=44) 43% objective response rate Best Response Duration of Response o 2 of 44 best response was a complete response (CR) o 17 of 44 best response was a partial response (PR) Source: Kim ASCO 2025. Note: Data presented is as of a cutoff
Slide 45: Gedatolisib + Paclitaxel + Carboplatin in Patients with Solid Tumors (N=17)
Gedatolisib + Paclitaxel + Carboplatin in Patients with Solid Tumors (N=17)1 65% ORR in all patients, 82% ORR in patients with ovarian cancer Ovarian Cancer (N=11) ORR: 82% Clear cell ovarian cancer (CCOC) (N = 10) ORR: 80% - 5/10 PR, 3/10 CR Low grade serous ovarian (N=1) 1/1 PR Other
Slide 46: Leading cancer KOLs are participating in our research
Leading cancer KOLs are participating in our research Mark Pegram M.D. Ph.D. Sara Hurvitz M.D. Ben Ho Park M.D., Ph.D. Adam Brufsky M.D., Ph.D. Lee Schwartzberg M.D. Clinical Advisory Board Hung Khong M.D. Bora Lim M.D. Mothaffar Rimawi M.D. Alberto Montero M.D. Scientific Advisory z Board Carol Lan
Slide 47: Leadership Team: Track Record of Developing Approved Therapies and Building Companies
Leadership Team: Track Record of Developing Approved Therapies and Building Companies Brian Sullivan Lance Laing, PhD Vicky Hahne Chief Executive Officer Chief Scientific Officer Chief Financial Office Co-Founder Co-Founder Igor Gorbatchevsky, MD Eldon Mayer Brent Eilefson Chief Medical Officer Chie
Slide 48: Upcoming Milestones
Upcoming Milestones Present full data for VIKTORIA-1 PIK3CA wild-type cohort at a major medical conference later this year Submit New Drug Application for VIKTORIA-1 PIK3CA wild-type cohort indication in Q4 2025 Report topline data for VIKTORIA-1 PIK3CA mutation cohort by end of 2025 48
Slide 49: The Celcuity Opportunity
The Celcuity Opportunity Effectively treating PAM pathway driven tumors is one the largest opportunities in oncology 1 • Gedatolisib’s differentiated MOA and PK profile result in a highly potent, cytotoxic, and well tolerated PAM inhibitor 2 • Phase 3 VIKTORIA-1 WT cohort efficacy results showed unp
Slide 50: Celcuity is focused on unlocking the potential of treating cancers that involve the PI3K/AKT/mTOR pathway
Celcuity is focused on Our third-generation cellular unlocking the potential of analysis platform unravels complex oncogenic activity treating cancers that involve molecular tests can’t detect. the PI3K/AKT/mTOR pathway We harvest these insights to develop new targeted therapies for cancer patients