Celcuity Inc. · NASDAQ:CELC
Celcuity — Investor Presentation (May 2025)
54 slides from Celcuity's investor presentation, May 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 May 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial condition, including but not limited to current beliefs, expectations and assumptions rega
Slide 3: The Celcuity Opportunity
The Celcuity Opportunity Significant untapped potential to effectively treat PAM pathway involved cancers 1 • Gedatolisib’s differentiated MOA and PK profile result in a highly potent, cytotoxic, and well tolerated PAM inhibitor 2 • Very compelling data in 1L (mPFS 48 months) and 2L (mPFS 12.9 month
Slide 4: 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 5: 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 6: 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 7: Gedatolisib is a Potential First-in-Class PAM Inhibitor
Gedatolisib is a Potential First-in-Class PAM (PI3K, AKT, mTOR) Inhibitor Breakthrough Therapy Designation granted for 2L HR+/HER2- advanced breast cancer indication Differentiated Compelling Potential to Address Well-Tolerated Mechanism of Action Preliminary Results Large Unmet Need • Inhibits all
Slide 8: Gedatolisib Has a Highly Differentiated Mechanism of Action and Potency
Gedatolisib Has a Highly Differentiated Mechanism of Action and Potency Results in superior cytotoxicity vs. single node PAM inhibitors Cell-Free Biochemical Dose Response Analysis Live Cell Proliferation Rate Dose Response Analysis7 IC50 (nM)1 Average values for 14 PIK3CA MT and 14 PIK3CA WT breast
Slide 9: 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 10: 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 11: 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 12: 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 13: Addressable Patient Population in Breast and Prostate Cancer
Addressable Patient Population in Breast and Prostate Cancer US, EU5, and Japan Patient Populations 615,000 Metastatic HSPC Aggregate Patient Populations 458,000 404,000 nmCRPC nmCRPC HR+/HER2- HR+/HER2- HR+/HER2- Stage II/III Stage II/III Stage II/III 288,000 Early BC Early BC Early BC Metastatic M
Slide 14: 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 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: Limited Benefit for 1st Line ET Resistant or 2nd Line HR+/HER2- ABC Patients
Limited Benefit for 1st Line ET Resistant or 2nd Line HR+/HER2- ABC Patients Significant need for better therapeutic options st 1 Line Treatment Outcomes 2nd Line Treatment Outcomes (post CDK4/6 treatment) 27.6 25.3 mPFS 14.5 (mo) 7.3 7.3 5.5 3.8 1.9 Palbociclib Ribociclib Palbociclib 3.8 +months Al
Slide 19: Review of Phase 1b Data
Review of Phase 1b Data Gedatolisib + Palbociclib + Fulvestrant/Letrozole 19
Slide 20: 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 21: Phase 1b Dose Escalation and Expansion Study (B2151009)
Phase 1b Dose Escalation and Expansion Study (B2151009) Key eligibility criteria: patients with HR+, HER2-, advanced breast cancer Expansion (gedatolisib 180 mg) N = 103 Dose Escalation Arm A (gedatolisib 180 mg weekly) 1st Line: N = 35 palbo + letrozole + gedatolisib (weekly) Letrozole Cohort Arm B
Slide 22: B2151009 Expansion Arms: Baseline Characteristics
B2151009 Expansion Arms: Baseline Characteristics Arm A Arm B Arm C Arm D (N=31) (N=13) (N=32) (N=27) Tumor, Node, Metastasis (TNM) Current Stage, n (%) Stage IV 31 (100) 13 (100) 32 (100) 27 (100) Prior therapies for ABC, n (%) Prior Chemotherapy 1 (3.2) 4 (30.8) 15 (46.9) 5 (18.5) Prior Endocrine
Slide 23: ORR and PFS in Each Expansion Arm Was Superior to SOC
ORR and PFS in Each Expansion Arm Was Superior to SOC Results from Arm D - 63% ORR and 12.9 months PFS – provide basis for Phase 3 clinical trial B2151009 Expansion Arms Efficacy Summary (N=103) Arm A Arm B Arm C Arm D 2L+ 2L/3L 2L/3L Prior Therapy 1L CDKi-naive CDKi-pretreated CDKi-pretreated n (Fu
Slide 24: Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line HR+/HER2- ABC Patients
Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line HR+/HER2- ABC Patients mPFS and ORR from Arm D with Phase 3 regimen compares favorably to published data for current SOC Median Progression Free Survival Tumor Size Change 12.9 Months ORR = 63% (17/27) 100 Progression free survival (%) 80 60 40
Slide 25: Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line: Overall Survival
Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line HR+/HER2- ABC Patients mOS from Arm D with Phase 3 regimen compares favorably to published data for current SOC Median Overall Survival 33.9 months1 Relevant OS data in 2L post-CDK4/6 setting Overall survival (%) Alpelisib + fulvestrant: 27.3
Slide 26: 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 27: Gedatolisib Combo and SOC Data for 2L HR+ / HER2- ABC Post-CDKi
Gedatolisib Combo and SOC Data for 2L HR+ / HER2- ABC Post-CDKi Gedatolisib Combo Offers Potential for Superior Efficacy Compared to Alternatives Patient Population 2nd Line ER+/HER2- ABC mPFS 12.9 months All Gedatolisib + Fulvestrant + Palbociclib 1 ORR 63% mPFS 8.0 months PIK3CA+ Alpelisib + Fulve
Slide 28: Efficacy in Treatment-Naïve Population Superior to SOC
Efficacy in Treatment-Naïve Population Superior to SOC mPFS of 48.6 months, mDOR of 46.9 months, and ORR of 79% B2151009 Treatment-Naïve Patients (N=41) Escalation Arm A Expansion Arm A Total Treatment Naïve Progression-Free Survival n = 11 n = 30 n = 41 (full analysis set) 45.8 48.6 48.6 Median PFS
Slide 29: Gedatolisib + Palbociclib + Letrozole in 1st Line HR+/HER2- ABC
Gedatolisib + Palbociclib + Letrozole in 1st Line HR+/HER2- ABC (N=41)1 mPFS and ORR for treatment-naïve 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 Baseline 80 Pr
Slide 30: Gedatolisib Combo vs. SOC for 1L HR+ / HER2- ABC
Gedatolisib Combo vs. SOC for 1L HR+ / HER2- ABC 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 14.5 mos ORR 44% Source
Slide 31: Gedatolisib + Palbociclib + Letrozole in 1st Line: Overall Survival
Gedatolisib + Palbociclib + Letrozole in 1st Line HR+/HER2- ABC Patients mOS data for treatment-naïve patients 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 PALOMA
Slide 32: Phase 3 Study Designs VIKTORIA-1 and VIKTORIA-2
Phase 3 Study Designs VIKTORIA-1 and VIKTORIA-2 32
Slide 33: VIKTORIA-1: Trial Design Considerations for 2nd Line HR+/HER2- ABC
VIKTORIA-1: Trial Design Considerations for 2nd Line HR+/HER2- ABC • Standard-of-care 2nd line treatment is based on PIK3CA status Supports design with multiple • ~35-40% of patients have disease with PIK3CA mutations primary endpoints in different sub-groups • PFS is accepted primary end point for
Slide 34: VIKTORIA-1: Phase 3 Study Features for 2L HR+/HER2- ABC
VIKTORIA-1: Phase 3 Study Features for 2L HR+/HER2- ABC Global open-label randomized study (>200 sites) Key eligibility criteria: • ER+/HER2- advanced or metastatic breast cancer Phase 3 vs. Phase 1b Arm D Key Eligibility Criteria Differences • Prior CDK4/6i + NSAI • Bone-only with measurable lesi
Slide 35: 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 Q3 PFS 1:1:1 N = 117 ’25 A vs. C N = 351 B vs. C Arm C1 Fulvestrant Patients with N = 117 HR+/HER2- ABC who P
Slide 36: Relevant Comparisons to VIKTORIA-1 Controls
Relevant Comparisons to VIKTORIA-1 Controls B2151009 study results compared to published data for patients who received a prior CDK4/6i Gedatolisib + Palbo + Alpelisib + Alpelisib + Fulvestrant Fulvestrant Fulvestrant Fulvestrant Fulvestrant Fulvestrant N=1653 N=375 N=1216 N=271,2 N=1268 N=1219 Tria
Slide 37: 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 38: 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 39: 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 40: Clinical Trial Results Provide POC in this 1L ABC Patient Population
Clinical Trial Results Provide POC in this 1L ABC Patient Population1 Results for a less potent PAM inhibitor in small fraction of population highlights opportunity for gedatolisib Line of Patient Overall Results Study Regimens N Comments Therapy Population (Months rPFS) Inavolisib shows clinical
Slide 41: Gedatolisib for Prostate Cancer
Gedatolisib for Prostate Cancer 41
Slide 42: 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 43: 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 44: 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 45: 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 46: CELC-G-201: Phase 1b/2 Trial Design Overview
CELC-G-201: Phase 1b/2 Trial Design Overview Evaluating gedatolisib combined with darolutamide, a potent next generation androgen receptor inhibitor Phase 1b Phase 2 Determine RP2D, assess Dose Expansion safety and tolerability Arm 1 Gedatolisib 120 mg + Darolutamide 600 mg BID Primary Endpoint: N =
Slide 47: Clinical Trial Results Provide POC for PAM Inhibitors in 2L mCRPC post ARi
Clinical Trial Results Provide POC for PAM Inhibitors in 2L mCRPC post ARi Less potent PAM inhibitors combined with AR an inhibitor reported favorable results Line of Patient Overall Results Study Regimens N Comments Therapy Population (Months rPFS) 10.5 vs. 5.5 months Samotolisib efficacious desp
Slide 48: Additional Early Phase Clinical Data
Additional Early Phase Clinical Data 48
Slide 49: Gedatolisib + Paclitaxel + Carboplatin in Patients with Solid Tumors
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 50: Gedatolisib + Trastuzumab Biosimilar in 3L+ HER2+ ABC Patients
Gedatolisib + Trastuzumab Biosimilar in 3L+ HER2+ ABC Patients (N=17) 59% ORR and 83% clinical benefit rate Best Response Duration of Response * * Target lesion decreased by 63% but a new leptomeningeal seeding occurred. o 10 of 17 (59%) showed partial response (PR) o Median duration of response 7.1
Slide 51: 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 52: 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 53: The Celcuity Opportunity
The Celcuity Opportunity Significant untapped potential to effectively treat PAM pathway involved cancers 1 • Gedatolisib’s differentiated MOA and PK profile result in a highly potent, cytotoxic, and well tolerated PAM inhibitor 2 • Very compelling data in 1L (mPFS 48 months) and 2L (mPFS 12.9 month
Slide 54: Celcuity is focused on unlocking the potential of treating cancers
Celcuity is focused on Our third-generation cellular unlocking the potential analysis platform unravels complex oncogenic activity of treating cancers that molecular tests can’t detect. involve the PI3K/mTOR pathway We harvest these insights to develop new targeted therapies for cancer patients 54