Celcuity Inc. · NASDAQ:CELC
Celcuity — Investor Presentation (May 2022)
41 slides from Celcuity's investor presentation, May 16, 2022
Slide 1: Developing Potentially First-in-Class Rx using 3rd Generation Dx
Developing Potentially First-in- Class Rx using 3rd Generation Dx May 16, 2022
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: Developing Potentially First-in-Class Rx using 3rd Generation Dx
Developing Potentially First-in-Class Rx using 3rd Generation Dx Our CELsignia platform Detects oncogenic Enables discovery of Leveraging our platform creates a “movie” of pathway activity that new cancer drivers and to develop gedatolisib, a signaling activity in live molecular tests cannot expands
Slide 4: Gedatolisib is a Potential First-in-Class PI3K/mTOR Inhibitor
Gedatolisib is a Potential First-in-Class PI3K/mTOR Inhibitor Phase 3 expected to be initiated in mid-2022 for 2L HR+ / HER2- advanced breast cancer Highly Differentiated o First small molecule inhibitor of the PI3K/mTOR pathway administered intravenously Mechanism o Inhibits all isoforms of PI3K an
Slide 5: PI3K/mTOR is One of Most Important and Complex Oncogenic Pathways
PI3K/mTOR is One of Most Important and Complex Oncogenic Pathways Key oncogenic driver and resistance mechanism for multiple oncogenic pathways PIK3CA PTEN Loss Tumor type PI3K/mTOR regulates cell mutation or Mutated growth and metabolism ER+ BC1,2 ~39%1 ~46% Endometrial2 ~37% ~82% o Linked to multi
Slide 6: Difficult to Safely and Efficaciously Inhibit the PI3K/mTOR Pathway
Difficult to Safely and Efficaciously Inhibit the PI3K/mTOR Pathway Maximum efficacy requires inhibition of all Class 1 PI3K isoforms and mTORC1 and mTORC2 Multiple pathway components Therapeutic window for oral PI3K or must be targeted mTOR inhibitors is narrow o Feedforward and feedback loops betw
Slide 7: Gedatolisib Has a Highly Differentiated Mechanism of Action
Gedatolisib Has a Highly Differentiated Mechanism of Action Only pan-PI3K/mTOR inhibitor known to be under active development Gedatolisib vs. Approved Solid Tumor PI3Ki or mTORi IC50 (nM)1 Target Gedatolisib2 Alpelisib3 Everolimus4 Gedatolisib differentially targets one of the most important and com
Slide 8: Gedatolisib PK Properties vs. Other Approved PI3K Inhibitors
Gedatolisib PK Properties vs. Other Approved PI3K Inhibitors Differentiated chemical structure results in favorable PK profile and lower toxicity Gedatolisib1 Alpelisib2 Copanlisib2 Duvelisib2 Idelalisib2 Umbralisib2 Pan-PI3K PI3K-δ Gedatolisib vs. PI3K-α and pan-PI3K drugs Target(s) PI3K-α Pan-PI3K
Slide 9: Gedatolisib for Advanced Breast Cancer (ABC)
Gedatolisib for Advanced Breast Cancer (ABC) 9
Slide 10: Limited Benefit for 2nd Line HR+/HER2- ABC Patients Post-CDK4/6 Treatment
Limited Benefit for 2nd Line HR+/HER2- ABC Patients Post-CDK4/6 Treatment Finding more effective treatment for these patients is the biggest unmet need in breast cancer 2nd Line SOC HR+/HER2- Metastatic Breast Cancer (Post CDK4/6 inhibitor) Treatment guidelines recommend use of Treatment mPFS 1 sequ
Slide 11: Clinical Development Plan
Clinical Development Plan Phase 3 study for patients with HR+/HER2- Significant potential indications based on ABC who progressed on CDK4/6 therapy POC and nonclinical study data o Expect to initiate a pivotal Phase 3 clinical trial for ▪ Treating hormonally driven cancers has strong biological rati
Slide 12: Review of Preliminary Phase 1b Data
Review of Preliminary Phase 1b Data 12
Slide 13: PI3K/mTOR, ER, and CDK4/6 are Interdependent Signaling Pathways
PI3K/mTOR, ER, and CDK4/6 are Interdependent Signaling Pathways PI3K/mTOR is a key resistance mechanism to estrogen and CDK4/6 therapies Treatment Strategy o Simultaneously blocking interdependent ER, PI3K, mTOR & CDK signaling pathways in ER+ breast cancer addresses ER and CDKi resistance mechanism
Slide 14: B2151009: Phase 1b Study (138 patients)
B2151009: Phase 1b Study (138 patients) Dose escalation and safety/efficacy expansion (early signals of clinical activity) Expansion (4 Arms) Dose Escalation N = 103 (2 cohorts) Arm A N = 35 1st Line: palbo + letrozole + gedatolisib Letrozole Cohort palbociclib + letrozole + gedatolisib Arm B May 20
Slide 15: 63% ORR and 12.9 months PFS in Arm D with Phase 3 Dosing Schedule
63% ORR and 12.9 months PFS in Arm D with Phase 3 Dosing Schedule ORR and PFS was superior to SOC in each arm for their respective lines of therapy B2151009 Efficacy Summary (N=103) Patients 1L: CDKi-Naïve 2L+: CDKi-naïve 2L/3L: CDKi-pretreated 2L/3L: CDKi-pretreated A B C D Arm (N=31) (N=13) (N=32)
Slide 16: Gedatolisib + Palbociclib + Letrozole in 1st Line HR+/HER2- ABC
Gedatolisib + Palbociclib + Letrozole in 1st Line HR+/HER2- ABC (N=31)1 Data compares favorably to published data for SOC palbociclib + letrozole therapy from PALOMA-22 PFS Tumor Size Change 31.1 months ORR= 85% % CHANGE FROM BASELINE 20 0 -20 -40 -60 -80 -100 CR orange; PR = dark blue. PD/SD = ligh
Slide 17: Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line HR+/HER2- ABC
Gedatolisib + Palbociclib + Fulvestrant in 2nd/3rd Line HR+/HER2- ABC Patients Data from Phase 1b study with Phase 3 regimen (Arm D) compares favorably to published data with current SOC (N=27) PFS Tumor Size Change 12.9 months ORR= 63% 60 % CHANGE FROM BASELINE 40 20 0 -20 -40 * * -60 -80 ORR inclu
Slide 18: Adding Gedatolisib to Palbociclib + ET Resulted in Higher ORR
Adding Gedatolisib to Palbociclib + ET Resulted in Higher ORR (1.5-2.5x) Arm D vs. PALOMA-3 ORR and PFS results are particularly significant since PALOMA-3 patients were CDKi-naïve 1L 1L+ 2L/3L Patients CDKi-naïve CDKi-naïve Prior CDKi Study PALOMA-21 Arm A2 PALOMA-33 Arm D2 ▪ Arm A ORR 1.55 times h
Slide 19: Gedatolisib Combo vs. SOC Benchmarks for 2L HR+/HER2- ABC Post-CDKi
Gedatolisib Combo vs. SOC Benchmarks for 2L HER+ / HER2- ABC Post-CDKi Gedatolisib Combo Offers Potential for Superior Efficacy Compared to SOC Options 2nd Line ER+/HER2- MBC (post-CDKi) Drug Regimen Efficacy Gedatolisib + Palbociclib + Fulvestrant1 PFS 12.9 months (PIK3CA WT and MT patients) ORR 63
Slide 20: Arm D: Duration of Treatment in Patients' Refractory to Prior Therapy
Arm D: Duration of Treatment in Patients’ Refractory to Prior Therapy Gedatolisib treatment duration significantly greater than patient’s prior line of therapy Duration of Immediate Prior Treatment (DIPT) DIPT <180 Days DIPT <365 Days Arm D D # Evaluable patients with DIPT <185 or 365 days 7 (27%) 1
Slide 21: Arm D: High ORR Irrespective of Number of Prior Lines of Therapy
Arm D: High ORR Irrespective of Number of Prior Lines of Therapy Number of Prior Lines of Therapy for Advanced Disease > 2 Prior Lines 1 Prior Line # of Evaluable Patients 9 18 # of Partial Responses 7 10 Objective Response Rate 78% 56% Source: Layman 2021 SABCS Data presented for gedatolisib is fro
Slide 22: Safety Summary: Treatment-Emergent Adverse Events
Safety Summary: Treatment-Emergent Adverse Events G + P + ET was well tolerated overall; < 4% discontinuation rate with Phase 3 dosing (Arm D) Phase 1 Trial: Gedatolisib alone1 Phase 1b Trial – Arm D: G + P + F2 Phase 1b Trial – Arm D: G + P + F2 (154 mg weekly IV) (180 mg IV, 3 weeks, one week off)
Slide 23: 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 24: 56% ORR for Patients Receiving Gedatolisib + Trastuzumab Biosimilar
56% ORR for Patients Receiving Gedatolisib + Trastuzumab Biosimilar1 Best Response Duration of Response *Patient whose target lesion decreased by 63% but a new leptomeningeal seeding occurred. o 9 of 16 (56%) showed partial response (PR) Swimmer plot of the treatment duration o 4 of 16 (25%) had sta
Slide 25: Phase 3 Study Design VIKTORIA-1
Phase 3 Study Design VIKTORIA-1 25
Slide 26: Pivotal Trial Design Considerations for 2nd Line HR+/HER2- ABC
Pivotal Trial Design Considerations for 2nd Line HR+/HER2- ABC ▪ Standard-of-care 2nd line treatment differs based on PIK3CA status ▪ PIK3CA wildtype (WT): Fulvestrant or everolimus + exemestane ▪ PIK3CA mutated (MT): Alpelisib + fulvestrant ▪ 35% of patients have PIK3CA mutations in HR+/HER2- breas
Slide 27: VIKTORIA-1 Pivotal Phase 3 Trial Design Overview
VIKTORIA-1 Pivotal Phase 3 Trial Design Overview Arm A Gedatolisib + Palbociclib + Fulvestrant N = 117 PIK3CA Wild-type Arm B Primary Endpoints (WT) 65% Gedatolisib + Fulvestrant 2H ‘24 PFS N = 351 N = 117 A vs. C B vs. C Arm C1 Patients with Fulvestrant HR+/HER2- ABC N = 117 who received prior CDK4
Slide 28: Relevant Clinical Trial Results for VIKTORIA-1 Study Arms
Relevant Clinical Trial Results for VIKTORIA-1 Study Arms Each trial evaluated patients who received prior treatment with a CDK4/6 therapy Gedatolisib + Palbociclib Alpelisib + Alpelisib + Fulvestrant Fulvestrant + Fulvestrant N=1654 N=526 Fulvestrant Fulvestrant N=271 N=1267 N=1218 WT / M WT / MT P
Slide 29: VIKTORIA-1 Pivotal Study Features
VIKTORIA-1 Pivotal Study Features • Global open-label randomized study • Key eligibility criteria: • Any PIK3CA status Designed to support • Progressed on prior CDK4/6 treatment indications for gedatolisib and • Any menopausal status fulvestrant with or without • < 2 prior endocrine therapy and < 1
Slide 30: ~175 Sites Across 15+ Countries
~175 Sites Across 15+ Countries Europe (not Ukraine or Russia) North America Asia Pacific Group name 30
Slide 31: Experienced drug development team
Experienced drug development team CMO VP Clin Ops VP Pharma Dev VP Quality VP Program Mgmt. VP Medical Affairs Igor Gorbatchevsky, MD Nadene Zack Bernhard Lambert, PhD David Bridge Michael Snitkovsky Pratima Nayak, MD R Red Oak 31
Slide 32: 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 33: Celcuity Leadership Team
Celcuity Leadership Team Co-Founder and CEO Co-Founder and CSO CFO CBO Brian Sullivan Lance Laing, PhD Vicky Hahne Eric Lindquist CEO, Founder - PUR Water Scientist at Scriptgen/Anadys CFO – SimonDelivers (on-line 17% Global VP of BD at Natera Filters (purchased by Novartis) grocery) (Signatera) o S
Slide 34: Gedatolisib – A Phase 3 Ready Asset with Multiple Potential Indications
Gedatolisib – A Phase 3 Ready Asset with Multiple Potential Indications Phase 1b data in HR+/HER2- MBC reported better ORR and PFS than SOC in 1st and 2nd lines Compelling Efficacy in Multiple Potential Financial Key Milestones Advanced Breast Cancer Indications Resources Very promising results Nume
Slide 35: Live tumor cells contain infinitely more data than fragmented cells
Live tumor cells contain infinitely more data than the fragmented cells current cancer diagnostics use The CELsignia platform captures this data 35
Slide 36: Researchers recognize need for alternatives to genomic analysis
Researchers recognize need for alternatives to genomic analysis Complexity of signaling pathway networks requires much greater data to characterize than genomics can provide “It is becoming increasingly clear that pathways rather than individual genes govern the course of tumorigenesis.” Kornelia Po
Slide 37: CELsignia – the first 3rd generation diagnostic
– the first 3rd generation diagnostic Measures dynamic cell signaling activity to identify cancer drivers genomic tests cannot detect Live Tumor Cell Signaling Algorithmic Cells Isolated Quantified Analysis >100,000 patient tumor cells are Cell pathways are activated to A proprietary algorithm analy
Slide 38: Current Molecular Diagnostics vs. CELsignia – HER2 Example
Current Molecular Diagnostics vs. CELsignia – HER2 Example CELsignia identifies new sub-group of patients with HER2 driven cancer FISH HER2 Dx CELsignia HER2 Activity FISH+ CELsignia+ (1 pathway gene ) (4 hours of pathway signaling events) 15% 15%-20% $9 billion CELsignia identifies new $Billions ad
Slide 39: Key research discoveries drive test development
Key research discoveries drive test development CELsignia platform provides powerful tool to discover new cancer sub-types and mechanisms Specific target mutations Mutations often don’t lead to Drug resistance mechanisms (e.g. HER2+) not required for oncogenic signaling characterized oncogenic signa
Slide 40: CELsignia CDx identifies new patients for targeted therapies
CELsignia CDx identifies new patients for targeted therapies Pharma Pathway Targets Targeted Therapies Unique Patient Pool Impact Genentech Pfizer HER2 Herceptin $4B1 PI3K Perjeta Tepotinib available market Roche for Celcuity Nerlynx Piqray Merck c-MET 2x more Tepmetko Tafinlar Novartis MEK Aliqopa
Slide 41: Celcuity is a clinical stage biotechnology company
Celcuity is a clinical Our third-generation cellular stage biotechnology analysis platform unravels complex oncogenic activity company that molecular tests can’t detect. discovers previously undetectable cancer We harvest these insights to drivers and develops develop new targeted therapies drugs to