Carsgen Therapeutics Holdings Ltd. · HKEX:2171
Carsgen Therapeutics — Investor Presentation (June 2025)
53 slides from Carsgen Therapeutics's investor presentation, June 2025
Slide 1: CARsgen Therapeutics
CARsgen Therapeutics (HKEX: 02171) June 2025 Making Cancer Curable
Slide 2: Disclaimer
Disclaimer THIS DOCUMENT IS FOR INFORMATIONAL PURPOSES ONLY AND IS NOT INTENDED TO BE, AND SHALL NOT BE CONSTRUED AS, AN OFFER, INDUCEMENT, INVITATION, SOLICITATION, COMMITMENT OR ADVERTISEMENT WITH RESPECT TO THE PURCHASE, SUBSCRIPTION OR SALE OF ANY SECURITY AND NO PART OF IT SHALL FORM THE BASIS
Slide 3: We Develop Innovative and Differentiated Cell Therapies to Make Cancer and Other Diseases Curable
We Develop Innovative and Differentiated Cell Therapies to Make Cancer and Other Diseases Curable 1 Marketed product: 3 CAR-T Products at IND stage: 4+ Core technology platforms: • zevorcabtagene autoleucel • Satri-cel (Claudin18.2) • CycloCAR®, THANK-uCAR®, (zevor-cel, CT053) • CT011 (GPC3) THANK-u
Slide 4: Global Reach with Integrated R&D and Manufacturing Capabilities Complemented with Synergistic Partnership
Global Reach with Integrated R&D and Manufacturing Capabilities Complemented with Synergistic Partnership CGMP Facility Beijing Office Headquarter (Xuhui) Shanghai Headquarter, research, clinical development, GMP Durham Beijing commercial and clinical manufacturing facility Shanghai Durham, North Ca
Slide 5: Continuous Innovation and Technology Advancement to Tackle the Major Challenges with CAR-T therapies Since 2014
Continuous Innovation and Technology Advancement to Tackle the Major Challenges with CAR-T therapies Since 2014 Enabling Technologies Allogeneic CAR-T • THANK-uCAR®, THANK-u CycloCAR® Plus platforms co-expression of IL-7 + CCL21 LADAR® (precise targeting) Autologous CAR-T • BCMA CAR-T (zevor-cel) Ly
Slide 6: Competitive Product Pipeline with Global Rights
Competitive Product Pipeline with Global Rights Product BLA Target Indication Pre-clinical Phase I Phase II/III2 Candidate1 NDA R/R MM LUMMICAR 1 (China) launched Zevor-cel (CT053)3 BCMA R/R MM LUMMICAR 2 (US, Canada) G/GEJA ST-01 (China) Autologous GC/PC ST-02 (US, Canada) CAR-T Satri-cel (CT041) C
Slide 7: Autologous CAR-T Against Hematologic Malignancies
Autologous CAR-T Against Hematologic Malignancies
Slide 8: Zevor-cel: Differentiated Fully-human BCMA CAR-T for R/R MM (Approved in China)
Zevor-cel: Differentiated Fully-human BCMA CAR-T for R/R MM (Approved in China) Zevor-cel Highlights China Pivotal Phase II (LUMMICAR-1) 1 N=102 Follow-up, median (range), Month 20.3 (0.4-27) • Optimized scFv ISS stage III, No. (%) 39 (38.2%) • Enhanced binding affinity High risk Cytogenetic, No. (%
Slide 9: Zevor-cel: Outstanding Efficacy and Manageable Safety
Zevor-cel: Outstanding Efficacy and Manageable Safety ISS-based subgroups Higher safety, lower incidence 100 93.7% 93.7% Long-term survival with deep response 89.7% 87.2% of SAE 80 77.8% Response rate (%) 61.5% 60 40 It has been reported that ISS-III and high risk ◆ In IIT, Phase I, and Phase II stu
Slide 10: CT071: Differentiated GPRC5D CAR-T with CARcelerate Platform
CT071：Differentiated GPRC5D CAR-T with CARcelerate® Platform Product Clinical Development Status • Fully-human scFV generated by CARsgen • For relapsed R/R Multiple Myeloma or R/R Primary Plasma Cell Leukemia • Proprietary CARcelerate® platform • China investigator-initiated trial for R/R MM and Man
Slide 11: CT071: Baseline Characteristics
CT071: Baseline Characteristics 0.1 106 cells/kg 0.3 106 cells/kg All Patients Patient Characteristics (n=8) (n=9) (n=17) R-ISS disease stage, No. (%) II 4 (50.0) 8 (88.9) 12 (70.6) III 4 (50.0) 0 4 (23.5) ECOG PS, No. (%) 1 4 (50.0) 5 (55.6) 9 (52.9) 2 1 (12.5) 0 1 (5.9) Extramedullary Disease a, N
Slide 12: CT071: Early and Deep Responses with Promising Safety Profile
CT071: Early and Deep Responses with Promising Safety Profile 0.1 106 cells/kg 0.3 106 cells/kg All Patients (n=8) (n=9) (n=17) ORR, No. (%) 8 (100) 8 (88.9) 16 (94.1) CR/sCR rate, No. (%) 5 (62.5) 4 (44.4) 9 (52.9) VGPR or better rate, No. (%) 5 (62.5) 5 (55.6) 10 (58.8) Time to CR or better, Media
Slide 13: CT071: Rapid and Durable Responses
CT071: Rapid and Durable Responses • Seven patients achieved CR or better at week 4. # • All 4 patients with previous exposure to BCMA or BCMA/CD19 CAR-T responded (2 sCR and # 2 PR). • One patient with SD demonstrated ongoing tumor shrinkage of a large EMD (125 mm 99 * mm at baseline) with 38.2% de
Slide 14: Autologous CAR-T Against Solid Tumors
Autologous CAR-T Against Solid Tumors
Slide 15: Unmet Medical Needs in Solid Tumors, Including Gastric and Pancreatic Cancers
Unmet Medical Needs in Solid Tumors, Including Gastric and Pancreatic Cancers Incidence ~25.6K1 Incidence ~358.7K1 Gastric Resectable ~10.0K • • Resectable ~300.0K Cancer Mortality ~11.0K1 Mortality ~260.4K1 5-year survival rate of advanced GC is 5-20%; For advanced GC (3L+), ORR is 4.5%, mPFS < 2mo
Slide 16: CARsgen Proprietary Claudin18.2 IHC Test
CARsgen Proprietary Claudin18.2 IHC Test Claudin18.2 IHC test kit with high sensitivity Gastric Cancer Pancreatic Cancer Bile Duct Cancer Medium & high positivity rate Medium & high positivity rate Medium & high positivity rate (≥2+, ≥40%) (≥2+, ≥40%) (≥2+, ≥40%) 68% 55% 25% Confidential Copyrights
Slide 17: Satri-cel (CT041): Global First-in-Class CAR-T for Claudin18.2-Positive Solid Tumors
Satri-cel (CT041): Global First-in-Class CAR-T for Claudin18.2- Positive Solid Tumors Product Designations Clinical Development Plan • Breakthrough Therapy (NMPA) • Optimized scFv1 • GC (3L+) confirmatory Phase II trial in ✓ High binding affinity • RMAT (FDA) China achieved positive results; plan to
Slide 18: Satri-cel China Pivotal Phase II: Trial Design
Satri-cel China Pivotal Phase II: Trial Design An open-label, multicenter, randomized controlled trial conducted in China (CT041-ST-01). Study population n=104 Satri-cel Disease progression Bridging • 18-75 years of age Apheresis Lymphodepletion or intolerable toxicity, therapy (250 ×106 cells) etc.
Slide 19: Satri-cel China Pivotal Phase II: Patient Disposition
Satri-cel China Pivotal Phase II: Patient Disposition 266 patients were screened 110 excluded ITT population 156 randomly assigned (2:1) 104 assigned to satri-cel group 52 assigned to TPC group 48 received apheresis* 104 received apheresis 101 received bridging therapy 4 did not receive TPC 16 did n
Slide 20: Satri-cel China Pivotal Phase II: Baseline Characteristics
Satri-cel China Pivotal Phase II: Baseline Characteristics Satri-cel group TPC group Satri-cel group TPC group Characteristics Characteristics (n=104) (n=52) (n=104) (n=52) Age, median (IQR), years 53.5 (45.0, 60.0) 50.5 (43.0, 58.0) CLDN18.2 expression, n (%)† Sex, n (%) Medium expression 24 (23.1)
Slide 21: Satri-cel China Pivotal Phase II: Primary Endpoint—PFS by IRC
Satri-cel China Pivotal Phase II: Primary Endpoint—PFS by IRCa Satri-cel demonstrated statistically significant PFS improvement Events/patients 80/104 37/52 Median PFS, months 3.25 1.77 (95% CI) 2.86-4.53 (1.61-2.04) HR (95% CI) 0.366 (0.241-0.557) P value (one-sided) <0.0001 6-month PFS rate: 24% v
Slide 22: Satri-cel China Pivotal Phase II: Key Secondary Endpoint OS
Satri-cel China Pivotal Phase II: Key Secondary Endpoint OS Satri-cel demonstrated clinically meaningful OS benefit Satri-cel TPC Events/patients 70/104 35/52 Median OS, months 7.92 5.49 (95% CI) 5.78-10.02 (3.94-6.93) HR (95% CI) 0.693 (0.457-1.051) P value (one-sided) 0.0416 12-month OS rate: 35%
Slide 23: Satri-cel China Pivotal Phase II: PFS and OS in Treated Population
Satri-cel China Pivotal Phase II: PFS and OS in Treated Population In treated population (mITT), PFS per IRC and OS were obviously longer in Satri-cel group vs TPC group PFS assessed by IRCa OS in mITT population Satri-cel TPC Satri-cel TPC Events/patients 68/88 35/48 Events/patients 58/88 32/48 Med
Slide 24: Satri-cel China Pivotal Phase II: Adjusting OS for Treatment Switching in TPC
Satri-cel China Pivotal Phase II: Adjusting OS for Treatment Switching in TPC The estimated mOS was 1.81-2.06 fold longer with satri-cel vs TPC by RPSFT model, providing a 53% and 63% reduction in risk of mortality in the ITT and mITT populations, respectively. OS (ITT) analyzed by RPSFT model OS (m
Slide 25: Satri-cel China Pivotal Phase II: Manageable Safety
Satri-cel China Pivotal Phase II: Manageable Safety Satri-cel group (n=88) TPC group (n=48) Safety, n (%) All grade Grade ≥3 All grade Grade ≥3 All treatment-emergent adverse events 88 (100%) 87 (98.9%) 44 (91.7%) 30 (62.5%) (TEAEs) TEAEs related to treatment (TRAEs) 88 (100%) 87 (98.9%) 44 (91.7%)
Slide 26: Satri-cel China Pivotal Phase II: Conclusions
Satri-cel China Pivotal Phase II: Conclusions ✓ It is the world's first confirmatory randomized controlled trial (RCT) of a CAR-T cell therapy in solid tumors. It is also the first RCT in this field to demonstrate statistically superior efficacy on its primary endpoint. ✓ Satri-cel demonstrated stat
Slide 27: Satri-cel: Clinical Data from China and the US (Single-arm Study)
Satri-cel: Clinical Data from China and the US (Single-arm Study) China investigator-initiated Phase Ib in China Phase 1b in the US trial (NCT03874897)1,2 (NCT04581473)3 (NCT04404595)4 Sample size, No. 51 G/GEJA* 14 G/GEJA 7 G/GEJA 12 PC Median follow-up, Month 32.4* 8.8 8.9 ORR 54.9%* 57.1% 42.9% 1
Slide 28: Satri-cel: Extension to GC/PC Earlier Line / Adjuvant Settings
Satri-cel: Extension to GC/PC Earlier Line / Adjuvant Settings Promising greater clinical value in earlier line / earlier disease stage and providing better chances of cure for a much broader patient population More Accessible Tumor Better Tolerability • Low disease burden & • Mild CRS aggressivenes
Slide 29: Satri-cel as 1L Consolidation Delivered 100% Response Rate
Satri-cel as 1L Consolidation Delivered 100% Response Rate BOR of Satri- No. Age/Gender BOR of 1st line TTR cel Satri-cel Efficacy Highlights 1 50/F SD PR W4 • ORR 100% in 4 patients with target lesions, 2 55/F PR PR W4 TTR (Time-To-Response) Week 4 • 1 NN patient remained stable beyond 15 months 3
Slide 30: Following Satri-cel Infusion, Two Patients Underwent Surgical Resection, and Remain in Long-term Survival
Following Satri-cel Infusion, Two Patients Underwent Surgical Resection, and Remain in Long-term Survival at the Latest Follow-up Case 1 Case 2 1L Treatment 1L Treatment • Regimen: POS regimen (6 cycles) + S-1/Paclitaxel/TNF • Regimen: SOX regimen (4 cycles) intraperitoneal perfusion (4 cycles) • BO
Slide 31: Liver Cancer: The Third Leading Cause of Cancer Mortality Worldwide
Liver Cancer: The Third Leading Cause of Cancer Mortality Worldwide 2022 Liver Cancer Epidemiology in the US and China1 Incidence ~43.5K Incidence ~367.7K Mortality ~30.9K Mortality ~316.5K Liver Cancer 5-year survival rate Global2 US3 China4 Liver Cancer, all stages 18% 20% 12% 1. International Age
Slide 32: CT011: First-in-class CAR-T in Hepatocellular Carcinoma with PoC Clinical Results
CT011: First-in-class CAR-T in Hepatocellular Carcinoma with PoC Clinical Results GPC3: high expression and specificity CT011 • Overexpression in hepatocellular carcinomas (HCC), Product and is associated with poor disease prognosis ✓ an autologous GPC3 CAR-T product CARsgen’s GPC3 IHC test kit Clin
Slide 33: GPC3 CAR-T in Combination with Small Molecules Against HCC
GPC3 CAR-T in Combination with Small Molecules Against HCC: First publication in Molecular Therapy As of Dec 2021 (last follow-up at publication) • CR status has been over 24 months and continues (Photo taken in Jun 2023) 1. Wu X, Mol Ther. 2019 Aug 7;27(8):1483-1494 Confidential Copyrights reserved
Slide 34: CAR-T in Combination with Local Therapy Against HCC
CAR-T in Combination with Local Therapy Against HCC: Cover Story in Cancer Communications; Both Patients Achieved Disease-free Survival around 9 Years • Case: a 54-year-old male Diagnosed with Ib- tumor free survival Multiple Metastasis; stage HCC. Liver GKRS (> 5 years) GKRS and 2* MWA resection of
Slide 35: Next-Gen CAR-T Development: Tackling Key Challenges in Solid Tumors
Next-Gen CAR-T Development: Tackling Key Challenges in Solid Tumors Next-gen Claudin18.2 CAR-T shows enhanced anti- Next-gen NKG2DL CAR-T shows robust anti-tumor tumor activity in GC models activity in HCC models Satri-cel-derived Armored CAR-T demonstrates Proprietary CAR-NKG2DL T cells achieve 100
Slide 36: Allogeneic CAR-T Platforms and Pipeline Products
Allogeneic CAR-T Platforms and Pipeline Products
Slide 37: Existing Efficacy Gap between Allogeneic CAR-T and Autologous CAR-T
Existing Efficacy Gap between Allogeneic CAR-T and Autologous CAR-T Allogeneic BCMA CAR-T Autologous BCMA CAR-T Treatment and outcomes ALLO-715 P-BCMA-ALLO12 cilta-cel 3.2 x108 cells, N=241 All Arm**: 0.25-6 x106 cells/kg, Arm C**:2 x106 cells/kg 0.5-1 x106 cells/kg, N=973 N=72 N=23 23 (including 2
Slide 38: Relatively Limited Expansion of Allogeneic CAR-T in Patients
Relatively Limited Expansion of Allogeneic CAR-T in Patients • In allogeneic CAR-T, donor T cells will be recognized and rapidly eliminated by the host immune system (HvGR), which impacts CAR-T cell survival and results in limited expansion. • Compared to autologous CAR-T, in vivo persistence of all
Slide 39: THANK-uCAR: Innovative Allogeneic CAR-T Platform Aimed to Address Immune Rejection
THANK-uCAR® : Innovative Allogeneic CAR-T Platform Aimed to Address Immune Rejection Target and Hinder the Attack of NK cells on Universal CAR-T cells (THANK-uCAR®) HvGR is the major challenge faced by Allogeneic CAR-T • B2M knockout to overcome HvGR from host T cells, but NK cells attack uCAR-T cel
Slide 40: CT0590 (BCMA CAR-T, THANK-uCAR): Baseline Characteristics and Outcomes from the IIT
CT0590 (BCMA CAR-T, THANK-uCAR® ): Baseline Characteristics and Outcomes from the IIT • An open-label, single-arm, phase 1, first-in-human trial in China (NCT05066022). • Lymphodepletion: F: Fludarabine (30mg/m2/day x 3days), C: Cyclophosphamide (500 mg/m2/day x 3 days). • Doses: 50 106, 150 106, 30
Slide 41: CT0590: Manageable Safety Profile, Deep and Durable Responses
CT0590: Manageable Safety Profile, Deep and Durable Responses Safety Efficacy • Two patients experienced CRS • 3 subjects achieved confirmed responses including 2 ✓ One patient each at Grade 1 and with sCR and 1 with PR. 1 Patient achieved PR but it Grade 2; no ≥ Grade 3 CRS; could not be confirmed
Slide 42: A Case of CT0590 to Treat R/R MM
A Case of CT0590 to Treat R/R MM Baseline Characteristics • A 71-year-old male diagnosed with MM, • Double-refractory, with 94.5% plasma cells in bone marrow. • 2 prior lines of therapies, including 3 regimens. • Received 3×108 CT0590 CAR-T cells infusion. 482749 Safety • 1 Grade CRS • Only 1 subjec
Slide 43: A Case of CT0590 to Treat R/R pPCL
A Case of CT0590 to Treat R/R pPCL Baseline Characteristics 280863 • A 52-year-old male diagnosed with pPCL • Double-refractory • 3 prior lines, including 3 regimens (ASCT, etc.) • Received an infusion of 3×108 CT0590 CAR-T cells. Safety • 1 Grade CRS • Grade 1 infection (pneumonia), unrelated to tr
Slide 44: Baseline NKG2A Expression on NK cells may be Predictive of CT0590 Responses
Baseline NKG2A Expression on NK cells may be Predictive of CT0590 Responses % Baseline Patient Dose NKG2A Best overall (Diagnosis) (cells) expression response • 4 patients had baseline NKG2A data available. NK cells • Both patients who attained sCR, Patient 2 and PT 1 (MM) 50 106 Patient 5, had rela
Slide 45: CT0596: Allogeneic BCMA-Targeted CAR-T (THANK-u Plus)
CT0596: Allogeneic BCMA-Targeted CAR-T (THANK-u Plus ) THANK-u Plus Platform Clinical Development • THANK-u Plus exhibits significantly improved • CT0596 is under evaluation in an IIT for the expansion compared to THANK-uCAR® treatment of R/R MM or R/R PCL: • THANK-u Plus demonstrates sustained ✓ As
Slide 46: CT0596: Enhanced and Sustained Allogeneic CAR-T Expansion Across Different NKG2A Expression Levels
CT0596: Enhanced and Sustained Allogeneic CAR-T Expansion Across Different NKG2A Expression Levels NKG2A expression: High Medium Low • CT0590 (THANK-uCAR®): exhibits a decrease in expansion within 72 hours in NK cells with relatively low NKG2A expression. • CT0596 (THANK-u Plus ): ✓ In the presence
Slide 47: CT0596 IIT Preliminary Data: Favorable Safety and Efficacy
CT0596 IIT Preliminary Data: Favorable Safety and Efficacy • CT0596 demonstrated favorable tolerability: Safety ✓ NO ≥Grade 3 CRS ✓ NO ICANS or GvHD ✓ NO DLTs, no patients discontinuing treatment due to AE • As of May 6, 2025, 8 R/R MM patients (3L+) received infusion (Lymphodepletion: fludarabine 2
Slide 48: Allogeneic CD19/20X CAR-T (THANK-u Plus) Exhibits Robust Anti-lymphoma Activity in the Presence of NK Cells
Allogeneic CD19/20X CAR-T (THANK-u Plus ) Exhibits Robust Anti-lymphoma Activity in the Presence of NK Cells uUTD+NK uCD19/20 uCD19/20+NK CD19/20X CD19/20X +NK Week 1 Week 2 Week 3 Week 4 Death Week 5 Death In the presence of NK cells, allogeneic CD19/20X CAR-T (THANK-u Plus platform) demonstrates m
Slide 49: KJ-C2320, Allogeneic CD38 CAR-T (THANK-uCAR) Exhibits Enhanced Antitumor Activity in Mice
KJ-C2320, Allogeneic CD38 CAR-T (THANK-uCAR® ) Exhibits Enhanced Antitumor Activity in Mice in the Presence of NK Cells uUTD uCD38 uCD38+NK KJ-C2320 KJ-C2320+NK Week 1 Week 2 Week 3 Week 4 Death In the presence of NK cells, allogeneic CD38 CAR-T (THANK-uCAR® platform) demonstrates much better anti-t
Slide 50: Summary of CARsgen's Allogeneic CAR-T Platform
Summary of CARsgen’s Allogeneic CAR-T Platform • Allogeneic CAR-T products are currently in development: CT0596 – targeting BCMA, for R/R MM and R/R PCL, an IIT is ongoing. KJ-C2219 – targeting CD19/CD20, for B-cell malignancies, an IIT is ongoing; for SLE and SSc, an IIT is ongoing. KJ-C2320
Slide 51: Multiple Value Inflection Milestones in the Near Future
Multiple Value Inflection Milestones in the Near Future • H1 2025: Planned NDA submission for satri-cel to the NMPA. • H2 2025: Anticipated data release for satri-cel in pancreatic cancer adjuvant therapy. • H2 2025: Anticipated IND application for CT0596. Anticipated disclosure of CT0596 clinical d
Slide 52: Experienced Senior Management Team
Experienced Senior Management Team Zonghai Li, MD, PhD Huamao Wang, PhD Hua Jiang, MD, PhD Co-founder, Chairman of Co-founder and Vice President, the Board, CEO, CSO COO Early Discovery Yi Luo, MD, PhD Nishan Andy (Peng) Zang, PhD Vice President, Clinical Rajakumaraswamy, MD Vice President, Head of
Slide 53: Making Cancer Curable
Making Cancer Curable oS cARSGEN
CARsgen Therapeutics — Investor Presentation (June 2025) on 6ix