Syncona Ltd GBP · LSE:SYNC
Syncona Ltd GBP — Achilles Therapeutics — Investor Presentation (November 2023)
23 slides from Syncona Ltd GBP's investor presentation, November 2023
Slide 1: Achilles Therapeutics
Achilles Therapeutics Precision Cell Therapy Targeting All Tumor Cells November 2023 Non-Confidential © Achilles Therapeutics plc 2023
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains “forward-looking statements,” including statements regarding the proposed development plans and timelines for the Company’s product candidates and the success, cost and timing of its research activities and clinical trials. Forward-looking statem
Slide 3: Clinical-stage precision targeting for solid tumors using clonal neoantigen-reactive T cells
Clinical-stage precision targeting for solid tumors using clonal neoantigen-reactive T cells (cNeT) Targeting clonal neoantigens: a novel class of cancer target present on all tumor cells We have developed a proprietary patent protected AI platform (PELEUS®) that is validated on real world patient d
Slide 4: Working to transform the treatment of solid tumors with precision T cell therapy
Working to transform the treatment of solid tumors with precision T cell therapy Company Nasdaq IPO: Early clinical Clinical founded ACHL proof of concept Update 2016 2021 2022 Q1 2024 Global Headquarters Two active clinical Catapult Cell and Gene Therapy Centre London, UK programs with near-term St
Slide 5: Unlocking efficacy in the solid tumors through precision targeting
Unlocking efficacy in the solid tumors through precision targeting T cells Present on all cancer Clonal neoantigens: cells & absent from the ideal cancer target healthy tissue Validated method to Clonal neoantigens PELEUS: a proprietary identify immunogenic AI capability Sub-clonal neoantigens targe
Slide 6: Experienced leadership with decades in cell therapy drug development
Experienced leadership with decades in cell therapy drug development Sergio Quezada Karl Peggs CSO CMO Iraj Ali CEO Robert Coutts Daniel Hood CFO General Counsel Shree Patel Ed Samuel EVP, Patient Supply EVP, Technical Operations Operations Jim Taylor CBO Non-Confidential © Achilles Therapeutics plc
Slide 7: Differentiated pipeline of precision T cell therapies across multiple solid tumors
Differentiated pipeline of precision T cell therapies across multiple solid tumors Upcoming IND enabling Phase I/II Phase III Milestones Lung Cancer Clinical data update (NSCLC) CHIRON monotherapy Q1 2024 Melanoma Clinical data update Monotherapy THETIS & CPI combo Cohort A & Cohort B Q1 2024 Non-Co
Slide 8: Clonality has been convincingly demonstrated in the landmark TRACERx study
Clonality has been convincingly demonstrated in the landmark TRACERx study Sub-clonal neoantigens Clonal neoantigens Clonal neoantigens remain as genetic diversity increases Achilles can identify Original, clonal Tumors constantly clonal mutations for mutations passed evolve and acquire each patient
Slide 9: Clinical evidence supports clonal neoantigens as the best targets in the solid tumors
Clinical evidence supports clonal neoantigens as the best targets in the solid tumors Clonals are the only known targets present on all Multiple clinical modalities validate neoantigens tumor cells & absent from healthy tissue but only clonals drive overall survival Tumor Neoantigen-reactive T cells
Slide 10: TRACERx is a unique asset that enables Achilles' neoantigen identification capability
TRACERx is a unique asset that enables Achilles’ neoantigen identification capability 819 patients enrolled with early stage to advanced NSCLC and followed up to nine years Early-stage disease Late-stage metastatic Largest longitudinal real-world patient data set of its kind1-4 Extensive sequencing
Slide 11: PELEUS: A patent protected world-leading AI-platform
PELEUS™: A patent protected world-leading AI-platform for identifying the most potent and immunogenic targets Superior clonal calling: only platform to use multi-region analysis proven to overcome limitations of traditional VAF based methods1 PELEUS Most immunogenic targets: our proprietary and vali
Slide 12: Clonal neoantigens can be targeted with a range of therapeutic modalities
Clonal neoantigens can be targeted with a range of therapeutic modalities TIL-based cNeT Blood-based cNeT Current Achilles approach Clinically Blood as source of validated across cNeT, without the multiple solid need for surgery tumor settings TCR-therapy Clonal neoantigen vaccines Alternative modal
Slide 13: Multiple lines of clinical evidence show neoantigen reactive T cells driving efficacy in TIL
Multiple lines of clinical evidence show neoantigen reactive T cells driving efficacy in TIL TIL-based studies have demonstrated impressive clinical responses in multiple solid tumour settings1-3 Targeting neoantigens has been Improved clinical outcomes in Efficacious TIL products found shown to dri
Slide 14: Achilles process delivers precision clonal neoantigen targeting T cell therapy (cNeT)
Achilles process delivers precision clonal neoantigen targeting T cell therapy (cNeT) Cutting edge personalized genomics and machine learning enable targeting of all cancer cells Tumor T cells PELEUS Clonal Neoantigen- reactive T cell (cNeT) product Genomic analysis identifies targets present on all
Slide 15: Two studies open in advanced NSCLC and melanoma
Two studies open in advanced NSCLC and melanoma Monotherapy Advanced NSCLC Evaluating safety, tolerability • Advanced unresectable or metastatic Stage III-Stage IV NSCLC and activity (RECIST) and CHIRON • Never-smokers and EGFR/ALK/Ros-1 mut excluded biomarkers of clinical activity • Open-label Ongo
Slide 16: VELOS manufacturing process delivering median 144M cNeT to patients in 2023
VELOS™ manufacturing process delivering median 144M cNeT to patients in 2023 VELOS Manufactured Cell Doses Process 1 Process 2 10 B 1B Pre-Expansion Optimized upfront TIL T cells are harvested extraction + cytokines Median = 144M from the tumor boost for tumor-reactive 100 M Reactive TIL cells3 T ce
Slide 17: cNeT were generally well tolerated in the fourteen patients treated in CHIRON & THETIS
cNeT were generally well tolerated in the fourteen patients treated in CHIRON & THETIS Heavily pretreated patients with advanced cancer cNeT tolerability profile1 • Eight advanced unresectable or • Tolerability similar cNeT to standard tolerability profileTIL 1 metastatic NSCLC patients (CHIRON) • L
Slide 18: CHIRON (NSCLC) patients dosed with best response of PR and SD
8 CHIRON (NSCLC) patients dosed with Best Response of PR and SD1 100 Best response to cNeT PR Best Key SD Response 50 PD Early proof-of-concept demonstrated in Sum of target lesion % NSCLC change from baseline § Disease control at >12 weeks observed in 5 0 of 7 evaluable patients (71%), including on
Slide 19: Patient C-17: 56% reduction in total target lesion size vs. baseline at week 36
Patient C-17: 56% reduction in total target lesion size vs. baseline at week 36 Target lesion (TL) size1 Re-screening Week 6 Week 12 mm, C-17 Lesion 1 50 Target TL1 45 TL2 40 35 30 Lesion 2 Target 25 20 15 10 5 Week 18 Week 24 Week 36 0 Lesion 1 Re-screening Week 12 Week 18 Week 24 Week 36 Week 6 Ta
Slide 20: Patient C-17: cNeTs expand and persist coincident with tumor regression
Patient C-17: cNeTs expand and persist beyond week 12 coincident with tumor regression Clonal neoantigen peptide-reactive cells in blood (normalized spot count) Detection of T cell receptors from the product vs. blood IL-6 level (pg/ml) % of sample, C-17 Clonal NeoAg peptide reactive cells TCR clone
Slide 21: Efficient scale-up of GMP manufacturing to align with clinical and commercial need
Efficient scale-up of GMP manufacturing to align with clinical and commercial need Flexible manufacturing GMP facilities at Royal Free Identified and initiated tech allows efficient alignment Hospital in London and Catapult transfer to CDMO in of scale-up site in Stevenage, UK support Philadelphia,
Slide 22: Focus for CHIRON and THETIS data readouts
Focus for CHIRON and THETIS data readouts Dose 15-20 additional patients in 2023 & deliver data in Q1 2024 with cNeT monotherapy (lung & melanoma) and CPI combo (melanoma) Clinical Drive additional confirmed responses in CHIRON and THETIS patients with higher cNeT doses Translational Leverage world-
Slide 23: Achilles Therapeutics
Achilles Therapeutics AI-Powered Precision Cell Therapy Targeting All Tumor Cells November 2023 Non-Confidential © Achilles Therapeutics plc 2023
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