Autolus Therapeutics Plc · NASDAQ:AUTL
Autolus Therapeutics — Investor Presentation (June 2022)
44 slides from Autolus Therapeutics's investor presentation, June 2022
Slide 1: Developing Next Generation Programmed T Cell Therapies
Developing Next Generation Programmed T Cell Therapies June 2022
Slide 2: Disclaimer
Developing Next Generation Programmed T Cell Therapies Autolus.com Disclaimer These slides contain forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical
Slide 3: Autolus Overview
Developing Next Generation Programmed T Cell Therapies Autolus.com 3 Autolus Overview Building a fully integrated CAR T company Best-in-class Pipeline Scalable Collaboration lead asset manufacturing • Lead product obe-cel • Pipeline built on • In house cell • Collaboration with potentially best-in-
Slide 4: obe-cel
LEAD CLINICAL PROGRAM obe-cel A standalone, potentially best-in-class CD19 CAR T cell therapy
Slide 5: obe-cel has a unique mechanism of action
Autolus EHA Webinar Autolus.com 5 obe-cel has a unique mechanism of action • Fast off-rate obe-cel has a shorter half-life of interaction CD19 binder On-Rate: Ka [M-1S-1] ] 1 - Other CD19 Binders Obe-cel Binder compared to binders used in approved S with fast 1 - [M K a products : off-rate e Ra t •
Slide 6: obe-cel for adult ALL: high unmet need
Developing Next Generation Programmed T Cell Therapies Autolus.com 6 obe-cel for adult Acute Lymphoblastic Leukemia (ALL): high unmet need Successful therapy requires high level of activity and sustained persistence paired with good tolerability • Median overall survival is < 1 year in r/r adult ALL
Slide 7: obe-cel is a potentially transformational therapy for adult ALL
Developing Next Generation Programmed T Cell Therapies Autolus.com 7 obe-cel is a potentially transformational therapy for adult ALL Unique CAR T design drives differentiated product profile obe-cel • Unique mechanism of action built on a fast off-rate from CD19 target antigen Orphan Drug designatio
Slide 8: obe-cel shows consistent clinical profile across three clinical studies
Developing Next Generation Programmed T Cell Therapies Autolus.com 8 obe-cel shows consistent clinical profile across three clinical studies Data from 3 studies - range of ages and patient conditions CARPALL #1 ALLCAR19 #2 FELIX 1b #3 Peds ALL Adult ALL Adult ALL • obe-cel has a favourable n 14 20 1
Slide 9: obe-cel shows sustained event-free survival beyond months
Developing Next Generation Programmed T Cell Therapies Autolus.com 9 obe-cel shows sustained event-free survival beyond 30 months Long term CAR T persistence drives durability of effect Median CAR T cell levels in peripheral blood ALLCAR19 Event-Free Survival Median CAR T cell levels by flow cytomet
Slide 10: Unmet medical need in r/r adult ALL despite approved agents
Developing Next Generation Programmed T Cell Therapies Autolus.com 10 Unmet medical need in r/r adult ALL despite approved agents Current standard of care and recently approved agents in r/r adult ALL Standard of Care Recently FDA approved Blincyto1 Besponsa2 Tecartus3 N 271 109 54 ORR 44% 81% 65% E
Slide 11: obe-cel could launch into an expanding ALL market
Developing Next Generation Programmed T Cell Therapies Autolus.com 11 obe-cel could launch into an expanding ALL market Blincyto®, current market leader, shows annual revenue growth of 25% Reported Blincyto® sales1 • Blincyto® sales price estimated to be $178k3 (for 2 cycles) supporting approx. >2,0
Slide 12: Next steps: obe-cel initial data (FELIX) expected in H2 2022
Developing Next Generation Programmed T Cell Therapies Autolus.com 12 Next steps: obe-cel initial data (FELIX) expected in H2 2022 obe-cel is the first Autolus program to move into a pivotal program: full data in H1 2023 Primary endpoint: H2 2022 overall complete Pivotal Phase 2 trial in adult ALL I
Slide 13: Building obe-cel into a franchise
Building obe-cel into a franchise Deep value program with broad applicability
Slide 14: Capitalising on the unique profile of obe-cel
Developing Next Generation Programmed T Cell Therapies Autolus.com 14 Capitalising on the unique profile of obe-cel • Clinical data supports differentiated product profile ― High degree of activity and persistence -> drives long term outcomes ― Potential best-in-class safety profile -> will drive ad
Slide 15: EHA data: encouraging efficacy and duration of response in NHL/CLL
Developing Next Generation Programmed T Cell Therapies Autolus.com 15 EHA data: encouraging efficacy and duration of response in NHL/CLL Long term persistence drives durable outcomes • Of 20 patients evaluable for efficacy, the ORR was 18/20 N (%) (90%) Follicular Lymphoma CR + PR 7 (100%) • In the
Slide 16: Primary CNS Lymphoma: favorable tolerability profile
Autolus EHA Webinar Autolus.com 16 Primary CNS Lymphoma: favorable tolerability profile • No grade high grade CRS was observed using IV or intraventricular obe-cel Adverse Events of Special Interest administration Event N = 6 All Grades Grade 1 Grade 2 Grade 3 Grade 4 patients n (%) n (%) n (%) n (%
Slide 17: obe-cel shows excellent T cell expansion and engraftment
Autolus EHA Webinar Autolus.com 17 obe-cel shows excellent T cell expansion and engraftment Persistence of obe-cel demonstrated by qPCR CAR qPCR (copies/ug gDNA) 10 6 10 5 10 4 10 3 10 2 • Persistence in all treated patients at 10 1 last follow-up 10 0 10 -1 10 -2 M 2 M 3 M 4 M 5 6 6 10 17 21 M 8 5
Slide 18: obe-cel shows encouraging initial efficacy and durability in PCNSL
Autolus EHA Webinar Autolus.com 18 obe-cel shows encouraging initial efficacy and durability in PCNSL Overall response rate was 4/6 (67%) and these patients are without disease progression at last follow up N (%) Pre-obe-cel Post-obe-cel CR + PR 4 (67%) CR 2 (33%) (1 SD -> CR) PR 2 (33%) (1 SD -> PR
Slide 19: Summary and next steps for obe-cel
Developing Next Generation Programmed T Cell Therapies Autolus.com 19 Summary and next steps for obe-cel Building a franchise through broad applicability ✓ Favourable and consistent safety profile demonstrated across all indications ✓ Of patients evaluable for efficacy across MCL, DLBCL, FL and CLL
Slide 20: Pipeline
Pipeline A broad portfolio of next generation modular T cell therapies
Slide 21: A broad toolkit which is core to our strategy of modular innovation
Developing Next Generation Programmed T Cell Therapies Autolus.com 21 A broad toolkit which is core to our strategy of modular innovation Advanced T cell programming Viral Vector Advanced Targeting Modules Pharmacological Control Modules Activity Enhancement Modules Allogeneic Programming Modules Vi
Slide 22: Pipeline
Developing Next Generation Programmed T Cell Therapies Autolus.com 22 Pipeline Designed to address limitations of current T cell therapies PRODUCT INDICATION TARGET STUDY PRE PHASE 1 PHASE 2/ BLA CLINICAL PIVOTAL obe-cel Adult ALL CD19 FELIX obe-cel B-NHL & CLL CD19 ALLCAR19 Ext* obe-cel Primary CNS
Slide 23: AUTO1/22: Pediatric Acute Lymphoblastic Leukemia
Autolus EHA Webinar Autolus.com 23 AUTO1/22: Pediatric Acute Lymphoblastic Leukemia CD19 negative antigen escape is a common cause of treatment failure Screening for high sensitivity CARPALL Study CD22 binders n 14 CR Rate 86% 52% EFS 12m (95% CI, 16% to 72%) CD19 CD22 No. of CD19 negative relapses
Slide 24: AUTO1/22 in pediatric Acute Lymphoblastic Leukemia
Autolus EHA Webinar Autolus.com 24 AUTO1/22 in pediatric Acute Lymphoblastic Leukemia Patient characteristics Total n=11 (%) Median age at registration 12 yrs (range = 3.7-20.5) Indication Post SCT relapse 6 (55%, 3 isolated extramedullary) 1st relapse 2 (18%) 2nd relapse 8 (73%) >2nd relapse 1 (9%)
Slide 25: EHA data: AUTO1/22 in pediatric Acute Lymphocytic Leukemia
Developing Next Generation Programmed T Cell Therapies Autolus.com 25 EHA data: AUTO1/22 in pediatric Acute Lymphocytic Leukemia Phase 1 trial: Dose escalation trial – safety profile qPCR (blood) • AUTO1/22: reproducibly generate product balanced in CD19 vs CD22CAR with predominantly central memory
Slide 26: AUTO1/22 in pediatric Acute Lymphoblastic Leukemia
Autolus EHA Webinar Autolus.com 26 AUTO1/22 in pediatric Acute Lymphoblastic Leukemia Efficacy data Total N=11 Molecular MRD neg CR/Cri by d60 9 (82%) Disease progression 2 (18%) Events in responders 3 Emergence of molecular MRD 1 CD19+/CD22+ relapse 2 • No antigen-ve relapse was seen in responding
Slide 27: Summary for AUTO1/22 phase 1 study in r/r pALL
Autolus EHA Webinar Autolus.com 27 Summary for AUTO1/22 phase 1 study in r/r pALL Initial data presented at EHA, June 2022 ✓ CD19/CD22 targeting CAR T cells generated by co-transduction show robust expansion and persistence ✓ Favorable safety profile demonstrated, with no severe CRS ✓ Demonstration
Slide 28: AUTO4 and AUTO5 for Peripheral T-Cell Lymphoma
Autolus EHA Webinar Autolus.com 28 AUTO4 and AUTO5 for Peripheral T-Cell Lymphoma T-Cell Lymphoma is an aggressive disease with a very poor prognosis • A large portion of T-Cell Lymphoma patients are refractory/relapse 9,200 following first-line treatment (68%)3 new cases of T-Cell Lymphomas • Stand
Slide 29: Mature T cells express either TRBC1 or TRBC2
Developing Next Generation Programmed T Cell Therapies Autolus.com 29 Mature T cells express either TRBC1 or TRBC2 T-Cell Lymphomas are also clonal and express either TRBC1 or TRBC2 Structure of the T Cell Receptor or TCR Healthy T Cells Contain mixture of TRBC1+ cells TRBC2+ cells TRBC2+ normal T
Slide 30: TRBC1/TRBC2 targeting approach applicable to 95% of TCL subtypes
Autolus EHA Webinar Autolus.com 30 TRBC1/TRBC2 targeting approach applicable to 95% of TCL subtypes Patient subtypes addressable with TRBC1/2 targeting Distribution of cases by subtype • High and homogeneous expression of TRBC1 or TRBC2 is seen in the majority of TCL subsets - 95% of cases • TRBC1 a
Slide 31: Three key elements to address T-Cell Lymphomas
Developing Next Generation Programmed T Cell Therapies Autolus.com 31 Three key elements to address T-Cell Lymphomas A companion diagnostic: AUTO4 and AUTO5 Current Development > ~40% of T cell lymphomas are TRBC1+ Milestone AUTO4 – TRBC1+ TCL Phase 1 dose escalation Multiple Stratify into approache
Slide 32: Companion Diagnostic
Autolus EHA Webinar Autolus.com 32 Companion Diagnostic Multiple approaches de-risked for development Next Generation Sequencing Immunohistochemistry Flow Cytometry TRBC1 positive T-cell Lβx Vβx Dβ1 Jβ1x6 Cβ1 Dβ2 Jβ2x7 Cβ2 Prolymphocytic TRBC1 TRBC2 Leukemia 52x TRBC1 or TRBC2 NGS sequencing TRBC2 p
Slide 33: EHA data: Key Safety Data
Developing Next Generation Programmed T Cell Therapies Autolus.com 33 EHA data: Key Safety Data AUTO4 findings / clinical data Cohort 1 Cohort 2 Cohort 3 Cohort 4 Total 25x106 cells 75x106 cells 225x106 cells 450x106 cells (N = 3) (N = 2) (N = 1) (N = 4) (N = 10) Dose Limiting Toxicity (DLT) 0 0 0 0
Slide 34: Initial data encouraging
Autolus EHA Webinar Autolus.com 34 Initial data encouraging All patients treated at highest dose level had a complete metabolic response 450 x 106 cells 225 x 106 cells * 75 x 106 cells 25 x 106 cells Efficacy assessments were performed by the Investigators according to the Lugano Classification. †
Slide 35: PET scans for patient given 450 x 10^6 CAR T cells
Autolus EHA Webinar Autolus.com 35 PET scans for patient given 450 x 106 CAR T cells Patient refractory to prior therapies achieved a metabolic complete response Baseline Day 28 post-infusion 3 months post-infusion Data Cutoff: 26 April 2022
Slide 36: AUTO4 summary and next steps
Autolus EHA Webinar Autolus.com 36 AUTO4 summary and next steps • AUTO4 treatment generally well tolerated • Early efficacy is encouraging, particularly with all patients responding at higher dose levels • CAR T-cells detected in lymph node but no expansion observed in peripheral blood • Study ongoi
Slide 37: Manufacturing
Manufacturing
Slide 38: Manufacturing capabilities to accelerate assets into the clinic and beyond
Developing Next Generation Programmed T Cell Therapies Autolus.com 38 Manufacturing capabilities to accelerate assets into the clinic and beyond Building a fully integrated manufacturing and logistics platform that aims to bring assets to market Product Delivery • Highly skilled Autolus manufacturin
Slide 39: Blackstone Collaboration
Blackstone Collaboration
Slide 40: Blackstone Life Sciences to invest up to $250m to develop obe-cel in adult ALL
Developing Next Generation Programmed T Cell Therapies Autolus.com 40 Blackstone Life Sciences to invest up to $250m to develop obe-cel in adult ALL Investment of $100m in equity and up to $150 million in product financing • Blackstone agreed to purchase $100 million of Autolus’ American Depositary
Slide 41: Summary
Summary Multiple catalysts in H2 2022
Slide 42: Autolus poised for potential value inflection
Autolus EHA Webinar Autolus.com 42 Autolus poised for potential value inflection obe-cel pivotal data in adult ALL in 2022 • obe-cel – FELIX Phase 2 study in adult ALL ongoing; initial data expected in H2 2022 and full data in H1 2023 – Evaluation in r/r B-NHL and CLL ongoing – Evaluation in Primary
Slide 43: Autolus key newsflow timeline
43 Autolus key newsflow timeline EHA – June 2022 • obe-cel in r/r B-NHL and CLL (Phase 1) • obe-cel in primary CNS lymphoma (Phase 1) H2 2023 • AUTO4 in T Cell Lymphoma H2 2022 Potentially submit BLA for (interim Phase 1) FELIX Phase 2 initial data obe-cel in ALL • AUTO1/22 in pediatric ALL (Phase 1
Slide 44: Thank you
Thank you autolus.com
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