Autolus Therapeutics Plc · NASDAQ:AUTL
Autolus Therapeutics — Investor Presentation (October 2023)
37 slides from Autolus Therapeutics's investor presentation, October 2023
Slide 1: Developing Next Generation Programmed T Cell Therapies
Developing Next Generation Programmed T Cell Therapies October 2023
Slide 2: Disclaimer
Developing Next Generation Programmed T Cell Therapies 2 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 facts, and
Slide 3: Building a leading CAR T company developing transformational therapies for cancer and autoimmune diseases
Developing Next Generation Programmed T Cell Therapies 3 Building a leading CAR T company developing transformational therapies for cancer and autoimmune diseases Established excellence in R&D and Manufacturing; scaling company toward commercialization $ Obe-cel approaching Pipeline expansion Scalab
Slide 4: Obe-cel
LEAD CLINICAL PROGRAM Obe-cel A standalone, potentially best-in-class CD19 CAR T cell therapy candidate
Slide 5: Obe-cel has a unique mechanism of action
Developing Next Generation Programmed T Cell Therapies 5 Obe-cel has a unique mechanism of action Designed for increased activity and reduced toxicity Differentiated CD19 binder Fast off-rate Shorter half-life of interaction compared On-Rate: Ka [M-1S-1] ]1 CD19 binder with - - S1 Other CD19 Binders
Slide 6: FELIX data at ASCO and EHA 2023
Developing Next Generation Programmed T Cell Therapies 6 FELIX data at ASCO and EHA 2023 Trial design and patient baseline characteristics Total infused Key eligibility criteria Baseline Characteristics (N = 94) • R/R adult B-ALL* • Aged ≥18 years Age years, median (range) 50 (20–81) • ≥5% BM blasts
Slide 7: FELIX: disease response per IRRC assessment
Developing Next Generation Programmed T Cell Therapies 7 FELIX: disease response per IRRC assessment 76% of infused patients achieved CR/CRi 100 90 80 70 21.3% 60 CRi ORR: 76% ORR, % 50 95% CI (66, 84) 40 p<0.0001* 30 54.3% 20 CR 10 0 97% of responders with evaluable samples were MRD negative at 10-
Slide 8: FELIX: duration of remission
Developing Next Generation Programmed T Cell Therapies 8 FELIX: duration of remission 61% responders in ongoing remission without subsequent anti-cancer therapies 100 90 Median follow-up: 9.5 months 80 70 Probability, % 60 50 40 30 20 10 Events, n = 18; Median DoR: 14.1 months (95% CI: 5.9, NE) 0 0
Slide 9: FELIX: CRS and ICANS profile
9 Developing Next Generation Programmed T Cell Therapies 99 FELIX: CRS and ICANS profile Low rates of Grade ≥3 CRS and/or ICANS were observed BM blasts ≤20% BM blasts >20% All infused patients at pre-conditioning at pre-conditioning (N = 37) (N = 57) (N = 94) CRS Any grade, n (%) 24 (64.9) 47 (82.5)
Slide 10: FELIX: subgroup analysis of CR/CRi (IRRC assessment)
Developing Next Generation Programmed T Cell Therapies 10 FELIX: subgroup analysis of CR/CRi (IRRC assessment) Benefits observed across all patient subgroups; high risk subgroups include EMD and high BM blasts at pre-conditioning Total ORR Subgroup N (%) % (95% CI) Overall 94 (100) 76 (66, 84) Age,
Slide 11: FELIX: obe-cel expansion and persistence
Developing Next Generation Programmed T Cell Therapies 11 FELIX: obe-cel expansion and persistence CAR T cellular kinetics are consistent with the ALLCAR19 study1 Mean (SE) for CAR T therapy by PCR in peripheral blood FELIX ALLCAR19 100,000 (N = 94) (N = 20) Cmax, copies/ug 114,982 127,152 copies/ug
Slide 12: Obe-cel Phase 1 long term follow up demonstrates durable responses
Developing Next Generation Programmed T Cell Therapies 12 Obe-cel Phase 1 long term follow up demonstrates durable responses Long term follow up from Phase 1 ALLCAR19 study in r/r ALL of up to 4 years ALLCAR19 Swim plot ALLCAR19 Median persistence Median CAR T-cell levels by PCR • Of the 20 infused
Slide 13: FELIX: conclusions
Developing Next Generation Programmed T Cell Therapies 13 FELIX: conclusions • CR/CRi rate of 76%, with 97% of responders becoming MRD negative – With a median of 9.5 months’ follow-up, 61% of responders remain in remission • Very low rates of Grade ≥3 CRS (3.2%) and low rates of Grade ≥3 ICANS (7.4
Slide 14: ALL: unmet need and market overview
ALL: unmet need and market overview
Slide 15: Obe-cel could launch into an expanding ALL market if approved
Developing Next Generation Programmed T Cell Therapies 15 Obe-cel could launch into an expanding ALL market if approved Blincyto®, current market leader, sales increased 48% year-over-year to $206 million for the second quarter 2023 Reported Blincyto® sales1 • Blincyto® sales price estimated to be $
Slide 16: Over 8,000 new cases of adult ALL annually worldwide
Developing Next Generation Programmed T Cell Therapies 16 Over 8,000 new cases of adult ALL annually worldwide 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 • Combination chemotherapy
Slide 17: Critical drivers for market adoption
Developing Next Generation Programmed T Cell Therapies 17 Critical drivers for market adoption PRODUCT PROFILE TREATMENT EXPERIENCE Durable and robust response Timely & reliable product supply • CR/CRi rate of 76%, with 97% of responders • Quality product with low out-of-spec rates becoming MRD nega
Slide 18: Commercial Launch Readiness
Commercial Launch Readiness
Slide 19: Product supply
Developing Next Generation Programmed T Cell Therapies 19 Product supply Critical success factors for a personalized cell therapy Reliable and timely delivery of every batch with consistent quality is critical for each patient • Process – Consistent manufacturing process performance over a wide rang
Slide 20: The Nucleus
Developing Next Generation Programmed T Cell Therapies 20 The Nucleus State of the art design and operations established – validation on track Design Build Operations • ~70,000 sq ft facility • Nov 8, 2021 ground breaking • Dec 14, 2022 first Prodigy operational • Modular build using PAMs • Nov 25,
Slide 21: Obe-cel steps to commercialization
21 Obe-cel steps to commercialization Roadmap to a 2024 commercial launch H2 2023 H1 2024 Manufacturing BLA filing GMP license EU marketing Facility authorization application Filing of Biologics Filing of EU marketing License Application authorization application Operating at 2,000+ (BLA) to U.S. Fo
Slide 22: Expanding the obe-cel opportunity
Expanding the obe-cel opportunity Deep value program with potentially broad applicability
Slide 23: The obe-cel product family and franchise opportunity
Developing Next Generation Programmed T Cell Therapies 23 The obe-cel product family and franchise opportunity • Optimal CD19 CAR design B-ALL and B-NHL Obe-cel • Potential best-in-class response and tolerability CD19 • Supported by state-of-the-art manufacturing B-cell mediated • Supported by matur
Slide 24: Obe-cel in B-NHL/CLL: High level clinical activity with durable outcomes
B-ALL and B-NHL Developing Next Generation Programmed T Cell Therapies 24 Obe-cel in B-NHL/CLL: High level clinical activity with durable outcomes Long term persistence driving durable outcomes ALLCAR19 – B-NHL and CLL Median follow-up 12.9 months N 25 ORR All patients 92% Follicular Lymphoma 100% M
Slide 25: AUTO1/22 in pediatric ALL
B-ALL and B-NHL Developing Next Generation Programmed T Cell Therapies 25 AUTO1/22 in pediatric ALL No antigen negative relapse seen in responding patients CARPALL Disease Response (n=12) • Favorable adverse event profile with no severe CRS Molecular MRD neg CR/Cri by d30 10 (83%) Disease progressi
Slide 26: AUTO8: combining a sensitive BCMA CAR with the CD19 CAR from obe-cel
Confidential Multiple Myeloma Developing Next Generation Programmed T Cell Therapies 26 AUTO8: combining a sensitive BCMA CAR with the CD19 CAR from obe-cel Designed to induce deep and durable responses NT Screening for high sensitivity BCMA 72h cytotoxicity binders 72h cytotoxicity APRIL BCMA CAR 1
Slide 27: Opportunity and unmet need in B-cell autoimmune disease
AUTOIMMUNE Developing Next Generation Programmed T Cell Therapies 27 Opportunity and unmet need in B-cell autoimmune disease Systemic Lupus Erythematosus (SLE) Rationale Why SLE • Autoimmune diseases are often driven by auto-reactive • SLE is a multi-organ systemic autoimmune disease that antibodies
Slide 28: Uniquely positioned to deliver CAR T therapy in autoimmune disease
Developing Next Generation Programmed T Cell Therapies 28 Uniquely positioned to deliver CAR T therapy in autoimmune disease Lead opportunity for obe-cel in SLE with potential to explore and broaden application across other AI indications Potential best in class therapy Speed to launch Attractive ec
Slide 29: Other pipeline programs and technologies
Other pipeline programs and technologies A broad portfolio of potential next generation modular T cell therapies
Slide 30: Autolus pipeline
Developing Next Generation Programmed T Cell Therapies 30 Autolus pipeline Obe-cel product family PRODUCT INDICATION TARGET STUDY NAME COLLABORATION PHASE UPCOMING CATALYST Q4 2023: FELIX data updates Obe-cel Adult B-ALL CD19 FELIX Pivotal Q4 2023: BLA filing with FDA Systemic Lupus Obe-cel CD19 TB
Slide 31: A broad toolkit which is core to our strategy of modular innovation
Private & Confidential | Developing Next Generation Programmed T Cell Therapies 31 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 Activity Enhancement
Slide 32: Leveraging our industry leading technology platform via partnerships
Developing Next Generation Programmed T Cell Therapies 32 Leveraging our industry leading technology platform via partnerships Technology partnerships Leveraging our Tumor targeting, Validating Potential for value modular programming pharmacological collaborations creation through near technology to
Slide 33: Upcoming news flow
Upcoming news flow
Slide 34: Autolus planned news flow
Developing Next Generation Programmed T Cell Therapies 34 Autolus planned news flow Milestone or Data Catalysts Timing Obe-cel Biologics License Application (BLA) to FDA By end of 2023 Obe-cel FELIX data update expected at ASH Dec 2023 AUTO8 update (McCarty study) expected at ASH Dec 2023 Obe-cel in
Slide 35: Summary
Summary
Slide 36: Building a leading CAR T company developing transformational therapies for cancer and autoimmune diseases
Developing Next Generation Programmed T Cell Therapies 36 Building a leading CAR T company developing transformational therapies for cancer and autoimmune diseases Established excellence in R&D and Manufacturing; scaling company toward commercialization $ Obe-cel approaching Pipeline expansion Scala
Slide 37: Thank you
Thank you autolus.com
Autolus Therapeutics — Investor Presentation (October 2023) on 6ix