Autolus Therapeutics Plc · NASDAQ:AUTL
Autolus Therapeutics — Investor Presentation (September 2024)
39 slides from Autolus Therapeutics's investor presentation, Sep 26, 2024
Slide 1: Developing Next Generation Programmed T Cell Therapies
Developing Next Generation Programmed T Cell Therapies September 2024 Autolus.com
Slide 2: Disclaimer
Developing Next Generation Programmed T Cell Therapies 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 i
Slide 3: Autolus overview – scaling towards commercialization
Developing Next Generation Programmed T Cell Therapies Autolus overview – scaling towards commercialization Building a leading CAR T company developing therapies for cancer and autoimmune diseases $ Obe-cel: a potentially best- Pipeline expansion Scalable manufacturing Strategic Strong cash in-class
Slide 4: Obe-cel
LEAD CLINICAL PROGRAM Obe-cel A standalone, potentially best-in-class CD19 CAR T cell therapy candidate
Slide 5: We believe obe-cel has a unique mechanism of action
Developing Next Generation Programmed T Cell Therapies We believe 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 to binders used in On-Rate: Ka [M-1S-1] ]1 Other CD19 Bin
Slide 6: The FELIX phase 1b/2 pivotal study
Developing Next Generation Programmed T Cell Therapies The FELIX phase 1b/2 pivotal study Reliable obe-cel supply for FELIX despite the COVID–19 pandemic • US international airline flights decreased by 41% compared to flights from pre-COVID–19 pandemic1 • BUT international flights are reliable and o
Slide 7: Overview of FELIX 1b/2 clinical experience to date
Developing Next Generation Programmed T Cell Therapies Overview of FELIX 1b/2 clinical experience to date FELIX pooled analysis of all cohorts presented ASH 2023 and ASCO/EHA 2024 Durable and robust response rates 1 • The ORR (CR/CRi) in all patients who received obe-cel in the FELIX study was 78% •
Slide 8: ASCO/EHA 2024
OBE-CEL IN ADULTS WITH R/R B-ALL ASCO/EHA 2024 FELIX Phase 1b/2 trial
Slide 9: FELIX study all cohorts: Majority of responders show durable response
Developing Next Generation Programmed T Cell Therapies FELIX study all cohorts: Majority of responders show durable response (n=127) 40% of responders are in ongoing remission without consolidative SCT and 18% had consolidative SCT Infused Data cut-off date: N = 127 February 7, 2024 No response or n
Slide 10: FELIX study all cohorts: Event-free survival
Developing Next Generation Programmed T Cell Therapies FELIX study all cohorts: Event-free survival (n=127) Subset of patients benefit from standalone treatment with obe-cel 100 90 Censoring for SCT (main analysis) Without censoring for SCT 80 Probability (%) 70 60 50 40 30 20 Censoring for SCT With
Slide 11: FELIX study all cohorts: Overall survival
Developing Next Generation Programmed T Cell Therapies FELIX study all cohorts: Overall survival (n=127) Potential long-term plateau 100 90 Censoring for SCT 80 Without censoring for SCT (main analysis) Probability (%) 70 60 50 40 30 20 Censoring for SCT Without censoring for SCT 10 Median (95% CI),
Slide 12: FELIX study all cohorts: CAR T persistence and predicted relapse
Developing Next Generation Programmed T Cell Therapies FELIX study all cohorts: CAR T persistence and predicted relapse Ongoing CAR T persistence correlates with long-term EFS 1.0 1.0 Ongoing CAR T persistence Ongoing B-cell aplasia Loss of CAR T persistence at Month 12 B-cell recovery at Month 12 0
Slide 13: ASCO 2024 takeaway messages
Developing Next Generation Programmed T Cell Therapies ASCO 2024 takeaway messages FELIX study - pooled analysis of all cohorts • 40% of responders in ongoing remission without subsequent SCT/other therapy, with a median follow-up of 21.5 months • Survival outcomes show potential of long-term platea
Slide 14: ALL: unmet need and market overview
ALL: unmet need and market overview
Slide 15: If approved, obe-cel could launch into an expanding ALL market
Developing Next Generation Programmed T Cell Therapies If approved, obe-cel could launch into an expanding ALL market Blincyto®, current market leader, sales increased 48% year-over-year to $861 million for the full year 2023 Reported Blincyto® sales¹ • Blincyto® sales price estimated to be $103.5k²
Slide 16: Over 8,000 new cases of adult ALL annually worldwide
Developing Next Generation Programmed T Cell Therapies 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 ena
Slide 17: Commercial Launch Readiness
Commercial Launch Readiness
Slide 18: Obe-cel steps to commercialization
Developing Next Generation Programmed T Cell Therapies Obe-cel steps to commercialization Roadmap to a commercial launch in r/r adult ALL 2024 Q1 Q2 Q3 Q4 Obe-cel BLA Nucleus MHRA Obe-cel FDA accepted by FDA inspection MHRA PDUFA & approval authorization Obe-cel EMA MAA application Obtained MHRA MIA
Slide 19: US treatment center timelines for authorization and first patient readiness
Developing Next Generation Programmed T Cell Therapies US treatment center timelines for authorization and first patient readiness 2024 2025 Now-Oct Nov Dec Jan Feb Mar Activation Qualification (activities ongoing) Activation Activities Complete = Authorization Trigger PDUFA* (Nov 16th) Centers have
Slide 20: The Nucleus – Our Commercial Manufacturing Facility
Developing Next Generation Programmed T Cell Therapies The Nucleus – Our Commercial Manufacturing Facility State of the art design and operations established – groundbreaking to complete validation in 2 years Design Build Operations • Facility ~70,000 sq ft • Modular build (70% built off-site) • Tim
Slide 21: Expanding the obe-cel opportunity
Expanding the obe-cel opportunity Deep value program with potentially broad applicability
Slide 22: The obe-cel product family and franchise opportunity
Developing Next Generation Programmed T Cell Therapies The obe-cel product family and franchise opportunity • Optimized CD19 CAR design B-ALL and B-NHL Obe-cel • Potential best-in-class efficacy and safety profile CD19 • Supported by state-of-the-art manufacturing B-cell mediated • Supported by matu
Slide 23: Uniquely positioned to develop CAR T therapy candidate in autoimmune disease
Autoimmune Developing Next Generation Programmed T Cell Therapies Uniquely positioned to develop CAR T therapy candidate in autoimmune disease Obe-cel’s potential characteristics Supporting evidence • Potential best-in-class risk/benefit profile in pivotal FELIX trial in adult ALL Favorable tolerabi
Slide 24: Phase 1 study in r/r SLE – enrollment ongoing
Autoimmune Developing Next Generation Programmed T Cell Therapies Phase 1 study in r/r SLE – enrollment ongoing Primary goal of the Phase 1 study will be confirming the fixed dose in adult SLE patients CARLYSLE Study A Single-Arm, Open-Label, Phase I Study to Determine the Safety, Tolerability and P
Slide 25: Initial clinical experience in r/r NHL, r/r pALL and in r/r MM
Oncology Developing Next Generation Programmed T Cell Therapies Initial clinical experience in r/r NHL, r/r pALL and in r/r MM Obe-cel ALLCAR19 study, AUTO1/22 CARPALL study and AUTO8 McCARTY study Obe-cel in NHL* AUTO1/22 in pALL* AUTO8 in r/r MM* • 22 r/r NHL patients treated • Kymriah ineligible
Slide 26: Partnerships, pipeline programs and technologies
Partnerships, pipeline programs and technologies A broad portfolio of potential next generation modular T cell therapies
Slide 27: Autolus pipeline
Developing Next Generation Programmed T Cell Therapies Autolus pipeline Obe-cel product family Product Indication Target Study Name Partner Phase Status/Expected Milestones Submitted to EMA, MHRA and FDA Obe-cel Adult B-ALL CD19 FELIX Pivotal (PDUFA November 16, 2024) Obe-cel Systemic Lupus Erythema
Slide 28: Leveraging our industry leading technology platform via partnerships
Developing Next Generation Programmed T Cell Therapies Leveraging our industry leading technology platform via partnerships Technology partnerships Potential for value Leveraging our Tumor targeting, Validating creation through near modular programming pharmacological collaborations with term option
Slide 29: Upcoming news flow
Upcoming news flow
Slide 30: Autolus planned news flow
Developing Next Generation Programmed T Cell Therapies Autolus planned news flow Anticipated Milestone or Data Catalysts Anticipated Timing Obe-cel U.S. FDA PDUFA target action date November 16, 2024 Obe-cel FELIX data update at ASH 2024 December 2024 Obe-cel in autoimmune disease – initial data fro
Slide 31: Summary
Summary
Slide 32: Autolus overview – scaling towards commercialization
Developing Next Generation Programmed T Cell Therapies Autolus overview – scaling towards commercialization Building a leading CAR T company developing therapies for cancer and autoimmune diseases $ Obe-cel: a potentially best- Pipeline expansion Scalable manufacturing Strategic Strong cash in-class
Slide 33: Thank you
Thank you Autolus.com
Slide 34: Appendix
Appendix Autolus.com
Slide 35: FELIX Phase 1b/2 pooled analysis: patient disposition
Developing Next Generation Programmed T Cell Therapies FELIX Phase 1b/2 pooled analysis: patient disposition 127/153 (83%) enrolled patients received obe-cel* n (%) Enrolled** Discontinued 26 (17) N = 153 Death 15 (10) Manufacturing-related 7 (5) Adverse event 2 (1) Morphologic disease*** (n = 98) P
Slide 36: ASH2023: EFS by leukemic burden prior to lymphodepletion
Developing Next Generation Programmed T Cell Therapies ASH2023: EFS by leukemic burden prior to lymphodepletion* Lower leukemic burden is associated with better outcomes <5% (n = 36) 100 ≥5−≤75% (n = 51) 90 >75% (n = 40) 80 70 Probability (%) 60 50 40 Events, n: <5% 10 30 ≥5−≤75% 22 >75% 27 20 Media
Slide 37: ASH2023 FELIX Phase 1b/2 pooled analysis: CRS and ICANS
Developing Next Generation Programmed T Cell Therapies ASH2023 FELIX Phase 1b/2 pooled analysis: CRS and ICANS Low rates of Grade ≥3 CRS and/or ICANS were observed CRS and ICANS in all patients CRS by % BM blasts ICANS by % BM blasts 100 100 100 88% 80 80 80 69% 69% Incidence, % Incidence, % Inciden
Slide 38: ASH 2023 takeaway messages
Developing Next Generation Programmed T Cell Therapies ASH 2023 takeaway messages • Obe-cel successfully manufactured in 95% of leukapheresed patients • High remission rates independent of leukemic burden at lymphodepletion • 50% EFS estimate at 12 months, with only 17% of responders proceeding to S
Slide 39: Strategic multi-platform R&D collaboration with BioNTech
Developing Next Generation Programmed T Cell Therapies Strategic multi-platform R&D collaboration with BioNTech CAR T Cell Therapies Commercial Infrastructure Access Deal Financials BioNTech to financially support obe-cel BioNTech to receive option to access Upfront Payments planned/potential commer
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