Autolus Therapeutics Plc · NASDAQ:AUTL
Autolus Therapeutics — Investor Presentation (May 2024)
40 slides from Autolus Therapeutics's investor presentation, May 2024
Slide 1: Developing Next Generation Programmed T Cell Therapies
Developing Next Generation Programmed T Cell Therapies May 2024
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: Building a leading CAR T company developing therapies for cancer and autoimmune diseases
Developing Next Generation Programmed T Cell Therapies 3 Building a leading CAR T company developing therapies for cancer and autoimmune diseases Scaling company toward commercialization $ Obe-cel potentially best in Pipeline expansion Scalable manufacturing Strategic Strong cash class CAR T for r/r
Slide 4: A strategic multi-platform R&D collaboration with BioNTech
Developing Next Generation Programmed T Cell Therapies A strategic multi-platform R&D collaboration with BioNTech CAR T Cell Therapies Commercial Infrastructure Access Deal Financials Upfront Payments ◼ BioNTech to financially support obe- ◼ BioNTech to receive option to access ◼ $200 million upfron
Slide 5: Obe-cel
LEAD CLINICAL PROGRAM Obe-cel A standalone, potentially best-in-class CD19 CAR T cell therapy candidate
Slide 6: We believe obe-cel has a unique mechanism of action
Developing Next Generation Programmed T Cell Therapies 6 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 On-Rate: Ka [M-1S-1] ]1 CD19 binder with - - S1 Other C
Slide 7: Obe-cel pooled analysis: FELIX Phase 1b/2 trial
ASH 2023 Obe-cel pooled analysis FELIX Phase 1b/2 trial
Slide 8: FELIX Phase 1b/2 pooled analysis: patient disposition
Developing Next Generation Programmed T Cell Therapies 8 FELIX Phase 1b/2 pooled analysis: patient disposition 127/153 (83%) enrolled patients received obe-cel* n (%) Discontinued Enrolled** 26 (17) N = 153 Death 15 (10) Manufacturing-related 7 (5) Morphologic disease*** (n = 98) Adverse event 2 (1)
Slide 9: FELIX Phase 1b/2 pooled analysis: EFS in all treated patients
Developing Next Generation Programmed T Cell Therapies 9 FELIX Phase 1b/2 pooled analysis: EFS in all treated patients* The event-free survival estimate at 12 months was 50% 100 90 Overall (N = 127) All treated patients (N = 127) 80 70 Median EFS 11.7 (95% CI), Probability (%) 60 (7.2, NE) months 50
Slide 10: FELIX Phase 1b/2 pooled analysis: leukemic burden in all treated patients
Developing Next Generation Programmed T Cell Therapies 10 FELIX Phase 1b/2 pooled analysis: leukemic burden in all treated patients Leukemic burden at screening is not predictive of leukemic burden prior to lymphodepletion BM blasts % 118/127 (93%) patients received BM blasts % prior at screening br
Slide 11: FELIX Ph1b/2 pooled: EFS by leukemic burden prior to lymphodepletion
Developing Next Generation Programmed T Cell Therapies 11 FELIX Ph1b/2 pooled: 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 >
Slide 12: FELIX Phase 1b/2 pooled analysis: CRS and ICANS
Developing Next Generation Programmed T Cell Therapies 12 FELIX Phase 1b/2 pooled analysis: CRS and ICANS Low rates of Grade ≥3 CRS and/or ICANS were observed CRS and ICANS CRS by ICANS by in all patients % BM blasts % BM blasts 100 100 100 88% 80 80 80 69% 69% Incidence, % Incidence, % Incidence, %
Slide 13: Obe-cel pooled analysis: ALLCAR19 Phase 1b / FELIX Ph 1b
ASH 2023 Obe-cel pooled analysis ALLCAR19 Phase 1b /FELIX Ph 1b
Slide 14: Long-term follow up in R/R B-ALL demonstrates favorable EFS and OS
Developing Next Generation Programmed T Cell Therapies 14 Long-term follow up in R/R B-ALL demonstrates favorable EFS and OS Median follow up 36.5 months; pooled analysis Phase 1b ALLCAR19/Phase 1b FELIX With censoring*: Median OS: 16.4 months (95% CI: 7.1–N/A) • Median EFS: 9.0 months (95% CI: 5.1–
Slide 15: Durable remissions and prolonged persistence in patients with R/R B-ALL
Developing Next Generation Programmed T Cell Therapies 15 Durable remissions and prolonged persistence in patients with R/R B-ALL Pooled analysis Phase 1b ALLCAR19 / Phase 1b FELIX No. Months since infusion • ORR: 80.6% (95% CI: 64.0–91.8) • Ongoing CAR T persistence • All patients in ongoing remiss
Slide 16: ALL: unmet need and market overview
ALL: unmet need and market overview
Slide 17: If approved, obe-cel could launch into an expanding ALL market
Developing Next Generation Programmed T Cell Therapies 17 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,
Slide 18: Over 8,000 new cases of adult ALL annually worldwide
Developing Next Generation Programmed T Cell Therapies 18 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 19: Critical drivers for potential market adoption if approved
Developing Next Generation Programmed T Cell Therapies 19 Critical drivers for potential market adoption if approved CLINICAL DATA1 TREATMENT EXPERIENCE GOALS Durable and robust response Timely & reliable product supply • Morphological disease: CR/CRi rate of 74%, with • Quality product with low out
Slide 20: Commercial Launch Readiness
Commercial Launch Readiness
Slide 21: Obe-cel steps to commercialization in r/r adult B-ALL
Developing Next Generation Programmed T Cell Therapies 21 Obe-cel steps to commercialization in r/r adult B-ALL Roadmap to a 2024 commercial launch Q1 Q2 Q3 Q4 Obe-cel BLA MHRA Nucleus inspection & approval Obe-cel FDA accepted by MHRA PDUFA FDA Obe-cel EU authorization application authorization app
Slide 22: The Nucleus
Developing Next Generation Programmed T Cell Therapies 22 The Nucleus State of the art design and operations established – groundbreaking to complete validation in 2 years Design Build Operations • ~70,000 sq ft facility • Modular build using PAMs • 70% built off-site • 60% reduced build time • BREE
Slide 23: Expanding the obe-cel opportunity
Expanding the obe-cel opportunity Deep value program with potentially broad applicability
Slide 24: The obe-cel product family and franchise opportunity
Developing Next Generation Programmed T Cell Therapies 24 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 m
Slide 25: Obe-cel in B-NHL/B-CLL: High response rates with durable remissions
B-NHL/CLL Developing Next Generation Programmed T Cell Therapies 25 Obe-cel in B-NHL/B-CLL: High response rates with durable remissions Data from ALLCAR19 extension: Long term persistence driving durable outcomes Responses by subtype Median follow-up 20.9 months (range 0.9 – 36.2) CMR SD MRD-negativ
Slide 26: AUTO1/22 in pediatric ALL
B-ALL and B-NHL Developing Next Generation Programmed T Cell Therapies 26 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 27: AUTO8: combining a sensitive BCMA CAR with the CD19 CAR from obe-cel
Confidential Multiple Myeloma Developing Next Generation Programmed T Cell Therapies 27 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 28: Initial data from MCARTY Phase 1 showed clinical responses in all patients
Multiple Myeloma Developing Next Generation Programmed T Cell Therapies 28 Initial data from MCARTY Phase 1 showed clinical responses in all patients Both D8 BCMA CAR and AUTO8 associated with high response rate • ORR 100%; 3 PR*, 1 VGPR*, 7 CR*/sCR* (all evaluable MRD-) • Two patients remained in s
Slide 29: Initial safety data Phase 1 MCARTY study
Multiple Myeloma Developing Next Generation Programmed T Cell Therapies 29 Initial safety data Phase 1 MCARTY study D8 BCMA CAR and AUTO8 did not result in severe ICANS/CRS and were well tolerated with no DLTs • CRS in 10 patients (91%) and all low grade; no patients reported ICANS • Tocilizumab giv
Slide 30: Uniquely positioned to develop CAR T therapy candidate in autoimmune disease
Autoimmune Developing Next Generation Programmed T Cell Therapies 30 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 toler
Slide 31: Phase 1 study in r/r SLE – open for enrollment
Autoimmune Developing Next Generation Programmed T Cell Therapies Phase 1 study in r/r SLE – open for enrollment 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 an
Slide 32: Other pipeline programs and technologies
Other pipeline programs and technologies A broad portfolio of potential next generation modular T cell therapies
Slide 33: 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 and FDA Obe-cel Adult B-ALL CD19 FELIX Pivotal (PDUFA November 16, 2024) Obe-cel Systemic Lupus Erythematosus
Slide 34: A broad toolkit which is core to our strategy of modular innovation
Private & Confidential | Developing Next Generation Programmed T Cell Therapies 34 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 35: Leveraging our industry leading technology platform via partnerships
Developing Next Generation Programmed T Cell Therapies 35 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 36: Upcoming news flow
Upcoming news flow
Slide 37: Autolus planned news flow
Developing Next Generation Programmed T Cell Therapies 37 Autolus planned news flow Anticipated Milestone or Data Catalysts Anticipated Timing Obe-cel FELIX data update at ASCO, EHA & ASH 2024 June & December 2024 Obe-cel Marketing Authorization Application to MHRA Second half 2024 Obe-cel U.S. FDA
Slide 38: Summary
Summary
Slide 39: Building a leading CAR T company developing therapies for cancer and autoimmune diseases
Developing Next Generation Programmed T Cell Therapies 39 Building a leading CAR T company developing therapies for cancer and autoimmune diseases Scaling company toward commercialization $ Obe-cel potentially best in Pipeline expansion Scalable manufacturing Strategic Strong cash class CAR T for r/
Slide 40: Thank you
Thank you autolus.com
Autolus Therapeutics — Investor Presentation (May 2024) on 6ix