Autolus Therapeutics Plc · NASDAQ:AUTL
Autolus Therapeutics — Investor Presentation (June 2023)
38 slides from Autolus Therapeutics's investor presentation, June 2023
Slide 1: Developing Next Generation Programmed T Cell Therapies
Developing Next Generation Programmed T Cell Therapies June 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 fully integrated CAR T company
Developing Next Generation Programmed T Cell Therapies 3 Building a fully integrated CAR T company Expanding excellence in R&D and manufacturing to commercialization $ Obe-cel met primary Pipeline Scalable manufacturing Collaboration Strong cash position endpoint in pivotal study • Lead product cand
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 background – a fast-off CD19 CAR T therapy
Developing Next Generation Programmed T Cell Therapies 5 Obe-cel background – a fast-off CD19 CAR T therapy • CD19 CAR-T therapy has revolutionized the field of R/R B-ALL1 • Obe-cel is an autologous CD19 CAR with a fast off-rate CD19 binding domain designed to mitigate safety concerns and improve pe
Slide 6: Obe-cel has a unique mechanism of action
Developing Next Generation Programmed T Cell Therapies 6 Obe-cel has a unique mechanism of action Designed for increased activity and reduced toxicity Fast off-rate CD19 binder with Obe-cel has a shorter half-life of interaction On-Rate: Ka [M-1S-1] ] Obe-cel Binder fast off-rate 1 - S 1 - Other CD1
Slide 7: FELIX study: obe-cel for adults with r/r CD19+ B-ALL
Developing Next Generation Programmed T Cell Therapies 7 FELIX study: obe-cel for adults with r/r CD19+ B-ALL Obe-cel manufacturing Leukapheresis Split dose infusion Day 1 Day 10 Bridging therapy Safety and efficacy follow-up Day -6 Screening Enrollment Pre-conditioning Flu 30 mg/m2 Cy 500 mg/m2 Pre
Slide 8: FELIX: eligibility, endpoints, and disposition
Developing Next Generation Programmed T Cell Therapies 8 FELIX: eligibility, endpoints, and disposition 84% of enrolled patients were infused with obe-cel Key eligibility criteria Enrolled • R/R adult B-ALL* N = 112 • Aged ≥18 years • ≥5% BM blasts at screening (Cohort IIA) Discontinued: 18 • Death
Slide 9: FELIX: baseline characteristics
Developing Next Generation Programmed T Cell Therapies 9 FELIX: baseline characteristics Heavily pre-treated patients with high disease burden Total infused (N = 94) Age years, median (range) 50 (20–81) Gender male/female, n 47/47 Philadelphia chromosome-positive, n (%) 25 (26.6) Prior therapies, me
Slide 10: FELIX: disease response per IRRC assessment
Developing Next Generation Programmed T Cell Therapies 10 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 11: FELIX: duration of remission
Developing Next Generation Programmed T Cell Therapies 11 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 12: FELIX: subgroup analysis of CR/CRi (IRRC assessment)
Developing Next Generation Programmed T Cell Therapies 12 FELIX: subgroup analysis of CR/CRi (IRRC assessment) 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, years 18–39 31 (33) 58 (39, 75) 40–64 42 (45) 79
Slide 13: FELIX: safety – CRS and ICANS
13 Developing Next Generation Programmed T Cell Therapies 13 13 FELIX: safety – CRS and ICANS 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 (
Slide 14: FELIX: obe-cel expansion and persistence
Developing Next Generation Programmed T Cell Therapies 14 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 15: Obe-cel at ASH 2022 – B-ALL long term follow up from Ph1 ALLCAR19 trial
Developing Next Generation Programmed T Cell Therapies 15 Obe-cel at ASH 2022 – B-ALL long term follow up from Ph1 ALLCAR19 trial ‘Safety, Efficiency and Long-Term Follow-up of obe-cel, a Fast-Off Rate CD19 CAR in Relapsed/Refractory B-Cell Acute Lymphoblastic Leukaemia and Other B-Cell Malignancies
Slide 16: FELIX: conclusions
Developing Next Generation Programmed T Cell Therapies 16 FELIX: conclusions • Obe-cel infusion resulted in a 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 • Obe-cel infusion resulted in very low rates
Slide 17: Over 8,000 new cases of adult ALL annually worldwide
Developing Next Generation Programmed T Cell Therapies 17 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 18: FELIX study suggests improved safety and efficacy profile vs SoC
Developing Next Generation Programmed T Cell Therapies 18 FELIX study suggests improved safety and efficacy profile vs SoC Current standard of care for r/r adult ALL1 STANDARD OF CARE FELIX Blincyto®2 Besponsa®3 Obe-cel (blinatumomab) (inotuzumab ozogamicin) (obecabtagene autoleucel) N 271 109 94 OR
Slide 19: High grade ICANS are indicative of intensity of patient management
Developing Next Generation Programmed T Cell Therapies 19 High grade ICANS are indicative of intensity of patient management Relapsed/refractory ALL patients need therapies with high level of clinical activity and well manageable safety profile1 Tecartus(R) Obe-cel ZUMA-32 ROCCO3 FELIX4 N (dosed) 55
Slide 20: Obe-cel could launch into an expanding ALL market if approved
Developing Next Generation Programmed T Cell Therapies 20 Obe-cel could launch into an expanding ALL market if approved Blincyto®, current market leader, shows annual revenue growth of c.24% driven by well manageable safety profile Reported Blincyto® sales1 • Blincyto® sales price estimated to be $2
Slide 21: Obe-cel next steps to commercialization
Developing Next Generation Programmed T Cell Therapies 21 Obe-cel next steps to commercialization Preparing for launch in 2024 Data and path to approval Manufacturing Commercialization • Filing of Biologics License • Complete full characterization of • Focus in 2023 on Medical affairs, Application (
Slide 22: Product supply
Developing Next Generation Programmed T Cell Therapies 22 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 – Manufacturing process to perform consistently with wide ran
Slide 23: FELIX: obe-cel manufacturing
Developing Next Generation Programmed T Cell Therapies 23 FELIX: obe-cel manufacturing Manufacturing quality and logistics were reliable and consistent 96% of products reached Median transduction Median 21 days from vein target dose efficiency of 72% to release 1600 100 CAR-T manufactured (x 106 cel
Slide 24: Commercial manufacturing facility – The Nucleus
Developing Next Generation Programmed T Cell Therapies 24 Commercial manufacturing facility – The Nucleus State of the art design and operations established – validation on track Design Build Operations Manufacturing • Modular build using PAMs • Nov 8, 2021 ground breaking • Dec 14, 2022 first Prodi
Slide 25: Building the obe-cel opportunity
Building the obe-cel opportunity Deep value program with potentially broad applicability
Slide 26: Obe-cel lifecycle outlook - NHL/CLL and Pediatric ALL - ASH 2022
Developing Next Generation Programmed T Cell Therapies 26 Obe-cel lifecycle outlook - NHL/CLL and Pediatric ALL - ASH 2022 High level of clinical activity with well manageable safety profile • Clinical data supports differentiated product profile in Adult ALL, B-NHL (obe-cel) and Pediatric ALL (AUTO
Slide 27: Pipeline
Pipeline A broad portfolio of potential next generation modular T cell therapies
Slide 28: A broad toolkit which is core to our strategy of modular innovation
Developing Next Generation Programmed T Cell Therapies 28 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 Modules Viral Vector TAR
Slide 29: Leveraging our industry leading technology platform via partnerships
Developing Next Generation Programmed T Cell Therapies 29 Leveraging our industry leading technology platform via partnerships Technology partnerships • Leveraging our modular programming • Moderna Tx technology to generate safer and more effective – Access to propriety binders for the therapies dev
Slide 30: Early-stage pipeline
Developing Next Generation Programmed T Cell Therapies 30 Early-stage pipeline Leveraging academic collaborations to generate opportunity for non-dilutive funding opportunities Next data readouts expected in 2023/2024
Slide 31: AUTO4 and AUTO5 for Peripheral T-Cell Lymphoma
Developing Next Generation Programmed T Cell Therapies 31 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 following first- line treatment (68%)1 9,200 • Standard of
Slide 32: AUTO4 for Peripheral T-Cell Lymphoma: ASH 2022
Developing Next Generation Programmed T Cell Therapies 32 AUTO4 for Peripheral T-Cell Lymphoma: ASH 2022 Patients achieve durable metabolic complete responses • AUTO4 treatment was well tolerated with no dose-limiting toxicities • Ongoing responses at 9 and 12 months post-dosing at the highest dose
Slide 33: Strong cash position with key financing partner
Strong cash position with key financing partner
Slide 34: Strong cash position to deliver on current strategy into 2025
Developing Next Generation Programmed T Cell Therapies 34 Strong cash position to deliver on current strategy into 2025 Q4 2022 Cash inflows $343.4m $164m $19.3m R&D tax credits December public Cash at March 31, 2023 offering from HMRC Blackstone collaboration Autolus is funded into 2025 $70m • $100
Slide 35: Summary
Summary
Slide 36: Autolus planned news flow
Developing Next Generation Programmed T Cell Therapies 36 Autolus planned news flow Obe-cel Pipeline Manufacturing • FELIX - Phase 2 clinical presentation • Updates on AUTO1/22 and AUTO 4 • Qualification of Nucleus facility ASCO and EHA in June planned for 2023 in H1 2023 • Biologics License Applica
Slide 37: The Autolus opportunity
Developing Next Generation Programmed T Cell Therapies 37 The Autolus opportunity Building a fully integrated CAR T company - Expanding excellence in R&D and manufacturing to commercialization • Obe-cel, a potentially best in class product • Purpose-built commercial manufacturing candidate, met prim
Slide 38: Thank you
Thank you autolus.com
Autolus Therapeutics — Investor Presentation (June 2023) on 6ix