Full Press Release Details
Developing Next Generation Programmed
T Cell Therapies February 2024 Exhibit 99.1
Disclaimer Developing Next Generation
Programmed T Cell Therapies 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 in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements
regarding Autolus' development of its product candidates, including the obe-cel program; the profile and potential application of obe-cel in additional disease settings; the future
clinical development, efficacy, safety and therapeutic potential of the Company's product candidates, including progress, expectations as to the reporting of data, conduct and timing and potential future clinical and
preclinical activity and milestones; expectations regarding the initiation, design and reporting of data from clinical trials and preclinical studies; the extension of the pipeline beyond obe-cel; expectations regarding the regulatory
approval process for any product candidates; the benefits of the collaboration between Autolus and BioNTech, including the potential and timing to receive milestone payments and royalties under the terms of the strategic
collaboration; the Company's current and future manufacturing capabilities; and the Company's anticipated cash runway. Any forward-looking statements are based on management's current views and assumptions and involve
risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, the risks
that Autolus' preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results;
the cost, timing and results of clinical trials; that many product candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; and possible safety and
efficacy concerns. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Autolus' actual results to differ from those contained in the forward-looking statements, see the
section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission, or the SEC, on March 7, 2023 and in Autolus' Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission on November 9, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Autolus' subsequent filings with the Securities and Exchange Commission. All information in
this presentation is as of the date of the presentation, and Autolus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise,
except as required by law. You should, therefore, not rely on these forward-looking statements as representing the Company's views as of any date subsequent to the date of this presentation.
Building a leading CAR T company
developing therapies for cancer and autoimmune diseases FELIX pivotal trial showed high ORR, encouraging EFS and favorable tolerability with low levels of high-grade CRS and ICANS PDUFA date 16 Nov 2024 EMA submission planned for 1H 2024
Strategic multi-platform R&D collaboration with BioNTech Established technology collaborations with Moderna, BMS and Cabaletta Long-standing academic collaboration with University College London Expand obe-cel opportunity in B cell malignancies,
autoimmune diseases & life cycle strategy SLE B-NHL indications Bi-specific therapies (CD19 /CD22; CD19/BCMA) Expand to additional indications with novel CAR T therapies, alone or with partners Demonstrated reliable clinical trial supply (96%
target dose reached in FELIX pivotal study) New commercial cell manufacturing facility in qualification stage; planned annual capacity 2,000+ batches Expected vein-to-delivery time at launch of ~16 days Obe-cel potentially best in class CAR T for
r/r adult ALL Strategic collaborations Pipeline expansion strategy Scalable manufacturing and in-house facility Cash $256.4M (Q3 2023) Runway into 2025* Enables execution on current strategy through expected approval of obe-cel Strong cash
position Scaling company toward commercialization $ Developing Next Generation Programmed T Cell Therapies Abbreviations and notes: r/r ALL - relapsed/refractory acute lymphoblastic leukemia; B-NHL - B-cell non-Hodgkin's lymphoma; SLE
- systemic lupus erythematosus. *Does not include proceeds from BioNTech
A strategic multi-platform R&D
collaboration with BioNTech CAR T Cell Therapies BioNTech to financially support obe-cel planned/potential commercial launch in adult ALL (Acute Lymphoblastic Leukemia) and expansion of development program Commercial Infrastructure Access
BioNTech to receive option to access Autolus' GMP product supply and commercial infrastructure for their CAR T program, BNT211 Development Product Options BioNTech to receive co-development and co-commercialization options for AUTO1/22
(CD19/22) and AUTO6NG (GD2) programs Technology Platform License BioNTech to receive license and options to access proprietary binders, safety switches and technologies for certain BioNTech programs Deal Financials Upfront Payments $200
million upfront for equity $50 million upfront cash Downstream Economics Up to $580 million in further option exercise and milestones payments BioNTech to receive up to mid-single digit royalty on obe-cel project financing Autolus eligible for an
additional equity investment of $20m, an option exercise payment and profit share based on products manufactured for BioNTech's BNT211 program BioNTech has option to co-fund and co-commercialize AUTO1/22 and AUTO6NG, if approved, in
return for profit share Technology license and options provided in exchange for milestones and royalties Developing Next Generation Programmed T Cell Therapies
Obe-cel Lead Clinical Program A
standalone, potentially best-in-class CD19 CAR T cell therapy candidate
We believe obe-cel has a unique
mechanism of action Avoided over-activation of CAR T cells Increased CAR T peak expansion Avoided exhaustion of CAR T-cells Enhanced cytotoxicity and proliferation Off Rate: Kd [S-1] ] 1 - S 1 - [M a K : e Rat - On Other CD19 Binders
Obe-cel Binder Off Rate: Kd [S-1] On-Rate: Ka [M-1S-1] Shorter half-life of interaction compared to binders used in approved products obe-cel = 9.8 seconds Kymriah = 21 minutes % Tumor Cell Killing Proliferation Ghorashian et al. Nature
Medicine 2019 Fast off-rate CD19 binder with fast off-rate Designed for increased activity and reduced toxicity Developing Next Generation Programmed T Cell Therapies Differentiated CD19 binder Potential for improved potency, reduced toxicity
Reduced toxicities Improved persistence Improved engraftment Improved persistence
Obe-cel pooled analysis ASH 2023 FELIX
FELIX Phase 1b/2 pooled analysis:
patient disposition Developing Next Generation Programmed T Cell Therapies 127/153 (83%) enrolled patients received obe-cel* Discontinued n (%) 26 (17) Death 15 (10) Manufacturing-related 7 (5) Adverse event 2 (1) Physician decision 1
(0.7) Progressive disease 1 (0.7) *Seven patients received Dose 1 only; **All eligibility criteria met and the leukapheresate accepted for manufacturing; obe-cel, obecabtagene autoleucel; Roddie et al., ASH 2023, Data cut-off date: September 13,
2023 Infused N = 127 (83%) Enrolled** N = 153 Cohort A n = 107 (84%) 5% BM blasts at screening Cohort B n = 13 (10%) MRD-positive at screening Cohort C n = 7 (6%) Isolated EMD at screening Morphologic disease*** (n = 98) 74% of
patients had CR/CRi (n = 73) 95% of evaluated responders were MRD-negative No morphologic disease (n = 29) 100% of evaluable patients were MRD-negative ***Morphologic disease defined as 5% BM blasts or presence of EMD regardless
of BM blast status; MRD status available for 64/73 patients, as assessed by NGS or flow cytometry; MRD status available for 27/29 patients, as assessed by NGS or flow cytometry; BM, bone marrow; CR, complete remission; CRi, CR with
incomplete hematologic recovery; EMD, extramedullary disease; MRD, measurable residual disease; NGS, next-generation sequencing; obe-cel, obecabtagene autoleucel
FELIX Phase 1b/2 pooled analysis: EFS
in all treated patients* Developing Next Generation Programmed T Cell Therapies The event-free survival estimate at 12 months was 50% Median follow-up time was 16.6 months (range: 3.7 36.6 months) 17/99 (17%) responders proceeded to SCT while
in remission *Censoring new non-protocol anti-cancer therapies including SCT with disease assessment by IRRC (data cut-off date: September 13, 2023); Median EFS: ITT population 9.8 months (95% CI: 5.9, 12.9); CI, confidence interval; EFS,
event-free survival; IRRC, Independent Response Review Committee; ITT, intent-to-treat; NE, not evaluable; obe-cel, obecabtagene autoleucel; SCT, stem cell transplant; Roddie et al., ASH 2023 All treated patients (N = 127) Total infused (N = 126)
Median EFS (95% CI), months 11.7 (7.2, NE) 6-month EFS (95% CI), % 65 (56, 73) 12-month EFS (95% CI), % 50 (39, 59) Events, n: Overall Median (95% CI): Overall 59 11.7 (7.2, NE) Probability (%) 0 10 20 30 40 50 60 70 80 90 100 Time (months) 0
1 2 3 4 5 6 7 8 9 10 1 1 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 Overall (N = 127) Patients at risk 127 104 94 85 78 68 60 55 52 45 34 33 29 27 27 22 18 18 16 12 12 6 5 4 4 4 3 3 3 3 2 2 2 2 2 2 1 0
FELIX Phase 1b/2 pooled analysis:
leukemic burden in all treated patients Developing Next Generation Programmed T Cell Therapies Leukemic burden at screening is not predictive of leukemic burden prior to lymphodepletion *Bridging therapy per physician's choice, including
inotuzumab ozogamicin; BM, bone marrow; Roddie et al., ASH 2023 16% 28% 53% 40% 31% 31% BM blasts % at screening BM blasts % prior to lymphodepletion 118/127 (93%) patients received bridging therapy* <5% 5 75% >75% 53% 31%
31% <5% 5 75% >75% 28% 41% 16%
FELIX Ph1b/2 pooled: EFS by
leukemic burden prior to lymphodepletion* Developing Next Generation Programmed T Cell Therapies Lower leukemic burden is associated with better outcomes BM blasts % prior to lymphodepletion <5% (n = 36) 5 75% (n = 51)
>75% (n = 40) Median EFS (95% CI), months NE 15.0 (6.6, NE) 4.5 (1.5, 9.0) 6-month EFS (95% CI), % 83 (65, 92) 72 (57, 82) 40 (23, 56) 12-month EFS (95% CI), % 65 (44, 80) 55 (38, 69) 27 (12, 44) Events, n: <5% 10 5 75% 22
>75% 27 Median (95% CI): <5% NE 5 75% 15 (6.6, NE) >75% 4.5 (1.5, 9.0) Patients at risk <5% (n = 36) 5 75% (n = 51) >75% (n = 40) <5% (n = 36) 5 75% (n = 51) >75% (n =
40) 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 36 34 31 28 25 24 22 20 19 18 14 13 11 11 11 11 8 8 7 6 6 2 2 2 2 2 1 1 1 1 1 1 1 1 1 1 0 0 51 43 41 39 36 31 28 25 23 18 15 15 13 12 12 9 8
8 7 4 4 2 1 1 1 1 1 1 1 1 1 1 1 1 1 1 1 0 40 27 22 18 17 13 10 10 10 9 5 5 5 4 4 2 2 2 2 2 2 2 2 1 1 1 1 1 1 1 0 0 0 0 0 0 0 0 Time (months) 0 10 20 30 40 50 60 70 80 90 100 Probability (%) *Censoring new non-protocol anti-cancer therapies including
SCT with disease assessment by IRRC (data cut-off date: September 13, 2023); BM, bone marrow; CI, confidence interval; EFS, event-free survival; IRRC, Independent Response Review Committee; NE, not evaluable; SCT, stem cell transplant; Roddie et
FELIX Phase 1b/2 pooled analysis:
CRS and ICANS Developing Next Generation Programmed T Cell Therapies Low rates of Grade 3 CRS and/or ICANS were observed BM blasts % at lymphodepletion 20 0 40 60 80 100 Incidence, % CRS ICANS 20 0 40 60 80 100 Incidence, % <5%
5 75% >75% 20 0 40 60 80 100 Incidence, % <5% 5 75% >75% CRS and ICANS in all patients CRS by % BM blasts ICANS by % BM blasts Light colors = grade 2 Dark colors = grade 3 69% 23% 47%
69% 88% 8% 18% 43% No grade 3 CRS and/or ICANS were observed in patients with <5% BM blasts at lymphodepletion Vasopressors were used to treat CRS in 2.4% of patients The treatment was generally well tolerated Two deaths were considered
treatment-related per investigator assessment: neutropenic sepsis (n = 1); acute respiratory distress syndrome and ICANS (n = 1) BM, bone marrow; CRS, cytokine release syndrome; ICANS, immune effector cell-associated neurotoxicity syndrome; ICU,
intensive care unit; Roddie et al., ASH 2023
Obe-cel pooled analysis ASH 2023
ALLCAR19 Phase 1b /FELIX Ph 1b
Long-term follow up in R/R B-ALL
demonstrates favorable EFS and OS Developing Next Generation Programmed T Cell Therapies Median follow up 36.5 months; pooled analysis Phase 1b ALLCAR19/Phase 1b FELIX *Censored for allo-HSCT and other anti-cancer treatment. Investigator-assessed
disease evaluations were performed locally by CT and BM biopsy for B-ALL. Allo-HSCT, allogeneic hematopoietic stem cell transplant; B-ALL, B-cell acute lymphoblastic leukemia; BM, bone marrow; CI, confidence interval; CT, computed tomography; EFS,
event-free survival; N/A, not available; obe-cel, obecabtagene autoleucel; OS, overall survival; R/R, relapsed/refractory. Roddie et al, ASH 2023, Poster 2114. Median OS: 16.4 months (95% CI: 7.1-N/A) 36-month OS rate: 41% (95% CI:
24-56) With censoring*: Median EFS: 9.0 months (95% CI: 5.1-N/A) 36-month EFS rate: 45% (95% CI: 27-62) Without censoring: Median EFS: 9.6 months (95% CI: 5.1-N/A) 36-month EFS rate: 36% (95% CI: 21-51)
Durable remissions and prolonged
persistence in patients with R/R B-ALL Developing Next Generation Programmed T Cell Therapies Pooled analysis Phase 1b ALLCAR19 / Phase 1b FELIX MRD status was determined using flow cytometry or IgH PCR/NGS (MRD-negative: <10-4 [<0.01%]). Loss
of CAR T persistency was defined as the time from first obe-cel infusion to undetectable CAR T transgene (copies/ g DNA) in peripheral blood. Patients who proceeded to allo-HSCT with ongoing CAR T persistency were censored at the last result
prior to receiving allo-HSCT. Allo-HSCT, allogeneic hematopoietic stem cell transplant; B-ALL, B-cell acute lymphoblastic leukemia; BM, bone marrow; CI, confidence interval; CR, complete remission; CT, computed tomography; DOR, duration of response;
MRD, measurable residual disease; N/A, not available; NGS, next-generation sequencing; ORR, overall response rate; PCR, polymerase chain reaction; R/R, relapsed/refractory. Roddie et al, ASH 2023, Poster 2114. Months since infusion ORR: 80.6% (95%
CI: 64.0-91.8) All patients in ongoing remission were MRD-negative at last assessment Median DOR: Not reached (95% CI: 5.1-N/A) Safety: No grade 3 CRS reported; 4/36 grade 3 ICANS; No new safety signals or deaths related
to obe-cel Ongoing CAR T persistence 12 months: 60.6% (95% CI: 38.9-76.8) 24 months: 55.1% (95% CI: 33.1-72.6) No.
ALL: unmet need and market overview
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 Blincyto sales price estimated to be $103,5k2 (for 1 cycle) supporting approx >2,500
commercial adult ALL patients across all lines of ALL treatment. Sales increased 48% year-over-year to $861 million for the full year 2023 Kymriah is priced at $508k in pediatric ALL. Breyanzi is priced at $447k in DLBCL3. Tecartus
is priced at $424k3 for adult ALL Breyanzi and other CAR T cell therapies are expanding delivery center footprint Tecartus is expected to establish CAR T use in adult ALL If approved, obe-cel has the potential to be best-in-class
curative therapy and expanding use beyond academic transplant centers 3121 3791 4721 +21% Reported Blincyto sales1 Notes As per Amgen quarterly SEC filings
https://www.cms.gov/medicare/payment/all-fee-service-providers/medicare-part-b-drug-average-sales-price/asp-pricing-files 3. Red Book pricing database https://www.ibm.com/products/micromedex-red-book/pricing +25% +24% Developing Next Generation
Programmed T Cell Therapies 5831 8611 +48%
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 enables 90% of adult ALL patients to
experience Complete Response (CR) Only 30% to 40% achieve long-term remission Current T cell therapies for adult patients are Blincyto and Tecartus Both therapies are highly active, but frequently followed by subsequent treatments (e.g.
alloSCT) Blincyto : favorable safety profile, few patients experiencing severe CRS and ICANS, but limitations on convenience - continuous i.v. infusion during 4-week treatment cycles Tecartus more challenging to manage - induces
elevated levels of severe CRS, a high levels of severe ICANS, and requires vasopressors for many patients Opportunity to expand the addressable patient population in earlier lines of therapy Notes SEER and EUCAN estimates (respectively) for US
and EU 8,4001 New cases of adult ALL diagnosed yearly 3,000 Addressable patient population Developing Next Generation Programmed T Cell Therapies
Critical drivers for potential