Financial Data and Key Metrics Changes - Cash, cash equivalents, and marketable securities at year-end 2024 totaled $588 million, up from $239 million at the end of 2023, primarily due to a collaboration with BioNTech and equity financing [27][28] - Loss from operations for the year ending December 31, 2024, was $241.4 million, compared to $179.7 million for 2023 [28] - Net loss was $220.7 million for the year-end December 31, 2024, compared to $208.4 million for the same period in 2023 [30] Business Line Data and Key Metrics Changes - Cost of sales totaled $11.4 million following the BLA approval for obe-cel, representing costs associated with commercially available plant capacity [28] - Research and development expenses increased to $138.4 million for the year ending December 31, 2024, compared to $130.5 million in 2023, driven by increases in employee salaries and manufacturing costs [29] - Selling, general, and administrative expenses increased to $101.1 million for the year, compared to $46.7 million for the same period in 2023, primarily due to increased headcount for commercialization activities [29] Market Data and Key Metrics Changes - As of March 19, 2025, 33 centers were authorized to deliver AUCATZYL, expected to reach approximately 60% of the target patient population in the U.S. [13][21] - The company aims to have approximately 60 centers ready to deliver AUCATZYL by the end of the year [14] Company Strategy and Development Direction - The primary objective for 2025 is to execute a successful commercial launch for AUCATZYL and explore additional indications for obe-cel [8][10] - The company is planning an R&D event on April 23, 2025, to outline future growth opportunities [9] - The company is also moving through regulatory steps in the UK and Europe, with expected decisions in the second half of 2025 [14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the product's safety profile and its potential for outpatient administration, depending on physician experience and patient suitability [41][42] - The company noted a strong interest from centers to activate and deliver the product, indicating a positive dynamic in the launch [39] - Management highlighted the importance of ongoing experience with the product to build confidence among physicians [42] Other Important Information - The company received a $30 million milestone payment from Blackstone following FDA approval and made a regulatory milestone payment of GBP10 million [31][32] - The manufacturing facility is operational and is expected to reliably deliver products to centers [23] Q&A Session Summary Question: Initial demand and outpatient administration for AUCATZYL - Management noted a positive dynamic around center activation and interest in outpatient administration based on safety data [39][41] Question: Ramp-up of authorized centers and impact of tariffs - Management expects a steady ramp-up of centers, driven by individual center capabilities, and noted uncertainty regarding potential tariffs [48][53] Question: Time from site activation to first patient treatment - The time varies significantly, with some centers quickly treating patients while others may experience delays [60] Question: Manufacturing success rate and BioNTech's decision on AUTO6NG - Initial production experience mirrors clinical studies, and BioNTech's option on AUTO6NG will be exercised after pivotal study results [71][72] Question: SLE data context and resource allocation for launch - Upcoming SLE data will focus on product properties, safety, and B-cell depletion, with resources primarily directed towards service support for centers [78][85] Question: Treatment adoption among centers and sales guidance - Both experienced and new centers are adopting the treatment, and the company will not provide sales guidance due to multiple variables affecting sales trajectories [91][110]

AUCATZYL Launch and Expansion - Autolus' AUCATZYL received FDA approval on November 8, 2024, triggering a $30 million milestone payment from Blackstone[12] - As of March 19, 2025, 33 treatment centers are authorized for AUCATZYL, covering over 60% of the target patient population, with a goal to reach approximately 60 centers covering 90% by the end of 2025[12, 23] - Over 85% of total U S medical lives are covered for AUCATZYL[22] - The anticipated payor mix for AUCATZYL is approximately 60% commercial and 40% government/other[23] Obe-cel Development - The FELIX trial data for obe-cel was published in the New England Journal of Medicine in December 2024[12] - The FELIX trial showed an overall remission rate of 77% in all patients, 75% in morphological disease, 96% in measurable residual disease, and 71% in isolated extramedullary disease[13] - In the FELIX trial, the estimated 6- and 12-month overall survival rates were 80 3% and 61 1%, respectively, with a median OS of 15 6 months[19, 20] - Regulatory decisions from MHRA and EMA regarding obe-cel are expected in H2 2025[12] Financial Performance - Autolus' cash, cash equivalents, and marketable securities totaled $588 023 million for FY 2024, compared to $239 566 million for FY 2023[35] - Total revenue, net, for FY 2024 was $10 120 million, compared to $1 698 million for FY 2023[35] - The net loss for FY 2024 was $(220 844) million, compared to $(208 383) million for FY 2023[35]

Financial Data and Key Metrics Changes - Cash, cash equivalents, and marketable securities at year-end 2024 totaled $588 million, up from $239 million at the end of 2023, primarily due to a collaboration with BioNTech and equity financing [25] - Loss from operations for the year ending December 31, 2024, was $241.4 million, compared to $179.7 million for 2023 [26] - Net loss was $220.7 million for the year ended December 31, 2024, compared to $208.4 million for the same period in 2023 [27] Business Line Data and Key Metrics Changes - Cost of sales totaled $11.4 million following the BLA approval for Obicel, representing the cost of commercially available plant capacity [26] - Research and development expenses increased to $138.4 million for the year ended December 31, 2024, from $130.5 million in 2023, driven by increases in employee salaries and manufacturing costs [27] - Selling, general, and administrative expenses rose to $101.1 million for the year compared to $46.7 million in 2023, primarily due to increased salaries and employee-related costs [27] Market Data and Key Metrics Changes - As of March 19, 2025, 33 centers were authorized to deliver Ocassol, expected to reach approximately 60 centers by the end of the year, covering about 60% of the target patient population in the U.S. [12][13][20] - The product was included in the NCCN guidelines shortly after clinical results were published, enhancing confidence in its commercial use [12] Company Strategy and Development Direction - The primary objective for 2025 is to execute a successful commercial launch of Ocassol and explore opportunities for expanding its utility in additional indications [7][8] - The company plans to hold an R&D event on April 23, 2025, to outline future growth opportunities [9] - The company is also moving through regulatory steps in the UK and Europe, with expected decisions in the second half of 2025 [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the product's safety profile and its potential for outpatient administration, contingent on physician experience and patient suitability [35][36] - The company noted a strong dynamic in the activation of treatment centers, driven by physician interest and patient need [34] - Management highlighted the importance of ongoing data collection and analysis to support the product's positioning and adoption in the market [66] Other Important Information - The company received a $30 million milestone payment from Blackstone following FDA approval and made a regulatory milestone payment of GBP 10 million [28] - The manufacturing facility is operational and is expected to reliably deliver products to treatment centers [22] Q&A Session Summary Question: Can you provide any color on the initial demand or the book of orders for Ocassol? - Management noted a positive dynamic around center activation, driven by interest and patient need, but stated a fuller picture would be provided in the Q1 update [34] Question: What is the ramp-up plan for the authorized centers? - Management indicated a steady trajectory for onboarding centers, with expectations for gradual growth throughout the year [41][42] Question: Can you comment on the spread of time from site activation to treating the first patient? - Management stated that the time varies significantly based on patient suitability and center readiness, with some centers achieving quick onboarding [50] Question: What is the sentiment among prescribers regarding the need for subsequent therapy after using Ocassol? - Management indicated that initial experiences suggest a low percentage of patients require subsequent stem cell transplants, which resonates positively with physicians [55] Question: What has been the manufacturing success rate and turnaround time for Ocassol? - Management reported that production experiences are aligning well with clinical study data, providing reassurance for future batches [57]

Financial Results Announcement - Autolus Therapeutics plc announced its financial results for the year ended December 31, 2024, during a conference call on March 20, 2025[5] - The press release detailing the financial results is included as Exhibit 99.1[9] Corporate Presentation - The company provided an updated corporate presentation alongside the financial results, which is available as Exhibit 99.2[7]

Core Insights - Autolus Therapeutics achieved significant milestones in 2024, including FDA approval and commercial launch of AUCATZYL, a CAR T therapy for adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) [2][5] - The company is focused on expanding its commercial launch and advancing its clinical pipeline in 2025, with plans to enter new markets and invest in the obe-cel opportunity [2][4] Financial Performance - As of December 31, 2024, cash, cash equivalents, and marketable securities totaled $588.0 million, up from $239.6 million in 2023, primarily due to a strategic collaboration with BioNTech [8] - The net loss for the year ended December 31, 2024, was $220.7 million, compared to a loss of $208.4 million in 2023, with a basic and diluted net loss per share of $(0.86) [12][18] - Research and development expenses increased to $138.4 million from $130.5 million in 2023, while selling, general, and administrative expenses rose significantly to $101.1 million from $46.7 million [10][11] Product Development and Regulatory Updates - AUCATZYL was approved by the FDA on November 8, 2024, and is progressing well in the U.S. commercial launch with 33 authorized treatment centers as of March 19, 2025 [4][5] - The company expects marketing authorizations for obe-cel from the MHRA and EMA in the second half of 2025 [4][6] - Initial data from the Phase 1 trial in systemic lupus erythematosus (SLE) will be presented at an R&D event on April 23, 2025 [7][20] Clinical Trials and Pipeline - The pivotal Phase 1b/2 FELIX clinical trial of obe-cel demonstrated high rates of durable responses with low incidence of severe immune-related toxicity [5][9] - Autolus is also advancing early-stage pipeline programs, including AUTO8 and AUTO6NG, with updates planned for the upcoming R&D event [10][20] Market Access and Coverage - Patient access to AUCATZYL is strong, with coverage secured for over 85% of total U.S. medical lives, and the company aims to authorize 60 treatment centers by the end of 2025 [5][8] - The National Comprehensive Cancer Network (NCCN) has included AUCATZYL in its Clinical Practice Guidelines for the treatment of adult patients with relapsed/refractory B-ALL [5]

Core Insights - Autolus Therapeutics plc is set to release its fourth quarter and full year 2024 financial results on March 20, 2025, before the US markets open [1] - A conference call and webcast will be held on the same day at 8:30 am EDT/12:30 pm GMT to discuss the financial results and provide a business update [2] Company Overview - Autolus Therapeutics plc is an early commercial stage biopharmaceutical company focused on developing next-generation T cell therapies for cancer and autoimmune diseases [3] - The company utilizes proprietary T cell programming technologies to create targeted and controlled T cell therapies aimed at effectively recognizing and eliminating target cells [3] - Autolus has an FDA-approved product, AUCATZYL, and is developing a pipeline of candidates for treating hematological malignancies, solid tumors, and autoimmune diseases [3]

Core Insights - Autolus Therapeutics plc is a commercial-stage biopharmaceutical company focused on developing next-generation programmed T cell therapies for cancer and autoimmune diseases [3] Group 1: Upcoming Events - The company will participate in the Jefferies Biotech on the Beach Summit on March 11, 2025, in Miami, FL, hosting investor meetings [2] - Autolus will also present a fireside chat at the Leerink Global Biopharma Conference on March 12, 2025, at 8:40 am EDT / 12:40 pm GMT, led by CEO Dr. Christian Itin [2] - A webcast of the fireside chat will be available on the company's website, with a replay archived for 90 days [2] Group 2: Company Overview - Autolus utilizes proprietary and modular T cell programming technologies to create targeted and controlled T cell therapies [3] - The company has an FDA-approved product, AUCATZYL, and is developing a pipeline for treating hematological malignancies, solid tumors, and autoimmune diseases [3]

Core Insights - Autolus Therapeutics plc is set to present new data on its CAR-T cell therapy AUCATZYL® at the 2025 Tandem Meetings, highlighting its cost-effectiveness and clinical outcomes compared to other therapies [1][2]. Group 1: Presentations and Findings - The company will deliver an oral presentation and three poster presentations at the upcoming conference, focusing on the economic model comparing AUCATZYL® with other CAR-T therapies [1]. - An updated health economic cost model indicates that AUCATZYL® has a shorter duration and lower incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), leading to reduced healthcare costs [2]. - A poster presentation will detail the economic model comparing costs associated with CRS and ICANS among patients treated with CAR-T therapies for relapsed/refractory B-Cell Acute Lymphoblastic Leukemia (R/R B-ALL), showing that AUCATZYL® is associated with lower healthcare costs compared to brexu-cel [4]. Group 2: Clinical Outcomes - An oral presentation will discuss risk factors associated with sub-optimal outcomes and hematotoxicity following treatment with obe-cel for R/R B-ALL, emphasizing the potential for risk stratification to identify patients who may benefit from treatment [3]. - A poster will compare the efficacy and safety outcomes of obe-cel versus non-CAR T-cell standard of care therapies, demonstrating higher overall response rates (ORR) and longer overall survival (OS) and event-free survival (EFS) for obe-cel [5]. - Another poster will present findings that deep molecular remission predicts better clinical outcomes in adults treated with obe-cel, with 84% of responders achieving measurable residual disease (MRD) <10 leukemic cells, correlating with improved EFS and OS rates [6][7]. Group 3: Company Overview - Autolus Therapeutics plc is a commercial-stage biopharmaceutical company focused on developing next-generation T cell therapies for cancer and autoimmune diseases, with an FDA-approved product, AUCATZYL, and a pipeline of candidates in development [8]. - The company utilizes proprietary T cell programming technologies to create targeted and effective therapies designed to overcome the limitations of existing treatments [8]. - AUCATZYL is specifically indicated for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, showcasing the company's commitment to addressing significant unmet medical needs in oncology [11].

Core Insights - Autolus Therapeutics is progressing with the commercial launch of AUCATZYL, having 24 treatment centers authorized as of January 10, 2025, with plans to reach 30 by the end of Q1 2025 [1][2] - AUCATZYL has been included in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines for treating adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL) [3] - The company will provide updates on its clinical development program, including expansion into autoimmune diseases, at an R&D investor event scheduled for April 23, 2025 [1][4] Company Updates - The FDA approved AUCATZYL on November 8, 2024, and the company anticipates completing authorization for 60 treatment centers by the end of 2025, covering approximately 90% of the target patient population [2][4] - Autolus is also under regulatory review for AUCATZYL in the European Union and the United Kingdom, with expected marketing approvals in the second half of 2025 [4] Clinical Development - Initial data from the ongoing CARLYSLE Phase 1 trial in systemic lupus erythematosus (SLE) will be shared at the upcoming R&D investor event [2][4] - The company plans to present initial data from the PY01 trial of AUCATZYL in pediatric ALL in the second half of 2025 [4] Product Information - AUCATZYL is a CD19-directed CAR T cell therapy designed to improve clinical activity and safety compared to existing therapies, specifically for adult patients with relapsed or refractory B-ALL [7][8] - The therapy has a fast target binding off-rate to minimize excessive activation of T cells [7] Safety and Efficacy - The safety profile of AUCATZYL was evaluated in the FELIX study, which included 100 patients, with a median dose of 410 × 10^6 CD19 CAR-positive viable T cells [27] - Common serious adverse reactions included infections, febrile neutropenia, and cytokine release syndrome (CRS) [28]

Financial Performance - For the three months ended September 30, 2024, the company incurred a net loss of $82.1 million, compared to a net loss of $45.8 million for the same period in 2023, representing an increase of 79.3%[111] - For the nine months ended September 30, 2024, the net loss was $193.1 million, up from $131.2 million in 2023, indicating a year-over-year increase of 47.2%[111] - The net loss for the three months ended September 30, 2024, was $82.1 million, a 79% increase from the net loss of $45.8 million in the same period of 2023[131] - License revenue was $0 for the three months ended September 30, 2024, compared to $0.4 million in the same period of 2023[132] - License revenue for the nine months ended September 30, 2024, was $10.1 million, a 494% increase from $1.7 million for the same period in 2023[142] Cash and Cash Equivalents - As of September 30, 2024, the company had an accumulated deficit of $1,071.6 million, compared to $878.6 million as of December 31, 2023[111] - The company had cash and cash equivalents of $657.1 million as of September 30, 2024, which is expected to fund operations for at least the next twelve months[111] - As of September 30, 2024, the company had cash and cash equivalents of $657.1 million, up from $239.6 million as of December 31, 2023[169] Operating Expenses - Research and development expenses increased by 25% to $40.3 million for the three months ended September 30, 2024, compared to $32.3 million in the same period of 2023[131] - General and administrative expenses surged by 158% to $27.3 million for the three months ended September 30, 2024, up from $10.6 million in the prior year[131] - Total operating expenses rose by 58% to $67.9 million for the three months ended September 30, 2024, compared to $42.9 million in the same period of 2023[131] - General and administrative expenses rose by $36.4 million to $67.4 million for the nine months ended September 30, 2024, compared to $31.0 million for the same period in 2023, marking a 117% increase[145] - Total research and development expenses for the nine months ended September 30, 2024, were $107.6 million, up 16% from $92.9 million for the same period in 2023[144] Interest and Foreign Exchange - Interest income increased by 128% to $8.3 million for the three months ended September 30, 2024, compared to $3.6 million in the prior year[131] - Interest expense rose by 113% to $10.7 million for the three months ended September 30, 2024, up from $5.0 million in the same period of 2023[131] - Foreign exchange losses increased significantly to $11.9 million for the three months ended September 30, 2024, compared to $1.7 million in the same period of 2023, marking a 586% increase[131] - For the nine months ended September 30, 2024, foreign exchange losses were $12.4 million, up from $0.4 million in the same period of 2023[171] - An immediate hypothetical one percentage point change in interest rates would have resulted in a $1.8 million increase in interest income for the nine months ended September 30, 2024[169] Regulatory Approvals and Clinical Trials - The FDA granted marketing approval for AUCATZYL (obe-cel) for adult relapsed/refractory B-cell Acute Lymphoblastic Leukemia on November 8, 2024[112] - The approval of AUCATZYL triggered a $30.0 million milestone payment from Blackstone, resulting in a net increase to cash and cash equivalents of $16.6 million after a £10.0 million regulatory milestone payment[113] - The ongoing Phase 1 trial (CARLYSLE) for obe-cel in refractory systemic lupus erythematosus is expected to complete enrollment and present initial data in Q1 2025[112] - Clinical programs AUTO8, AUTO6NG, and AUTO1/22 are progressing as expected, with data updates planned for 2025[112] Future Outlook and Funding - The company expects significant expenses and operating losses as it markets AUCATZYL and advances other product candidates through development and regulatory approval[151] - The company plans to establish and expand its sales, marketing, and distribution infrastructure for AUCATZYL and other product candidates[159] - Future funding requirements will depend on the commercialization of AUCATZYL and the progress of clinical trials for other product candidates[161] - The company anticipates increased expenses related to hiring additional personnel and expanding infrastructure to support growth[159] Miscellaneous - The company has no revenue from commercial product sales as of September 30, 2024, with total revenue generated from outlicensing agreements[114] - The company has raised an aggregate of $1.7 billion from various capital sources since its inception in 2014 through September 30, 2024[151] - The company does not currently engage in currency hedging activities but may consider it in the future to manage currency exposure[171] - The effective interest rate for the BioNTech Liability may fluctuate due to discretionary contractual payments[169] - The carrying amount of the Blackstone Collaboration Agreement Liability is based on estimated future royalties and milestones[169] - The company maintains its accounting records in pounds sterling and presents consolidated financial statements in U.S. dollars[171] - Changes in exchange rates have caused material fluctuations in the Consolidated Statements of Operations and Comprehensive Loss[171]