Autolus Therapeutics (AUTL) Q4 2024 Earnings Call March 20, 2025 08:30 AM ET Company Participants Amanda Cray - Executive Director - IR & External CommunicationsChristian Itin - CEORob Dolski - CFOJames Shin - Director of Biopharma Equity ResearchRajan Sharma - Executive DirectorSimon Baker - Partner Conference Call Participants Asthika Goonewardene - AnalystMatthew Phipps - Equity Research AnalystGil Blum - Senior AnalystKelly Shi - SVP & Senior Research Analyst - BiotechnologyYanan Zhu - AnalystJacob Mekh ...

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]

Financial Data and Key Metrics Changes - Cash and cash equivalents increased to $657.1 million as of September 30, 2024, compared to $239.6 million at the end of December 2023 [37] - Total net operating expenses for Q3 2024 were $67.9 million, up from $42.9 million in Q3 2023 [38] - Research and development expenses rose from $32.3 million to $40.3 million year-over-year [39] - General and administrative expenses increased significantly from $10.6 million to $27.3 million, primarily due to increased headcount for pre-commercialization activities [40] - Net loss for Q3 2024 was $82.1 million, compared to $45.8 million in the same period last year [41] Business Line Data and Key Metrics Changes - The approval of AUCATZYL is a significant milestone, marking the first CAR-T program approved without a REMS requirement by the FDA [7][8] - The company has onboarded approximately 60 centers for AUCATZYL, with 30 ready to activate, covering about 60% of the target patient population in the U.S. [11] - The commercial manufacturing facility, The Nucleus, is expected to produce approximately 2,000 products annually, targeting two-thirds of relapsed or refractory patients in the U.S. and Europe [14] Market Data and Key Metrics Changes - The price for AUCATZYL is set at $525,000, based on extensive evaluations of its clinical value and safety profile [19] - The company is actively engaging with regulatory authorities in Europe and the UK for the approval of obe-cel, with expectations for mid-2025 [21][46] Company Strategy and Development Direction - The company is focused on launching AUCATZYL and expanding its CAR-T therapy offerings, with plans to add 30 more centers in 2025 to reach approximately 90% of the target population [12] - The company is also exploring broader applications for obe-cel beyond ALL, including studies in pediatric patients and systemic lupus [29][30] - The strategy includes a country-by-country approach for the European launch, starting with Germany [85] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the approval of AUCATZYL and the potential impact on ALL patients [9][34] - The company anticipates initial data from the Phase 1 SLE trial by the end of Q1 2025, with further updates on pediatric ALL and long-term observations later in the year [43][35] - Management highlighted the importance of a strong safety profile for obe-cel, which may facilitate outpatient administration [47] Other Important Information - The company expects to receive a $30 million milestone payment from Blackstone due to the FDA approval, along with a £10 million regulatory milestone payment [42] - The company is preparing for presentations at the upcoming ASH meeting, with four abstracts selected for presentation [24] Q&A Session Summary Question: Is obe-cel's EU approval still on track for mid-25? - Management confirmed that the EU approval process is on track for mid-2025, with similar expectations for the UK [46] Question: Can you expand on the baseline characteristics that might suggest obe-cel's superior efficacy? - Management noted that patients with less than 75% tumor burden at lymphodepletion showed an ORR of 85-86%, indicating high efficacy [51] Question: How are you maintaining a low out-of-spec rate in a commercial setting? - Management expressed confidence in maintaining low out-of-spec rates due to established specifications based on clinical performance [57] Question: Can you discuss any feedback from physicians regarding Ticardis versus Obe-cel? - Management reported positive feedback on obe-cel's product profile, highlighting its easier management and lower toxicity [74] Question: Can you provide insights into the SLE trial patient numbers and data disclosure? - The SLE trial is enrolling six patients initially, with data expected in Q1 and longer-term follow-up in the second half of the year [78]

Autolus Q3 2024 Financial Results and Business Updates 12 November 2024 For Investor communication only. Not for use in product promotion. Not for further distribution. Autolus.com 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 in some cases can be identified by terms such as "may," "will," "could," "expects," "pl ...