Core Insights - Autolus Therapeutics plc announced updated long-term data from the FELIX study of its programmed T cell therapy, obecabtagene autoleucel (obe-cel), for adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) [1][2] - The data will be presented at the European Hematology Association Congress in Milan, Italy, from June 12-15, 2025 [1] Efficacy and Safety - At a median follow-up of 32.8 months, 38.4% of responders remained in ongoing remission without subsequent therapy, showing a slight decrease from 40% at 21.5 months [3] - The 24-month probability of Event Free Survival was reported at 43%, while Overall Survival was at 46%, indicating a long-term plateau in outcomes [3] - No new safety signals or Grade ≥3 secondary malignancies were observed during the extended follow-up, suggesting a favorable safety profile for obe-cel [3][6] Patient Demographics and Treatment Outcomes - Obe-cel treatment demonstrated deep and durable remissions across different age groups, with low incidence of Grade ≥3 cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) [5] - The multivariate analysis indicated that factors such as Philadelphia chromosome-positive disease and lower disease burden at lymphodepletion were associated with higher remission rates [4] Clinical Trial Background - The FELIX clinical trial is a Phase 1b/2 study that enrolled over 100 adult patients with r/r B-precursor ALL across 30 leading centers in the US, UK, and Europe [9] - The primary endpoint was overall response rate, with secondary endpoints including duration of response and safety [9] Company Overview - Autolus Therapeutics is focused on developing next-generation T cell therapies for cancer and autoimmune diseases, with obe-cel being FDA approved and MHRA licensed [8]

Summary of Autolus Conference Call Company Overview - Autolus is a biotechnology company focused on autologous CAR T cell therapies, with its lead program, ObiCell (now branded as Alcatsol), recently approved in the U.S. in November 2022 [4][5][6] - The company is in the launch phase for Alcatsol in the U.S. and is undergoing regulatory processes in the U.K. and Europe [4][5] Key Differentiation and Clinical Data - ObiCell's unique design allows for a "fast on, fast off" mechanism, leading to lower levels of high-grade adverse events such as ICANS and CRS, which are common in CAR T therapies [5][6] - Clinical studies show a plateau in event-free survival and overall survival curves, with about 40% of patients remaining in remission without additional therapy [7] - The company has built a unique manufacturing facility in the U.K. to support commercial capacity for CAR T delivery [8][9] Market Launch and Performance - In Q1, Autolus reported $9 million in revenue, with a significant portion logged as deferred revenue [23] - The onboarding of treatment centers has exceeded expectations, with over 40 centers activated and a target of 60 by year-end, aiming for 90% coverage of addressable patients [24][36] - The company has achieved over 90% coverage of total medical lives in terms of access and reimbursement [25] Challenges and Considerations - Potential impacts of tariffs and most favored nation pricing in the U.S. are being monitored, with the company actively planning for various scenarios [16][19] - Discussions with European health authorities regarding pricing and reimbursement are ongoing, with a focus on the German market initially [46][47] Future Pipeline and Expansion - Autolus is exploring additional indications for ObiCell, including lupus nephritis and multiple sclerosis, with plans for pivotal studies [11][59] - The company is also conducting a pediatric trial for acute lymphoblastic leukemia (ALL) and aims for label expansion based on the data [48][50] Financial Outlook and Investment Thesis - The company targets a gross margin of 15% to 20% of U.S. sales as production volume increases [43] - The investment case for Autolus emphasizes execution of the launch, reliable product delivery, and potential growth in the autoimmune space, despite current market challenges [63][64] Conclusion - Autolus is positioned to leverage its unique CAR T therapy and manufacturing capabilities, with a strong focus on expanding its market presence and addressing unmet medical needs in both oncology and autoimmune diseases [64][65]

Group 1 - The article discusses the importance of staying updated on stocks in the biotech, pharma, and healthcare industries, highlighting key trends and catalysts that influence market valuations [1] - Autolus Therapeutics plc (NASDAQ: AUTL) was previously covered with a bullish outlook due to an upcoming PDUFA date, indicating potential positive developments for the company [1] - The investing group Haggerston BioHealth offers resources for both novice and experienced biotech investors, including sales forecasts and financial analyses for major pharmaceutical companies [1] Group 2 - The article emphasizes the expertise of Edmund Ingham, a biotech consultant with over five years of experience in the sector, who has produced detailed reports on more than 1,000 companies [1]

Summary of Autolus Therapeutics (AUTL) 2025 Conference Call Company Overview - **Company**: Autolus Therapeutics - **Product**: Ocatsil (formerly ObiCell) - **Approval Date**: November 8, 2024 - **Indication**: Approved for patients with relapsed/refractory Acute Lymphoblastic Leukemia (ALL) in the adult population - **Unique Selling Point**: First CAR T therapy approved without a Risk Evaluation and Mitigation Strategy (REMS) obligation, indicating a strong safety profile [4][5] Key Achievements and Launch Strategy - **Launch Progress**: Initiated launch at the end of 2024, currently has 40 active centers, expecting to reach 60 by year-end, providing approximately 90% patient access across the US [5][6] - **Market Access**: Achieved about 90% of lives covered, ensuring reimbursement for patients interested in therapy [6] - **Support Services**: Emphasized the importance of qualifying centers and providing comprehensive support, including training, product management, and patient support [7][8] Manufacturing and Quality Control - **Manufacturing Facility**: Located in the UK, underwent rigorous inspections by MHRA and FDA, allowing for high control over manufacturing processes [8] - **Quality Metrics**: Approximately 5-6% of manufactured products were out of specification during pivotal studies, indicating a low failure rate [29] Product Safety and Efficacy - **Adverse Events**: Reported low rates of high-grade cytokine release syndrome (2%) and neurological toxicities (7%), significantly lower than other T cell-mediated therapies [15][16] - **Dosing Strategy**: Tailored dosing based on tumor burden, allowing for better management of adverse events and improved patient tolerability [12][14] Financial Performance and Revenue Guidance - **Q1 Sales**: Reported $9 million in sales for Q1 2025, with cautious optimism about future revenue growth due to increasing center openings and physician adoption [19][24] - **Revenue Guidance**: Company refrained from providing specific revenue guidance, citing the complexity of launch dynamics and the need for more data over the next three quarters [19][24] Future Opportunities and Trials - **Expansion of Indications**: Plans to expand indications to pediatric ALL and autoimmune diseases, with ongoing trials and discussions with regulatory agencies [34][38] - **Lupus Nephritis Trials**: Focus on patients with advanced lupus nephritis, planning a pivotal study in the second half of 2025 [41][46] - **Multiple Sclerosis Exploratory Study**: Investigating the potential of CAR T cells to cross the blood-brain barrier for treating progressive multiple sclerosis [46][48] Regulatory Considerations - **Single Arm Trials**: Regulatory agencies may consider single-arm trials for high medical need settings, provided there is a high treatment effect and sufficient safety data [51][55] Upcoming Milestones - **Key Data Releases**: Anticipated updates from ongoing studies at upcoming conferences, including EHA and ASH, with a focus on leukemia and lupus nephritis [57] Conclusion - **Overall Sentiment**: The company expressed confidence in its product's safety and efficacy, with a strong focus on execution and market penetration as it navigates the complexities of launching a new CAR T therapy [10][24]

Core Viewpoint - Autolus Therapeutics plc has received a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use for the approval of its therapy, obecabtagene autoleucel (obe-cel), for treating adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia [1][6]. 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 [9]. - The company has a pipeline of product candidates and has received FDA approval and MHRA authorization for obe-cel [9]. Product Details - Obe-cel is an autologous CD19 CAR T cell therapy designed to target B-cell precursor acute lymphoblastic leukemia [4]. - The therapy has shown a Complete Response/Complete Response with Incomplete Hematological Recovery (CR/CRi) rate of 76.6% in the pivotal cohort of the FELIX study [2]. - The median response duration for patients treated with obe-cel was 21.2 months, with median event-free survival (EFS) of 11.9 months [2]. Clinical Study Insights - The CHMP recommendation was based on the results of the FELIX study, which was an open-label, multi-center, single-arm study involving adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia [2][10]. - The study enrolled over 100 patients across 30 leading academic and non-academic centers in the U.S., U.K., and Europe [10]. Safety Profile - The most common non-laboratory Grade 3 or higher adverse reactions included unspecified infections (32%), febrile neutropenia (24%), and bacterial infectious disorders (11%) [3]. - Cytokine release syndrome occurred in 68.5% of patients, with severe cases in 2.4% [3]. Market Context - Acute lymphoblastic leukemia (ALL) is an aggressive blood cancer with approximately 6,000 new cases diagnosed annually in Europe [5]. - Conventional treatments for adult B-ALL have a median overall survival of only eight months, highlighting the need for effective therapies like obe-cel [5]. Regulatory Status - The positive CHMP opinion serves as a scientific recommendation for marketing authorization, with the European Commission expected to make a final decision within approximately two months [6][8]. - Obe-cel has already received FDA approval in November 2024 and MHRA conditional marketing authorization in April 2025 [4][6].

Core Insights - Autolus Therapeutics plc is presenting three abstracts at the upcoming European Hematology Association Congress, focusing on the efficacy of its programmed T cell therapy, obecabtagene autoleucel (obe-cel), for adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) [1] Group 1: Clinical Findings - The oral presentation titled "Can CAR T-cell therapy be a definitive treatment for adult r/r B-ALL without transplant?" indicates that 40% of responders to obe-cel are in ongoing remission without subsequent stem cell therapy, suggesting its potential as a definitive treatment [2] - Another oral presentation discusses the efficacy and safety of obe-cel across different age groups, showing favorable outcomes with low incidence of severe cytokine release syndrome (CRS) and neurotoxicity, indicating a positive benefit-risk profile [3] - A poster presentation highlights the potential of the ALL-Hematotox model to better predict outcomes in patients treated with obe-cel compared to the CAR-Hematotox model, suggesting a need for further analysis [4] Group 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, utilizing proprietary T cell programming technologies [5] - The company has an FDA-approved product, AUCATZYL, and a pipeline of candidates for treating hematological malignancies and solid tumors [5][7] Group 3: Clinical Trial Information - The FELIX clinical trial of obe-cel enrolled over 100 adult patients with r/r B-precursor ALL, with primary endpoints focused on overall response rate and secondary endpoints including duration of response and safety [6]

[Part I – Financial Information](index=6&type=section&id=Part%20I%20%E2%80%93%20Financial%20Information) [Financial Statements](index=6&type=section&id=Item%201.%20Financial%20statements) The company reported its first AUCATZYL product revenue, a $70.2 million net loss, and increased liabilities for Q1 2025, confirming going concern [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets were $746.3 million, liabilities increased to $375.2 million, and equity decreased to $371.1 million as of March 31, 2025 Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $95,799 | $227,380 | | Marketable securities | $420,776 | $360,643 | | Inventories, net | $14,647 | $4,138 | | Total assets | $746,338 | $782,725 | | **Liabilities & Equity** | | | | Total current liabilities | $66,615 | $60,743 | | Liabilities related to future royalties and milestones, net (non-current) | $253,437 | $244,600 | | Total liabilities | $375,230 | $355,400 | | Total shareholders' equity | $371,108 | $427,325 | [Condensed Consolidated Statements of Operations and Comprehensive Income (Loss)](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Income%20(Loss)) Q1 2025 saw first product revenue of $9.0 million, with net loss increasing to $70.2 million from $52.7 million in Q1 2024 Q1 2025 vs Q1 2024 Statement of Operations (in thousands, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Product revenue, net | $8,982 | $— | | License revenue | $— | $10,091 | | **Total revenue, net** | **$8,982** | **$10,091** | | Cost of sales | ($17,951) | $— | | Research and development expenses, net | ($26,734) | ($30,671) | | Selling, general and administrative expenses | ($29,534) | ($18,177) | | **Loss from operations** | **($65,240)** | **($38,757)** | | **Net loss** | **($70,161)** | **($52,690)** | | **Basic and diluted net loss per share** | **($0.26)** | **($0.24)** | [Condensed Consolidated Statements of Changes in Shareholders' Equity](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Shareholders%27%20Equity) Shareholders' equity decreased to $371.1 million by March 31, 2025, primarily due to a $70.2 million net loss - Total shareholders' equity decreased by **$56.2 million** in Q1 2025, mainly driven by the quarterly net loss of **$70.2 million**[26](index=26&type=chunk) - In Q1 2024, the company raised net proceeds of **$520.6 million** from the issuance of ordinary shares[26](index=26&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $75.6 million in Q1 2025, with $59.5 million used in investing and no financing activities Cash Flow Summary (in thousands) | Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($75,565) | ($40,514) | | Net cash used in investing activities | ($59,547) | ($533) | | Net cash provided by financing activities | $— | $561,441 | - The total cash, cash equivalents, and restricted cash decreased by **$131.6 million** during Q1 2025, ending the period at **$97.3 million**[28](index=28&type=chunk)[29](index=29&type=chunk) [Notes to the Unaudited Condensed Consolidated Interim Financial Statements](index=12&type=section&id=Notes%20to%20the%20Unaudited%20Condensed%20Consolidated%20Interim%20Financial%20Statements) Notes detail the company's commercial transition with AUCATZYL, outlining revenue recognition policies and significant royalty liabilities - AUCATZYL (obe-cel) was approved by the FDA on November 8, 2024, for adult r/r B-ALL, with the U.S. commercial launch in January 2025. U.K. MHRA granted conditional marketing authorization in April 2025[32](index=32&type=chunk) - The company recognized its first product revenue of **$9.0 million** in Q1 2025, entirely from the U.S. This revenue is recognized when the product is administered to the patient and is net of estimated gross-to-net deductions[55](index=55&type=chunk)[77](index=77&type=chunk) - Liabilities related to future royalties and milestones from agreements with Blackstone and BioNTech totaled **$258.2 million** as of March 31, 2025. Interest expense accrued on these liabilities was **$10.1 million** for the quarter[125](index=125&type=chunk) - The company has capital commitments of **$16.4 million** for capital expenditures and **$2.9 million** for master supply commitments as of March 31, 2025[143](index=143&type=chunk)[144](index=144&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=32&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's commercial transition with AUCATZYL, reporting $9.0 million net product sales and a $70.2 million net loss in Q1 2025 [Recent Developments](index=33&type=section&id=MD%26A_Recent_Developments) Key Q1 2025 developments include AUCATZYL's U.S. launch, U.K. authorization, and progress in obe-cel clinical trials for LN and MS - Reported Q1 2025 net product sales of **$9.0 million** for AUCATZYL[173](index=173&type=chunk) - As of May 7, 2025, **39 U.S. cancer treatment centers** are activated, with over **90%** of U.S. medical lives having secured coverage for AUCATZYL[173](index=173&type=chunk) - The U.K. MHRA granted conditional marketing authorization for AUCATZYL on April 25, 2025[173](index=173&type=chunk) - The company plans to initiate a Phase 2 pivotal trial for obe-cel in lupus nephritis (LN) and a Phase 1 trial in progressive multiple sclerosis (MS) by year-end 2025[173](index=173&type=chunk)[171](index=171&type=chunk) [Results of Operations](index=39&type=section&id=MD%26A_Results_of_Operations) Q1 2025 total revenue decreased to $9.0 million, with new cost of sales, increased SG&A, and decreased R&D, widening the net loss to $70.2 million Comparison of Operations for Three Months Ended March 31 (in thousands) | Item | 2025 | 2024 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Total revenue, net | $8,982 | $10,091 | ($1,109) | (11)% | | Cost of sales | ($17,951) | $— | ($17,951) | 100% | | R&D expenses, net | ($26,734) | ($30,671) | $3,937 | (13)% | | SG&A expenses | ($29,534) | ($18,177) | ($11,357) | 62% | | **Net loss** | **($70,161)** | **($52,690)** | **($17,471)** | **33%** | - The decrease in R&D expenses was primarily due to a **$6.1 million** reduction from reallocating IT and support costs and a **$2.0 million** reduction in personnel costs to commercial manufacturing activities (cost of sales and inventory) following FDA approval[222](index=222&type=chunk) - The increase in SG&A expenses was driven by a **$7.9 million** rise in personnel costs from increased headcount and a **$1.7 million** increase in commercial readiness costs[223](index=223&type=chunk) [Liquidity and Capital Resources](index=41&type=section&id=MD%26A_Liquidity_and_Capital%20Resources) As of March 31, 2025, the company held $516.6 million in cash and marketable securities, sufficient for twelve months, with significant future capital needs Cash Position (in millions) | Item | March 31, 2025 | | :--- | :--- | | Cash and cash equivalents | $95.8 | | Available-for-sale debt securities | $420.8 | | **Total** | **$516.6** | - The company believes its existing cash and marketable securities will fund operating expenses and capital requirements for at least **twelve months** from the report's issuance date[237](index=237&type=chunk) - Future capital needs will be driven by AUCATZYL commercialization, advancing other product candidates, expanding manufacturing, and hiring additional personnel[236](index=236&type=chunk)[238](index=238&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=44&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risks are interest rate and foreign currency fluctuations, with a 1% interest rate change impacting income by $1.0 million - Primary market risks are interest rate and foreign currency exchange risk[252](index=252&type=chunk) - A hypothetical one percentage point change in interest rates would have resulted in a **$1.0 million** change in interest income for Q1 2025[253](index=253&type=chunk) - As of March 31, 2025, approximately **55%** of cash held by the main U.K. subsidiary was in GBP and **40%** in USD. The company recorded a foreign exchange gain of **$1.2 million** in Q1 2025[256](index=256&type=chunk)[257](index=257&type=chunk) [Controls and Procedures](index=45&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of March 31, 2025, with no material changes to internal control over financial reporting - The CEO and CFO concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[261](index=261&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter ended March 31, 2025[262](index=262&type=chunk) [Part II – Other Information](index=45&type=section&id=Part%20II%20%E2%80%93%20Other%20Information) [Legal Proceedings](index=45&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings, nor is it aware of any pending or threatened actions - As of the filing date, the company is not a party to any material legal proceedings[263](index=263&type=chunk) [Risk Factors](index=46&type=section&id=Item%201A.%20Risk%20Factors) Key risks include government price controls, CMS reimbursement policies delaying AUCATZYL revenue, and international trade policies impacting supply chain and costs - A new CMS policy splits the AUCATZYL dose into two administrations for billing, which may delay the company's and treatment centers' ability to recognize revenue[265](index=265&type=chunk) - International trade policies, including tariffs and sanctions, pose a risk as the company depends on a global supply chain and manufactures all commercial and clinical supplies in the U.K.[267](index=267&type=chunk) - The company's ability to pass on increased costs from tariffs is limited by fixed-price contracts with payors, which could negatively impact profitability[270](index=270&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=47&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities during the period - None[274](index=274&type=chunk) [Defaults Upon Senior Securities](index=47&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) There were no defaults upon senior securities during the period - None[275](index=275&type=chunk) [Mine Safety Disclosures](index=47&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[276](index=276&type=chunk) [Other Information](index=47&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted or terminated Rule 10b5-1 trading arrangements during Q1 2025 - No directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during Q1 2025[277](index=277&type=chunk) [Exhibits](index=48&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Quarterly Report on Form 10-Q, including the 2025 Inducement Plan and officer certifications

Financial Data and Key Metrics Changes - The company reported recognized revenue of $9 million for Q1 2025, marking a strong start for the U.S. launch of Ocassel [8][42] - Cost of sales totaled $18 million, which includes costs for all commercial products delivered to authorized treatment centers [44] - The net loss for Q1 2025 was $70.2 million, compared to a net loss of $52.7 million for the same period in 2024 [46] Business Line Data and Key Metrics Changes - The company aims to increase the number of centers authorized to deliver Ocassel from 39 to approximately 60, targeting 90% access to patients across the U.S. [11] - Research and development expenses decreased to $26.7 million from $30.7 million in the same period in 2024, primarily due to cost shifts following the approval of Ocassel [44] Market Data and Key Metrics Changes - As of April 1, CMS published codes for inpatient and outpatient use for Ocassel, formalizing reimbursement for patients on government programs [9][43] - The company is preparing for market expansion in the UK and Europe, having received conditional marketing authorization from the MHRA in the UK [12] Company Strategy and Development Direction - The company plans to expand its market share for CAR T products and is exploring opportunities in acute lymphoblastic leukemia and pediatric populations [12][14] - The strategy includes a fast-to-market approach for lupus nephritis, focusing on a refractory patient population with a clear objective endpoint [36] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial momentum seen in Q1 and expects this to carry into the second quarter [10] - The company is well-capitalized with $516.6 million in cash and equivalents, which supports ongoing commercialization efforts [46][52] Other Important Information - The company is evaluating the implications of the April 1 coding update on revenue recognition policies [57] - The safety profile of Ocassel is favorable, with no high-grade cytokine release syndrome observed in patients [19] Q&A Session Summary Question: Can you clarify the revenue recognition for Ocassel? - Management confirmed that revenue recognition for Q1 was based on patients receiving both doses, and they are evaluating the implications of the new coding update [54][56] Question: What is the potential exposure to UK or pharma-specific tariffs? - Management noted that blood products are typically exempt from tariffs, but the final impact remains uncertain pending government announcements [60][62] Question: Can you provide details on the cost of goods sold (COGS) for the quarter? - Management indicated that the majority of COGS is driven by the cost of products sold and those delivered but not yet booked as sales, with smaller contributions from patient assistance programs [71][73] Question: What is the median turnaround time for manufacturing success? - Management reported that the turnaround time is tracking towards the target of 16 days, consistent with previous trials [86]

Financial Data and Key Metrics Changes - The company reported recognized revenue of $9 million for Q1 2025, marking a strong start for the U.S. launch of Ocassel [7][42] - Cost of sales totaled $18 million, which includes costs for all commercial products delivered to authorized treatment centers [43][44] - Research and development expenses decreased to $26.7 million from $30.7 million in the same period in 2024, primarily due to cost shifts following Ocassel's approval [44] - Selling, general, and administrative expenses increased to $29.5 million from $18.2 million in the same period in 2024, driven by increased headcount for U.S. commercialization [45] - The net loss for Q1 2025 was $70.2 million, compared to $52.7 million for the same period in 2024 [46] Business Line Data and Key Metrics Changes - The company is focusing on expanding the number of centers authorized to deliver Ocassel therapy from 39 to approximately 60, aiming for 90% patient access across the U.S. [9][11] - The company is also planning to present key updates based on long-term follow-up from the FELIC study, which is expected to provide encouraging data for Ocassel [10] Market Data and Key Metrics Changes - As of April 1, CMS published codes for Ocassel, formalizing reimbursement for patients on government programs, which is expected to enhance market access [7][43] - The company has received conditional marketing authorization from the MHRA in the UK and is engaging with NICE for reimbursement processes [11] Company Strategy and Development Direction - The company aims to increase the number of treatment centers and expand market share for CAR T products, with a focus on geographic expansion into the UK and Europe [11][12] - The company is exploring the utility of its products in earlier stages of treatment and in pediatric populations, with plans for investigator-sponsored trials [13][14] - The company is also looking into opportunities beyond acute lymphoblastic leukemia, targeting a broader range of B cell malignancies and autoimmune diseases [20][21] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial momentum seen in Q1 and expects this to carry into Q2 [8] - The company is well-capitalized with $516.6 million in cash and equivalents, which is expected to support the launch and commercialization of its products [46][52] - Management highlighted the importance of medical need in regulatory assessments and the potential for accelerated access to therapies [66] Other Important Information - The company is preparing for updates and data presentations for various studies, including the Carlyle study for lupus nephritis and a phase I study for progressive MS [47][48] - The company is also evaluating the implications of recent coding updates on revenue recognition policies [56] Q&A Session Summary Question: Revenue recognition for Ocassel and impact of coding update - Management confirmed that revenue recognition for Q1 was based on patients receiving both doses, and they are evaluating the implications of the April 1 coding update on revenue recognition [54][56] Question: Potential exposure to tariffs - Management noted that blood products are typically exempt from tariffs, and the customs value used for tariff calculations is linked to manufacturing costs rather than product sales [61][62] Question: Manufacturing turnaround time and success rate - Management indicated that the turnaround time is tracking towards the target of 16 days, and while it is premature to discuss success rates, initial results indicate good quality products [87][88]

AUCATZYL Launch and Growth - AUCATZYL achieved net product sales of $9 million in Q1 2025[8] - Approximately 90% of total U S medical lives are covered for AUCATZYL[10] - 39 Treatment Centers Authorized as of 05/07/25[8] - Conditional marketing authorization in the UK received April 25, 2025, with EMA decision expected in H2 2025[12] Obe-cel Development and Potential - Obe-cel demonstrated a high MRD-negative complete remission rate of 94% in r/r adult and pediatric acute B cell lymphoblastic leukemia (ALL) patients[17] - In non-Hodgkin lymphoma, obe-cel showed a high metabolic complete remission rate of 88% in r/r LBCL and 95% in r/r FL[17] - Preliminary CARSLYLE SLE P1 trial data shows 10+ point drop in SLEDAI-2K scores and 3 of 6 patients with renal CRs by month 3[22] Financial Status - The company's cash, cash equivalents, and marketable securities totaled $516 6 million as of Q1 2025[41] - Net product revenue for Q1 2025 was $8 982 million[40] Upcoming Milestones - The company anticipates dosing the first patient in the Phase 2 trial for lupus nephritis by year-end 2025[32] - The company anticipates dosing the first patient in the progressive MS Phase 1 trial by year-end 2025[37]