On May 8, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported its operational and financial results for the first quarter ended March 31, 2025 (Press release, Autolus, MAY 8, 2025, View Source [SID1234652733]).
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"We had a great first quarter of launch and are highly encouraged by physician enthusiasm for AUCATZYL in the U.S. We believe this speaks to the product profile and significant unmet need for patients," said Dr. Christian Itin, Chief Executive Officer of Autolus. "Building on that momentum in the U.S., we recently obtained marketing authorization from the UK’s MHRA, and we are working in collaboration with National Institute for Health and Care Excellence (NICE) to bring this much-needed therapy to patients in the UK. Our goal to expand into new markets is underpinned by our proprietary manufacturing and commercial infrastructure which has positioned us for strong execution."
"In the second quarter we are planning to share longer-term follow-up data from the FELIX study, and in the second half of the year we plan to announce data from the pediatric PY1 trial. Building on strong data with obe-cel in r/r B-ALL, we are looking beyond ALL and recently highlighted at an R&D investor event our potential for value creation driven by obe-cel in autoimmune diseases, including lupus nephritis (LN) and multiple sclerosis (MS). Supporting our plans to pursue LN, we reported encouraging early clinical data that show obe-cel’s potential to treat advanced and relapsed lupus patients. We have aligned with the U.S. Food and Drug Administration (FDA) on a compact Phase 2 trial design and potential registrational path to approval and we look forward to dosing the first patient in the Phase 2 trial before year-end."
Key updates and anticipated milestones:
AUCATZYL Launch
Autolus reported Q1 2025 net product sales of $9.0 million.
The Company has 39 centers fully activated in the U.S. as of May 7, 2025.
Patient access to AUCATZYL continues to increase, with coverage secured for approximately 90% of total U.S. medical lives.
On April 1, 2025, the Centers for Medicare and Medicaid Services (CMS) included AUCATZYL in their published Healthcare Common Procedure Coding System (HCPCS) coding determinations and Hospital Outpatient Prospective Payment System (OPPS) payment rates, formalizing reimbursement for patients on government programs. The CMS policy splits the therapeutic dose of AUCATZYL into two administrations for coding and billing purposes. The Company is working with the treatment centers on implementing the coding and payment policy from CMS and is assessing any potential impact on the timing of revenue recognition.
On April 25, 2025, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization for AUCATZYL (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). The Company will work with the National Institute for Health and Care Excellence (NICE) on patient access to therapy and a meeting is planned for May 2025.
Obe-cel is under regulatory review in the EU and the Company expects to receive notification of approval status from the European Medicines Agency (EMA) in the second half of 2025.
Obe-cel in lupus nephritis (LN)
Preliminary data from the Phase 1 dose confirmation clinical trial (CARLYSLE) in refractory systemic lupus erythematosus (SLE) patients were reported on April 23, 2025, and support progressing into a planned Phase 2 pivotal study. Out of six patients in the cohort, three patients had complete renal response, all by month three. Complement normalized in all patients by month one. Rash, alopecia and mucosal ulcers resolved by month three and arthritis resolved by month one in all patients. Data show high peak expansion and deep B cell aplasia consistent with known obe-cel characteristics in oncology indications. No dose limiting toxicities (DLTs) or immune effector cell-associated neurotoxicity syndrome (ICANS) were observed in the study to date. Grade one cytokine release syndrome (CRS) was observed in three out of six patients. Hypertension, a typical sign of advanced lupus nephritis, pre-existed in three patients. On study, five of six patients experienced a transient hypertension, including Grade 3, well managed by anti-hypertensive agents.
The Company has aligned with U.S. Food and Drug Administration (FDA) on the Phase 2 trial design and potential registrational path to approval and anticipates dosing the first patient in a Phase 2 trial before the end of 2025.
Full data with longer term follow-up from the Phase 1 CARLYSLE clinical trial is targeted for presentation at a medical conference in the second half of 2025.
Obe-cel in progressive MS
Autolus plans to advance obe-cel into clinical development in progressive MS. The Company expects to dose its first patient in a Phase 1 dose escalation study by year-end 2025.
Early-stage pipeline programs and collaborations support longer-term growth
Autolus’ translational programs with UCL continue to fuel its early-stage pipeline, providing a cost-efficient path to development.
Summary of Anticipated News Flow:
ALL: FELIX clinical trial longer-term follow up Mid-Year
ALL: Notification from EU EMA regarding approval in r/r adult ALL H2 2025
ALL: PY01 trial in pediatric ALL first clinical data H2 2025
SLE: Phase 1 CARLYSLE trial presentation at medical conference H2 2025
LN: Expect to dose first patient in Phase 2 trial By year-end 2025
MS: Expect to dose first patient in Phase 1 trial in progressive MS
By year-end 2025
ALA: Expect to dose first patient in Phase 1 trial in AL amyloidosis By year-end 2025
ALL: adult lymphoblastic leukemia
SLE: systemic lupus erythematosus
LN: lupus nephritis
MS: multiple sclerosis
ALA: light-chain amyloidosis
Financial Results for the Quarter Ended March 31, 2025
Product revenue, net for the three months ended March 31, 2025 was $9.0 million.
Cost of sales for the three months ended March 31, 2025 totaled $18.0 million. This amount includes the cost of all commercial product delivered to the authorized treatment centers, including product delivered but not yet recorded as product revenue which is captured as deferred revenue. Additionally, cost of sales includes any cancelled orders in the period, patient access program product, and 3rd party royalties for certain technology licenses.
Research and development expenses decreased from $30.7 million to $26.7 million for the three months ended March 31, 2025, compared to the same period in 2024. This change was primarily due to commercial manufacturing related employee and infrastructure cost shifting to cost of sales and inventory, partially offset by an increase in obe-cel clinical trial and supply costs.
Selling, general and administrative expenses increased from $18.2 million to $29.5 million for the three months ended March 31, 2025, compared to the same period in 2024. This increase was primarily due to salaries and other employment-related costs, driven by increased headcount supporting U.S. commercialization activities.
Loss from operations for the three months ended March 31, 2025 was $65.2 million, as compared to $38.8 million for the same period in 2024.
Net loss was $70.2 million for the three months ended March 31, 2025, compared to $52.7 million for the same period in 2024. Basic and diluted net loss per ordinary share for the three months ended March 31, 2025, totaled $(0.26), compared to basic and diluted net loss per ordinary share of $(0.24) for the same period in 2024.
Cash, cash equivalents and marketable securities at March 31, 2025, totaled $516.6 million, as compared to $588.0 million at December 31, 2024. The decrease was primarily driven by net cash used in operating and investing activities and impacted by a delayed cash receipt of approximately $20 million in R&D tax credit expected from the UK HMRC.
Autolus estimates that, with its current cash and cash equivalents and marketable securities, the Company is well capitalized to drive the launch and commercialization of obe-cel in r/r B-ALL and to obtain data in the LN pivotal trial and MS Phase 1 trial.
Financial Results for the Period Ended March 31, 2025
Selected Consolidated Balance Sheet Data
(In thousands)
March 31 December 31
2025 2024
Assets
Cash and cash equivalents $ 95,799 $ 227,380
Marketable securities – Available-for-sale debt securities $ 420,776 $ 360,643
Total current assets $ 615,773 $ 660,929
Total assets $ 746,338 $ 782,725
Liabilities and shareholders’ equity
Total current liabilities $ 66,615 $ 60,743
Total liabilities $ 375,230 $ 355,400
Total shareholders’ equity $ 371,108 $ 427,325
Selected Consolidated Statements of Operations and Comprehensive Loss Data
(In thousands, except share and per share amounts)
Three months ended March 31,
2025 2024
Product revenue, net $ 8,982 $ —
License revenue — 10,091
Cost and operating expenses:
Cost of sales (17,951 ) —
Research and development expenses, net (26,734 ) (30,671 )
Selling, general and administrative expenses (29,534 ) (18,177 )
Loss on disposal of property and equipment (3 ) —
Loss from operations (65,240 ) (38,757 )
Total other expenses, net (2,696 ) (13,941 )
Net loss before income tax (67,936 ) (52,698 )
Income tax (expense) benefit (2,225 ) 8
Net loss attributable to ordinary shareholders (70,161 ) (52,690 )
Other comprehensive income, net of tax 11,068 58
Total comprehensive loss $ (59,093 ) $ 52,632 )
Basic and diluted net loss per ordinary share $ (0.26 ) $ (0.24 )
Weighted-average basic and diluted ordinary shares 266,126,548 222,170,707
Conference Call
Management will host a conference call and webcast today at 8:30am EDT/13:30pm BST to discuss the company’s financial results. Conference call participants should pre-register using this link to receive the dial-in numbers and a personal PIN, which are required to access the conference call. A simultaneous audio webcast and replay will be accessible on the events section of Autolus’ website at View Source