On May 17, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported its operational and financial results for the first quarter ended March 31, 2024 (Press release, Autolus, MAY 17, 2024, View Source [SID1234643422]).
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"We continue to engage with the FDA in the regulatory review process for obecabtagene autoleucel (obe-cel) in adult ALL as we head towards the PDUFA target action date of November 16, 2024, and are driving commercial readiness activities across the Company," said Dr. Christian Itin, Chief Executive Officer of Autolus. "We’re also delighted that our abstracts from the pivotal FELIX Phase 2 trial have been accepted for oral presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) this year and we look forward to sharing further long-term data and additional subset analyses."
"In addition, the first two patients have been enrolled into our dose confirmation trial (CARLYSLE) of obe-cel in Systemic Lupus Erythematosus (SLE) and the study is on track for initial data by end of 2024."
Key obe-cel updates and anticipated milestones:
Obe-cel in relapsed / refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL) – The FELIX Study
Obe-cel Biologics License Application (BLA) for r/r B-ALL submitted to the FDA in November 2023; PDUFA target action date of November 16, 2024. A marketing authorization application (MAA) to the European Medicines Agency (EMA) was accepted in April 2024. For the UK we are evaluating a filing based on an international recognition procedure.
Pooled analysis of the FELIX Phase 1b/2 study presented at ASH (Free ASH Whitepaper) in December 2023 demonstrated prolonged event free survival and low overall immunotoxicity across all cohorts in r/r B-ALL, and particularly in patients with low leukemic burden at lymphodepletion.
Further long-term data from the FELIX study including additional subset analysis will be presented in oral and poster presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting (ASCO – May 31 – June 4, 2024), and European Hematology Association (EHA) (Free EHA Whitepaper) congress (EHA – June 13 – 16, 2024) respectively.
Obe-cel in B-cell mediated autoimmune diseases
The Phase 1 dose confirmation study (CARLYSLE) in refractory systemic lupus erythematosus (SLE) patients is ongoing. Two patients have been enrolled and Autolus continues to expect initial clinical data in late 2024.
Pipeline clinical trials, in collaboration with University College London (UCL), updates and anticipated milestones:
AUTO8 in Multiple Myeloma – Phase 1 MCARTY Study
AUTO8 is a next-generation product candidate for multiple myeloma, which includes two CARs for the multiple myeloma targets, BCMA and CD19. Initial data from the MCARTY Phase 1 study in multiple myeloma presented at ASH (Free ASH Whitepaper) in December 2023 showed AUTO8 was well tolerated, with responses observed in all patients. Enrollment of the initial cohorts are complete and further updates from the MCARTY study are anticipated in H2 2024.
AUTO6NG in Neuroblastoma – Phase 1 MAGNETO Study
AUTO6NG contains a CAR that targets GD2 alongside additional programming modules to enhance the activity and persistence. A Phase 1 clinical study in children with r/r neuroblastoma was opened for enrollment in the fourth quarter of 2023.
Strategic developments:
In February 2024, BioNTech and Autolus announced a strategic CAR T cell therapy collaboration to advance their pipelines and expand late-stage programs, for $50 million cash upfront and up to $582 million in potential option exercise and milestone payments. Additionally, Autolus sold $200 million of ADSs to BioNTech in a concurrent private placement financing transaction.
In February 2024, Autolus completed an underwritten offering in the United States at a price of $6.00 per ADS, for total gross proceeds of $350 million before underwriting fees and offering expenses.
Operational Updates:
In March 2024, The Nucleus manufacturing facility in Stevenage obtained a Manufacturer’s Importation Authorization (MIA), together with the accompanying GMP certificate. This authorization enables Autolus to manufacture products for global commercial and clinical supply at The Nucleus, effective as of March 18, 2024.
In April 2024, Autolus announced that the European Medicines Agency (EMA) had accepted its Marketing Authorization Application (MAA) for obe-cel for patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL). The MAA submission was based on data from the pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.
In April 2024, Autolus entered into a distribution services agreement with a subsidiary of Cardinal Health to support the ordering and distribution of obe-cel in the United States, following the receipt of regulatory approval.
In April 2024, Autolus announced the appointment of Mike Bonney as Chairman of the Board, and Ravi Rao M.D., as Non-Executive Director. John H. Johnson advised the Board of his decision to step down from his role as Chairman of the Board and Non-Executive Director, effective April 1, 2024.
Scientific Publications:
In January 2024, Autolus announced the publication of a paper in ACS Chemical Biology entitled: ‘Designer small molecule control system based on Minocycline induced disruption of protein-protein interaction’ – Jha et al., ACS Chemical Biology (2024) doi:10.1021/acschembio.3c00521; [Link]
In February 2024, Autolus announced the publication of a paper in Nature Communications entitled: ‘Structure-Guided Engineering of Immunotherapies Targeting TRBC1 and TRBC2 in T Cell Malignancies’ – Ferrari et al., Nat Commun 15, 1583 (2024) doi:10.1038/s41467-024-45854-3; [Link]
In March 2024, Autolus announced the publication of a paper in Blood Cancer Journal entitled: ‘Dual T-cell constant β chain (TRBC)1 and TRBC2 staining for the identification of T-cell neoplasms by flow cytometry – Horna et al., Blood Cancer J. 14, 34 (2024) doi: 10.1038/s41408-024-01002-0; [Link]
2024 Expected News Flow:
Obe-cel FELIX data update at ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper) & ASH (Free ASH Whitepaper) May, June & Dec 2024
Obe-cel Marketing Authorization Application to MHRA Second half 2024
Obe-cel U.S. FDA PDUFA target action date November 16, 2024
Obe-cel in autoimmune disease – initial data from SLE Phase 1 study Late 2024
Financial Results (Unaudited) for the Quarter Ended March 31, 2024
Cash and cash equivalents at March 31, 2024, totaled $758.5 million, as compared to $239.6 million at December 31, 2023.
Total operating expenses, net for the three months ended March 31, 2024, were $38.8 million, as compared to $39.1 million, for the same period in 2023.
Research and development expenses increased from $27.4 million to $30.7 million for the three months ended March 31, 2024, compared to the same period in 2023. This change was primarily due to increases in operating costs related to the Company’s new commercial manufacturing facility, employee salaries and related costs, clinical trial costs related to obe-cel, and a decrease in our U.K. reimbursable R&D tax credits claimable through the U.K. small and medium-sized entity (SME) scheme. These were partially offset by decreases in professional consulting fees, legal fees, manufacturing costs related to obe-cel clinical supply, information technology infrastructure fees and general office expenses.
General and administrative expenses increased from $9.3 million to $18.2 million for the three months ended March 31, 2024, compared to the same period in 2023. This increase was primarily due to salaries and other employment-related costs driven by an increase in general and administrative headcount supporting the overall growth of the business, primarily relating to pre-commercialization activities.
Net loss was $52.7 million for the three months ended March 31, 2024, compared to $39.8 million for the same period in 2023. The basic and diluted net loss per ordinary share for the three months ended March 31, 2024, totaled $(0.24), compared to a basic and diluted net loss per ordinary share of $(0.23) for 2023.
Autolus estimates that, with its current cash and cash equivalents and proceeds received from the strategic alliance with BioNTech and the private placement and underwritten equity financing, it is well capitalized to drive the full launch and commercialization of obe-cel in r/r adult ALL as well as to advance its pipeline development plans, which includes providing runway to data in the first pivotal study of obe-cel in autoimmune disease.
Financial Results for the Quarter Ended March 31, 2024
Selected Unaudited Condensed Consolidated Balance Sheet Data
(In thousands)
March 31 December 31
2024 2023
Assets
Cash and cash equivalents $ 758,529 $ 239,566
Total current assets $ 804,298 $ 275,302
Total assets $ 901,436 $ 375,381
Liabilities and shareholders’ equity
Total current liabilities $ 43,985 $ 44,737
Total liabilities $ 319,406 $ 263,907
Total shareholders’ equity $ 582,030 $ 111,474
Selected Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss Data
(In thousands, except share and per share amounts)
Three Months Ended March 31,
2024 2023
License revenues $ 10,091 $ 1,292
Operating expenses:
Research and development (30,671 ) (27,388 )
General and administrative (18,177 ) (9,284 )
Loss on disposal of property and equipment - (3,768 )
Total operating expenses, net (38,757 ) (39,148 )
Total other expenses, net (13,941 ) (677 )
Net loss before income tax (52,698 ) (39,825 )
Income tax benefit 8 14
Net loss (52,690 ) (39,811 )
Other comprehensive income (loss):
Foreign currency exchange translation adjustment 58 5,641
Total comprehensive loss $ (52,632 ) $ (34,170 )
Basic and diluted net loss per ordinary share $ (0.24 ) $ (0.23 )
Weighted-average basic and diluted ordinary shares 222,170,707 173,825,825
Conference Call
Management will host a conference call and webcast at 08:30 am EDT/13:30 pm BST to discuss the Company’s financial results and provide a general business update. 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.