Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates
Autolus Therapeutics plc announces strategic collaboration and equity investment from BioNTech, submits BLA for obecabtagene autoleucel, and completes underwritten offering of ADSs. Financial results show increased expenses and net loss for the year ended December 31, 2023.
Positive
Strategic collaboration and equity investment from BioNTech for $250 million upfront and underwritten offering of ADSs for $350 million, totaling $600 million in gross proceeds
Submission of Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel) for r/r adult B-cell Acute Lymphoblastic Leukemia (ALL) to the FDA with PDUFA target action date of November 16, 2024
Completion of first facility inspection and obtaining Manufacturer’s Importation Authorization (MIA) from MHRA for commercial product supply of obe-cel
Market Authorization Application (MAA) submission for obe-cel in r/r adult ALL with EMA
Increased total operating expenses, net for the year ended December 31, 2023, to $179.7 million from $143.4 million in 2022
Net loss attributable to ordinary shareholders of $208.4 million for the year ended December 31, 2023, compared to $148.8 million in 2022
Negative
Decrease in cash and cash equivalents from $382.4 million to $239.6 million at December 31, 2023
Increase in research and development expenses from $117.4 million to $130.5 million for the year ended December 31, 2023
Increase in general and administrative expenses from $31.9 million to $46.7 million for the year ended December 31, 2023
The strategic collaboration and equity investment by BioNTech in Autolus Therapeutics, combined with the additional $350 million from an underwritten offering of ADSs, reflect a significant influx of capital, totaling $600 million. This financial boost is a strong indicator of investor confidence and positions Autolus favorably for the commercialization of their lead CAR T therapy candidate, obe-cel, targeting relapsed/refractory adult B-cell Acute Lymphoblastic Leukemia (ALL). The financial support from a prominent player like BioNTech not only provides substantial funding but also infers a strategic endorsement of Autolus’ technology and pipeline.
From a financial perspective, the company’s reported cash and cash equivalents at the end of 2023, amounting to $239.6 million, coupled with the recent capital raise, suggests robust financial health and the ability to sustain operations and R&D activities. However, it is crucial to monitor the burn rate closely, as the increased R&D expenses, which rose to $130.5 million, indicate significant investment in the development of their therapies. The net loss widening to $208.4 million year-over-year raises questions about the sustainability of current expenditure levels without generating revenue. Stakeholders should consider both the potential of the pipeline and the financial prudence of the company’s strategy.
The submission of a Biologics License Application (BLA) for obe-cel to the FDA and the Market Authorization Application (MAA) to the EMA signifies a critical regulatory milestone for Autolus. Obe-cel’s potential as a transformative treatment for relapsed/refractory adult B-cell ALL could address a significant unmet medical need, given the aggressive nature of this cancer and the limited options for patients who have relapsed or are refractory to existing therapies.
The data from the FELIX study indicating prolonged event-free survival and low overall immunotoxicity is promising, particularly in the context of CAR T therapies where safety concerns, such as cytokine release syndrome and neurotoxicity, have been paramount. The durability of the response, as suggested by the >3-year follow-up data, is particularly encouraging for long-term patient outcomes.
Furthermore, the expansion of obe-cel into autoimmune diseases, with a Phase 1 trial in refractory systemic lupus erythematosus (SLE) underway, highlights the versatility of Autolus’ technology platform and could open additional market opportunities. The initial data from this study, expected in late 2024, will be pivotal in assessing the broader applicability of obe-cel beyond oncology.
The strategic alliance between Autolus and BioNTech is likely to accelerate the clinical development and commercialization efforts of Autolus’ pipeline. This partnership could enhance Autolus’ market position in the competitive landscape of cell therapy. The right granted to BioNTech to utilize Autolus’ manufacturing capacity and technologies may also streamline the production process, potentially reducing costs and improving efficiency.
Moreover, the commercialization rights and royalty arrangements on net sales of obe-cel could provide a recurring revenue stream for Autolus, contingent upon successful product launch and market adoption. It is essential to evaluate the market size for r/r adult B-cell ALL and the competitive landscape, including other CAR T therapies, to estimate the potential market share and revenue obe-cel might capture. Additionally, the exploration of obe-cel’s use in autoimmune diseases represents a strategic move to diversify the application of their CAR T platform, potentially tapping into a broader patient population and generating additional revenue streams.
03/14/2024 - 07:00 AM
Announced strategic collaboration and equity investment from BioNTech for aggregate proceeds of $250 million upfront, plus underwritten offering of ADSs for $350 million , for gross proceeds of $600 million received in February 2024 Submitted a Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel), a potentially transformational treatment for relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL), to the US Food & Drug Administration (FDA); Prescription Drug User Fee Act (PDUFA) target action date November 16, 2024 Successfully completed first facility inspection and obtained a Manufacturer’s Importation Authorization (MIA) from the Medicines and Healthcare products Regulatory Agency (MHRA), enabling the commercial product supply for obe-cel at The Nucleus manufacturing facility Submitted a Market Authorization Application (MAA) for obe-cel in r/r adult ALL with the European Medicines Agency (EMA) Conference call to be held today at 08:30 am EDT/12:30 pm GMT: Conference call participants should pre-register using the link at the bottom of this press release LONDON, March 14, 2024 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced its operational and financial results for the full year ended December 31, 2023.
“We’re delighted to be starting 2024 in such a strong financial position; our recently announced strategic alliance with BioNTech, coupled with two equity financing transactions, raised gross proceeds of $600 million . Combined with our 2023 ending cash of $240 million , this enables us to drive the full launch and commercialization of obe-cel in r/r adult ALL and establish Autolus as a potential leader in the delivery of CAR T therapy to patients with autoimmune diseases,” said Dr. Christian Itin, Chief Executive Officer of Autolus.
“2023 was a transformational year for the Company. Our lead program, obe-cel, demonstrated strong data in B-ALL in the pivotal FELIX study, we fully validated our commercial manufacturing facility, The Nucleus, to support our regulatory submissions and we submitted our first BLA for obe-cel to the United States Food and Drug Administration (FDA) in November, with a PDUFA target action date of November 16, 2024. We also just submitted an MAA to the European Medicines Agency (EMA).
“Beyond B-ALL, we see a significant opportunity for obe-cel in autoimmune disease. Our Phase 1 dose confirmation trial in refractory SLE is now open for enrollment. We believe obe-cel’s clinical profile, together with our commercial product delivery base and infrastructure, will help to drive an accelerated and differentiated expansion in autoimmune diseases and we look forward to sharing initial data from the study in late 2024.
“For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company. Pre-commercial and product delivery activities are well underway, and we are on track to make obe-cel available to B-ALL patients as soon as possible, following a potential approval.”
Key obecabtagene autoleucel (obe-cel) updates and anticipated milestones:
Obe-cel in relapsed / refractory (r/r) adult ALL – The FELIX Study Obe-cel 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 just submitted and an MAA submission to the MHRA in the UK is planned for the second half of 2024. Pooled analysis of the FELIX Phase 1b/2 study presented at ASH 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. Additionally, data from a pooled analysis from the ALLCAR19 study and FELIX Phase 1b in r/r B-ALL showed durable remissions with obe-cel as a stand-alone therapy in a subset of patients after a median follow up of >3 years. Further long-term data from the FELIX study is anticipated at medical conferences in 2024. Obe-cel in B-cell mediated autoimmune diseases The Phase 1 dose confirmation study in refractory systemic lupus erythematosus (SLE) patients has the first site open for enrollment; initial clinical data expected 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 in December 2023 showed AUTO8 was well tolerated, with responses observed in all patients. Further updates from the MCARTY study are anticipated during 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. Post-period:
Strategic developments:
Strategic alliance with BioNTech SE
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.
Overview:
BioNTech has right to utilize Autolus’ manufacturing capacity, know-how and cost-efficiencies to accelerate the planned clinical development and commercialization of BNT211 BioNTech to support launch and expansion of development program of Autolus’ lead cell therapy candidate obe-cel and will receive a royalty on net sales BioNTech has co-commercialization options for Autolus’ AUTO1/22 and AUTO6NG programs, and an option to access a suite of Autolus target binders and cell programming technologies Underwritten offering
In February 2024, Autolus completed an underwritten registered direct offering in the United States at a price of $6.00 per ADS, for total gross proceeds of $350 million .
Recent Operational Updates:
In March 2024, following the most recent GMP inspection by the MHRA in February 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 February 2024, Autolus promoted Dr. Chris Williams to Chief Business Officer and Alex Driggs to Senior Vice President, Legal Affairs and General Counsel. Chris has been with the Company since its inception, having negotiated on behalf of UCL the spin off and formation of Autolus. Alex has been with Autolus since 2018 in the role of General Counsel. Dr. Edgar Braendle has notified the Company that he will step down as Chief Development Officer to pursue other opportunities. Edgar will continue to advise the company during the BLA and MAA review process. Miranda Neville, SVP and obe-cel Program Leader will run the Development team. Autolus announced the appointment of Elisabeth (Lis) Leiderman, M.D. and Robert W. Azelby to its Board of Directors. Dr. Leiderman brings extensive transactional and financial expertise, and Mr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board. 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, EHA & ASH 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 Full Year Ended December 31, 2023
Cash and cash equivalents at December 31, 2023 totaled $239.6 million , as compared to $382.4 million at December 31, 2023.
Total operating expenses, net for the year ended December 31, 2023, were $179.7 million , as compared to $143.4 million , for the year ended December 31, 2022.
Research and development expenses increased from $117.4 million to $130.5 million for the year ended December 31, 2023, compared to the same period in 2022. This change was primarily due to increases in operating costs related to the Company’s new commercial manufacturing facility, contractual milestone payments and employee salaries and related costs, a decrease in our U.K. reimbursable R&D tax credits claimable through the U.K. small and medium-sized entity (SME) scheme and partially offset by decreases in clinical and manufacturing costs related to the Company’s obe-cel clinical product candidate.
In prior years, Autolus reported the R&D tax credits as income tax benefit on its statements of operations. The Company has revised its financial presentation, including the prior years, and will now present such tax credits as a reduction in research and development expense. As a result, income tax benefit has reduced by $19.5 million and $24.6 million for the years ended December 31, 2023, and 2022, respectively, with corresponding reductions in research and development expenses and total operating expenses.
General and administrative expenses increased from $31.9 million to $46.7 million for the year ended December 31, 2023, compared to the same period in 2022. 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 attributable to ordinary shareholders was $208.4 million for the year ended December 31, 2023, compared to $148.8 million for the same period in 2022. The basic and diluted net loss per ordinary share for the year ended December 31, 2023, totaled $(1.20) , compared to a basic and diluted net loss per ordinary share of $(1.57) for 2022.
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 Year Ended December 31, 2023 Selected Unaudited Consolidated Balance Sheet Data (In thousands) December 31, 2023 2022 Assets Cash and cash equivalents $ 239,566 $ 382,436 Total current assets $ 275,302 $ 425,771 Total assets $ 375,381 $ 490,274 Liabilities and shareholders’ equity Total current liabilities $ 44,737 $ 46,366 Total liabilities $ 263,907 $ 191,600 Total shareholders' equity $ 111,474 $ 298,674
Selected Unaudited Consolidated Statements of Operations and Comprehensive Loss Data (In thousands, except share and per share amounts) Year Ended December 31, 2023 2022 Grant income $ — $ 166 License revenues 1,698 6,194 Operating expenses: Research and development1 (130,481 ) (117,354 ) General and administrative (46,745 ) (31,899 ) Loss on disposal of property and equipment (3,791 ) (515 ) Impairment of operating lease right-right-of-use and related property equipment (382 ) — Total operating expenses, net (179,701 ) (143,408 ) Total other expenses, net (28,701 ) (5,159 ) Net loss before income tax (208,402 ) (148,567 ) Income tax benefit (expense)1 19 (272 ) Net loss attributable to ordinary shareholders (208,383 ) (148,839 ) Other comprehensive income (loss): Foreign currency exchange translation adjustment 9,906 (30,328 ) Total comprehensive loss $ (198,477 ) $ (179,167 ) Basic and diluted net loss per ordinary share $ (1.20 ) $ (1.57 ) Weighted-average basic and diluted ordinary shares 173,941,926 94,993,400
Conference Call
Management will host a conference call and webcast at 08:30 am EDT/12:30 pm GMT 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.
About Autolus Therapeutics plc www.autolus.com
About obe-cel (AUTO1)
About obe-cel FELIX clinical trial
About AUTO1/22 NCT02443831 ]
About AUTO6NG
About AUTO8
Forward-Looking Statements
Contact:
Olivia Mansero.manser@autolus.com
Julia Wilsonj.wilson@autolus.com
Susan A. Noonansusan@sanoonan.com
_____________________________________ Includes the presentation of our U.K. SME R&D Tax Credit with Income tax benefit as contra research and development expense in the amounts of $19.5 million and $24.6 million for the years ended December 31, 2023, and 2022, respectively.
What was the total gross proceeds raised through the strategic collaboration and equity investment from BioNTech and underwritten offering of ADSs?
The total gross proceeds raised were $600 million.
What is the target action date for the Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel) submitted to the FDA?
The Prescription Drug User Fee Act (PDUFA) target action date is November 16, 2024.
What authorization did Autolus obtain from MHRA for commercial product supply of obe-cel?
Autolus obtained a Manufacturer’s Importation Authorization (MIA) from the Medicines and Healthcare products Regulatory Agency (MHRA).
What was the net loss attributable to ordinary shareholders for the year ended December 31, 2023?
The net loss attributable to ordinary shareholders was $208.4 million for the year ended December 31, 2023.
How much did the cash and cash equivalents decrease from December 31, 2022, to December 31, 2023?
The cash and cash equivalents decreased from $382.4 million to $239.6 million.
What was the increase in research and development expenses for the year ended December 31, 2023, compared to 2022?
Research and development expenses increased from $117.4 million to $130.5 million for the year ended December 31, 2023.
What was the increase in general and administrative expenses for the year ended December 31, 2023, compared to 2022?
General and administrative expenses increased from $31.9 million to $46.7 million for the year ended December 31, 2023.
