Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates
- 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
- 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
Insights
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.
- 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
“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
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
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
Total operating expenses, net for the year ended December 31, 2023, were
Research and development expenses increased from
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
General and administrative expenses increased from
Net loss attributable to ordinary shareholders was
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
Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer and autoimmune disease. Using a broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize target cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies, solid tumors and autoimmune diseases. For more information, please visit www.autolus.com
About obe-cel (AUTO1)
Obe-cel is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. Obe-cel is designed with a fast target binding off-rate to minimize excessive activation of the programmed T cells. In clinical trials of obe-cel, this “fast off-rate” profile reduced toxicity and T cell exhaustion, resulting in improved persistence and leading to high levels of durable remissions in r/r Adult ALL patients. The results of the FELIX trial, a pivotal trial for adult ALL, have been submitted and accepted by the FDA with a PDUFA target action date of November 16, 2024. A regulatory submission to the EMA was made in the first half of 2024. In collaboration with Autolus’ academic partner, UCL, obe-cel is currently being evaluated in a Phase 1 clinical trials for B-NHL.
About obe-cel FELIX clinical trial
Autolus’ Phase 1b/2 clinical trial of obe-cel enrolled adult patients with relapsed / refractory B-precursor ALL. The trial had a Phase 1b component prior to proceeding to the single arm, Phase 2 clinical trial. The primary endpoint was overall response rate, and the secondary endpoints included duration of response, MRD negative CR rate and safety. The trial enrolled over 100 patients across 30 of the leading academic and non-academic centers in the United States, United Kingdom and Europe. [NCT04404660]
About AUTO1/22
AUTO1/22 is a novel dual targeting CAR T cell-based therapy candidate based on obe-cel. It is designed to combine the enhanced safety, robust expansion and persistence seen with the fast off rate CD19 CAR from obe-cel with a high sensitivity CD22 CAR to reduce antigen negative relapses. This product candidate is currently in a Phase I clinical trial for patients with r/r pediatric ALL. [NCT02443831]
About AUTO6NG
AUTO6NG is a next generation programmed T cell product candidate in development for the treatment of both neuroblastoma and other GD2-expressing solid tumors. AUTO6NG builds on preliminary proof of concept data from AUTO6, a CAR targeting GD2-expression cancer cell currently in clinical development for the treatment of neuroblastoma. AUTO6NG incorporates additional cell programming modules to overcome immune suppressive defense mechanisms in the tumor microenvironment, in addition to endowing the CAR T cells with extended persistence capacity. A Phase 1 clinical trial of AUTO6NG in children with relapsed/refractory neuroblastoma was opened for enrollment in the fourth quarter of 2023.
About AUTO8
AUTO8 is a next-generation product candidate for multiple myeloma which comprises two independent CARs for the multiple myeloma targets, BCMA and CD19. We have developed an optimized BCMA CAR designed for improved killing of target cells that express BCMA at low levels. This has been combined with fast off rate CD19 CAR from obe-cel, with the aim of inducing deep and durable responses and extending the durability of effect over other BCMA CARs currently in development. This product candidate is currently in a Phase I clinical trial for patients with r/r multiple myeloma. [NCT04795882]
Forward-Looking Statements
This press release contains 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," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus’ development and commercialization of its product candidates, timing of data announcements and regulatory submissions, its cash resources and the market opportunity for obe-cel. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, the risks that Autolus’ preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the results of early clinical trials are not always being predictive of future results; the cost, timing and results of clinical trials; that many product candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; and possible safety and efficacy concerns. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Autolus’ actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission, or the SEC, on March 7, 2023 and in Autolus' Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Autolus' subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Autolus undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law. You should, therefore, not rely on these forward-looking statements as representing Autolus’ views as of any date subsequent to the date of this press release.
Contact:
Olivia Manser
+44 (0) 7780 471 568
o.manser@autolus.com
Julia Wilson
+44 (0) 7818 430877
j.wilson@autolus.com
Susan A. Noonan
S.A. Noonan Communications
+1-917-513-5303
susan@sanoonan.com
_____________________________________
1 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
FAQ
What was the total gross proceeds raised through the strategic collaboration and equity investment from BioNTech and underwritten offering of ADSs?
What is the target action date for the Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel) submitted to the FDA?
What authorization did Autolus obtain from MHRA for commercial product supply of obe-cel?
What was the net loss attributable to ordinary shareholders for the year ended December 31, 2023?
How much did the cash and cash equivalents decrease from December 31, 2022, to December 31, 2023?
What was the increase in research and development expenses for the year ended December 31, 2023, compared to 2022?
What was the increase in general and administrative expenses for the year ended December 31, 2023, compared to 2022?
