- 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 inFebruary 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 dateNovember 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
“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
“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
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 ofNovember 16, 2024 . A marketing authorization application (MAA) to theEuropean Medicines Agency (EMA) was just submitted and an MAA submission to the MHRA in theUK 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 BLA for r/r B-ALL submitted to the FDA in
- 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
- 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.
- 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
- 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
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
Recent Operational Updates:
- In
March 2024 , following the most recent GMP inspection by the MHRA inFebruary 2024 , The Nucleus manufacturing facility inStevenage obtained a Manufacturer’s Importation Authorization (MIA), together with the accompanying GMP certificate. This authorization enablesAutolus to manufacture products for global commercial and clinical supply at The Nucleus, effective as ofMarch 18, 2024 . - In
February 2024 ,Autolus promoted Dr.Chris Williams to Chief Business Officer andAlex 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 ofAutolus . Alex has been withAutolus 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 ofElisabeth (Lis) Leiderman , M.D. andRobert W. Azelby to its Board of Directors.Dr. Leiderman brings extensive transactional and financial expertise, andMr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board.
- 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 inNature 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 inBlood 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 & |
Obe-cel Marketing Authorization Application to MHRA | Second half 2024 |
Obe-cel | |
Obe-cel in autoimmune disease – initial data from SLE Phase 1 study | Late 2024 |
Financial Results (Unaudited) for the Full Year Ended
Cash and cash equivalents at
Total operating expenses, net for the year ended
Research and development expenses increased from
In prior years,
General and administrative expenses increased from
Net loss attributable to ordinary shareholders was
Financial Results for the Year Ended | |||||
Selected Unaudited Consolidated Balance Sheet Data | |||||
(In thousands) | |||||
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 | |||||||
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/
A simultaneous audio webcast and replay will be accessible on the events section of Autolus’ website.
About
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
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
Contact:
+44 (0) 7780 471 568
o.manser@autolus.com
+44 (0) 7818 430877
j.wilson@autolus.com
+1-917-513-5303
susan@sanoonan.com
_____________________________________
1 Includes the presentation of our
Source:
2024 GlobeNewswire, Inc., source