Autolus Therapeutics announces acceptance of Biologics License Application for obecabtagene autoleucel (obe-cel) as a potential treatment for relapsed/refractory Adult B-cell Acute Lymphoblastic Leukemia (ALL)
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Insights
The acceptance of Autolus Therapeutics' BLA for obecabtagene autoleucel (obe-cel) by the FDA marks a significant progression in the regulatory pathway for a novel therapy in the treatment of Adult B-Cell Acute Lymphoblastic Leukemia (ALL). The critical aspect of this development lies in the potential of obe-cel to address the unmet needs in relapsed/refractory (r/r) ALL, a patient group that typically has limited treatment options and a poor prognosis. The FELIX study's data, which underpin the BLA, have already been scrutinized by the medical community at prestigious conferences, suggesting a promising efficacy and safety profile.
As obe-cel has received Orphan Drug and RMAT designations, this implies that the therapy targets a condition affecting a small patient population and represents a significant advance over existing therapies. The Orphan Drug Designation also provides certain benefits, such as tax credits and market exclusivity upon approval, which could have substantial commercial implications for Autolus. However, the competitive landscape for CAR T therapies is evolving and the long-term commercial success of obe-cel will depend on its clinical differentiation from other treatments and the company's ability to navigate market access challenges, including reimbursement.
The PDUFA date set for November 16, 2024, provides a definitive timeline for investors and the company regarding the FDA's decision, which is crucial for forecasting the potential revenue generation and market entry. The absence of a planned FDA advisory committee meeting could be interpreted as a sign of straightforward regulatory proceedings, although it does not guarantee approval. For the stock market, this event is a critical inflection point that could significantly affect Autolus' stock performance, depending on the outcome.
Additionally, the planned submission to the EMA indicates Autolus' intention to commercialize obe-cel in Europe, expanding the potential market. However, investors should be mindful of the capital requirements and operational execution needed to launch a new therapy. The market's response will likely hinge on further updates regarding the BLA's progress, any partnership announcements for distribution or commercialization and the anticipation of market dynamics post-approval.
Understanding the market dynamics for CAR T therapies is essential for evaluating the potential impact of obe-cel. The designation of obe-cel as a PRIME medicine by the EMA is indicative of the therapy's potential to benefit patients with significant unmet medical needs and may expedite the review process in Europe. This is particularly relevant given the high stakes in the oncology market, where early access and adoption can lead to a strong market position.
It is also important to consider the payer landscape and the reimbursement strategies that Autolus will need to employ. Given the high costs typically associated with CAR T therapies, reimbursement negotiations will be a critical factor in the therapy's adoption. Furthermore, the company's ability to scale up manufacturing and assure supply chain integrity will be pivotal in meeting the anticipated demand, should the therapy gain approval. These factors will ultimately influence the therapy's market penetration and the company's financial performance.
- PDUFA Goal date is November 16, 2024
- Company on track to submit a marketing authorization application to the European Medicines Agency (EMA) in the first half of 2024
LONDON, Jan. 22, 2024 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announces that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel) for patients with relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (ALL). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of November 16, 2024, a standard review timeline consistent with recently approved CAR T therapies. The FDA is not currently planning to hold an advisory committee meeting to discuss this application.
The BLA submission is based on data from the Pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL. The data were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in June 2023, with updated data presented at the Annual Meeting of the American Society for Hematology Meeting (ASH) in December 2023.
“Acceptance of the BLA filing is an important milestone for Autolus and we look forward to continuing our collaboration with the FDA during the review cycle,” commented Dr. Christian Itin, Chief Executive Officer of Autolus. “With the PDUFA date set for November, we remain focused on preparing for the potential launch of obe-cel.”
Autolus plans to submit a Marketing Authorization Application for obe-cel in relapsed/refractory ALL to the European Medicines Agency (EMA) in the first half of 2024.
Obe-cel has been granted Orphan Drug Designation by the FDA, Orphan Medical Product Designation by the EMA, Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA for adult r/r B-ALL.
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, the Company 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. Clinical trials of obe-cel have demonstrated that this “fast off-rate” profile reduces 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, are being prepared for regulatory submissions with the FDA and EMA. Autolus is conducting a Phase 1b study in paediatric patients with ALL and aggressive B-NHL and iIn collaboration with UCL, obe-cel is currently being evaluated in a Phase 1 clinical trials for B-NHL.
About obe-cel FELIX clinical trial
Autolus’ Phase Ib/II clinical trial of obe-cel enrolled adult patients with relapsed / refractory B-precursor ALL. The trial had a Phase Ib component prior to proceeding to the single arm, Phase II clinical trial. The primary endpoint is overall response rate, and the secondary endpoints include 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]
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 the Company’s anticipated transition plans and timing from a clinical to commercial stage company. 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 on March 7, 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.
Contact:
Olivia Manser
+44 (0) 7780 471568
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
Lauren Williams
Investase
+44 23 9438 7760
lauren@investase.com
FAQ
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What is the status of the Biologics License Application for obecabtagene autoleucel (obe-cel) by Autolus Therapeutics plc (AUTL)?
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When does Autolus Therapeutics plc (AUTL) plan to submit a Marketing Authorization Application to the European Medicines Agency (EMA) for obe-cel?
