Autolus Therapeutics plc (Nasdaq: AUTL) has announced that the U.S. Food and Drug Administration (FDA) approved AUCATZYL® (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) on November 8, 2024, ahead of the target PDUFA date. This approval marks a significant milestone for Autolus, triggering a $30 million milestone payment from Blackstone.
Financial Highlights
The company's Q3 2024 financial results show a robust cash position of $657.1 million, a substantial increase from $239.6 million at the end of 2023. Operating expenses for the quarter were $67.9 million, with a net loss of $82.1 million, or $0.31 per share. The increased expenses reflect commercial preparation activities and pipeline development.
Clinical Data and Regulatory Updates
"With the U.S. Food and Drug Administration (FDA) having approved AUCATZYL® (obe-cel) for the treatment of adult B-cell Acute Lymphoblastic Leukemia (B-ALL) patients, we are all systems go with our commercial efforts in the US across the Company," said Dr. Christian Itin, Chief Executive Officer of Autolus. "This first FDA approval is just the beginning for Autolus; we have great belief in our pipeline and our manufacturing capabilities and are excited for the future."
The approval was based on data from the pivotal Phase 1b/2 FELIX study (NCT04404660), which evaluated the efficacy and safety of obecabtagene autoleucel in adult patients with r/r B-precursor ALL. The study enrolled over 100 patients across 30 leading academic and non-academic centers in the United States, United Kingdom, and Europe. Updated data presented at the Society of Hematologic Oncology (SOHO) meeting in August 2024 demonstrated the rationale for tumor burden (TB)-guided dosing, highlighting the importance of administering a split dose based on the unique binding properties of obecabtagene autoleucel.
Marketing authorization submissions for obecabtagene autoleucel are currently under review by both the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA).
Expansion into Autoimmune Diseases
Autolus is also exploring the potential of obecabtagene autoleucel in B-cell mediated autoimmune diseases. The Phase 1 dose confirmation study (CARLYSLE) in refractory systemic lupus erythematosus (SLE) patients is ongoing, with initial data expected in Q1 2025. Full data with adequate follow-up is targeted for presentation at a medical conference in the second half of 2025.
Pipeline Development
In addition to obecabtagene autoleucel, Autolus is advancing several other clinical programs, including AUTO8, AUTO6NG, and AUTO1/22, in collaboration with University College London. Data updates for all programs are planned for 2025.
Leadership Appointment
In September 2024, Autolus appointed Matthias Will M.D. as Chief Development Officer. Dr. Will brings extensive experience in clinical development, particularly in hematologic cancers and CAR T-cell therapies.
About Obecabtagene Autoleucel
Obecabtagene autoleucel is a CD19 CAR T-cell therapy designed with a fast target binding off-rate to minimize excessive activation of programmed T cells. It is currently being evaluated in a Phase 1 clinical trial for B-cell non-Hodgkin lymphoma (B-NHL) in collaboration with University College London.
