Autolus Therapeutics plc (Nasdaq: AUTL) has announced that the U.S. Food and Drug Administration (FDA) approved AUCATZYL® (obecabtagene autoleucel) on November 8, 2024, ahead of its target PDUFA date. This approval marks a significant milestone for adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). The company has initiated its commercial launch in the US.
Regulatory and Financial Milestones
The FDA approval of AUCATZYL® triggers a $30 million milestone payment to Autolus from Blackstone, according to their collaboration agreement. Additionally, Autolus will make a £10 million regulatory milestone payment to UCL Business Ltd., in accordance with their license agreement.
Marketing authorizations for obe-cel are currently under review with both the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK and the European Medicines Agency (EMA).
Clinical Data and Updates
Updated data from the pivotal Phase 1b/2 FELIX study were presented at the Society of Hematologic Oncology (SOHO) meeting in August 2024. The data supported tumor burden (TB)-guided dosing by analyzing the impact of bone marrow (BM) blast percentage in patients treated with obe-cel. The findings underscored the importance of administering a split dose and highlighted the differentiation of obe-cel based on its unique binding properties and tumor burden-guided approach.
Further data presented at the 2024 Lymphoma, Leukemia & Myeloma Congress suggested that adult patients with r/r B-ALL achieve comparable outcomes irrespective of the timing of stem cell transplant (SCT) pre or post obe-cel, suggesting no further benefit of consolidative transplant based on this post-hoc analysis. Additionally, obe-cel given as a sole treatment to patients with lower Tumor Burden (TB) at Lymphodepletion (LD) was associated with better outcomes.
Pipeline Development
The Phase 1 dose confirmation study (CARLYSLE) in refractory systemic lupus erythematosus (SLE) patients is ongoing, with initial data expected in Q1 2025 and full data targeted for 2H 2025. Clinical programs AUTO8, AUTO6NG, and AUTO1/22 are progressing, and the company plans to provide data updates for all programs in 2025.
Leadership Appointment
In September 2024, Autolus appointed Matthias Will M.D. as Chief Development Officer, effective September 30, 2024. Dr. Will brings experience from Dren Bio, Inc. and CRISPR Therapeutics, where he led clinical team expansions and oversaw IND submissions for hematologic cancer treatments.
Financial Position
As of September 30, 2024, Autolus reported cash and cash equivalents totaling $657.1 million, compared to $239.6 million at December 31, 2023. The company believes it is well-capitalized to support the commercial launch of obe-cel in r/r adult B-ALL and advance its pipeline development plans.
Total operating expenses for the three months ended September 30, 2024, were $67.9 million, compared to $42.9 million for the same period in 2023. The net loss for the quarter was $82.1 million, compared to $45.8 million for the same period in 2023.
About AUCATZYL® (obecabtagene autoleucel)
AUCATZYL® is a CD19 CAR T-cell therapy approved for the treatment of relapsed/refractory adult B-cell acute lymphoblastic leukemia (B-ALL). Obe-cel is designed with a fast target binding off-rate to minimize excessive activation of programmed T cells. It is also being evaluated in a Phase 1 clinical trial for B-cell non-Hodgkin lymphoma (B-NHL) in collaboration with University College London.
