Autolus Therapeutics presented data from its CATULUS Phase 1 trial of obe‑cel in 22 pediatric patients with relapsed or refractory B‑ALL at the American Society of Hematology Annual Meeting on December 8 2025. The study achieved an overall response rate of 95.5 % (21 of 22 patients) and a complete response rate of 90.9 % (20 of 22), with only 8.7 % of patients experiencing high‑grade cytokine release syndrome and no cases of severe neurotoxicity.
The safety profile in children mirrored that seen in adults, where obe‑cel has already received FDA approval for adult r/r B‑ALL in November 2024 and conditional marketing authorizations in the UK and EU in 2025. The low incidence of high‑grade CRS and ICANS is consistent with the engineered safety features of the product and supports its use in a vulnerable population.
These results reinforce Autolus’ strategy to expand obe‑cel into high‑unmet‑need pediatric indications and position the therapy as a potential first‑line treatment. The company will transition the study into a Phase 2 expansion later in 2025, a move that signals confidence in the drug’s efficacy and safety and addresses the critical need for effective options in pediatric B‑ALL. The data also give Autolus a competitive edge, as obe‑cel is the only CAR‑T therapy for B‑ALL that does not require a U.S. FDA‑mandated REMS program, potentially simplifying access and reimbursement.
Investors responded positively to the presentation, reflecting confidence in the drug’s clinical promise and the company’s broader pipeline. The high remission rates, coupled with a favorable safety profile, align with the regulatory momentum already achieved for obe‑cel and underscore the potential for accelerated future approvals and market access in the United States and abroad.
Dr. Matthias Will, Chief Development Officer, noted that the data “confirm the robust efficacy of obe‑cel in a population with a poor prognosis and demonstrate that the safety profile is consistent with adult experience.” He added that the transition to Phase 2 will “address the significant unmet need for effective therapies in pediatric B‑ALL and could position obe‑cel as a first‑line option.”
Autolus continues to build its commercial pipeline, with ongoing studies in autoimmune diseases and other hematologic malignancies. The strong pediatric data strengthen the company’s overall value proposition and support its strategy to bring obe‑cel to a broader patient population while maintaining a solid financial foundation with a substantial cash runway and no debt.
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