Autolus Surges on 95% Remission Rate for Leukemia Therapy Obe-cel
Shares in the biotech firm climb as new data shows its CAR-T therapy has a superior safety and efficacy profile in pediatric patients.
Shares of Autolus Therapeutics (NASDAQ: AUTL) jumped in active trading Monday after the company presented highly encouraging clinical data for its lead cancer therapy, obecabtagene autoleucel (obe-cel), in pediatric leukemia patients. The results, unveiled at the American Society of Hematology (ASH) Annual Meeting, suggest a potentially best-in-class profile for the treatment.
In data from its CATULUS Phase 1 trial, Autolus reported that its CAR-T therapy demonstrated a 95% overall remission rate in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). According to the company's announcement, 20 out of 21 patients in the trial responded to the treatment, with 17 achieving a deep, measurable residual disease (MRD)-negative remission.
This strong efficacy data was coupled with a favorable safety profile, a critical differentiator in the competitive landscape of cellular therapies. The trial showed low rates of high-grade cytokine release syndrome (CRS) and neurotoxicity, side effects that can be severe and have complicated the rollout of earlier CAR-T treatments.
The positive pediatric results follow a major milestone for Autolus. In November 2024, the U.S. Food and Drug Administration (FDA) approved obe-cel for the treatment of adult patients with the same condition. The new pediatric data could pave the way for a label expansion, significantly broadening the therapy's addressable market.
Obe-cel is designed to be a safer alternative to existing CD19 CAR-T therapies like Novartis's Kymriah and Gilead Sciences' Yescarta. While effective, these treatments carry higher risks of severe side effects. The data presented by Autolus suggests obe-cel could offer a compelling balance of high efficacy and improved tolerability, a factor that could drive significant clinical adoption.
Investors and analysts have taken note of the company's progress. Autolus, with a market capitalization of approximately $412 million, has a consensus "Buy" rating from analysts, who have set an average price target of $9.52—representing a substantial premium to its current trading price. In November, analysts at Redburn-Atlantic upgraded the stock to a "Buy" with a $13 price target, citing growing confidence in obe-cel's commercial trajectory following its initial FDA approval.
The path forward for Autolus involves completing the CATULUS trial and engaging with regulators to add the pediatric indication to obe-cel's label. Success in this high-risk patient population could position Autolus as a key player in the rapidly growing cell therapy market, as it aims to provide a safer, more effective treatment for a devastating childhood cancer.