Autolus Therapeutics has announced plans to advance obecabtagene autoleucel (obe-cel) into a pivotal Phase 2 clinical trial for systemic lupus erythematosus (SLE) following promising preliminary data from its Phase 1 dose confirmation study. The company shared these developments during a recent R&D investor event highlighting its expanding autoimmune disease pipeline.
Promising Phase 1 Results Support Pivotal Trial
Preliminary data from the CARLYSLE Phase 1 dose confirmation trial in six patients with refractory SLE demonstrated encouraging efficacy and safety signals. Three patients achieved complete renal response by month three, while all patients showed normalized complement levels by month one. The trial also documented resolution of key lupus symptoms, including rash, alopecia, and mucosal ulcers by month three, with arthritis resolving by month one in all patients.
"The preliminary data from our Phase 1 trial provides strong rationale for advancing obe-cel into a pivotal study for lupus," said a company representative. "We're particularly encouraged by the rapid resolution of symptoms and the complete renal responses observed in half of the patients."
The CAR-T therapy demonstrated high peak expansion and deep B cell aplasia consistent with its known characteristics in oncology indications. From a safety perspective, no dose-limiting toxicities or immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. Grade one cytokine release syndrome (CRS) occurred in three of six patients, while transient hypertension, including grade three, was seen in five patients.
Regulatory Alignment and Development Timeline
Autolus has successfully aligned with the U.S. Food and Drug Administration (FDA) on the Phase 2 trial design and potential registrational path to approval. The company anticipates dosing the first patient in this pivotal trial before the end of 2025.
Full data with longer-term follow-up from the Phase 1 CARLYSLE clinical trial is expected to be presented at a medical conference in the second half of 2025, providing additional insights into the durability of response and long-term safety profile.
Expanding into Multiple Sclerosis
Beyond lupus, Autolus is broadening obe-cel's potential in autoimmune conditions by planning a Phase 1 dose escalation study in progressive multiple sclerosis (MS). The company expects to dose its first patient in this trial by year-end 2025, marking another significant expansion of its autoimmune disease portfolio.
This strategic move into MS represents an important diversification of obe-cel's potential applications, targeting a condition with significant unmet needs, particularly in progressive forms of the disease where effective treatment options remain limited.
AUCATZYL Commercial Launch Progress
Alongside its autoimmune disease developments, Autolus reported that the U.S. commercial launch of AUCATZYL (obe-cel) in relapsed/refractory adult B-cell acute lymphoblastic leukemia (B-ALL) is progressing on track. As of April 22, 2025, the company had fully activated 38 treatment centers.
AUCATZYL received FDA approval in November 2024 for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The therapy is currently under regulatory review in both the European Union and the United Kingdom, with approval decisions expected from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in Q2 2025 and the European Medicines Agency (EMA) in the second half of 2025.
Financial Outlook
Autolus stated that its current cash position and marketable securities provide sufficient runway to support both the commercial launch of AUCATZYL in the U.S., UK, and EU markets and its pipeline development plans, including funding through data readouts from the first pivotal clinical trial of obe-cel in autoimmune disease.
The company plans to report AUCATZYL's first-quarter sales during its Q1 2025 financial results announcement on May 8, 2025, providing the first glimpse of the therapy's commercial performance since approval.
CAR-T in Autoimmune Disease: A Growing Field
Autolus' advancement of obe-cel into autoimmune indications comes amid growing interest in CAR-T cell therapy applications beyond oncology. Several companies are exploring CAR-T approaches for autoimmune conditions, with early clinical data suggesting potential for deep and durable responses in diseases characterized by B-cell dysfunction.
Obe-cel's unique design features, including its fast target binding off-rate that minimizes excessive T cell activation, may provide advantages in the autoimmune setting where controlling inflammation while maintaining efficacy is crucial.
Looking to the Future
With a dual focus on oncology and autoimmune applications, Autolus is positioning obe-cel as a potential platform therapy across multiple indications. The company's translational programs with University College London (UCL) continue to fuel its early-stage pipeline, providing what the company describes as a cost-efficient path to development.
As Autolus advances its autoimmune disease programs while commercializing AUCATZYL in oncology, the company is emerging as a significant player in the cell therapy landscape with applications spanning both cancer and autoimmune conditions.
