Imugene’s Azer-cel Shows 82% Response Rate, FDA Backs Phase 3 Trial Design

FDA Alignment Clears Path for Azer-cel’s Pivotal Trial

Imugene Limited (ASX – IMU) has taken a significant step forward in the development of its off-the-shelf CAR T therapy, azer-cel, with the US Food and Drug Administration (FDA) providing written confirmation supporting its progression into a Phase 3 registrational study. The FDA’s recent Type C meeting minutes endorse the company’s proposed dosing strategy, patient population, and clinical endpoints, marking a crucial milestone for this promising immuno-oncology candidate.

The FDA agreed that azer-cel’s regimen; comprising augmented lymphodepletion followed by a fixed dose of 500 million cells and a 14-day course of low-dose subcutaneous interleukin-2 (IL-2); is appropriate for the pivotal study. Importantly, the agency accepted third-line and later diffuse large B-cell lymphoma (DLBCL) patients, including those who have relapsed after autologous CAR-T therapy, as the target population. This patient group represents a high unmet medical need with limited treatment options and poor outcomes.

Strategic Endpoints and Manufacturing Readiness

Imugene’s dual endpoint approach received FDA endorsement – Overall Response Rate (ORR) with durability will support accelerated approval, while Progression Free Survival (PFS) will underpin full approval. The FDA also agreed that a single randomized trial incorporating investigator choice therapies as the control arm can satisfy both endpoints, streamlining the regulatory pathway. Additionally, the FDA confirmed that Imugene’s Chemistry Manufacturing Controls (CMC) program is sufficiently mature to initiate a registrational study, with only minor refinements needed.

Compelling Clinical Data Bolster Confidence

Recent clinical results reinforce azer-cel’s potential. The therapy has demonstrated an 82% ORR in patients who relapsed after CAR-T treatment and an 83% ORR in CAR-T naïve patients across multiple CD19-positive blood cancers. These response rates are notable given the aggressive nature of DLBCL and related lymphomas. Durability of response is also developing positively, with ongoing patient follow-up indicating sustained benefit.

Imugene’s CEO Leslie Chong highlighted the importance of the FDA’s feedback, emphasizing that the alignment on dosing, patient selection, endpoints, and manufacturing readiness provides a clear framework for designing the pivotal study. The company is now focused on finalizing the study protocol and operational plans, with continued patient enrollment progressing rapidly, especially in the CAR-T naïve cohort.

Broader Implications for Blood Cancer Treatment

Azer-cel’s advancement into late-stage development could address a critical gap in treatment for patients with relapsed or refractory DLBCL, a disease with approximately 160,000 global cases annually and limited effective therapies after CAR-T failure. The therapy’s off-the-shelf nature offers potential advantages over autologous CAR-T products, including faster availability and scalability. Moreover, Imugene’s expanding clinical program into other B-cell lymphomas, such as primary central nervous system lymphoma and chronic lymphocytic leukemia, suggests a broader commercial opportunity.

As Imugene prepares for subsequent FDA interactions and finalizes its pivotal trial design, the oncology community will be watching closely to see if azer-cel can fulfill its promise as a differentiated and scalable immunotherapy option for high-need patient populations.