Imugene (ASX:IMU) has taken a significant step toward potential commercialisation of its allogeneic CAR-T therapy azercabtagene zapreleucel (azer-cel), after receiving supportive written minutes from the US Food and Drug Administration (FDA) outlining a clear route to a Phase 3 registrational trial.
The company said the update represents the strongest regulatory endorsement to date for its lead cell therapy and marks a pivotal shift in its development trajectory.
The FDA confirmed that data emerging from Imugene’s ongoing Phase 1b trial, which evaluates azer-cel in combination with augmented lymphodepletion and low-dose IL-2, together with its safety profile, are sufficient to progress toward a pivotal study. The regulator also accepted third-line and later diffuse large B-cell lymphoma (DLBCL), including patients who relapsed following autologous CAR-T therapy, as the target population/
The FDA endorsed Imugene’s proposal for a single randomised registrational study, using overall response rate (ORR) with durability to support accelerated approval and progression-free survival (PFS) for full approval. The FDA further advised that Imugene’s Chemistry, Manufacturing, and Controls (CMC) program is suitable for initiating a registrational study, with only standard refinements required.
Recent Phase 1b data have shown an 82 per cent ORR in CAR-T–relapsed DLBCL and an 83 per cent ORR in CAR-T–naïve patients across several CD19-positive cancers.
Imugene CEO Leslie Chong said the latest regulatory update delivers pivotal clarity as the company moves toward late-stage development. “The alignment across dosing regimen, patient population selection, accelerated approval endpoint strategy and CMC readiness gives us confidence as we continue to design our pivotal study plans,” she said. “Combined with our growing clinical data set, we believe azer-cel is well positioned to address high-need patient populations.”
Azer-cel, administered as a flat 500-million cell dose with 14 days of low-dose subcutaneous IL-2, continues to show durable responses, with additional patients maintaining meaningful disease control over time. The therapy is being studied in DLBCL as well as a range of other non-Hodgkin lymphoma subtypes, including PCNSL, CLL/SLL, marginal zone lymphoma, Waldenström macroglobulinemia, and follicular lymphoma, all areas where relapse or refractory disease remains a persistent challenge.