Imugene's azer-cel trial yields strong results in Phase 1b for DLBCL, FDA meeting planned.

Imugene's azer-cel CAR T-cell therapy has shown promising results in a Phase 1b trial for relapsed/refractory diffuse large B-cell lymphoma (DLBCL). The overall response rate is 75%, with six complete responses and three partial responses. The company plans to meet with the FDA to discuss pivotal study design and has expanded the trial to include patients with other B-cell lymphoma subtypes.

July 02, 2025

In recent developments, Mustang Bio, Lexeo Therapeutics, and Immix Biopharma have made significant strides in their respective cell therapy programs, each receiving notable designations from the FDA.

Mustang Bio's MB-101, a targeted CAR-T cell therapy for brain cancer, received Orphan Drug Designation from the FDA. MB-101 is indicated for the treatment of recurrent diffuse and anaplastic astrocytoma and glioblastoma. The therapy has shown promising results in an ongoing Phase I trial, with 50% of patients achieving stable disease or better. Mustang Bio plans to enhance the efficacy of MB-101 by combining it with its MB-108 oncolytic virus, presented as MB-109, to treat malignant glioma [1].

Lexeo Therapeutics' LX2006, an AAV-based gene therapy for Friedreich Ataxia (FA), received Breakthrough Therapy designation. The designation followed encouraging interim data where LX2006 demonstrated improvements in cardiac and neurological function and increased frataxin expression. The FDA's recognition highlights the potential of LX2006 and the strength of the clinical evidence generated to date [1].

Immix Biopharma's NXC-201, a CAR-T therapy for relapsed/refractory AL Amyloidosis, reported no neurotoxicity in low-volume disease. The company is progressing toward submitting its first Biologics License Application (BLA) for NXC-201, with plans for future indication expansions. NXC-201 has received both Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation (ODD) from the FDA, which may expedite its development and approval process [2].

These advancements underscore the growing potential of cell therapies in addressing complex and challenging-to-treat diseases. The FDA's designations and the promising trial results highlight the commitment of these companies to exploring new treatment options for patients.

References:
[1] https://www.regmednet.com/cell-therapy-weekly-orphan-drug-designation-for-brain-cancer-car-t-cell-therapy/
[2] https://www.nasdaq.com/articles/immix-biopharma-reports-class-leading-safety-profile-car-t-nxc-201-low-volume-al