AstraZeneca’s Dual-Targeting CAR-T Shows Encouraging Efficacy

(ASH Congress) – AstraZeneca (AZ) announced positive clinical data for its leading cell therapy candidate, AZD0120 (formerly GC012F), for the treatment of relapsed/refractory multiple myeloma at this year’s American Society of Hematology (ASH) conference.

AZD0120 is a CAR-T (Chimeric Antigen Receptor T-cell) therapy designed to dual-target both BCMA and CD19antigens. The therapy was acquired by AZ through its purchase of Chinese biotech Gracell in 2024.

DURGA-1 Trial Results

The Phase 1b/2 DURGA-1 study was an open-label, single-arm trial conducted in US patients, some of whom had previously received anti-BCMA treatment.

• Objective Response Rate (ORR): Achieved 96%.

• Combined Complete Response (CR) and stringent CR (sCR) rate: Reached 78.3%.

• The median time to response was a rapid 28 days.

• These results are considered robust, given that the trial subjects had undergone a median of four prior lines of therapy.

• Median progression-free survival (PFS) and overall survival (OS) were not reached after a follow-up period exceeding 36 months.

Safety Profile and Manufacturing

• Safety: The therapy was generally well-tolerated, with no reported deaths, Grade 4 or higher infections, or dose-limiting toxicities.

• Cytokine Release Syndrome (CRS): Approximately 62% of recipients experienced CRS, but most cases were Grade 1, with only one case of Grade 2 (the milder end of the severity spectrum).

• Neurotoxicity: No cases of neurotoxicity, another recognized adverse event with CAR-Ts, were reported.

• Outpatient Potential: The drug’s safety profile suggests it may be suitable for outpatient administration.

• FasTCAR Technology: AZD0120 utilizes Gracell’s FasTCAR platform, which enables the therapy to be manufactured in days, a significant speed advantage over other CAR-Ts that typically require weeks of production time.

In addition to multiple myeloma, AZD0120 is also being tested in clinical trials for the autoimmune disorder systemic lupus erythematosus (SLE) and amyloid light chain (AL) amyloidosis.

Source: https://pharmaphorum.com/news/ash-astrazenecas-dual-wielding-car-t-shows-promise