FDA approved Breyanzi as first and only CAR-T for adults with relapsed or refractory CLL and SLL

CLINICAL AND REGULATORY

FDA approved Breyanzi as first and only CAR-T for adults with relapsed or refractory CLL and SLL

Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial. In relapsed or refractory CLL or SLL, Breyanzi is delivered through a treatment process which culminates in a one-time infusion with a single dose containing CAR-positive viable T cells. The Phase 1/2 open-label, single-arm TRANSCEND CLL 004 study was the first pivotal multicenter trial to evaluate a CAR-T cell therapy in patients with relapsed or refractory CLL or SLL. The CR rate associated with Breyanzi treatment was 20% (95% CI: 11.1-31.8). Among patients who achieved a CR, median duration of response was not reached (95% CI: 15 months-NR) at the time of data cutoff. Among all responders (ORR = 45%; 95% CI: 32.3-57.5), median duration of response was 35.3 months (95% CI: 12.4-NR). High rates of minimal residual disease (MRD) negative status were observed across patients treated with Breyanzi who achieved a CR, with an MRD-negativity rate of 100% in the blood (95% CI: 75.3-100) and 92.3% in the bone marrow (95% CI: 64-99.8).

NKarta deprioritized development of NKX101 following interim evaluation of Phase 1 data

Nkarta  has closed patient enrollment in its clinical trial of NKX101 and deprioritized the program as part of a pipeline realignment that directs primary resources to its lead pipeline program, NKX019, for the treatment of autoimmune disease. This follows a recent review of preliminary safety and response data from patients with relapsed or refractory AML that received NKX101 after LD comprising fludarabine and cytarabine (flu/Ara-C). The aggregate CR/CRi rate (5 of 20 patients) was lower than what had been observed in the first 6 patients in the cohort. The safety profile of NKX101 was consistent with previously reported data. This announcement reflects the NKX101 clinical update that Nkarta had planned to report in the first half of 2024. Nkarta plans to present these data at a future medical conference.

 DEALS AND FINANCING

Allogene and Arbor Biotech announced gene editing licensing agreement

Allogene and Arbor Biotechnologies announced a non-exclusive, global gene editing licensing agreement for use of Arbor’s proprietary CRISPR gene-editing technology in Allogene’s next generation AlloCAR T platform for the treatment of autoimmune disease. Allogene has applied its understanding of CAR T research and development to design next-generation allogeneic CAR T investigational products with a goal of reduced or chemotherapy-free conditioning that the Company believes can sustain the scale of the AID market while also meeting the unique requirements for these patients where they seek care. Allogene’s first AID AlloCAR T investigational product is expected to enter Phase 1 clinical trials in early 2025.

Legend and Novartis enter a commercial production agreement for Carvykti

J&J and Legend expanded a previous agreement, with Legend and J&J tapping Novartis to perform commercial manufacturing for Carvykti (cilta-cel). The new deal runs through the end of 2029, with Novartis agreeing to set aside a specified number of production suites at its facility in Morris Plains, New Jersey. Legend and J&J committed to meet specified volume targets and to a tiered pricing arrangement, depending on how many batches they order.

Anocca licenses gene editing technology from EmndoBio

Anocca, a swedish T cell receptor-engineered T cell cellular therapeutics company, and EmendoBio, a nuclease discovery and gene editing therapeutics company, announced a non-exclusive licensing agreement for the use of EmendoBio’s novel OMNI-A4 nuclease to accelerate the manufacture and development of Anocca’s deep pipeline of TCR-T cell therapies for difficult-to-treat solid cancers. Anocca recently received certification of GMP compliance and a manufacturing license from the Swedish regulators for their cell therapy production facility, the largest in the Nordics. EmendoBio’s technology strengthens Anocca’s manufacturing capability and provides a foundation for the ambitious goal of reaching more patients faster with personalized treatments targeting the underlying genetic drivers of hard-to-treat cancers. The licensed gene editing technology is part of EmendoBio’s portfolio of proprietary nucleases developed to be highly active and specific for cell therapy applications.