Breakthrough in vivo CAR-T therapy shows 100% response in early cancer trials

Legend Biotech has announced the first clinical proof-of-concept data for LB2501, an investigational in vivo CAR-T therapy. The treatment targets CD19 and CD20, offering a potential breakthrough in cancer care. The company is a leader in cell therapy and aims to transform how certain cancers are treated. LB2501 is designed to generate CAR-T cells directly in the patient after a single intravenous infusion. It is a first-in-class dual-targeting therapy for relapsed or refractory B-cell non-Hodgkin lymphoma (RR B-NHL). In a Phase 1 study, a single dose produced dose-dependent CAR-T cell expansion without the need for lymphodepletion.

At the higher dose level (DL2), the therapy achieved a 100% objective response rate in RR B-NHL patients. An 83.3% complete response rate was also observed, with all responses ongoing at the data cutoff. CAR-T cells remained detectable in the blood for up to 116 days after treatment.

The safety profile of LB2501 was described as favourable. The most common adverse events were infusion-related reactions and cytokine release syndrome, all graded as mild to moderate. No dose-limiting toxicities, serious adverse events, neurotoxicity, or deaths were reported. The results mark a significant step for in vivo CAR-T therapies. LB2501 demonstrated strong efficacy and a manageable safety profile in early testing. The findings suggest potential for broader applications in treating B-cell lymphomas, which make up the majority of non-Hodgkin lymphoma cases.