Key Takeaways:
Legend Biotech Corp.'s experimental in vivo CAR-T therapy LB2501 produced a 100% objective response rate at the higher dose level in 12 patients with relapsed or refractory B-cell non-Hodgkin lymphoma, with five of six patients achieving complete remission.
"By generating CAR-T cells directly within the patient, this approach has the potential to simplify treatment delivery and expand access for patients who may not be able to receive traditional CAR-T cell therapies," Ying Huang, chief executive officer of Legend Biotech, said. LB2501 is built on the TaVec platform, a proprietary lentiviral vector designed to enhance T-cell specificity while restricting transduction of non-T cells, she said.
At dose level 2, the complete response rate reached 83.3% among six patients with a median follow-up of 2.2 months. All responses were ongoing at the April 1 data cutoff. CAR-T cells were detected in peripheral blood for as long as 116 days after a single intravenous infusion, and the therapy generated dose-dependent in vivo expansion without requiring lymphodepleting chemotherapy — a standard preconditioning step for approved CAR-T products. No dose-limiting toxicities, serious adverse events, or deaths were reported. Cytokine release syndrome occurred in 66.7% of patients, all Grade 2 or lower, and no immune effector cell-associated neurotoxicity syndrome was observed. Grade 3 or higher adverse events linked to the lentiviral vector or CAR-T cells were limited to decreased lymphocyte and neutrophil counts.
The data position LB2501 as a potential first-in-class candidate in the emerging field of in vivo CAR-T, which aims to eliminate the weeks-long cell manufacturing process required by approved therapies such as Novartis AG's Kymriah, Gilead Sciences Inc.'s Yescarta, and Bristol Myers Squibb Co.'s Breyanzi. Those ex vivo products require harvesting a patient's T cells, genetically engineering them in a centralized facility, and reinfusing them — a process that can take two to four weeks and excludes patients with rapidly progressing disease. Legend's approach delivers a lentiviral vector intravenously that programs T cells inside the body, bypassing manufacturing entirely. The company plans to present full Phase 1 results during a late-breaking oral session at the European Hematology Association 2026 Congress in Stockholm on June 14.
Legend Biotech, which employs about 3,000 people and is the largest standalone cell therapy company, already markets CARVYKTI with partner Johnson & Johnson for multiple myeloma. The LB2501 program extends its CAR-T expertise into non-Hodgkin lymphoma, a cancer expected to be diagnosed in about 79,320 people in the US this year, according to the American Cancer Society. B-cell lymphomas account for roughly 85% of NHL cases. Legend shares surged 42% to $36.28 on June 2 following the data release, while shares of associate company Genscript Bio jumped 19% to HKD 14.48 in Hong Kong. The company has not disclosed a cash runway figure for the LB2501 program, which remains in early-stage development with additional dose escalation and expansion cohorts ongoing.
This article is for informational purposes only and does not constitute investment advice.
