Imviva Biotech, a clinical-stage biotechnology company developing next-generation allogeneic CAR-T cell therapies, announced that the U.S. Food and Drug Administration has granted orphan drug designation to CTD402, the company’s investigational allogeneic anti-CD7 CAR-T cell therapy for the treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma (T-ALL/LBL). This designation underscores the significant unmet need for new treatments in these hematologic conditions.

CTD402 is currently being evaluated in the global Phase 1b/2 TENACITY-01 clinical trial (NCT07070219). The ongoing global, single‑arm, open‑label TENACITY‑01 trial is enrolling adolescents and adults (≥12 years) to evaluate the safety, efficacy, and cellular pharmacokinetics of CTD402. Phase 1b/2 will enroll about 54 patients across the U.S., EU, and APAC. CTD402’s point‑of‑care platform enables rapid treatment without the 1–2-month manufacturing delays of autologous CAR‑T therapies. Early data show a 64.1% complete remission (CR) rate and 91.7% MRD‑negative status in R/R T‑ALL/LBL, supporting CTD402’s potential to address a critical unmet need.

The TENACITY-01 clinical trial successfully dosed the first patient in the United States in December 2025, with Phase 1b interim data expected by mid-2026 and study completion by late 2028. This timing enables the therapy to progress into a Phase 2 evaluation following Phase 1b readout, supporting the accelerated development pathway for a therapy benefiting a patient population where treatment timing is critical due to a high mortality rate.

"Receiving orphan drug designation for CTD402 is an important milestone for patients with relapsed or refractory T‑ALL/LBL, who urgently need more effective and accessible treatment options," said Imviva Biotech Chief Medical Officer Jan Davidson-Moncada, M.D., Ph.D. “This recognition provides regulatory support and extended market exclusivity to advance our development pathway, supporting our belief that a truly off‑the‑shelf CAR‑T therapy, available at the point of care, has the potential to change the treatment paradigm for these rapidly progressing diseases.”

Orphan drug designation is granted by the FDA for therapies treating rare disease affecting fewer than 200,000 patients in the U.S., and provides development incentives including extended market exclusivity, tax credits for clinical research, and waiver of prescription drug user fees.