CTD402 Receives Orphan Drug Designation For T-Cell Leukemia/Lymphoma

The FDA has granted orphan drug designation to CTD402, an allogenic anti-CD7 CAR-T cell therapy, as a monotherapy treatment for patients with relapsed/refractory T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma (T-ALL/LBL), according to a news release from the developer, Imviva Biotech.¹

Supporting the regulatory decision are data from the phase 1b/2 TENACITY-01 trial (NCT07070219), an ongoing, single-arm, open-label trial evaluating the safety, efficacy, and pharmacokinetics of the agent in patients 12 years and older with relapsed/refractory T-ALL/LBL. Specifically, findings have revealed that among patients who have been treated with the agent, 64.1% achieved a complete remission (CR) and 91.7% attained minimal residual disease (MRD)-negative status.

Moreover, the developers expect interim data from the phase 1b portion of the trial by the middle of 2026 and the completion of the study by late 2028. The timing, according to them, would support an accelerated development pathway for the therapy by enabling the therapy to progress into a phase 2 evaluation following the phase 1b readout.

"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," Jan Davidson-Moncada, MD, PhD, chief medical officer of Imviva Biotech, said in the news release.¹ “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.”

The single-arm study will enroll approximately 18 patients during the phase 1b portion of the study to evaluate the agent’s safety and establish its recommended phase 2 dose (RP2D).² An additional 36 patients will be enrolled in the phase 2 portion of the study to validate the safety and efficacy of the drug.

Patients on the trial will receive one CTD402 infusion on day 0 of study treatment and will undergo an evaluation for anti-tumor activity by independent review committee (IRC) assessment based on NCCN criteria for T-ALL and Lugano 2014 criteria for T-LBL. Prior to treatment, patients will undergo screening for up to 2 weeks prior and a lymphodepletion period within 7 days of treatment. Follow-up will continue for a maximum of 24 months and patients will enroll under a separate long-term follow-up protocol for up to 15 years.

The primary end points of the trial include safety and CR rate. A secondary end point is the cellular pharmacokinetic profile of CTD402.

The developers engineered CTD402 as a ‘ready-at-point of care’ therapy designed for T-cell mediated release. In addition to their proprietary ANSWER^TM inhibitory ligands, the agent incorporates T-cell receptor and HLA class II knockout to enhance resistance to host immune rejection. The robustness of the manufacturing process is intended to deliver an immediately available “off-the-shelf” allogenic platform.

The FDA has previously granted rare pediatric disease designation (RPDD), and regenerative medicine advanced therapy (RMAT) designation to CTD402 for the treatment of this relapsed/refractory group.

Enrollment will be open to patients 12 years and older weighing at least 40 kg with relapsed/refractory disease following 2 or more lines of therapy, or a first relapse occurring within 12 months of first remission, or relapse following allogenic hematopoietic stem cell transplantation (HSCT) within 100 days of HSCT prior to screening period. Additionally, patients must have a bone marrow lymphoblast presence of 5% or more or evidence of extramedullary disease, have eligible HLA-matched related donors (MRD) or unrelated donor (URD), adequate organ function, and a Karnofsky performance score of at least 60 in patients 16 years and older or a Lansky performance score of at most 60 for patients younger than 16.

References

1. Imviva Biotech receives FDA orphan drug designation for CTD402 for the treatment of T-Cell Leukemia and Lymphoma. News release. Imviva Biotech. January 27, 2026. Accessed January 28, 2026. https://tinyurl.com/59pwhjmw
2. A study of CTD402 in T-ALL/​LBL patients (TENACITY-01). ClinicalTrials.gov. Updated December 30, 2025. Accessed January 28, 2026. https://tinyurl.com/9km6mkz4