CTD402 Gains FDA Orphan Drug Designation in T-ALL and T-LBL

The FDA has granted orphan drug designation (ODD) to CTD402, a novel allogeneic chimeric antigen receptor T-cell (CAR T) therapy, for treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and lymphoblastic lymphoma (T-LBL).¹

The FDA’s ODD is awarded to medicines intended for the treatment of rare diseases, providing the developer with various incentives to support its development.

CTD402 is an investigational anti-CD7 CAR-T therapy. Unlike autologous CAR-T products, which require a multiweek manufacturing process using a patient’s own cells, CTD402 is derived from healthy donors and is available for immediate administration. The product utilizes Imviva Biotech’s proprietary ANSWER™ platform, which incorporates inhibitory ligands to enhance resistance against host immune rejection as well as other mechanisms including T-cell receptor and HLA class II knockout.

"Receiving [ODD] for CTD402 is an important milestone for patients with [R/R] T-ALL/LBL, who urgently need more effective and accessible treatment options," said Jan Davidson-Moncada, MD, PhD, chief medical officer of Imviva Biotech, in a 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.”

In March 2025, the FDA cleared the investigational new drug application for CTD402, commencing its clinical investigation in the global phase 1b/2 TENACITY-01 study (NCT07070219). The agent has also previously earned FDA rare pediatric disease designation and regenerative medicine advanced therapy designation in the same indication.¹

About TENACITY-01 and Preliminary Data

The ongoing single-arm, open-label TENACITY-01 study is spearheading the clinical development of CTD402. Its objective is to evaluate the safety and preliminary efficacy of CTD402 in adolescent and adult patients with R/R T-ALL and T-LBL.² Here, patients receive a single infusion of CTD402 on day 0 and are subsequently evaluated for antitumor activity for a total study follow-up of 24 months. Longer-term follow-up will be conducted under a separate protocol for up to 15 years.

The trial aims to enroll a total of 54 patients across sites in the United States, European Union, and Asia. Patients over the age of 12 years whose disease have relapsed within 12 months after the first remission or are refractory after 2 or more lines of systemic therapy are eligible for participation.

Preliminary data suggest that CTD402 can induce deep, rapid remissions and antileukemic activity in a heavily pretreated patient population, offering a potential bridge to consolidative stem cell transplantation for those who previously had few therapeutic alternatives.³ As of a data cutoff on June 30, 2025, 41 patients were treated with CTD402 at the recommended phase 2 dose, and 39 patients were evaluable for efficacy.

Among evaluable patients, the rate of complete remission (CR) plus CR with incomplete hematologic recovery reached 64.1%. Notably, a staggering 91.7% of these responders achieved minimal residual disease (MRD)-negative status, a key predictor of long-term survival.

The therapy also demonstrated encouraging efficacy in particularly challenging subgroups, such as those with extramedullary disease (CR rate, 59%) and high-risk molecular profiles including ETP phenotype or complex karyotypes (CR rate, 60.9%).

The safety profile was characterized as manageable, with 78% of patients experiencing cytokine release syndrome, primarily grade 1 or 2. No cases of immune effector cell-associated neurotoxicity syndrome or graft-vs-host disease were reported. Cytopenia was noted as the most commonly occurring adverse event posttreatment.

Further phase 1b interim data from TENACITY-01 are expected to be announced in mid-2026, according to the sponsor.

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 29, 2026. https://tinyurl.com/vw6ca3hy

2. A study of CTD402 in T-ALL/LBL patients (TENACITY-01). ClinicalTrials.gov. Updated December 30, 2025. Accessed January 29, 2026. https://clinicaltrials.gov/study/NCT07070219

3. Lu P, Hu Y, Zhang X, et al. CTD402, allogeneic anti-CD7 CAR t-cell, in relapsed or refractory (R/R) t-cell acute lymphoblastic leukemia/lymphoblastic lymphoma (T-ALL/LBL) - report of clinical outcomes at the recommended phase 2 dose (RP2D). Blood. 2025;146(Supplement 1):1042-1042. doi:10.1182/blood-2025-1042