NEWS

From October 24 to 29, 2025, the American College of Rheumatology (ACR) Convergence 2025 was grandly held in Chicago, USA. At this event, Bioheng Therapeutics presented the latest clinical results of CTA311, its allogeneic anti-CD19 CAR-T product for systemic lupus erythematosus (SLE) and ANCA-associated vasculitis (AAV), and CTA313, its allogeneic anti-CD19/BCMA CAR-T product for SLE and lupus nephritis (LN), in the form of academic posters.

The poster at ACR Convergence 2025 detailed the results of an investigator-initiated trial. This single-arm, open-label, dose-escalation study evaluated the safety and efficacy of CTA311 in patients with SLE and AAV. Four dose levels were explored (DL1: 0.2×10⁶ CAR+ T cells/kg; DL2: 1.0×10⁶ CAR+ T cells/kg; DL3: 3.0×10⁶ CAR+ T cells/kg; DL4: 6.0×10⁶ CAR+ T cells/kg). All patients received lymphodepletion with fludarabine (30 mg/m²/day × 3 days) and cyclophosphamide (300 mg/m²/day × 3 days) prior to CTA311 infusion. As of August 30, 2025, a total of 11 patients were enrolled, including 10 with SLE and 1 with AAV.Ten patients (91%) were female, with a median age of 31 years (range: 21-55), and 64% patients previously received biologics including belimumab and telitacicept.

Safety:
CTA311 was well tolerated with no dose-limiting toxicities (DLT), neurotoxicity or graft-versus-host disease (GvHD) events reported. Cytokine release syndrome (CRS) events occurred in 82% (n=9), all low grade (G) (7 G1 and 2 G2). Three patients experienced treatment related infections (1 pneumonia (G3) and CMV reactivation (G1), 1 fungal infection (G2), and 1 infection NOS (G1)), which all fully resolved with antimicrobial treatment.

Efficacy and Cellular Kinetics:
In SLE/LN, preliminary analysis indicates elimination of autoantibodies, improvement in C3 levels, and decline in proteinuria leading to improvement in disease activity scores SLEDAI-2K and PGA. Responses were dose dependent with 100% LLDAS and 50% DORIS at DL4.

In AAV, the patient achieved a BVAS score of 0 at M3 and M4 from baseline 8. A minor symptom (arthritis) reappeared at M6 but resolved at M9 without any adjustment in medication.

PK analysis revealed peak expansion of CAR-T cells approximately 7-21 days post-infusion, with a dose-dependent pattern, suggesting the potential for deeper remission at higher dose levels.

Pharmacodynamics:
Complete B cell depletion was observed by Day 4 post-infusion with recovery starting Day 42. In patients that achieved anti-dsDNA antibodies eradication, the anti-dsDNA antibodies remained undetected up to 12 months, and ongoing, despite B-cell recovery. 

The poster at ACR Convergence 2025 detailed the results of an investigator-initiated trial. This single-arm, open-label, dose-escalation study evaluated the safety and efficacy of CTA313 in patients with SLE/LN. Three dose levels were explored (DL1: 3×10⁶ CAR+ T cells/kg; DL2: 6×10⁶ CAR+ T cells/kg; DL3: 10×10⁶ CAR+ T cells/kg). All patients received lymphodepletion with fludarabine (30 mg/m²/day × 3 days) and cyclophosphamide (300 mg/m²/day × 3 days) prior to CTA313 infusion. As of September 20, 2025, a total of 15 patients were enrolled, including 6 (40%) with SLE and 9 (60%) with LN, with a median age of 37 years (range: 21-50).

In terms of safety, no immune effector cell-associated neurotoxicity syndrome (ICANS) or graft-versus-host disease (GvHD) was observed at any dose level. Only Grade 1 Cytokine release syndrome (CRS) was observed in 6 patients (40%), largely self-limiting. All Grade 3 infections fully resolved with antimicrobial treatment.

Regarding efficacy, with a median follow-up of 4 months, 100% of patients achieved the Systemic Lupus Erythematosus Responder Index-4 (SRI-4), 79% achieved Lupus Low Disease Activity State (LLDAS), and 43% achieved Definitions of Remission in SLE (DORIS). Robust in vivo expansion of CAR-T cells was observed, with 28-day persistence in 67% of patients. A single infusion of CAR-T cells induced complete B-cell depletion, sustained undectable anti-dsDNA antibodies, and notable reductions in other autoantibodies, including anti-Sm, anti-histone, and anti-nucleosome antibodies.

Preliminary results indicate that CTA313 can rapidly and durably control disease activity in patients with SLE and LN, with an acceptable safety profile. Ongoing clinical studies are further evaluating CTA313 in patients with other B cell-driven autoimmune diseases.