Telitacicept

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. Abstract Title: Preliminary Safety, Efficacy, and Cellular Kinetics of CTA311, a CD19 Targeted Universal CAR-T Therapy, for Active Autoimmune Diseases Abstract Code: 2129320 Poster Code: 2452 Session: Systemic Lupus Erythematosus – Treatment Poster III Presentation Time: Tuesday, October 28, 10:30 AM - 12:30 PM CT 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. CTA311 exhibited an acceptable safety profile in patients with SLE, LN and AAV, and displayed robust expansion coincided with profound B-cell depletion and concomitant elimination of pathogenic autoantibodies, leading to marked clinical improvement in these patients. Future clinical studies are warranted to further evaluate and characterize the efficacy and safety of CTA311 in patients with SLE or other B cell-driven autoimmune diseases. Abstract Title: Preliminary Safety, Efficacy, and Cellular Kinetics of CTA313, a CD19/BCMA Dual-Targeted Universal CAR-T Therapy, for Active Systemic Lupus Erythematosus Abstract Code: 2129706 Poster Code: 1532 Session: Systemic Lupus Erythematosus – Treatment Poster II Presentation Time: Monday, October 27, 10:30 AM - 12:30 PM CT 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. About CTA311 and CTA313 Both CTA311 and CTA313 are allogeneic CAR-T products derived from healthy donors. CTA311 targets CD19, whereas CTA313 targets both CD19 and BCMA. Both products are developed based on Bioheng Therapeutics’ proprietary allogeneic CAR-T platform—ANSWER®—which incorporates genetic modifications to prevent graft-versus-host disease (GvHD) and host-versus-graft rejection (HvG). These products can be manufactured in advance and stored for multiple patients, providing an "off-the-shelf" solution for patients in need of CAR-T cell therapy. Leveraging the ANSWER® platform, Bioheng Therapeutics is actively developing a broad pipeline of allogeneic CAR-T programs. These programs have demonstrated robust and durable persistence of allogeneic CAR-T cells, highlighting strong therapeutic potential in both autoimmune diseases and oncology.

CAMBRIDGE, Mass., Oct. 17, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that clinical data from Stage A of a Phase 3 study in China sponsored by Vor Bio’s collaborator RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), which evaluated telitacicept in adults with IgA neuropathy (IgAN), will be presented as a late-breaking oral presentation at American Society of Nephrology’s (ASN) Kidney Week 2025 being held Nov 5-9, 2025, in Houston, Texas. Late-Breaking Oral Presentation Details Abstract Title: Efficacy and Safety of Telitacicept in Patients with lgA Nephropathy: Results from Stage A of a Phase 3 Clinical Study Session: Hight-Impact Clinical Trials - 2 Date & Time: November 8, 2025 from 10:45-11:00am CT In August, Vor Bio announced topline results from the RemeGen-sponsored study, reporting that telitacicept achieved the primary endpoint of reducing proteinuria demonstrating a 55% reduction in 24-hour urine protein-to-creatinine ratio (UPCR) at 39 weeks compared with placebo (p<0.0001). UPCR is an objective and globally recognized regulatory marker of disease activity in IgAN. Telitacicept demonstrated a favorable safety profile. The full results of Part A of the study will be shared at ASN. RemeGen has submitted a Biologics License Application (BLA) to the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China for IgAN, which if approved would become telitacicept’s fifth approved indication in China. About Vor Bio Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and commercialization to address serious autoantibody-driven conditions worldwide. For more information visit www.vorbio.com. About Telitacicept Telitacicept is a novel, investigational recombinant fusion protein designed to treat autoimmune diseases by selectively inhibiting BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. In a Phase 3 clinical trial in generalized myasthenia gravis in China, telitacicept demonstrated a placebo adjusted 4.83-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living scale) at 24 weeks, the primary endpoint of the trial. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia-Pacific to support potential approval in the United States, Europe, and Japan. About IgAN Nephropathy IgA nephropathy (IgAN) is one of the most common primary glomerular diseases worldwide and a leading cause of chronic kidney disease (CKD) and end-stage renal disease (ESRD). It is characterized by IgA-containing immune complex deposition in the kidney, leading to inflammation, proteinuria, hypertension, and progressive loss of renal function. Up to 40% of patients progress to ESRD within 20 years of diagnosis, underscoring the significant unmet need for effective therapies. Current treatment approaches, including optimized blood pressure control, renin-angiotensin system blockade, and SGLT2 inhibitors, primarily slow disease progression but do not address the underlying immunopathology. The prevailing scientific consensus is that overproduction of galactose-deficient IgA1 (Gd-IgA1) is a central driver of IgAN. BAFF and APRIL, two cytokines critical to B-cell survival and function, promote the production of Gd-IgA1 and its pathogenic antibodies. Media & Investor Contacts:Carl Mauchcmauch@vorbio.com Sarah Spencerinvestors@vorbio.com