AbelZeta Receives FDA Clearance for Phase 1b/2 Clinical Trial of C-CAR168 in Progressive Multiple Sclerosis

AbelZeta Pharma, Inc. has received clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1b/2 clinical trial for C-CAR168, an anti-CD20/BCMA bispecific CAR-T therapy, in patients with progressive multiple sclerosis (PMS) who have not responded to standard therapy. This clearance marks an expansion of the company's clinical development program into refractory chronic autoimmune neurological diseases.

The approval follows recent regulatory milestones in the treatment of systemic lupus erythematosus (SLE), including the FDA's clearance of a multicenter registrational Phase II clinical trial and the European Medicines Agency's (EMA) PRIority Medicines (PRIME) designation. These achievements underscore AbelZeta's commitment to advancing innovative therapies for severe autoimmune diseases.

C-CAR168 is designed to target CD20 and BCMA, aiming to achieve deep depletion of disease-driving B cells and plasma cells. The therapy is part of AbelZeta's pipeline, which includes multiple CAR-T therapies for various autoimmune diseases such as progressive MS, systemic sclerosis (SSc), SLE, and lupus nephritis (LN).

The multi-center, Phase 1b/2 clinical trial will evaluate the safety, tolerability, and efficacy of C-CAR168 in patients with progressive MS who have inadequate response to standard therapies. The study is divided into two phases: Phase 1b will enroll approximately 6 to 24 patients with secondary progressive MS (SPMS) or primary progressive MS (PPMS) to identify the recommended Phase 2 dose. The Phase 2 portion will enroll approximately 95 patients in two independent cohorts, one with active SPMS and the other with PPMS/non-active progressing SPMS patients, to further assess the safety and clinical efficacy of C-CAR168.

Based on preliminary efficacy data from a Phase 1 first-in-human trial, presented at ACR 2025, C-CAR168 showed promising early efficacy signals in SPMS patients. Following treatment, patients demonstrated improvements in gait and neurological function, as well as reductions in brain inflammation and lesion size.

Tony (Bizuo) Liu, Chairman and CEO of AbelZeta, expressed cautious optimism about the potential of C-CAR168.

Multiple sclerosis (MS) is a chronic immune-mediated disease that damages the brain and spinal cord, leading to symptoms such as weakness, fatigue, mobility impairment, and cognitive dysfunction. MS affects approximately 2.8 million people worldwide, with a prevalence of 35.9 per 100,000 population and an incidence of 2.1 new cases per 100,000 people annually. Progressive MS, including primary progressive MS (PPMS) and secondary progressive MS (SPMS), is characterized by gradual worsening of neurological function and disability over time, with limited treatment options available.

The FDA clearance of this Phase 1b/2 study represents another important step in AbelZeta's mission to develop innovative and proprietary cell-based therapeutic products for chronic autoimmune diseases. The company is committed to advancing research and development in its own Good Manufacturing Practice (GMP) facilities at its centers of excellence in Rockville, Maryland, and Shanghai.