AT the 2025 American Academy of Neurology (AAN) Annual Meeting in San Diego, new late-breaking data from a Phase III clinical trial highlighted the efficacy and safety of telitacicept in patients with generalized myasthenia gravis (gMG).
Telitacicept is a recombinant fusion protein that simultaneously inhibits B lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL), reducing B cell activity. Building on prior phase 2 data, this placebo-controlled, multicenter study enrolled 114 adults with confirmed gMG who were positive for AChR or MuSK antibodies and met additional disease severity criteria.
Patients were randomized 1:1 to receive weekly subcutaneous telitacicept (240 mg) or placebo for 24 weeks. This was followed by a 24-week open-label period, which is ongoing. The primary endpoint was the change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24. Key secondary endpoints included changes in Quantitative Myasthenia Gravis (QMG) scores and response rates.
At Week 24, the mean change in MG-ADL score was –6.4 in the telitacicept group compared with –1.6 in the placebo group (p<0.001). Likewise, the mean QMG score improved by –7.8 versus –1.9 (p<0.001). A clinically meaningful ≥3-point reduction in MG-ADL was achieved by 98.1% of telitacicept-treated patients compared with 12.0% on placebo. Similarly, 87.0% of telitacicept patients saw a ≥5-point reduction in QMG score, versus 16.0% with placebo (p<0.001). Reductions in immunoglobulin G, A, and M levels were observed with telitacicept but not with placebo. The most frequently reported adverse event associated with telitacicept was a decrease in IgM levels. These results underscore telitacicept’s potential as a targeted immunomodulatory therapy for patients with gMG. The open-label extension will offer further insight into long-term outcomes. Reference: Yin J et al. Efficacy and Safety of Telitacicept in Patients with Generalized Myasthenia Gravis: Results of a Phase 3 Study. 2025 American Academy of Neurology (AAN) Annual Meeting, April 5-9, 2025, San Diego, California, USA.
