1st US patient enrolled in Phase 3 trial of gMG therapy telitacicept
Trial is recruiting 180 patients worldwide
Remegen enrolled the first U.S. patient in a Phase 3 trial testing the safety and efficacy of telitacicept to treat generalized myasthenia gravis (gMG), according to a company press release,
This Phase 3 clinical trial (NCT06456580) is a global, multi-center, randomized, double-blind, placebo-controlled study in which adults with gMG will receive either under-the-skin (subcutaneous) injections of telitacicept or a placebo for 24 weeks.
The trial is expected to recruit 180 patients worldwide. All trial participants will have to be diagnosed with gMG and test positive for self-reactive antibodies.
Telitacicept, previously known as RC18, works by reducing the inflammatory activity of B-cells, a type of immune cell. In myasthenia gravis (MG) patients, malfunctioning B-cells produce self-reactive antibodies that interfere with nerve-muscle communication, causing muscle weakness and other disease symptoms. Self-reactive antibodies targeting acetylcholine receptors are the most common type of disease-causing antibodies.
Telitacicept is designed to block two critical proteins involved in B-cell development: BLyS (a B-cell lymphocyte stimulator) and APRIL (A proliferating-inducing ligand). Specifically, the experimental therapy was created by fusing an antibody protein fragment with part of the TACI receptor, a protein that regulates B-cell development.
Daily living, quantitative measures tracked
The trial’s primary outcome is to evaluate if telitacicept improves scores on the MG Activities of Daily Living (MG-ADL) scale from baseline (the study start) to week 24 compared with the placebo-treated group. The MG-ADL scale assesses if the therapy improves engagement in daily activities.
Researchers will also test if the therapy reduces scores on the Quantitative Myasthenia Gravis (QMG) scale, a 13-item assessment of MG severity, and if it improves quality of life, measured at 24 weeks relative to initial baseline scores.
Results from a previous Phase 2 clinical trial (NCT04302103) in China showed that nearly six months of telitacicept treatment lessened disease severity in adults with gMG with antibodies targeting acetylcholine receptors.
In the course of the trial, QMG scores dropped for both the low (160 mg) and high (240 mg) doses tested, indicating less severe disease. Both groups also had reduced blood levels of different types of antibodies. Overall, the therapy was deemed safe and tolerable.
In 2022, telitacicept received orphan drug designation from the U.S. Food and Drug Administration (FDA) as a potential treatment for myasthenia gravis. The therapy also received fast track status in the U.S.
The therapy has been approved in China to treat other autoimmune conditions — systemic lupus erythematosus and rheumatoid arthritis — and has several ongoing trials examining its efficacy in treating other diseases.
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