In This Article:
On 9 April, during the Late-Breaking Science Session at the 2025 American Academy of Neurology (AAN) meeting, Remegen announced exciting results from a Phase III clinical trial evaluating the efficacy and safety of telitacicept. The results showed that telitacicept demonstrated a clinically meaningful efficacy and safety profile, displaying a significant improvement over 24 weeks with sustained reductions in total immunoglobulin G levels. This means that telitacicept has the potential to challenge currently marketed disease-modifying therapies (DMTs). Telitacicept reduces B-cell activity through a unique mechanism of action.
In generalised myasthenia gravis (gMG), B cells produce autoantibodies targeting the acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (MuSK), thus disrupting neuromuscular transmission and leading to muscle weakness. Telitacicept, a novel fusion protein, inhibits B lymphocyte stimulator and a proliferation-inducing ligand, reducing B-cell activity. The multicentre Phase III trial based in China enrolled 114 patients with gMG, confirmed as AChR+ or MuSK+, who were randomised to receive either subcutaneous telitacicept (240mg) or placebo for 24 weeks. Telitacicept demonstrated significant improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores after four weeks of treatment compared to placebo. At Week 24, telitacicept elicited a change in MG-ADL of -5.74, whereas the placebo group saw only a -0.91 change in MG-ADL score. Furthermore, 98.1% and 12.0% of Telitacicept and placebo group patients, respectively, had a three-point or higher reduction in MG-ADL. The key secondary endpoint was a change in QMG. At Week 24, change in QMG was -8.66 and -2.27 with the Telitacicept and placebo groups, respectively. Moreover, 87.0% and 16.0% of telitacicept and placebo group patients, respectively, had a five-point or higher reduction in QMG. This data demonstrated that patients treated with telitacicept achieved clinically meaningful reductions in disease severity. Telitacicept was also shown to be well-tolerated, with an overall adverse event (AE) incidence similar to that of the placebo group and an incidence of infection-related AEs that was lower than that of the placebo group (45.6% versus 59.6%). Displaying improvements in disease control, as measured by MG-ADL and QMG scores across a broad population that is seropositive for AChR or MuSK autoantibodies, is currently essential as most marketed therapies are indicated for AChR+ patients with gMG. As a DMT, telitacicept would be entering a highly competitive space in the market. According to leading data and analytics company GlobalData’s drugs database, there are six DMTs currently marketed across the seven major pharmaceutical markets (US, France, Germany, Italy, Spain, UK, and Japan) for AChR+ patients. These include complement inhibitors and neonatal Fc receptor inhibitors, both of which are treatment pathways that are well-established in the MG treatment paradigm.