Remegen Announces Exciting Results of Telitacicept Phase 3 Clinical Trial for Patients with Generalized Myasthenia Gravis

Rhea-AI Summary

RemeGen (REGMY) has announced breakthrough Phase 3 clinical trial results for Telitacicept in treating generalized myasthenia gravis (gMG). The trial, presented at the American Academy of Neurology Annual Meeting 2025, showed remarkable efficacy with 98.1% of patients in the Telitacicept 240 mg group achieving ≥3-point reduction in MG-ADL score and 87% showing ≥5-point reduction in QMG score at Week 24.

The multicenter, randomized, double-blind study involved 114 gMG patients, demonstrating significant improvements in clinical outcomes with a favorable safety profile. At Week 24, the Telitacicept group showed MG-ADL score changes of -5.74 versus -0.91 in the placebo group. The drug's Biologics License Application has been accepted by China's regulatory authorities, with approval expected in Q2 2025.

Positive

• Exceptional efficacy with 98.1% response rate in MG-ADL score reduction
• Strong safety profile with lower infection-related adverse events than placebo
• BLA acceptance by Chinese regulators with expected approval in Q2 2025
• Large market opportunity with 1.2 million global MG patients
• Sustained improvement in clinical outcomes through Week 24

Negative

• Global Phase 3 trial still ongoing, limiting immediate international market access
• Currently pending regulatory approval, no immediate revenue generation

YANTAI, China, April 8, 2025 /PRNewswire/ -- On April 8, 2025 (UTC-7), impressive results of the Phase 3 clinical trial (NCT05737160) evaluating the efficacy and safety of Telitacicept (synonym: RC18; brand name: 泰爱^®) in patients with generalized myasthenia gravis (gMG) were presented during the Late-Breaking Science Session at the American Academy of Neurology (AAN) Annual Meeting 2025 in San Diego, CA.

According to the presented data, among the participants in the Telitacicept 240 mg group, 98.1% of the patients demonstrated a ≥ 3-point reduction in Myasthenia Gravis Activities of Daily Living (MG-ADL) score and 87% of the patients demonstrated a ≥ 5-point reduction in Quantitative Myasthenia Gravis (QMG) score at Week 24. With the highest MG-ADL response rate among all drugs for gMG with phase 3 studies reported to date, Telitacicept will provide an important new treatment option gMG.

Telitacicept is a novel fusion protein targeting both BlyS and APRIL independently designed and developed by RemeGen Co.,Ltd. ("RemeGen"). The multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trial was conducted in China (NCT05737160) and led by Professor Jian Yin of Beijing Hospital. A total of 114 gMG patients with baseline MG-ADL score ≥6 and QMG score ≥8 were enrolled in the study, with 57 patients in the Telitacicept 240 mg group and the placebo group respectively. The study consisted of a 24-week double-blind treatment stage and an open-label treatment stage. The presented results herein were from the double-blind treatment study.

The data showed that ‌Telitacicept‌ achieved ‌sustained and significant improvement‌ in clinical outcomes for patients with gMG, ‌with a favorable safety profile：

• Telitacicept demonstrated significant improvements‌ in MG-ADL and QMG scores after 4 weeks' treatment compared to placebo.
• At Week 24, change in MG-ADL was -5.74 and -0.91 with Telitacicept and placebo group, respectively. 98.1% and 12.0% of Telitacicept and placebo group patients had ≥3-point reduction in MG-ADL, respectively. The MG-ADL scale quantifies the impact of MG symptoms on daily life quality, focusing on patients' subjective experience and daily function. These data showed that patients treated with Telitacicept achieved ‌clinically meaningful improvement‌‌.
• At Week 24, change in QMG was -8.66 and -2.27 with Telitacicept and placebo group, respectively. 87.0% and 16.0% of Telitacicept and placebo group patients had ≥ 5-point reduction in QMG, respectively. The QMG score assesses the strength and endurance of the whole body muscle group, focusing on objective measurement. These data demonstrated that patients treated with Telitacicept‌ achieved ‌clinically meaningful reductions in disease severity. There is a strong correlation between MG-ADL and QMG in evaluating treatment response, and the combination of MG-ADL and QMG can comprehensively reflect the disease severity.
• Sustained improvement: MG-ADL and QMG scores continued to decline over time in the Telitacicept group, with peak improvement at the last reported observation, Week 24.
• Telitacicept was well tolerated during the treatment, with an overall adverse event (AE) incidence similar to that in the placebo group and an incidence of infection-related AEs lower than that in the placebo group (45.6% vs. 59.6%).

The principal investigator Prof. Yin Jian of Beijing Hospital said "Telitacicept demonstrated rapid and significant clinical improvement in the phase 3 trial, and was well tolerated. Its dual-targeting mechanism not only inhibits abnormal B cells and plasma cells thoroughly and reduces the level of pathogenic antibodies, but also effectively slows down disease progression in the long term, and reduces steroid dosage as symptoms improve, providing a more precise, efficient, persistent, and safer option for gMG treatment."

Dr. Jianmin Fang, CEO of RemeGen, said "The phase 3 data of Telitacicept for MG is clinically meaningful. There is a large unmet medical need in MG because the disease presents significant threat to patients' health and life-span. This breakthrough means that we have an new, effective treatment for MG as Telitacicept can significantly improve patients' symptoms and bring long term benefits to MG patients. We look forward to working with global experts and scholars to overcome more complex autoimmune diseases and benefit more patients."

MG is an autoimmune disease caused by neuromuscular junction transmission disorders. It is characterized by fatigue and fluctuations in symptoms, long treatment cycles and high recurrence rate. More than 85% of patients develop to gMG within 24 months of onset. According to Frost & Sullivan report, there are about 1.2 million MG patients worldwide, including about 220,000 patients in China. At present, there is a great unmet medical demand.

The newly disclosed "best-in-class" data mark a breakthrough achievement of Chinese innovative drugs in the field of neuroimmunology. Currently, the BLA of Telitacicept for the treatment of gMG has been accepted by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration, and it is expected to be approved in China in the second quarter of this year.

Meanwhile, RemeGen is advancing a global multicenter Phase 3 trial of Telitacicept for the treatment of gMG, aiming to validate the efficacy and safety of Telitacicept in a wider patient population, and to provide a superior treatment option for gMG patients worldwide.

FAQ

What are the key efficacy results from REGMY's Telitacicept Phase 3 trial in gMG?

98.1% of patients achieved ≥3-point MG-ADL score reduction and 87% showed ≥5-point QMG score reduction at Week 24, demonstrating superior efficacy compared to placebo.

When is REGMY's Telitacicept expected to receive approval in China?

Telitacicept is expected to receive approval from China's National Medical Products Administration in Q2 2025.

How many patients were enrolled in REGMY's Telitacicept Phase 3 trial?

114 gMG patients were enrolled, with 57 patients each in the Telitacicept 240 mg group and placebo group.

What is the safety profile of REGMY's Telitacicept in the Phase 3 trial?

Telitacicept showed similar overall adverse event rates to placebo, with lower infection-related AEs (45.6% vs 59.6%).

What is REGMY's market potential for Telitacicept in gMG treatment?

There are approximately 1.2 million MG patients worldwide, including 220,000 in China, representing significant market potential.