RemeGen: Telitacicept for the Treatment of Generalized Myasthenia Gravis Approved in China

Rhea-AI Summary

RemeGen (REGMY) announced that Telitacicept has received NMPA approval in China for treating adult patients with anti-AChR antibody-positive generalized myasthenia gravis (gMG) in combination with conventional therapies. As the world's first innovative biologic drug targeting both BLyS and APRIL for gMG treatment, the drug showed exceptional Phase III results with 98.1% of patients achieving ≥3-point improvement in MG-ADL scores compared to 12.0% in the placebo group. The drug demonstrated strong safety profiles, with adverse event rates comparable to placebo. This approval marks Telitacicept's third approved indication in China, alongside systemic lupus erythematosus and rheumatoid arthritis. With approximately 220,000 MG patients in China and 1.2 million worldwide, this approval addresses a significant unmet medical need.

Positive

• Achieved highest MG-ADL response rate among gMG drugs in Phase III trials (98.1% vs 12.0% placebo)
• Strong efficacy with 87.0% of patients showing ≥5-point QMG score improvement vs 16.0% placebo
• Favorable safety profile with AE rates comparable to placebo
• Third approved indication for Telitacicept in China, expanding market potential
• Addresses large market with 220,000 MG patients in China

Negative

• None.

YANTAI, China, May 27, 2025 /PRNewswire/ -- On May 27, RemeGen Co., Ltd.(688331.SH / 09995.HK) announced that Telitacicept has officially been approved for marketing in China by the National Medical Products Administration (NMPA) for the treatment of adult patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in combination with conventional therapies. As the world's first innovative biologic drug targeting both BLyS and APRIL for the treatment of myasthenia gravis (MG), Telitacicept's approval not only brings new treatment options to patients, but also fills the gap of domestic innovative drugs in the field of MG treatment in China. It is expected to reshape the treatment landscape in this field. Previously, the indication of Telitacicept for MG was included in the breakthrough therapy category and priority review by the NMPA of China, and was granted orphan drug qualification and fast track designation by the FDA.

Myasthenia gravis is an autoimmune disease caused by neuromuscular junction transmission disorders. It is characterized by fluctuating muscle weakness and fatigue in symptoms, long  treatment cycles and high recurrence rate. Approximately 80%-85% of patients with MG patients are AChR antibody positive, and 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 approximately 220,000 patients in China. At present, there is a great unmet medical need.

Telitacicept is constructed with the extracellular domain of the human transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI) receptor and the fragment crystallizable (Fc) domain of human immunoglobulin G(IgG). It simultaneously targets B-cell lymphocyte stimulator (also known as BLyS) and a proliferation-inducing ligand (APRIL), and directly attack the source of pathenogenic antibody production – B cells and plasma cells.

According to the phase III data presented at the annual meeting of the American Academy of Neurology (AAN) on April 9, 2025, Telitacicept demonstrated a clinically meaningful efficacy and safety profile in patients with gMG. According to the data, after 24 weeks of treatment, 98.1% of patients in the Telitacicept group demonstrated a ≥ 3-point improvement in Myasthenia Gravis Activities of Daily Living ("MG-ADL") score, far exceeding that of the placebo group which was 12.0%. The MG-ADL score decreased by 5.74 points from baseline in the Telitacicept group, compared to a decrease of 0.91 point in the placebo group. 87.0% of the patients in the Telitacicept group demonstrated a ≥ 5-point improvement in Quantitative Myasthenia Gravis ("QMG") score, far exceeding that of the placebo group which was 16.0%. The QMG score decreased by 8.66 points from baseline in the Telitacicept, compared to a decrease of 2.27 points in the placebo group, thereby proving significant treatment benefit. In terms of safety, the overall adverse event (AE) rate in the Telitacicept group was comparable to that in the placebo group, indicating a good overall safety profile.

Among the drugs for gMG that have completed phase III clinical studies, Telitacicept had the highest MG-ADL response rate. The approval of Telitacicept in China will benefit more patients with MG in the country, thereby allowing more ambitious treatment goals to be achieved in terms of long-term disease management. Currently, the Company is advancing the global multi-center phase III trial of Telitacicept in patients with MG, aiming to validate the efficacy and safety of Telitacicept in a wider patient population.

The principal researcher, Professor Yin Jian from Beijing Hospital, said: "We sincerely congratulate the approval of the therapeutic indication of Telitacicept for MG! Telitacicept demonstrated rapid and significant clinical improvements in the Phase III trial with good tolerability. This is a major breakthrough and key progress for Chinese innovative drugs in the field of neuro-immune diseases. It provides a high profile basis for evidence-based clinical application of Telitacicept in the treatment of MG, and has also opened up a new paradigm of precise treatment in this field. We believe that with the inclusion of this indication in the medical insurance, Telitacicept will bring new and more effective treatment options to more patients."

Dr. Fang Jianmin, the CEO of RemeGen, said: "The approval of the MG indication for Telitacicept in China is of great significance. It provides clinicians with a new and more powerful 'weapon' to treat patients more effectively and precisely. We look forward to the inclusion of this indication in the medical insurance system to help patients and their families reduce the burden of treatment and further improve their quality of life. RemeGen is advancing the global Phase III clinical study of Telitacicept for MG. We are looking forward to working closely with global experts and scholars to contribute to the continuous advancement of treating autoimmune diseases."

Aside from Myasthenia Gravis, Telitacicept has been approved in China for the treatment of two other major indications, systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA).

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SOURCE RemeGen Co., Ltd

FAQ

What are the clinical trial results for RemeGen's Telitacicept in treating gMG?

In Phase III trials, 98.1% of patients showed ≥3-point MG-ADL score improvement vs 12.0% for placebo, and 87.0% achieved ≥5-point QMG score improvement vs 16.0% for placebo, with comparable safety to placebo.

What makes REGMY's Telitacicept unique in treating myasthenia gravis?

Telitacicept is the world's first innovative biologic drug that targets both BLyS and APRIL for myasthenia gravis treatment, attacking B cells and plasma cells that produce pathogenic antibodies.

How many indications has Telitacicept been approved for in China?

Telitacicept has been approved for three indications in China: generalized myasthenia gravis (gMG), systemic lupus erythematosus (SLE), and rheumatoid arthritis (RA).

What is the market size for myasthenia gravis treatment that REGMY's Telitacicept addresses?

The global market includes approximately 1.2 million MG patients, with about 220,000 patients in China specifically.

What regulatory designations has RemeGen's Telitacicept received for MG treatment?

Telitacicept received breakthrough therapy category and priority review status from China's NMPA, plus orphan drug qualification and fast track designation from the FDA.