On June 8, Remegen (688331.SH/09995.HK) announced that its self-developed global first-in-class BLyS/APRIL dual-target fusion protein innovative drug Telitacicept (RC18) has received approval from the China National Medical Products Administration (NMPA) for two new indications: the treatment of Sjögren's syndrome (dry disease) and IgA nephropathy. This marks the arrival of the world's first approved biologic for Sjögren's syndrome, while also ushering in a new era of precision treatment for IgA nephropathy.
To date, Telitacicept has been approved for marketing in China for five indications: systemic lupus erythematosus, rheumatoid arthritis, generalized myasthenia gravis, Sjögren's syndrome (dry disease), and IgA nephropathy.
Global first biologic approved for the treatment of Sjögren's syndrome (dry disease)
Sjögren’s syndrome (dry disease) indication approved for use in adult patients with active Sjögren’s syndrome (dry disease) (ESSDAI ≥ 5) receiving conventional treatment.
The core basis for this approval comes from a national multicenter, randomized, double-blind, placebo-controlled phase III confirmatory clinical study led by Professor Zeng Xiaofeng from Peking Union Medical College Hospital. This study is also the first Sjögren’s syndrome dual-target innovative drug project globally in the autoimmune biologics field to successfully complete a phase III clinical trial and achieve strong positive results.
381 patients with Sjögren’s syndrome (dry disease) were randomly assigned to the Telitacicept 160mg, Telitacicept 80mg, or placebo group, receiving subcutaneous injections once a week for a total of 48 doses. Between weeks 24 and 48, patients in the placebo group with an inadequate response could be randomly switched in a 1:1 ratio under blinded conditions to receive Telitacicept 160mg or Telitacicept 80mg. The primary efficacy endpoint was the change from baseline in the European League Against Rheumatism Sjögren’s Syndrome Disease Activity Index (ESSDAI) score at week 24. Results showed that compared with placebo, the Telitacicept 160mg group (least squares mean change: -4.4 vs -0.6, P<0.0001) and the 80mg group (least squares mean change: -3.0 vs -0.6, P<0.0001) had significantly reduced ESSDAI scores at week 24. The efficacy of both Telitacicept groups persisted until week 48, with the 160mg group showing better improvement. Other efficacy endpoints, including the proportion of subjects with a ≥3-point reduction in ESSDAI score from baseline, the proportion of subjects with ESSDAI < 5, the improvement rate in the European League Against Rheumatism Sjögren’s Syndrome Patient Reported Index (ESSPRI), and the Sjögren’s Syndrome Composite Responder Index (STAR) response rate, were consistent with the primary efficacy endpoint results.
Regarding this major breakthrough, Professor Zeng Xiaofeng from Peking Union Medical College Hospital stated: "The domestic approval of Telitacicept for Sjögren’s syndrome is a milestone. For patients, Telitacicept can reduce the risk of systemic damage caused by disease progression, thereby significantly improving patients’ quality of life and long-term survival prognosis; for doctors, it elevates the diagnosis and treatment approach for Sjögren’s syndrome from past ‘empirical treatment’ to the level of ‘precise targeting.’ Telitacicept is expected to reshape the future clinical treatment landscape and discipline development direction."
Dr. Fang Jianmin, CEO of Remegen, said: "For a long time, the treatment of Sjögren’s syndrome has been limited to symptom control, lacking effective and systematic treatments targeting the cause, resulting in a huge unmet clinical need. The approval of Telitacicept for this indication brings a new treatment option to a wide range of patients, and we are very excited. Currently, a global phase III clinical study for this indication is underway, and we are continuously exploring the clinical value of Telitacicept for more indications, hoping this blockbuster drug will benefit more patients with autoimmune diseases."
Ushering in a New Era of Precision Therapy for IgA Nephropathy
Indication for IgA nephropathy approved for reducing proteinuria in adult patients with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. This indication was granted conditional market approval by the National Medical Products Administration (NMPA) through the priority review and approval process, and the dosage form is a prefilled injection, which will make medication more convenient for patients and facilitate long-term disease management. This marks that Telitacicept is not only the first domestically developed innovative drug specifically for IgA nephropathy approved in China, but also the first dual-target biologic globally approved for the treatment of IgA nephropathy, potentially reshaping the treatment landscape for this disease.
This approval is based on the significant efficacy and sufficient safety demonstrated by Telitacicept in the completed Phase II clinical study (18C014) and Phase III clinical study (18C021) Stage A for the treatment of IgA nephropathy.
Phase II clinical study (18C014) data showed that the two experimental groups receiving Telitacicept demonstrated significant differences compared to the placebo group in reducing urinary protein, delaying the decline of estimated glomerular filtration rate (eGFR), and lowering immunoglobulin levels (IgA, IgG, and IgM), preliminarily validating the efficacy and safety of Telitacicept in the treatment of IgA nephropathy. The primary efficacy endpoint of Phase A of the Phase III clinical study (18C021) was the change in urinary protein-to-creatinine ratio (UPCR) from baseline at Week 39. The primary analysis method results showed that at Week 39, the UPCR in the Telitacicept 240 mg group decreased by 59% from baseline, with a placebo-adjusted reduction rate of 55%. This study achieved the primary efficacy endpoint of Phase A, and the safety profile was favorable. Specific study data were published in the internationally top-tier medical journal *New England Journal of Medicine* in May this year.
The lead researcher of this study, Professor Zhang Hong from the Department of Nephrology at Peking University First Hospital, commented: "As the first drug to target two pathways at once, Telitacicept stops harmful IgA from being produced right at the source. The clinical trial results show not only a clear benefit in lowering proteinuria and keeping kidney function stable, but also the possibility of cutting down or even replacing steroid use. The approval of Telitacicept for treating IgA nephropathy marks the beginning of a new era of precise, dual-target treatment, offering patients a more tailored option than ever before."
"We are incredibly proud to witness a landmark moment in kidney disease treatment—the domestic approval of Telitacicept for IgA nephropathy," said Dr. Fang Jianmin, CEO of RemeGen. "Over the past two years, RemeGen has achieved a series of groundbreaking advances in autoimmune disease therapy, underscoring our unwavering commitment to pioneering innovation and accelerating the clinical translation of cutting-edge treatments. Moving forward, we will continue to fast-track the development of additional indications, delivering more precise, effective, and safer therapeutic options to patients, and ultimately helping to improve their quality of life."
