Everest Medicines (HKEX 1952.HK, “Everest”, or the “Company”), a biopharmaceutical company focused on the development, manufacturing and commercialization of innovative medicines and vaccines, announced today that China’s National Medical Products Administration (NMPA) has approved Nefecon® for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk of disease progression. Nefecon® was the first ever treatment for IgAN approved by the U.S. Food and Drug Administration and European Medicines Agency. Today’s NMPA approval marks a new era of IgAN treatment in China.

China has the highest prevalence of primary glomerular diseases in the world with an estimated five million IgAN patients. Current non-targeted treatment options for IgAN, such as renin-angiotensin system (RAS) inhibitors, do not fundamentally alter disease progression. IgAN patients are at risk of progressing to end-stage renal disease, which may then require dialysis or kidney transplant. There is a very significant unmet medical need for novel effective therapies among IgAN patients in China and other Asian countries. On top of Nefecon’s approval in mainland China and Macau, it is expected to receive New Drug Application (NDA) approval in Singapore and Hong Kong in the near term.

“The NDA approval of Nefecon® in China is not only a significant milestone for Everest, but also a landmark for Chinese IgAN patients who will finally have an approved drug with an IgAN indication to treat their illness. Chinese patients have higher incidence rates of IgAN and faster disease progression than the rest of the world. They have an urgent need for innovative therapies like Nefecon® to target disease origin and delay disease progression. I would like to extend my gratitude to the regulators, experts, and our staff that made today’s approval possible,” said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. “The NDA approval further establishes our leadership position in nephrology. While the Company will actively prepare for the commercial launch of Nefecon® and bring this first-in-disease therapy to patients in China as soon as possible, we will also continue to develop other innovative drug candidates in renal and auto-immune diseases to benefit more Chinese and Asian patients.”

“IgA nephropathy is the most common primary glomerular disease in China and occurs at a young age with a high probability of progressing to end-stage renal disease, imposing a heavy burden on patients and society. Compared with European and American populations, Chinese IgAN patients have faster disease progression and worse prognoses. The full results of the NefIgArd study showed that Nefecon can protect renal function, delay patients’ progression to dialysis or kidney transplantation and significantly reduce urinary protein and hematuria. It is also safe and well-tolerated,” said Professor Zhang Hong with Peking University First Hospital, a member of the global steering committee for the Phase 3 clinical study NefIgArd. “In the Chinese population analysis, Nefecon had a numerically higher treatment effect in renal function protection. The approval of Nefecon in China helps fill the gap in targeted treatment of IgAN and allows this first-in-disease therapy to benefit more Chinese patients.”

“IgA nephropathy is highly prevalent in Asia and is one of the main causes of kidney failure in young adults in China. For a long time, the disease lacked targeted treatment options, resulting in significant unmet clinical needs. Nefecon is the world’s first drug for IgAN approved in the United States and the European Union,” said Professor Xie Jingyuan, Chief Physician of the Department of Nephrology, Ruijin Hospital affiliated with Shanghai Jiao Tong University School of Medicine. “Through the early-access program, the first prescription of Nefecon in China was issued at the Hainan subsidiary of Ruijin Hospital in Bo’ao on April 24, 2023. During the follow-up period, Nefecon was found to be effective in stabilizing renal function, reducing proteinuria and hematuria, and was well tolerated by the patients. Nefecon’s approval in China is of great significance, bringing new hope to IgAN patients in the nation and helping doctors better manage patient compliance and disease management.”

The global Phase 3 NefIgArd clinical trial was a randomized, double-blind, multicenter study that evaluated the efficacy and safety of Nefecon® at a once-daily dose of 16 mg, compared to placebo in adult patients with primary IgAN on optimized RASi therapy. In the global study, Nefecon® demonstrated a highly statistically significant and clinically relevant benefit compared to placebo in estimated glomerular filtration rate (eGFR) over the two-year period of 9-months of treatment with Nefecon® and 15-months of follow-up off drug. The reduction in urine protein creatinine ratio (UPCR) observed with Nefecon® treatment was also durable and the proportion of patients with microhematuria in the Nefecon group declined.

Results from Chinese subpopulation analysis demonstrated numerically greater magnitude of Nefecon® treatment effect compared with placebo in kidney function, proteinuria and microhematuria. The mean absolute change from baseline in eGFR at 24 months showed approximately 66% less deterioration in kidney function with Nefecon® compared with the global data set which demonstrated a smaller 50% reduction in loss of kidney function. Patients treated with Nefecon® in mainland China showed a larger 43% (95% CI 8%, 65%) reduction in UPCR compared with placebo at 24 months and a greater 31%  reduction (95% CI 0, 53) at 9 months. This compares to the global data analysis in which patients treated with Nefecon® recorded an approximately 30% reduction in proteinuria at both 24 months and 9 months compared to placebo. Nefecon® was generally well tolerated, with a safety profile in the Chinese population generally consistent with the larger global study. The Chinese patient data were presented at the American Society of Nephrology (ASN) Kidney Week 2023.

As the first non-oncology medicine to receive Breakthrough Therapy Designation in China, Nefecon® was also granted Priority Review in the country. In August 2023, the U.S. FDA accepted the submission for a supplemental New Drug Application (sNDA) for full approval of Nefecon® (marketed in the US under the brand name TARPEYO) from our partner Calliditas Therapeutics AB, granting Priority Review and setting a Prescription Drug User Fee Act (PDUFA) goal date of Dec. 20, 2023.