The board of directors of the Shanghai Junshi Biosciences Co., Ltd. has received a notice from the Drug Office, Department of Health, the Government of the Hong Kong Special Administration Region (the ?DO?) that the New Drug Application (the ?NDA?) for toripalimab (product code: TAB001/JS001) in combination with cisplatin and gemcitabine, for the first-line treatment of adults with metastatic or recurrent locally advanced nasopharyngeal carcinoma (?NPC?), and for toripalimab, as a single agent, for the treatment of adults with recurrent, unresectable, or metastatic NPC with disease progression on or after platinum-containing chemotherapy has been accepted by the DO. NPC is a malignant tumor that occurs in the nasopharyngeal mucosal epithelium and is one of the most common types of head and neck cancer. According to the World Health Organization, the number of newly diagnosed NPC cases in 2022 exceeded 120,000 worldwide.

Due to the location of the primary tumor, surgery is rarely an option, while radiotherapy alone or in combination with chemotherapy are the main treatment options for localized cancers. This NDA is supported by the results from JUPITER-02, a randomized, double blind, placebo- controlled, multinational multi-center Phase III clinical study (NCT03581786), for the first-line treatment of NPC and the results from POLARIS-02, a multi-center, open-label, pivotal Phase II clinical study (NCT02915432), for second-line or more prior treatments for recurrent or metastatic NPC. The results of JUPITER-02, the first multinational multi-center, double-blind, randomized, placebo- controlled Phase III clinical study with the largest sample size in the field of immunotherapy for NPC, were published at the plenary session of the 2021 American Society of Clinical Oncology (ASCO) annual meeting (#LBA2), and in Nature Medicine (IF: 82.9) and the Journal of the American Medical Association (IF: 120.7).

The results showed that as compared to chemotherapy alone, toripalimab in combination with chemotherapy for the first-line treatment of recurrent or metastatic NPC significantly improved progression-free survival (? PFS?) and overall survival (?OS?), with a median PFS of 21.4 months, and a 3-year OS rate of 64.5%, reduced the risks of disease progression or death by 48%, and the risk of death by 37% and demonstrated a manageable safety profile. The POLARIS-02 results were published online in January 2021 in the Journal of Clinical Oncology (IF: 45.3).

The results showed that toripalimab demonstrated durable antitumor activity in patients with recurrent or metastatic NPC who failed previous chemotherapy, with an objective response rate (ORR) of 20.5%, a median duration of response (DoR) of 12.8 months, and a median OS of 17.4 months with a manageable safety profile. Toripalimab injection is the first domestic anti-PD-1 monoclonal antibody approved for marketing in China, and has won the ?Chinese Patent Gold Award ?, the top award in China 's patent field. Over forty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally, including in China, the United States, Southeast Asia, and Europe.

Ongoing or completed pivotal clinical studies evaluating the safety and efficacy of toripalimab cover a broad range of tumor types. As of the date of this announcement, there are eight approved indications for toripalimab in Chinese mainland. In December 2020, toripalimab injection was successfully negotiated into the National Reimbursement Drug List (the ?NRDL?) for the first time.

At present, six indications have been included in the NRDL (2023 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for the treatment of melanoma. In April 2024, the DO accepted the NDA for toripalimab in combination with cisplatin and gemcitabine, for the first-line treatment of adults with metastatic or recurrent locally advanced NPC, and for toripalimab, as a single agent, for the treatment of adults with recurrent, unresectable, or metastatic NPC with disease progression on or after platinum-containing chemotherapy.

In terms of international registration activities, toripalimab has been approved for marketing as the first nasopharyngeal cancer drug in the United States in October 2023. In addition, the European Medicines Agency (EMA) and the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) accepted the marketing authorization application (MAA) for toripalimab in combination with cisplatin and gemcitabine for the first-line treatment of patients with locally recurrent or metastatic NPC, and toripalimab in combination with paclitaxel and cisplatin for the first- line treatment of patients with unresectable locally advanced/recurrent or metastatic esophageal squamous cell carcinoma. The Australia Therapeutic Goods Administration (TGA) and the Singapore Health Sciences Authority (HSA) accepted the new chemical entity application and the New Drug Application for toripalimab in combination with cisplatin and gemcitabine, for the first- line treatment of adults with metastatic or recurrent locally advanced NPC, and for toripalimab, as a single agent, for the treatment of adults with recurrent, unresectable, or metastatic NPC with disease progression on or after platinum-containing chemotherapy, respectively.