(SUZHOU, China, June 21, 2021) GAVRETO^® (pralsetinib), a precision therapy discovered by CStone Pharmaceuticals’ partner Blueprint Medicines, had the first batch of prescriptions issued at Guangdong Province People’s Hospital and around 100 other hospitals across China. GAVRETO is also available at around 80 pharmacies in close to 70 cities across China. With this milestone, GAVRETO, as the first approved selective RET inhibitor in China, can benefit Chinese patients.

Currently, lung cancer is ranked the highest in both incidence and mortality among all types of cancer in China, of which non-small cell lung cancer (NSCLC) represents 80% to 85% ^[1]. RET fusions are oncogenic drivers in one to two percent of NSCLC patients^[2]. In China, about 11,000 new cases of RET fusion-positive NSCLC patients are diagnosed each year, and most of them are already in the advanced stage when diagnosed, posing great challenges to treatment.

Historically, the clinical efficacy of treatment options for RET fusion-positive NSCLC has been very limited. The approval of GAVRETO changed the treatment paradigm in this field and provides physicians with a new treatment option. GAVRETO is a selective RET inhibitor with robust and durable anti-tumor activity and has shown a high objective response rate, as well as safety and tolerability, in patients with RET fusion-positive NSCLC in the Phase 1/2 ARROW clinical study.

Dr. Yi-long Wu, Guangdong Province People’s Hospital said, “Prior to the approval of GAVRETO, treatment options for patients with RET fusion-positive non-small cell lung cancer were limited. GAVRETO, a once-daily treatment option, is expected to become the standard of care for patients with RET fusion-positive NSCLC in China. I am very glad that the therapy has been approved and is starting to benefit patients.”

In March 2021, the National Medical Products Administration of China approved GAVRETO (pralsetinib) capsules for the treatment of adult patients with locally advanced or metastatic RET fusion-positive NSCLC after platinum-based chemotherapy. GAVRETO was granted approval from the U.S. Food and Drug Administration in September 2020 for the treatment of adults with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, and was soon introduced in Hainan Boao Lecheng International Medical Tourism Pilot Zone in China under a pilot program, making it the first new drug to be launched in Boao Lecheng within the same month after it was approved in the U.S.

To accelerate the commercialization of GAVRETO in China, CStone has signed strategic partnerships with Sinopharm Group, Shanghai MediTrust Health Technology, and Beijing Yuanxin Technology Group, with the goal of improving the accessibility and affordability of GAVRETO as well as supporting patient compliance.

Ms. Shirley Zhao, General Manager of CStone Greater China, said, “GAVRETO is our first innovative precision therapy to benefit patients with RET fusion-positive NSCLC in China. We will work with our partners to further improve the accessibility and affordability of GAVRETO. Meanwhile, we will continue to explore the potential of GAVRETO in additional indications. In April 2021, the NMPA accepted the New Drug Application of selective inhibitor GAVRETO with priority review designation for the treatment of patients with advanced or metastatic RET-altered thyroid cancer. We look forward to potentially benefiting more patients in the future.”

References

[1] Lung cancer: New biological insights and recent therapeutic advances. Ramalingam SS, Owonikoko TK, Khuri FR,CA Cancer J Clin. 2011 Mar-Apr; 61(2):91-112.

[2] Subbiah V, Cote G J. Advances in Targeting RET-Dependent Cancers[J]. Cancer discovery, 2020, 10(4): 498-505

About RET fusion-positive NSCLC

In recent years, China has had rising lung cancer incidence. According to the latest estimates on the global burden of cancer released by International Agency for Research on Cancer (IARC), in 2020, an estimated 0.82 million new lung cancer cases and 0.71 million new lung cancer deaths occurred in China. Among all Chinese cancer patients, lung cancer is the leading cause of cancer-related deaths. NSCLC is the most common type of lung cancer.

In lung cancer, there are a number of somatic mutations, including EGFR, ALK, and ROS1, that can be targeted with approved therapies. RET fusions account for 1-2% of NSCLC patients, the majority of whom are non-smokers.

About GAVRETO (pralsetinib)

GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the NMPA of China for the treatment of adults with locally advanced or metastatic rearranged during transfection (RET) fusion-positive NSCLC after platinum-based chemotherapy.

GAVRETO is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC), and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

GAVRETO is not approved for the treatment of any other indication in China by the NMPA or in the U.S. by the FDA, or for any indication in any other jurisdiction by any other health authority.

GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In preclinical studies, GAVRETO inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2, and JAK2.

Blueprint Medicines and Roche are co-developing GAVRETO globally (excluding Greater China) for the treatment of patients with RET-altered NSCLC, thyroid cancer, and other solid tumors. Blueprint Medicines and Genentech, a member of the Roche Group, are co-commercializing GAVRETO in the U.S., and Roche has exclusive commercialization rights for GAVRETO outside of the U.S. (excluding greater China). The European Medicines Agency validated a marketing authorization application for GAVRETO for the treatment of RET fusion-positive NSCLC, and the review is ongoing. The FDA granted breakthrough therapy designation to GAVRETO for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no alternative treatments.

About CStone

CStone Pharmaceuticals (HKEX: 2616) is a biopharmaceutical company focused on researching, developing, and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 14 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, CStone has received three drug approvals in Greater China, including two in Mainland China and one in Taiwan. CStone's vision is to become globally recognized as a world-renowned biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone, please visit: www.cstonepharma.com

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