Suzhou, China, August 15^th, 2024 --- CStone Pharmaceuticals (HKEX: 2616), an innovation-driven biopharmaceutical company focused on the research and development of anti-cancer therapies, today announced the approval of the manufacturing localization registration application for AYVAKIT^® (avapritinib tablets, 100 mg) by the China National Medical Products Administration (NMPA). Following the approval of 300 mg strength earlier in June 2024, the 100 mg strength will further expand the treatment flexibility of AYVAKIT^®. Both strengths are expected to gradually replace the existing imported products, achieving domestic supply in late 2024 or early 2025.

Dr. Jason Yang, CEO, President of R&D and Executive Director of the Board at CStone, said, “The approval of the manufacturing localization application for both 100 mg and 300 mg strengths of AYVAKIT^® will not only better meet the needs of diverse patient populations and provide greater convenience, but also significantly enhance the accessibility and market competitiveness of AYVAKIT^® in China. Additionally, the manufacturing localization application for another of our precision medicines, GAVRETO^® (pralsetinib capsules), was accepted in April of this year and is currently under review by Center for Drug Evaluation, NMPA. We will continue to be committed to delivering high-quality medicines to the patients in China through advanced manufacturing technologies and rigorous production and quality management.”

AYVAKIT was approved by the NMPA of China in March 2021 for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA exon 18 mutation, including the PDGFRA D842V mutation. As the world’s first precision therapy for GIST approved based on a driver gene, AYVAKIT has demonstrated remarkable efficacy in multiple clinical trials. The inclusion of AYVAKIT in the National Reimbursement Drug List (NRDL) has further enhanced its accessibility and affordability. AYVAKIT is recommended in multiple domestic and international guidelines, including the 2023 CSCO Guidelines for Gastrointestinal Stromal Tumor Diagnosis and Treatment, the 2022 Clinical Practice Guidelines for the Pathological Diagnosis of Gastrointestinal Stromal Tumors, the Chinese Guidelines for the Diagnosis and Treatment of Systemic Mastocytosis, the 2023 NCCN Guidelines for Gastrointestinal Stromal Tumors, and the 2023 NCCN Guidelines for Systemic Mastocytosis.

About AYVAKIT^® (avapritinib tablets)

AYVAKIT is a precision therapy approved by the NMPA of China for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT was approved by the Hong Kong Department of Health (DOH), and Taiwan Food and Drug Administration (TFDA) for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.

AYVAKIT is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, adults with advanced systemic mastocytosis (advanced SM), including aggressive SM (ASM), and SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with indolent systemic mastocytosis (ISM). This medicine is approved in Europe (AYVAKYT^®) for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation, adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment.

AYVAKIT was discovered by CStone’s partner Blueprint Medicines. CStone entered into an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of AYVAKIT in the Greater China Region, including mainland China, Hong Kong, Macau and Taiwan.

In July 2024, CStone entered into an agreement with Hengrui to grant the exclusive promotion rights of precision therapy AYVAKIT in mainland China to Hengrui. Except for promotion, CStone retains all rights under its exclusive license agreement with Blueprint Medicines to AYVAKIT in mainland China, including rights to development, registration, manufacturing and distribution, etc.

About CStone

CStone (HKEX: 2616), established in late 2015, is an innovation-driven biopharmaceutical company focused on the research and development of anti-cancer therapies. Dedicated to addressing patient's unmet medical needs in China and globally, the company has made significant strides since its inception. To date, the company has successfully launched four innovative drugs and secured approvals for 15 new drug applications (NDAs) covering 9 indications. The company's pipeline is balanced by 12 promising candidates, featuring potentially first-in-class or best-in-class antibody-drug conjugates (ADCs), multispecific antibodies, immunotherapies and precision medicines. CStone also prides itself on a management team with comprehensive experiences and capabilities that span the entire drug development spectrum, from preclinical and translational research to clinical development, drug manufacturing, business development, and commercialization.

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

IR contact: ir@cstonepharma.com

PR contact: pr@cstonepharma.com

Forward-looking statements

The forward-looking statements made in this article only relate to events or information as of the date when the statements are made in this article. Except as required by law, we undertake no obligation to update or publicly revise any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. All statements in this article are made on the date of publication of this article and may change due to future developments.

Disclaimer: only for communication and scientific use by medical and health professionals.

Trademarks

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation outside of the United States.