CStone Pharmaceuticals first announced the data of Chinese patients with RET-mutated medullary thyroid cancer in the Pugethua®ARROW study in the form of an oral report at the 90th American Thyroid Association Annual Meeting in 2021

The main efficacy data show that Puyuhua® has shown superior and long-lasting anti-tumor activity in Chinese patients with advanced or metastatic transfection rearrangement (RET) mutant medullary thyroid carcinoma (MTC), confirmed objective response rate (ORR) ) Reached 73.
1%, 9-month duration of response (DOR) was 100%.
In April 2021, Pugeta® is used for the treatment of patients with advanced or metastatic RET mutant MTC and advanced or metastatic RET fusion-positive thyroid cancer.
The disease has been accepted by the China National Medical Products Administration (NMPA) and has been included in the priority review

Suzhou, China, October 2, 2021/PRNewswire/ - CStone Pharmaceuticals (Hong Kong Stock Exchange code: 2616), a leading biopharmaceutical company focusing on research, development and commercialization of innovative tumor immunotherapies and precision therapeutic drugs, It was announced today that at the 90th Annual Meeting of the American Thyroid Association 2021 (The 90th Annual Meeting of the American Thyroid Association 2021), the selective RET inhibitor Pugeta® (Platinib Capsules) will be in the global phase I/II In the ARROW trial, the registration study data for the treatment of patients with advanced or metastatic RET-mutant MTC in China has published detailed data in the form of a blockbuster oral report (Late Breaking Oral)
.

Research data shows that Pugeta® has shown extensive and long-lasting anti-tumor activity in Chinese patients with advanced or metastatic RET-mutant MTC who require systemic treatment, which is consistent with previous reports of ARROW in the global population

Research field: Thyroid cancer Date: October 2, 2021 00:00-1:00 (Beijing time) Report format: Clinical oral report Abstract Session title: The efficacy of selective RET inhibitor pratinib in the treatment of advanced RET mutant MTC And safety report number: Late Breaking Oral 3 Principal Investigator: Professor Gao Ming, Dean of Tianjin People’s Hospital Speaker: Professor Zheng Xiangqian, Tianjin Medical University Cancer Hospital

As of the data cut-off date (April 12, 2021), a total of 28 RET mutation-positive MTC patients from the China Research Center were enrolled in the global ARROW study Chinese MTC registration bridging cohort, and received a starting dose of 400 mg ( Once a day) treatment
.

Tumor remission was evaluated by the blind independent center review (BICR) using the "Solid Tumor Response Evaluation Criteria" (RECIST) version 1.

Effectiveness: Pugeta® has achieved superior and long-lasting anti-tumor activity in Chinese patients with advanced or metastatic RET-mutant MTC in need of systemic treatment

The confirmed ORR in 26 patients with RET mutations was 73.
1%, including 3 cases of complete remission and 16 cases of partial remission, and the disease control rate (DCR) was 84.
6%
.

Regardless of the RET mutation genotype, there is remission

Safety: Pugeta® is well tolerated and safety is controllable

Pugeta® is well tolerated, and is overall safe and controllable in Chinese patients, and no new safety signals have been found
.

Professor Gao Ming, the principal investigator of the ARROW study and the dean of Tianjin People’s Hospital, said: “In recent years, the incidence of thyroid cancer has continued to increase
.

There are limited clinical treatments for patients with RET-mutated MTC, and precise targeted treatment options are urgently needed

Dr.
Yang Jianxin, Chief Medical Officer of CStone Pharmaceuticals, said: “We are very pleased to see that Pujihua® has achieved excellent efficacy and safety for Chinese MTC patients with RET mutations
.

Currently Pujihua® targets this population and is positive for RET fusion.

Pujihua® is the first selective RET inhibitor approved for marketing in China.
It is used to treat locally advanced or metastatic NSCLC patients who have previously received platinum-containing chemotherapy and have positive RET gene fusion.
It was developed by CStone Pharmaceuticals partner Blueprint.
Developed by Medicines
.

CStone Pharmaceuticals and Blueprint Medicines have reached an exclusive cooperation and licensing agreement to obtain the exclusive development and commercialization rights of Phuket Hua ® in the Greater China region, including Mainland China, Hong Kong, Macau and Taiwan

About ARROW research

The ARROW study is a global clinical study aimed at evaluating the safety, tolerability and effectiveness of Pugeta® in patients with RET fusion-positive NSCLC, RET mutant medullary thyroid carcinoma and other advanced solid tumors with RET fusion
.

In August 2021, "The Lancet Diabetes and Endocrinology" published the global patient data of RET mutant MTC in the ARROW study
.

As of the data deadline of May 22, 2020, Pugeta® has shown potent and long-lasting anti-tumor activity in patients with RET-mutated thyroid cancer

About thyroid cancer

Thyroid cancer is the most common endocrine malignant tumor, and its incidence has increased significantly in recent years
.

According to the latest global cancer burden data released by the International Agency for Research on Cancer (IARC) of the World Health Organization (IARC), China has approximately 220,000 new cases of thyroid cancer in 2020, of which approximately 170,000 new cases of female cases[1]

Thyroid cancer is clinically divided into multiple subtypes such as papillary carcinoma, follicular carcinoma, undifferentiated carcinoma and medullary carcinoma.
Different types of thyroid cancer are different according to their tumor characteristics, treatment methods and prognosis
.

RET fusion and activating mutations are key disease drivers in many cancer types, including NSCLC and many types of thyroid cancer
.
About 10-20% of patients with papillary thyroid cancer (the most common thyroid cancer) carry RET fusion, and about 90% of patients with advanced medullary thyroid cancer (about 2-5% of thyroid cancer) carry RET mutations
.
There is currently no effective approved standard treatment for patients with RET mutant MTC in China
.

About Pugeta® (Platinib Capsules)

Pugeta® is an oral, once-a-day, potent and highly selective RET inhibitor.
It has been approved by the China National Medical Products Administration for the treatment of transfection rearrangement (RET) genes that have previously received platinum-containing chemotherapy Adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with positive fusion
.

Pugeta® targets advanced or metastatic RET-mutated medullary thyroid cancer (MTC) that requires systemic treatment, and advanced or metastatic RET fusion-positive thyroid cancer that requires systemic treatment and is refractory to radioiodine (if radioactive iodine is appropriate) The application for new indications of the company has also been accepted by the NMPA in April 2021 and has been included in the priority review
.

The US Food and Drug Administration approved it to be marketed under the trade name GAVRETO™.
The three indications are: for the treatment of adult patients with metastatic RET fusion-positive NSCLC confirmed by the FDA-approved test method, and systemic treatment is required.
Adults with advanced or metastatic RET-mutated medullary thyroid cancer and children 12 years and older, as well as adults with advanced or metastatic RET fusion-positive thyroid cancer and 12 years and older who require systemic treatment and are refractory to radioiodine (if applicable) Child patient
.
These indications are approved under the accelerated approval pathway based on ORR and DOR data
.
Continued approval for these indications may depend on the verification and description of clinical benefits in confirmatory trials
.

Phuket Hua® has not been approved for other indications in China and the United States
.

Pugeta® is designed to selectively and effectively target oncogenic RET mutations, including secondary RET mutations that may lead to treatment resistance
.
In pre-clinical studies, Pugeta® inhibits RET at a lower concentration than other drug-related kinases, including VEGFR2, FGFR2 and JAK2
.

Globally, the clinical development of Pugeta® for the treatment of patients with RET fusion-positive NSCLC, various types of thyroid cancer and other solid tumors is ongoing
.
In September 2021, the European Medicines Agency's Human Medicines Committee recommended approval of the marketing authorization application for Pugeta® for the treatment of RET fusion-positive NSCLC
.
The FDA granted Pugeta® Breakthrough Therapy Designation for the treatment of RET fusion-positive NSCLC that has progressed after platinum chemotherapy, and RET mutation-positive medullary thyroid cancer that requires systemic treatment and has no alternative therapy
.

About CStone Pharmaceuticals

CStone Pharmaceuticals (Hong Kong Stock Exchange code: 2616) is a biopharmaceutical company that focuses on research, development and commercialization of innovative tumor immunotherapy and precision treatment drugs to meet the ardent medical needs of cancer patients in China and around the world
.
Established at the end of 2015, CStone Pharmaceuticals has assembled a world-class management team with extensive experience in new drug development, clinical research and commercial operations
.
With tumor immunotherapy combination therapy as the core, the company has established a rich product pipeline consisting of 15 tumor drug candidates
.
At present, CStone Pharmaceuticals has obtained three new drug marketing approvals worldwide, two new drug marketing approvals in Mainland China, and one new drug marketing approval in Taiwan
.
Many late-stage drug candidates are in critical clinical trials or registration stages
.
CStone's vision is to become a world-renowned biopharmaceutical company, leading the way to overcome cancer
.

For more information about CStone Pharmaceuticals, please visit:
.

Forward-looking statement

The forward-looking statements made in this article only relate to events or information as of the date when the statement is made in this article
.
Except as required by law, we have no obligation to update or publicly revise any forward-looking statements and unexpected events after the date of forward-looking statements, regardless of whether new information, future events or other circumstances appear
.
Please read this article carefully and understand that our actual future results or performance may differ materially from expectations
.
All statements in this article are made on the date of publication of this article and may change due to future developments
.

trademark

Blueprint Medicines, GAVRETO and related trademarks are owned by Blueprint Medicines Corporation
.

[1] https://gco.
iarc.
fr/today/data/factsheets/populations/160-china-fact-sheets.
pdf

Source: CStone Pharmaceuticals