Selpercatinib, the First Highly Selective RET Inhibitor, Approved in China for RET-Driven Lung and Thyroid Cancers

Rockville, USA and Suzhou, ChinaOctober 10, 2022PR Newswire -- Innovent Bio (HKEX stock code: 01801), a biopharmaceutical company dedicated to the research, development, production, and commercialization of innovative drugs in major disease areas such as oncology, autoimmune diseases, metabolic disorders, and ophthalmology, is pleased to announce that the highly selective RET inhibitor Selpercatinib (40mg & 80mg capsules) has been approved by the National Medical Products Administration (NMPA) for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) positive for rearranged during transfection (RET) gene fusions, adult and pediatric patients aged 12 years and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) requiring systemic therapy, and adult and pediatric patients aged 12 years and older with advanced or metastatic RET fusion-positive thyroid cancer (TC) requiring systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is applicable).

Selpercatinib was developed by Eli Lilly and Company. In March 2022, Innovent Bio further deepened its strategic cooperation with Eli Lilly and Company in the field of oncology. This included Innovent Bio obtaining exclusive commercial rights to selpercatinib in China from Eli Lilly and Company, taking full responsibility for pricing, importation, marketing, distribution, and sales promotion of selpercatinib. Innovent Bio will leverage its extensive product pipeline in major cancer indications, a mature and comprehensive oncology commercialization platform, as well as widespread commercial coverage across hospitals and pharmacies nationwide in China, aiming to provide Chinese cancer patients with integrated solutions that offer strong combination synergies.

Selpercatinib is a potent, highly selective, oral RET tyrosine kinase inhibitor that received U.S. FDA approval in May 2020, becoming the world's first approved highly selective RET inhibitor.^[1]In November 2021, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) accepted the marketing application for Selpercatinib, which was included in the priority review and approval process, accelerating the approval process of Selpercatinib. This approval in China is based on data from the global study LIBRETTO-001 and the Chinese population data from the LIBRETTO-321 study.

LIBRETTO-001 Study: The Largest Global Phase I/II Clinical Trial Evaluating Selpercatinib for the Treatment of RET-Driven Cancer Patients. The primary efficacy endpoints are confirmed Objective Response Rate (ORR) and median Duration of Response (DoR). The latest findings in the NSCLC population were presented at the 2022 European Lung Cancer Conference (ELCC).^[2], and the latest research results on the thyroid cancer population were presented at the 2022 European Society for Medical Oncology (ESMO) Congress.^[3]。

The study results showed that, for patients with RET fusion-positive locally advanced or metastatic NSCLC, patients with RET-mutant advanced or metastatic MTC, and patients with RET fusion-positive advanced or metastatic TC,Selpercatinib demonstrated effective and durable anti-tumor activity along with favorable safety data.

LIBRETTO-321 Study^[5],[6]Is an open-label, multicenter, Phase II clinical trial aimed at evaluating the efficacy and safety of Selpercatinib in Chinese patients with advanced solid tumors harboring RET alterations. A total of 77 Chinese patients with advanced solid tumors carrying RET gene alterations were enrolled, including 47 patients with RET fusion-positive advanced NSCLC, 29 patients with RET mutation-positive advanced MTC, and 1 patient with RET fusion-positive advanced TC. The full text of this study was published separately in July 2022.^[5](NSCLC Section) and August^[6](MTC/TC section) published in the journal 'Therapeutic Advances in Medical Oncology'.

The study results show,China Data &The global data from LIBRETTO-001 demonstrate a high level of consistency, validating the efficacy and safety of selpercatinib in the Chinese population.

Professor Lu Shun from the Department of Thoracic Surgery, Shanghai Jiao Tong University Affiliated Chest Hospital"RET is a rare target in NSCLC, but rare targets are not 'rare.' Given the large population of NSCLC patients in China, there is a significant absolute number of these patients, and their survival status deserves attention. Selpercatinib has shown remarkable efficacy in treating RET fusion-positive NSCLC patients in the global LIBRETTO-001 Phase I/II clinical trial, with a median PFS of approximately 2 years."^[2]The LIBRETTO-321 study confirmed that selpercatinib significantly improved survival in Chinese patients with advanced RET fusion-positive NSCLC, including those with refractory brain metastases, all of whom experienced clinically meaningful responses when treated with selpercatinib.^[5]. Its approval brings a new treatment option for RET fusion-positive NSCLC patients in China, greatly benefiting Chinese patients."

Professor Cheng Ying from Jilin Province Cancer HospitalThe global incidence of RET fusion in NSCLC patients is currently 1-2%, and the incidence of RET fusion in NSCLC in China is 1.4%.^[7],[8]For a long time, there has been a lack of satisfactory treatment options for patients with advanced NSCLC positive for RET fusion. In recent years, the advent of RET inhibitors has ushered in an era of precision treatment for such patients. The LIBRETTO-321 study shows that Selpercatinib has demonstrated notable ORR in both previously treated and untreated Chinese patient populations. Additionally, this drug has been proven to have anti-tumor activity in the brain in preclinical models.^[5]We believe that the approval of Selpercatinib in China will bring more survival benefits to Chinese patients with RET fusion-positive NSCLC."

Professor Gao Ming from Tianjin People's Hospital"RET gene alterations are important driver genes in thyroid cancer, commonly seen in MTC."^[9]Selpercatinib has high selectivity for the RET target. The large-sample data from the global study LIBRETTO-001 confirmed that selpercatinib has a clinically significant high response rate for advanced MTC with RET mutations and advanced TC positive for RET fusion, while also demonstrating durable effects.^[3],[4]In addition, the LIBRETTO-321 study shows that the results of the Chinese population are highly consistent with global data, further verifying the efficacy and safety of Selpercatinib. Its approval for marketing is undoubtedly another "timely rain" for the vast number of thyroid cancer patients, and another milestone that can change treatment outcomes. In the future, more Chinese thyroid cancer patients will benefit from it."

Dr. Yongjun Liu, President of Innovent Bio"Selpercatinib is the world's first approved RET inhibitor, and we are very pleased to see its potent and sustained relief with good tolerability demonstrated in clinical studies. This approval in mainland China marks another milestone in China’s precision medicine, bringing international-quality treatment options to Chinese patients with RET fusion-positive cancers. For Innovent Bio, the small molecule TKI pipeline now includes another high-value commercial product, further enhancing synergy. We will work closely with Eli Lilly to accelerate the product's market launch, adhering to our original mission of developing affordable, high-quality biologics for the people, bringing new hope to more cancer patients in China."