Several years ago, an article titled "My Lung Cancer Treatment Journey in the U.S." went viral within cancer communities. The protagonist, 70-year-old Lou Qinyuan, was diagnosed with RET-mutated non-small cell lung cancer. His arduous yet resilient fight against cancer touched the hearts of countless patients and medical professionals.After trying various treatment options, Mr. Lou finally achieved significant disease control through a clinical trial in the U.S. for BLU-667 (pralsetinib), a novel targeted therapy specifically designed for RET-mutated tumors.
However, many more Chinese patients were still desperately searching for this urgently needed lifesaving drug without access.Fortunately, in the same month pralsetinib gained U.S. approval, this precision therapy that had transformed Mr. Lou's prognosis was introduced by CStone Pharmaceuticals. Through a pioneering pilot program, it became the first globally innovative drug to launch simultaneously in China at Boao Lecheng International Medical Tourism Zone that same month, bringing new hope to Chinese patients. Yet CStone's commitment to bringing this breakthrough treatment to China extends further.Within just six months, China's National Medical Products Administration (NMPA) granted conditional approval for Prutixar® (pralatrexate capsules) through its priority review and approval pathway, recognizing it as a Class I new drug. This marks China's first approved selective RET inhibitor.
On June 21, the first day of Pritinib's market launch, over 200 initial prescriptions were issued across more than 100 hospitals including Guangdong Provincial People's Hospital. This means over 200 lung cancer patients will gain extended lives through this new treatment option. Currently, Pritinib is available in nearly 80 pharmacies across nearly 70 cities nationwide.
【Puxihua? Available Nationwide in 80 Pharmacies Across Nearly 70 Cities】
China's First Approved Highly Selective RET Inhibitor Benefits Patients
Instone Pharmaceuticals' precision medicine Puxihua? is an oral, once-daily, potent and highly selective RET inhibitor demonstrating strong antitumor activity. It is also China's first approved selective RET inhibitor.According to the latest clinical data presented at the 2021 American Society of Clinical Oncology Annual Meeting, Puxihua® demonstrated potent antitumor activity with favorable safety and tolerability in RET fusion-positive NSCLC patients, achieving an overall response rate of 62% and a median progression-free survival of 17.1 months.
"Prior to Puxihua's approval, existing treatment options for RET fusion-positive NSCLC patients yielded suboptimal results. Puxihua balances efficacy and safety, delivering significant survival benefits and positioning itself as a potential standard treatment for such patients in China."As the principal investigator for Puxihua's global clinical trials in China, Professor Wu Yilong from Guangdong Provincial People's Hospital, a leading expert in lung cancer, expressed excitement over prescribing the first dose of Puxihua. "Puxihua's oral administration and once-daily dosing significantly improve patient compliance. I am delighted this drug is now benefiting patients and look forward to more individuals gaining from it."
Currently, authoritative clinical guidelines—including the 2020 Chinese Clinical Oncology Society Guidelines for Diagnosis and Treatment of Non-Small Cell Lung Cancer, the 2021 Clinical Practice Guidelines for Molecular Pathology Testing of Non-Small Cell Lung Cancer, and the Chinese Expert Consensus on Clinical Detection of RET Gene Fusion in Non-Small Cell Lung Cancer—all recommend RET gene fusion testing.With the approval of RET inhibitor targeted therapies and advances in detection technology, accurately identifying NSCLC patients harboring RET gene fusions is crucial for enabling earlier diagnosis and treatment.
To expand access to Prutiximab™ for more patients, CStone Pharmaceuticals is broadening its application scope by actively exploring additional indications. Currently, the expanded indication application for Prutiximab™ in advanced or metastatic RET-mutated thyroid cancer has been accepted by the China National Medical Products Administration (NMPA) and designated for priority review. This signals the drug's potential to benefit a wider patient population in the future.
A New Light Shines on Treatment Options for RET-Fusion-Positive Non-Small Cell Lung Cancer
Lung cancer remains China's leading cause of cancer incidence and mortality. Non-small cell lung cancer (NSCLC) accounts for 80%-85% of all lung cancer subtypes, with approximately 1%-2% of NSCLC patients harboring RET fusions.Data indicates that the RET gene, a key oncogene closely linked to cell growth, plays a significant role in the development of multiple tumors. Both RET gene mutations (intragenic alterations) and RET gene fusions (heterologous gene combinations) activate the RET protein. This activated protein then influences tumor proliferation, apoptosis, and invasion through various signaling pathways, thereby affecting tumorigenesis and progression.At that time, although the relationship between the RET gene and tumors was well established, no drugs targeting the RET pathway had yet been approved.
After his diagnosis, Mr. Lou conducted extensive research and actively explored various treatment options. Upon learning that the BLU-667 (pralsetinib) clinical trial specifically targeted patients with RET-mutated tumors, he immediately submitted an application and successfully enrolled in the trial.Six months later, Mr. Lou's tumor had significantly shrunk, symptoms like coughing disappeared, and his cardiopulmonary function largely recovered—marking a major milestone in his treatment. Mr. Lou's journey reflects the current landscape of diagnosing and treating RET fusion-positive non-small cell lung cancer (NSCLC).Although RET fusion-positive NSCLC patients constitute a relatively small proportion of all cancer patients, most are diagnosed at an advanced stage with rapid disease progression, making treatment exceptionally challenging. Previously, treatment options for this patient population were extremely limited and yielded unsatisfactory outcomes. Consequently, patients urgently require more effective and precise novel therapies.When Dr. Lou revealed that his condition was well-controlled through the BLU-667 (pralsetinib) clinical trial, patients and physicians worldwide expressed great excitement and attention. Even more exciting is that this globally leading precision therapy, introduced and developed by CStone Pharmaceuticals, offers a new treatment option for Chinese patients with RET fusion-positive NSCLC. Moving forward, CStone will continue to bring more globally innovative oncology therapies to Chinese patients through its CStone model.
Enhancing Comprehensive Accessibility: Bringing Pritinib Closer to Patients
While breakthroughs in drug development and regulatory approvals bring hope to patients, advancing drug accessibility is crucial for ensuring innovative therapies benefit those in need.To ensure patients can access this globally leading medication as quickly as possible, CStone Pharmaceuticals is accelerating efforts across multiple fronts—supply chain services, drug accessibility, payment accessibility, service accessibility, and medication adherence—by establishing strategic partnerships with leading industry players. This commitment aims to bring the product closer to patients.
Currently, Puxihua has been successfully included in the 2021 Chengdu Huirongbao Designated High-Cost Drug Directory, the upgraded 2021 special drug directory of Weibao Million Medical Insurance, and the 2021 special drug directory of Shuidihui Million Medical Insurance.CStone Pharmaceuticals has also established a green fundraising channel on the Shuidichou platform (fundraising hotline: 400-686-1179) to accelerate fundraising for patients facing extreme financial hardship. We believe these innovative payment methods, including commercial insurance and public welfare insurance, can help alleviate patients' financial burdens to a certain extent.
Additionally, the "Medicine Fund" patient assistance program, covering low-income individuals and those on pre-illness welfare, will launch in July. The patient care platform "Life Watch," initiated by Beijing Life Oasis Service Center, regularly provides public welfare patient education activities and updates on medication accessibility. Follow their official account for more details.
Regarding healthcare services, CStone Pharmaceuticals is actively expanding its nationwide network of hospital-adjacent pharmacies and DTP (Direct-to-Patient) self-operated pharmacies. Collaborations with internet hospitals and other platforms integrate online physician resources, enabling patients to access convenient telemedicine services, comprehensive disease knowledge, and high-quality, full-cycle disease management services—all aimed at enhancing their quality of life.