2020ASCO, Keystone Pharmaceuticals and Blueprint Medicines announce the latest research data from Pralsetinib

May 30, 2020, Blueprint Medicines, a partner of Keystone Pharmaceuticals (Suzhou) Co., Ltd("Keystone Pharmaceuticals," HKEx Code: 2616), released the latest data from its ongoing RESEARCH on the STUDY of the targeted cancer drug pralsetinib, a research study in which pralsetinib was used to treat RET fusion-positive non-small cell lung cancer (NSCLC), thyroid cancer, and other physical tumor patientsPreliminary data from the registered study showed strong and persistent clinical activity in RET fusion-positive NSCLC patients, with median remission duration (DOR) not being achievedOther results have shown that pralsetinib has extensive clinical anti-tumor activity for other RET fusion-positive tumors, including thyroid cancerPralsetinib's overall tolerance is good, and its safety results are consistent with the results of previous studies, with no new safety signals observedThe results will be presented at the 2020 Annual Meeting of the American Society of Clinical Oncology (ASCO20)Blueprint Medicines also announced that the new drug marketing application (NDA) and marketing authorization (MAA) for the treatment of late-localized or metastatic RET fusion-positive NSCLC have been accepted by the U.SFood and Drug Administration (FDA) and the European Drug Administration (EMA), respectivelyCurrently, pralsetinib has been granted priority review by the FDA, which has set a date for action on the Prescription Drug User Costs Act as November 23, 2020Blueprint Medicines plans to submit to the FDA in June 2020 a new drug market application for the treatment of late-stage RET mutations and RET fusion-positive thyroid cancer within the framework of the FDA's Real-Time Oncology Review Pilot Programclinical efficacy data
the report's data from a remission-assessable analysis set, including 116 Patients with NSCLC who received a daily (QD) pralsetinib starting dose of 400 mg (including 80 Patients with NSCLC who had previously received platinum-based chemotherapy, 26 patients with untreated NSCLC), 11 patients with RET fusion thyroid cancer, and 12 other RET-positive patientsThe results of tumor remission assessment were confirmed by the independent center blind evaluation according to the solid tumor evaluation standard (RECIST) version 1.1RET Fusion-positive NSCLCdata as of November 18, 2019, pralsetinib showed consistent and strong clinical anti-tumor activity in RET fusion-positive NSCLC patients, regardless of whether the patient had been treated, regardless of the type of RET fusion companion or whether the central nervous system (CNS) the objective remission rate (ORR) treated with pralsetinib was 61% (95% CI: 50-72%) among 80 patients who had previously received platinum-containing chemotherapy Partial remission (PR) efficacy assessment soutcomes in two patients at the data cut-off were in a pending state and subsequently confirmed Five percent of patients achieved confirmed complete remission (CR) and 14 percent had a complete subsidy of the target lesions tumor in 26 patients who did not receive systemic treatment, the objective remission rate of Pralsetinib was 73% (95% CI: 52-88%), and the proportion of patients with complete remission was 12% in all 116 patients, regardless of the previous treatment, did not reach the median DOR (95% CI: 11 months, the upper limit was not reached), the DOR reached 6 months of 86%. Overall, as of the data cut-off, 74% of patients with confirmed remission, including all CR patients, were still being treated Pralsetinib showed strong and persistent intracranial anti-tumor activity in 9 patients with assessable brain metastatic lesions at baselines All patients experienced a reduction in CNS metastasis, with 33% of patients achieving complete intracranial remission. There were no CNS progression events in patients with REmission of CNS. The median CNS DOR has not yet been achieved, and the duration of treatment for patients with assessable brain metastasis is currently up to 12 months As of the data deadline, no new CNS metastases had been found in patients with no previous CNS transfer other RET fusion-positive tumors
as of February 13, 2020, Pralsetinib showed significant clinical activity in a range of other RET fusion-positive tumors In 11 RET fusion-positive thyroid cancer patients (10 had received systemic treatment), the ORR confirmed by the center was 91% (95% CI: 59-100%) and the disease control rate was 100% (95% CI: 72-100%) Overall, as of the data, 70 percent of the patients in remission were still receiving treatment, which is currently lasting up to 22 months In 12 other RET fusion-positive tumor patients who had previously received systemic treatment, the researchers assessed an ORR of 50% (95% CI:21?u201279), of which 1 case was PR pending confirmation Varying degrees of relief were observed in all assessable patients with pancreatic cancer (n s 3) and bile duct cancer (n s 2), and these types of tumors generally had a poor prognosis safety data as of the previously published data, as of November 18, 2019, a total of 354 patients participated in the ARROW clinical trial and received Pralsetinib treatment at a starting dose of 400 mg QD Pralsetinib's overall security results are consistent with previously reported data results Pralsetinib showed good tolerance in all tumor strains, with most treatment-related adverse events (AE) level 1 or 2 the most common treatment-related adverse events reported by researchers include elevated athazole (AST), anemia, elevated alanine transaminase (ALT), constipation, hypertension, and neutrophil reduction The researchers reported level 3 or above treatment-related adverse events (up to 5%) including high blood pressure, neutropenomic cell reduction, and anemia Only 4% of patients terminated Pralsetinib because of adverse events associated with treatment pralsetinib Pralsetinib is an oral (once a day), highly efficient and highly selective targeted cancer-causing RET variant of the drug in the study Blueprint Medicines is undergoing clinical development of pralsetinib to treat patients with reT variants of non-small cell lung cancer, thyroid cancer and other solid tumors The FDA has granted pralsetinib breakthrough therapy to treat PATIENTs with RET fusion non-small cell lung cancer that progresses after platinum chemotherapy, and RET-positive thyroid myelin cancer who requires systematic treatment and has no alternative treatment Pralsetinib was designed by blueprint Medicines' research team based on its proprietary compound library In preclinical studies, pralsetinib consistently showed sub-nanomole levels for the most common RET gene fusion, activation mutations, and prediction of drug-resistant mutations Among them, the choice of PRalsetinib to RET is 80 times higher than that of VEGFR2 In addition, the selectivity of pralsetinib to RET was significantly improved compared to approved multikinase inhibitors By inhibiting primary and secondary mutations, pralsetinib is expected to overcome and prevent the occurrence of clinical drug resistance This therapy is expected to achieve long-lasting clinical remission and good safety in patients with different RET variants Keystone Pharmaceuticals has entered into an exclusive partnership and licensing agreement with Blueprint Pharmaceuticals, which has obtained exclusive rights to develop and commercialize a number of drugs, including pralsetinib, in Greater China Blueprint Medicines retains its right to develop and commercialize pralsetinib in other parts of the world