CDE proposed breakthrough therapy identified: cornerstone pharmaceutical platini, micro-core biosioroni and so on

On December 18, CDE announced three drugs to be identified as breakthrough therapies.
BLU-667 (Plaltini, pralsetinib) Platini is an oral, highly selective and powerful RET single target inhibitor.
is intended to be included in the systematic treatment of patients with advanced or metastatic thyroid myeloma (MTC) who are positive for trans-infected re-platoon (RET) mutations.
A key Phase I/II study code-named ARROW evaluated platinib's efficacy in patients with RET fusion-positive NSCLC, thyroid cancer, and other advanced solid tumors with RET variants.
of the 87 patients who had previously received platinum-containing chemotherapy, platini had ORR of 57% and CR of 5.7%.
of the 27 first-time patients who were not suitable for platinum-containing chemotherapy, the ORR was 70% and the total remission rate was 11%.
RET fusion of positive cancer and RET mutation-positive thyroid myelin-like cancer of tumor cell differentiation and proliferation is highly dependent on the activation of RET protein, which is often referred to as "carcinogenic gene addiction", so RET-positive tumors are highly sensitive to high-selective single-target RET inhibitors.
the incidence of RET gene fusion in NSCLC patients is about 1% to 2%, in thyroid papyroid cancer (about 85% of all thyroid cancer) incidence rate is 10% to 20%.
of the RET gene mutation in thyroid myelin-like cancer is about 60%.
September, Platinib was approved by the FDA to treat adult patients with RET fusion-positive localized late stage or metastasis NSCLC.
December 1, Platini obtained FDA approval for advanced or metastatic RET mutant thyroid myeloid cancer in children and adults over 12 years of age requiring systemic treatment, as well as advanced or metastatic RET fusion positive MTC in children 12 years and older who require systemic treatment and are difficult to treat with radioactive iodine.
Pratini is developed by Blueprint, which owns exclusive clinical development and commercial rights to the drug alone or combined therapy in Greater China.
Theo Ronnie Theo Ronnie capsule is a new class of drugs independently developed by microcore biology, this time to be included in the breakthrough therapy adaptation for single-drug treatment after 2-line system chemotherapy treatment after disease progression or recurrence of small cell lung cancer.
Theroni plays a role in inhibiting tumor angiogenesis (VEGFR1/2/3, PDGFRa/b, c-Kit) mediated tumor cell growth and silk/suline kinase (Aurora B)-mediated cell cycles.
also inhibits Aorora B kinase, which has the potential to reduce tumor tissue genomic instability and inhibit tumor cell metastasis.
Theo Ronnie structure in addition, Theoroni's pharmacy characteristics, target selectivity, effectiveness and off-target effect (off-target effect) are significantly better than the first-generation inhibitors Shonithini and Solafinib, and may have better clinical safety and a wider treatment window.
Phase I Clinical Study (NCT02122809) assessed the maximum to-dosage and initial anti-tumor activity of Theuroni in patients with refractic late-stage solid and lymphoma.
18 subjects were subjected to an incremental dose study of Theoroni 10 to 65 mg/d, one cycle every 28 days, 2 patients with 65 mg dose showed dose-limiting toxicity (grade 3 hypertension), and the most common treatment-related adverse events included fatigue (61.61. 1%), proteinuria (44.4%), blooduria (38.9%), hypothyroidism (38.9%), hyperglycerideemia (33.3%) and hypertension (33.3%).
no full or partial remission was observed, and 12 patients (66.7%) were in stable condition (SD).
according to the Medical Rubik's Cube NextPharma database, Therononi currently intends to develop small cell lung cancer, hepatocellular carcinoma, ovarian cancer, non-Hodgkin's lymphoma and other 4 adaptation certificates, are in phase II clinical research stage.
DZD9008 DZD9008 is a targeted EGFR/HER22 20 exon mutation inhibitor developed by Jiangsu Dizhe Pharmaceuticals, which is intended to incorporate breakthrough therapy adaptation to treat local late stage or metastasis NSCLC with EGFR 20 exon insertion mutations that have received at least one systemic chemotherapy in the past.
8FR exon 20 insertion mutation accounts for about 9% of all EGFR mutation non-small cell lung cancer patients.
patients with EGFR exon 20 insertion mutations in non-small cell lung cancer are generally insensitive to EGFR-TKI treatment and have a worse prognostic period than patients with more common EGFR mutations (exon 19 missing/L858R replacement).
, the world has not yet approved targeted therapies specifically for EGFR exon 20 insertion mutation NSCLC patients, the standard treatment for this type of patient is conventional cytotoxic drug chemotherapy.
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