Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
pralsetinib · 1 trial · 40 indications
MTD was defined as the highest tolerated dose of pralsetinib without causing dose limiting toxicities (DLTs). DLT was defined as any Grade ≥3 adverse event (AE) occurring during Cycle 1 during Phase 1 (dose escalation) that is not clearly caused by something other than pralsetinib. RP2D was defined as the highest dose with acceptable toxicity as determined from dose-escalation phase.
An AE was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.
ORR was defined as the percentage of participants with a confirmed complete response (CR) or partial response (PR) for at least two assessments with at least 28 days apart and no disease progression (PD) in between. Per Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1), CR was defined as the disappearance of all target lesions or any pathological lymph nodes (whether target or non-target) having a reduction in the short axis to \<10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters (SOD) of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline). ORR and its two-sided 95% CI, based on the exact binomial distribution (Clopper-Pearson), were presented.
| Arm | Type | Description |
|---|---|---|
| Phase 1 Dose Escalation | EXPERIMENTAL | Multiple doses of pralsetinib (BLU-667) for oral administration. |
| Phase 2 Dose Expansion | EXPERIMENTAL | Oral dose of pralsetinib (BLU-667) as determined during Dose Escalation. |
| Name | Type | Description |
|---|---|---|
| pralsetinib (BLU-667) | DRUG | pralsetinib (BLU-667) is a potent and selective inhibitor of the RET mutations, fusions, and predicted resistant mutants |
Key Inclusion Criteria: * Diagnosis during dose escalation (Phase 1) - Pathologically documented, definitively diagnosed non-resectable advanced solid tumor. * All participants treated at doses \> 120 mg per day must have MTC, or a RET-altered solid tumor per local assessment of tumor tissue and...
Pralsetinib is an investigational small molecule being studied for the treatment of RET-altered Non Small Cell Lung Cancer. It is also being evaluated in other RET-altered solid tumors, including thyroid cancer, colon cancer, and medullary thyroid cancer. The drug is currently in Phase 1 clinical development.
Pralsetinib targets RET, a receptor tyrosine kinase. It is a highly-selective RET inhibitor, designed to block the activity of RET fusion proteins and mutations that drive tumor growth. This makes it a targeted therapy for cancers with RET alterations, such as RET-altered Non Small Cell Lung Cancer.
Pralsetinib is being developed by Roche Holding AG, which trades under the ticker RHHBY. The drug is a small molecule kinase inhibitor in the -tinib class, and it is currently in Phase 1 clinical trials for RET-altered Non Small Cell Lung Cancer and other solid tumors.
Pralsetinib is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The drug is being studied in a Phase 1/2 trial for RET-altered Non Small Cell Lung Cancer and other advanced solid tumors, with the trial now completed.
Pralsetinib is being studied in clinical trial NCT03037385, a Phase 1/2 study of the highly-selective RET inhibitor pralsetinib (BLU-667) in participants with thyroid cancer, non-small cell lung cancer, and other advanced solid tumors. The trial enrolled 590 participants and has been completed.
Yes, pralsetinib is also known as BLU-667. The clinical trial NCT03037385 refers to the drug as pralsetinib (BLU-667), confirming that both names refer to the same investigational RET inhibitor being developed by Roche for RET-altered cancers.