Therapy Detail

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Therapy Name Pralsetinib
Synonyms
Therapy Description

BLU-667 selectively inhibits activated Ret, which may result in decreased tumor growth (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B151).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Pralsetinib Gavreto BLU667|BLU-667 RET Inhibitor 47 Gavreto (pralsetinib) is a small molecule inhibitor that selectively targets activated Ret, which may result in antitumor activity including inhibition of tumor growth and tumor regression (PMID: 29657135). Gavreto (pralsetinib) is FDA approved for use in patients with metastatic MET fusion-positive non-small cell lung cancer, in adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer, and in adult and pediatric patients 12 years of age and older with RET fusion-positive thyroid cancer who are radioactive iodine-refractory (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
RET C634W thyroid gland medullary carcinoma sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and proliferation of a medullary thyroid cancer cell line harboring RET C634W in culture, and inhibited tumor growth in xenograft models (PMID: 29657135). detail... 29657135
RET fusion differentiated thyroid gland carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for adult or pediatric patients 12 years or older with radioactive iodine-refractory, advanced or metastatic differentiated thyroid gland carcinoma harboring RET fusions (PMID: 31549998, PMID: 35491008; ESMO.org). 35491008 31549998 detail...
RET fusion thyroid gland follicular carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for patients with follicular thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET fusion extrahepatic bile duct carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) or subsequent-line therapy (category 2B) for patients with unresectable or metastatic extrahepatic cholangiocarcinoma harboring RET fusions (NCCN.org). detail...
RET fusion thyroid gland cancer sensitive Pralsetinib FDA approved Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 89% (8/9) in patients with advanced or metastatic RET fusion-positive thyroid cancer, with a median duration of response of 14.5 months, and 67% of responding patients continuing treatment (PMID: 34118198; NCT03037385). 34118198 detail...
RET M918T thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 61% of the patients harbored RET M918T (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
RET M918T thyroid gland medullary carcinoma sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I trial, a patient with sporadic medullary thryoid cancer harboring RET M918T demonstrated a partial response, with maximal tumor reduction of 47% following treatment with Gavreto (pralsetinib) (PMID: 29657135; NCT03037385). 29657135
RET M918T Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Erk phosphorylation and growth of transformed cells expressing RET M918T in culture (PMID: 32284345). 32284345
RET D898_E901del Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Erk phosphorylation and growth of transformed cells expressing RET D898_E901del in culture (PMID: 32284345). 32284345
RET fusion colon cancer predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in stable disease in 2 patients with RET fusion-positive colon cancer, with a duration of response of 7.3 and 9.3 months, respectively (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET fusion thyroid gland papillary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with papillary thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET fusion thyroid gland papillary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in an objective response rate of 75% (9/12) and a median duration of response of 14.5 months in patients with RET fusion positive papillary thyroid cancer (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET fusion thyroid gland Hurthle cell carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for patients with thyroid Hurthle cell carcinoma harboring RET fusions (NCCN.org). detail...
RET fusion intrahepatic cholangiocarcinoma sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in a partial response in a patient with RET fusion-positive intrahepatic cholangiocarcinoma, with a duration of response of 7.5 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET fusion intrahepatic cholangiocarcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) or subsequent-line therapy (category 2B) for patients with unresectable or metastatic intrahepatic cholangiocarcinoma harboring RET fusions (NCCN.org). detail...
RET rearrange lung non-small cell carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as a first-line therapy for non-small cell lung cancer patients with RET rearrangements (NCCN.org). detail...
RET C630del Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) treatment inhibited Ret phosphorylation and downstream signaling and resulted in decreased cell viability and colony formation in cells expressing RET C630del in culture (PMID: 35689816). 35689816
RET T930K Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited growth of transformed cells expressing RET T930K in culture (PMID: 32284345). 32284345
RET V591_G607del RET L1016S thyroid gland medullary carcinoma predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a clinical case study, Gavreto (pralsetinib) treatment resulted in a partial response lasting more than 1 year in a patient with medullary thyroid carcinoma harboring RET V591_G607del and germline RET L1016S (PMID: 36053791). 36053791
RET fusion gallbladder cancer sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) or subsequent-line therapy (category 2B) for patients with unresectable or metastatic gallbladder cancer harboring RET fusions (NCCN.org). detail...
RET C634X thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 28% of the patients harbored RET C634X (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
RET fusion thyroid gland medullary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with medullary thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET V804X thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 4% of the patients harbored RET V804X (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
EML4 - RET lung adenocarcinoma sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, a patient-derived lung adenocarcinoma cell line harboring EML4-RET was sensitive to treatment with Gavreto (pralsetinib), demonstrating decreased cell proliferation in culture, and inhibition of tumor growth in a xenograft model (PMID: 33795352). 33795352
RET T930P Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited growth of transformed cells expressing RET T930P in culture (PMID: 32284345). 32284345
RET L629P RET D631_R635delinsG RET V637R thyroid gland medullary carcinoma predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I trial, a patient with medullary thryoid cancer harboring RET D631_R635delinsG, L629P, and V637R demonstrated a partial response, with maximal tumor reduction of 47% at 10 months, following treatment with Gavreto (pralsetinib) (PMID: 29657135; NCT03037385). 29657135
RET fusion pancreatic cancer predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in a partial response in 2 of 2 patients with RET fusion positive pancreatic cancer, with a duration of response of 5.5 and 7.4 months, respectively (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET fusion lung non-small cell carcinoma sensitive Pralsetinib FDA approved - On Companion Diagnostic Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated, resulting in an objective response rate of 61% (53/87) and 70% (19/27) in previously treated and treatment-naive patients with RET fusion-positive non-small cell lung cancer, respectively (PMID: 34118197; NCT03037385). detail... 34118197 detail...
RET fusion Advanced Solid Tumor predicted - sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well tolerated in patients with advanced solid tumors harboring RET fusions, and led to an overall response rate of 57% (13/23, 3 complete and 10 partial responses), a clinical benefit rate of 70% (16/23), a disease control rate of 83% (19/23), a median duration of response of 11.7 months, a median progression-free survival of 7.4 months, and a median overall survival of 13.6 months (PMID: 35962206; NCT03037385). 35962206
RET mutant thyroid gland medullary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for adult or pediatric patients 12 years or older with advanced or metastatic medullary thyroid gland carcinoma harboring RET mutations (PMID: 31549998, PMID: 35491008; ESMO.org). 31549998 detail... 35491008
RET mutant thyroid gland medullary carcinoma sensitive Pralsetinib FDA approved Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated and resulted in an overall response rate (ORR) of 60% (3/55) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations who received prior treatments, ORR was 60% (32/53) in patients with prior therapies and 71% (15/21) in treatment-naive patients (PMID: 34118198; NCT03037385). 34118198 detail...
RET fusion thyroid gland anaplastic carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with anaplastic thyroid carcinoma harboring RET fusions (NCCN.org). detail...

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT04222972 Phase III Carboplatin + Pembrolizumab + Pemetrexed Disodium Carboplatin + Paclitaxel + Pembrolizumab Carboplatin + Nab-paclitaxel + Pembrolizumab Pralsetinib Cisplatin + Gemcitabine Carboplatin + Gemcitabine Cisplatin + Pemetrexed Disodium Cisplatin + Pembrolizumab + Pemetrexed Disodium Carboplatin + Pemetrexed Disodium A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC) Recruiting USA | ITA | FRA | ESP | DEU | CAN | BEL 18
NCT03037385 Phase Ib/II Pralsetinib Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) Active, not recruiting USA | ITA | ESP | DEU 7
NCT04760288 Phase III Cabozantinib Pralsetinib Vandetanib A Study of Pralsetinib Versus Standard of Care (SOC) for Treatment of RET-Mutated Medullary Thyroid Cancer (MTC). (AcceleRET-MTC) Not yet recruiting ESP 0
NCT05170204 Phase III Durvalumab Alectinib Pralsetinib Entrectinib A Study Evaluating the Efficacy and Safety of Multiple Therapies in Cohorts of Participants With Locally Advanced, Unresectable, Stage III Non-Small Cell Lung Cancer (NSCLC) Recruiting USA 7
NCT04302025 Phase II Cobimetinib + Vemurafenib Entrectinib Atezolizumab Alectinib Pralsetinib A Study of Multiple Therapies in Biomarker-Selected Patients With Resectable Stages IB-III Non-Small Cell Lung Cancer Recruiting USA 0


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