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| Therapy Name | Pralsetinib |
| Synonyms | |
| Therapy Description |
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 RET fusion-positive non-small cell lung 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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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
|---|---|---|---|---|
| Pralsetinib | Gavreto | BLU667|BLU-667 | RET Inhibitor 53 | 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 RET fusion-positive non-small cell lung 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 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 fusion | gallbladder cancer | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines as primary (category 2B) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including gallbladder cancer (NCCN.org). | detail... |
| RET D567Y | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET D567Y in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). | 36166639 |
| RET D631Y | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET D631Y in culture (PMID: 36166639). | 36166639 |
| RET E632_L633del | Advanced Solid Tumor | conflicting | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, a cell line expressing RET E632_L633del was less sensitive to Gavreto (pralsetinib) than cells expressing wild-type RET in culture (PMID: 36416226). | 36416226 |
| RET E632_L633del | Advanced Solid Tumor | conflicting | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET E632_L633del in culture (PMID: 36166639). | 36166639 |
| RET M918T | medullary thyroid 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 | medullary thyroid 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 G607C | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET G607C in culture (PMID: 36166639). | 36166639 |
| 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 D567N | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET D567N in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). | 36166639 |
| RET D584N | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Biochemical | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET D584N in culture (PMID: 36166639). | 36166639 |
| RET Y806C RET M918T | medullary thyroid carcinoma | predicted - resistant | Pralsetinib | Case Reports/Case Series | Actionable | In a retrospective analysis, a medullary thyroid carcinoma patient harboring RET M918T progressed on treatment with Gavreto (pralsetinib) and was found to have acquired RET Y806C (PMID: 38127829; NCT03157128, NCT03037385, NCT03780517). | 38127829 |
| RET rearrange | lung non-small cell carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines as a first-line therapy for patients with advanced or metastatic non-small cell lung cancer harboring RET rearrangements (NCCN.org). | detail... |
| 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 M918T | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET M918T in culture (PMID: 36166639). | 36166639 |
| RET L629P RET D631_R635delinsG RET V637R | medullary thyroid 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 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 V804X | medullary thyroid 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... |
| 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 mutant | medullary thyroid 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 | medullary thyroid carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines (category 2B) for patients with medullary thyroid carcinoma harboring RET mutations (NCCN.org). | detail... |
| RET mutant | medullary thyroid 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 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 |
| RET fusion | anaplastic thyroid carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines as neoadjuvant or systemic therapy for patients with anaplastic thyroid carcinoma harboring RET fusions (NCCN.org). | 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 over exp | pulmonary neuroendocrine tumor | predicted - sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited proliferation of a lung neuroendocrine tumor cell line with high RET expression in 3D spheroid culture (Cancer Res (2023) 83 (7_Supplement): 4039). | detail... |
| RET C634X | medullary thyroid 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... |
| HRAS A59T RET A883F RET A883T | medullary thyroid carcinoma | predicted - resistant | Pralsetinib | Case Reports/Case Series | Actionable | In a retrospective analysis, a medullary thyroid carcinoma patient harboring RET A883T progressed on treatment with Gavreto (pralsetinib) and was found to have acquired RET A883F and HRAS A59T (PMID: 38127829; NCT03157128, NCT03037385, NCT03780517). | 38127829 |
| RET fusion | lung non-small cell carcinoma | sensitive | Pralsetinib | Clinical Study | Actionable | In a retrospective study, Gavreto (pralsetinib) treatment resulted in an overall response rate of 55.6%, a disease control rate of 72.2%, a median progression-free survival of 10.7 months, and overall survival of 21.2 months in patients with metastatic non-small cell lung cancer harboring a RET fusion (n=36) including KIF5B-RET (n=13) and CCDC6-RET (n=2) (PMID: 39241296). | 39241296 |
| RET fusion | lung non-small cell carcinoma | sensitive | Pralsetinib | Clinical Study | Actionable | In a retrospective study, Gavreto (pralsetinib) treatment resulted in an objective response rate (ORR) of 61% (39/59, 2 complete responses (CR), 37 partial responses (PR)) and a disease control rate (DCR) of 79.7% in non-small cell lung cancer patients harboring RET fusions, and an intracranial ORR of 66.7% (4/6, 3 CR and 1 PR), an intracranial DCR of 100% in patients with measurable brain metastases (PMID: 36379124). | 36379124 |
| 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 | lung non-small cell carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines as a first-line therapy for patients with metastatic non-small cell lung cancer harboring RET fusions (PMID: 36872130; ESMO.org). | detail... 36872130 |
| 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 C620R | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET C620R in culture (PMID: 36166639). | 36166639 |
| RET C634W | medullary thyroid 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 | follicular thyroid carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines for patients with follicular thyroid carcinoma harboring RET fusions (NCCN.org). | detail... |
| RET D571N | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Biochemical | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET D571N in culture (PMID: 36166639). | 36166639 |
| RET fusion | papillary thyroid 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 | papillary thyroid 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 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 fusion | oncocytic carcinoma of the thyroid | 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 | extrahepatic bile duct carcinoma | sensitive | Pralsetinib | Guideline | Actionable | Gavreto (pralsetinib) is included in guidelines as primary (category 2B) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including extrahepatic cholangiocarcinoma (NCCN.org). | detail... |
| RET V591_G607del RET L1016S | medullary thyroid 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 C634R | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET C634R in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). | 36166639 |
| RET fusion | differentiated high-grade thyroid 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 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 D631_L633delinsE | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Cell culture | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET D631_L633delinsE in culture (PMID: 36166639). | 36166639 |
| RET fusion | medullary thyroid 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 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) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including intrahepatic cholangiocarcinoma (NCCN.org). | detail... |
| RET E574D | Advanced Solid Tumor | sensitive | Pralsetinib | Preclinical - Biochemical | Actionable | In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET E574D in culture (PMID: 36166639). | 36166639 |
| 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 |
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- Use quotes to match on a longer phrase with spaces (i.e. "mtor c1483f")
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
|---|---|---|---|---|---|---|
| NCT03037385 | Phase Ib/II | Pralsetinib | 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 (ARROW) | Completed | USA | NLD | ITA | GBR | FRA | ESP | DEU | BEL | 5 |
| 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 | TUR | SWE | POL | NZL | NOR | NLD | ITA | ISR | GBR | FRA | ESP | DEU | CAN | BRA | BEL | AUS | 13 |
| 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) (AcceleRET-Lung) | Completed | TUR | SWE | POL | NLD | ITA | IRL | GBR | FRA | ESP | DEU | CHE | BRA | BEL | AUS | ARG | 6 |
| 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) | Withdrawn | ESP | 0 |
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