Presentations & publications | Blueprint Medicines

ASCO 2019: Advancing Cancer Treatment with Precision Therapies

May 31, 2019

At the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting, a key focus will be precision therapy – targeting genetic drivers of cancer with highly selective medicines. By developing medicines for patients most likely to benefit, precision therapy offers...

Presentations and publications

Date	Focus	Journal title/Conference

1584342045 March 16, 2020	Avapritinib - SM	American Academy of Allergy, Asthma & Immunology (AAAAI) Virtual Oral Abstracts, Case Reports and Poster Sessions
PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy Read the full presentation

1575795946 December 08, 2019	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent or Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy Read the full presentation

1573772438 November 14, 2019	Avapritinib - GIST	Connective Tissue Oncology Society (CTOS) Annual Meeting
Clinical Response to Avapritinib by RECIST and Choi Criteria in ≥ 4th Line and PDGFRA Exon 18 Gastrointestinal Stromal Tumors (GIST) Clinical Response to Avapritinib by RECIST and Choi Criteria in ≥ 4^th Line and PDGFRA Exon 18 Gastrointestinal Stromal Tumors (GIST) Read the full presentation

1573702237 November 14, 2019	Avapritinib - GIST	Connective Tissue Oncology Society (CTOS) Annual Meeting
Avapritinib for the Treatment of GIST: Analysis of Efficacy, Safety, and Patient Management Strategies at the Recommended Phase 2 Dose Avapritinib for the Treatment of GIST: Analysis of Efficacy, Safety, and Patient Management Strategies at the Recommended Phase 2 Dose Read the full presentation

1573137683 November 07, 2019	Avapritinib - SM	ISPOR Europe
Psychometric Evaluation of the Advanced Systemic Mastocytosis Symptom Assessment Form (AdvSM-SAF) in patients with Advanced Systemic Mastocytosis Psychometric Evaluation of the Advanced Systemic Mastocytosis Symptom Assessment Form (AdvSM-SAF) in patients with Advanced Systemic Mastocytosis Read the full presentation

1573137287 November 07, 2019	Avapritinib - SM	ISPOR Europe
Psychometric Performance of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) Psychometric Performance of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) Read the full presentation

1569920764 October 01, 2019	Fisogatinib	Cancer Discovery
First-in-Human Phase I Study of Fisogatinib (BLU-554) Validates Aberrant Fibroblast Growth Factor 19 Signaling as a Driver Event in Hepatocellular Carcinoma First-in-Human Phase I Study of Fisogatinib (BLU-554) Validates Aberrant Fibroblast Growth Factor 19 Signaling as a Driver Event in Hepatocellular Carcinoma Read the full article

1569920650 October 01, 2019	Fisogatinib	Cancer Discovery
Acquired On-Target Clinical Resistance Validates FGFR4 as a Driver of Hepatocellular Carcinoma Acquired On-Target Clinical Resistance Validates FGFR4 as a Driver of Hepatocellular Carcinoma Read the full article

1569823542 September 30, 2019	Pralsetinib	European Society for Medical Oncology (ESMO) Annual Meeting
Treatment With Pralsetinib (BLU-667), a Potent and Selective RET Inhibitor, Provides Rapid Clearance of ctDNA in Patients With RET-Altered Non-Small Cell Lung Cancer (NSCLC) and Thyroid Cancer Treatment With Pralsetinib (BLU-667), a Potent and Selective RET Inhibitor, Provides Rapid Clearance of ctDNA in Patients With RET-Altered Non-Small Cell Lung Cancer (NSCLC) and Thyroid Cancer Read the full presentation

1569155420 September 22, 2019	BLU-782	ASBMR 2019 Annual Meeting
A clinical update on BLU-782, an investigational selective ALK2 inhibitor in development for fibrodysplasia ossificans progressiva (FOP) A clinical update on BLU-782, an investigational selective ALK2 inhibitor in development for fibrodysplasia ossificans progressiva (FOP) Read the full presentation

1568023113 September 09, 2019	Pralsetinib	IASLC World Conference on Lung Cancer
Pralsetinib (BLU-667) demonstrates robust activity in RET-fusion-driven intracranial tumor models Pralsetinib (BLU-667) demonstrates robust activity in RET-fusion-driven intracranial tumor models Read the full presentation

1560579358 June 15, 2019	Avapritinib - SM	European Hematology Association (EHA) Annual Meeting
Avapritinib, a Potent and Selective Inhibitor of KIT D816V, Induces Complete and Durable Responses in Patients (pts) with Advanced Systemic Mastocytosis (AdvSM) Avapritinib, a Potent and Selective Inhibitor of KIT D816V, Induces Complete and Durable Responses in Patients (pts) with Advanced Systemic Mastocytosis (AdvSM) Read the full presentation

1559552740 June 03, 2019	Pralsetinib	ASCO 2019
Clinical Activity and Tolerability of BLU-667, a Highly Potent and Selective RET Inhibitor, in Patients with Advanced RET-Fusion+ Non-small Cell Lung Cancer Clinical Activity and Tolerability of BLU-667, a Highly Potent and Selective RET Inhibitor, in Patients with Advanced RET-Fusion+ Non-small Cell Lung Cancer Read the full presentation

1559398538 June 01, 2019	Pralsetinib	ASCO 2019
Activity and Tolerability of BLU-667, a Highly Potent and Selective RET Inhibitor, in Patients with Advanced RET-altered Thyroid Cancers Activity and Tolerability of BLU-667, a Highly Potent and Selective RET Inhibitor, in Patients with Advanced RET-altered Thyroid Cancers Read the full presentation

1559379949 June 01, 2019	Avapritinib - GIST	ASCO 2019
Clinical Activity of Avapritinib in ≥4th Line (4L+) and PDGFRA Exon 18 Gastrointestinal Stromal Tumors (GIST) Clinical Activity of Avapritinib in ≥4th Line (4L+) and PDGFRA Exon 18 Gastrointestinal Stromal Tumors (GIST) Read the full presentation

1553970136 March 30, 2019	Pralsetinib	American Association for Cancer Research (AACR) Annual Meeting
Discovery of BLU-667 for RET-driven cancers Discovery of BLU-667 for RET-driven cancers Read the full presentation

1543789289 December 02, 2018	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Avapritinib, a Potent and Selective Inhibitor of KIT D816V, Improves Symptoms of Advanced Systemic Mastocytosis (AdvSM) (oral presentation) Avapritinib, a Potent and Selective Inhibitor of KIT D816V, Improves Symptoms of Advanced Systemic Mastocytosis (AdvSM) (oral presentation) Read the full presentation

1542320453 November 15, 2018	Avapritinib - GIST	Connective Tissue Oncology Society (CTOS) Annual Meeting
Avapritinib is Highly Active and Well-tolerated in Patients With Advanced GIST Driven by a Diverse Variety of Oncogenic Mutations in KIT and PDGFRA (oral presentation) Avapritinib is Highly Active and Well-tolerated in Patients With Advanced GIST Driven by a Diverse Variety of Oncogenic Mutations in KIT and PDGFRA (oral presentation) Read the full presentation

1540246817 October 22, 2018	Avapritinib - GIST	European Society for Medical Oncology (ESMO) Annual Meeting
Correlation of ctDNA and Response in Patients with PDGFRα D842 GIST Treated with Avapritinib (poster presentation) Correlation of ctDNA and Response in Patients with PDGFRα D842 GIST Treated with Avapritinib (poster presentation) Read the full presentation

1540246775 October 22, 2018	Avapritinib - GIST	European Society for Medical Oncology (ESMO) Annual Meeting
An Open-label, Randomized, Phase 3 Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Metastatic or Unresectable Gastrointestinal Stromal Tumor (poster presentation) An Open-label, Randomized, Phase 3 Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Metastatic or Unresectable Gastrointestinal Stromal Tumor (poster presentation) Read the full presentation

1538864323 October 06, 2018	Pralsetinib	American Thyroid Association (ATA) Annual Meeting
Clinical activity of BLU-667, a highly selective RET inhibitor, in advanced RET-altered thyroid cancers: updated results from the phase 1 ARROW study (oral presentation) Clinical activity of BLU-667, a highly selective RET inhibitor, in advanced RET-altered thyroid cancers: updated results from the phase 1 ARROW study (oral presentation) Read the full presentation

1538432296 October 01, 2018	Avapritinib - GIST	Clinical Cancer Research
Robust activity of avapritinib, potent and highly selective inhibitor of mutated KIT, in patient-derived xenograft models of gastrointestinal stromal tumors Robust activity of avapritinib, potent and highly selective inhibitor of mutated KIT, in patient-derived xenograft models of gastrointestinal stromal tumors Read the full article

1538345823 September 30, 2018	BLU-782	American Society for Bone and Mineral Research (ASBMR) Annual Meeting
BLU-782: A highly selective ALK2 inhibitor, designed specifically to target the cause of fibrodysplasia ossificans progressiva BLU-782: A highly selective ALK2 inhibitor, designed specifically to target the cause of fibrodysplasia ossificans progressiva Read the full presentation

1538000121 September 26, 2018	Pralsetinib	IASLC World Conference on Lung Cancer
Activity of osimertinib and the selective RET inhibitor BLU-667 in an EGFR-mutant patient with acquired RET rearrangement (oral presentation) Activity of osimertinib and the selective RET inhibitor BLU-667 in an EGFR-mutant patient with acquired RET rearrangement (oral presentation) Read the full presentation

1538000089 September 26, 2018	Pralsetinib	Cancer Discovery
Landscape of acquired resistance to osimertinib in EGFR-mutant NSCLC and clinical validation of combined EGFR and RET inhibition with osimertinib and BLU-667 for acquired RET fusion Landscape of acquired resistance to osimertinib in EGFR-mutant NSCLC and clinical validation of combined EGFR and RET inhibition with osimertinib and BLU-667 for acquired RET fusion Read the full article

1529100833 June 15, 2018	Avapritinib - SM	European Hematology Association (EHA) Annual Meeting
Avapritinib (BLU-285), a Selective KIT Inhibitor, is Associated with High Response Rate and Tolerable Safety Profile in Advanced Systemic Mastocytosis: Results of a Phase 1 Study (poster presentation) Avapritinib (BLU-285), a Selective KIT Inhibitor, is Associated with High Response Rate and Tolerable Safety Profile in Advanced Systemic Mastocytosis: Results of a Phase 1 Study (poster presentation) Read the full presentation

1523830394 April 15, 2018	Pralsetinib	American Association for Cancer Research (AACR) Annual Meeting
Highly potent and selective RET inhibitor, BLU-667, achieves proof of concept in ARROW, a phase 1 study of advanced, RET-altered solid tumors (oral presentation) Highly potent and selective RET inhibitor, BLU-667, achieves proof of concept in ARROW, a phase 1 study of advanced, RET-altered solid tumors (oral presentation) Read the full presentation

1523830353 April 15, 2018	Pralsetinib	Cancer Discovery
Precision targeted therapy with BLU-667 for RET-driven cancers Precision targeted therapy with BLU-667 for RET-driven cancers Read the full article

1512943899 December 10, 2017	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Clinical activity in a Phase 1 study of BLU-285, a potent, highly-selective inhibitor of KIT D816V in advanced systemic mastocytosis (plenary oral presentation) Clinical activity in a Phase 1 study of BLU-285, a potent, highly-selective inhibitor of KIT D816V in advanced systemic mastocytosis (plenary oral presentation) Read the full article

1510351812 November 10, 2017	Avapritinib - GIST	Connective Tissue Oncology Society (CTOS) Annual Meeting
Clinical activity of BLU-285, a highly potent and selective KIT/PDGFRα inhibitor designed to treat gastrointestinal stromal tumor (GIST) (oral presentation) Clinical activity of BLU-285, a highly potent and selective KIT/PDGFRα inhibitor designed to treat gastrointestinal stromal tumor (GIST) (oral presentation) Read the full presentation

1509574157 November 01, 2017	Avapritinib	Science Translational Medicine
A precision therapy against cancers driven by KIT/PDGFRA mutations A precision therapy against cancers driven by KIT/PDGFRA mutations Read the full article

1509314866 October 29, 2017	Pralsetinib	Molecular Targets and Cancer Therapeutics (AACR – NCI – EORTC)
BLU-667 is a Potent and Highly Selective RET Inhibitor Being Developed for RET-Driven Cancers BLU-667 is a Potent and Highly Selective RET Inhibitor Being Developed for RET-Driven Cancers Read the full presentation

1505685990 September 17, 2017	Fisogatinib	International Liver Cancer Association (ILCA) Annual Conference
(Clinical activity Of BLU-554, a potent, highly-selective FGFR4 inhibitor in advanced HCC with FGFR4 pathway activation (oral presentation) (Clinical activity Of BLU-554, a potent, highly-selective FGFR4 inhibitor in advanced HCC with FGFR4 pathway activation (oral presentation) Read the full presentation

1505081139 September 10, 2017	Fisogatinib	European Society for Medical Oncology (ESMO) Annual Meeting
Phase 1 Safety and Clinical Activity of BLU-554 in advanced hepatocellular carcinoma (oral presentation) Phase 1 Safety and Clinical Activity of BLU-554 in advanced hepatocellular carcinoma (oral presentation) Read the full presentation

1501452295 July 30, 2017	Pralsetinib	World Congress on Thyroid Cancer
BLU-667 is a potent and highly selective RET inhibitor in development for RET-driven thyroid cancers BLU-667 is a potent and highly selective RET inhibitor in development for RET-driven thyroid cancers Read the full presentation

1496700240 June 05, 2017	Avapritinib - GIST	American Society of Clinical Oncology (ASCO) Annual Meeting
Clinical activity of BLU-285 in advanced gastrointestinal stromal tumor (GIST) (oral presentation) Clinical activity of BLU-285 in advanced gastrointestinal stromal tumor (GIST) (oral presentation) Read the full presentation

1491170553 April 02, 2017	Avapritinib - GIST	American Association for Cancer Research (AACR) Annual Meeting
BLU-285: A potent and highly selective inhibitor designed to target malignancies driven by KIT and PDGFRα mutations (oral presentation) BLU-285: A potent and highly selective inhibitor designed to target malignancies driven by KIT and PDGFRα mutations (oral presentation) Read the full presentation

1491170447 April 02, 2017	Avapritinib - SM	American Association for Cancer Research (AACR) Annual Meeting
BLU-285: A potent and highly selective inhibitor designed to target malignancies driven by KIT and PDGFRα mutations (oral presentation) BLU-285: A potent and highly selective inhibitor designed to target malignancies driven by KIT and PDGFRα mutations (oral presentation) Read the full presentation

1480888795 December 04, 2016	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Preliminary Safety and Activity in a Phase 1 study of BLU-285, a Potent, Highly-Selective Inhibitor of KIT D816V in Advanced Systemic Mastocytosis (SM) (oral presentation) Preliminary Safety and Activity in a Phase 1 study of BLU-285, a Potent, Highly-Selective Inhibitor of KIT D816V in Advanced Systemic Mastocytosis (SM) (oral presentation) Read the full presentation

1480629552 December 01, 2016	Avapritinib - GIST	EORTC – NCI – AACR Molecular Targets and Cancer Therapeutics Symposium
Preliminary safety and activity in a first-in-human Phase 1 study of BLU-285, a potent, highly selective inhibitor of KIT and PDGFRα activation loop mutants in advanced gastrointestinal stromal tumor (GIST) (oral presentation) Preliminary safety and activity in a first-in-human Phase 1 study of BLU-285, a potent, highly selective inhibitor of KIT and PDGFRα activation loop mutants in advanced gastrointestinal stromal tumor (GIST) (oral presentation) Read the full presentation

1480456639 November 29, 2016	Fisogatinib	Molecular Targets and Cancer Therapeutics (EORTC – NCI – AACR)
First-in-human study of BLU-554, a potent, highly selective FGFR4 inhibitor designed for hepatocellular carcinoma (HCC) with FGFR4 pathway activation (poster presentation) First-in-human study of BLU-554, a potent, highly selective FGFR4 inhibitor designed for hepatocellular carcinoma (HCC) with FGFR4 pathway activation (poster presentation) Read the full presentation

1461016563 April 18, 2016	Pralsetinib	American Association for Cancer Research (AACR) Annual Meeting
The development of Potent and Selective RET inhibitors The development of Potent and Selective RET inhibitors Read the full presentation

1430516121 May 01, 2015	Research	Journal of Clinical Investigation
Targeting cancer with kinase inhibitors Targeting cancer with kinase inhibitors Read the full presentation

1426541525 March 16, 2015	Fisogatinib	Cancer Discovery
First Selective Small Molecule Inhibitor of FGFR4 for the Treatment of Hepatocellular Carcinomas with an Activated FGFR4 Signaling Pathway First Selective Small Molecule Inhibitor of FGFR4 for the Treatment of Hepatocellular Carcinomas with an Activated FGFR4 Signaling Pathway Read the full article

1411162202 September 19, 2014	Research	Nature Communications
Genomic analyses of gynaecologic carcinosarcomas reveal frequent mutations in chromatin remodelling genes Genomic analyses of gynaecologic carcinosarcomas reveal frequent mutations in chromatin remodelling genes Read the full article

1410384400 September 10, 2014	Research	Nature Communications
The landscape of kinase fusions in cancer The landscape of kinase fusions in cancer Read the full article