Presentations & publications | Blueprint Medicines

The importance of Rare Disease Day

February 28, 2023 by Georg Pirmin Meyer (he/him), Senior Vice President, Head of International

Rare Disease Day is a very important reminder of the struggles faced by those living with rare conditions across the world, including their family and loved ones. In my early years as a physician, I witnessed first-hand the impact rare...

Date	Focus	Journal title/Conference

1677423731 February 26, 2023	Avapritinib - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Avapritinib Improved Skin Findings In Patients With Indolent Systemic Mastocytosis (ISM) in the Registrational, Double-Blind, Placebo-Controlled PIONEER Study Read the full presentation

1677423727 February 26, 2023	Avapritinib - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Efficacy and Safety of Avapritinib in Indolent Systemic Mastocytosis (ISM): Results from the Double-Blind Placebo-Controlled PIONEER Study Read the full presentation

1677398570 February 26, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Development of Scalable, Electronic Health Record (EHR)-Based Screening for Undiagnosed Systemic Mastocytosis: PREDICT-SM Read the full presentation

1677225833 February 24, 2023	Avapritinib - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Avapritinib Improved Symptoms and Quality of Life in Patients with Indolent Systemic Mastocytosis in the PIONEER Study Read the full presentation

1677225776 February 24, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Symptomatology and Diagnostic Journey of Patients Diagnosed with Systemic Mastocytosis in The US Oncology Network Read the full presentation

1677225770 February 24, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Disease Worsening and Progression in Patients with Indolent Systemic Mastocytosis: A US Population-Level Analysis Using Health Claims-Based Dataset Read the full presentation

1677225747 February 24, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
PROSPECTOR: A Global, Prospective Study to Determine the Prevalence of the KIT D816V Mutation in Peripheral Blood From Patients With Evidence of Systemic Mast Cell Activation Read the full presentation

1677225730 February 24, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Real-world chart pull data on the clinical presentation and diagnosis of indolent systemic mastocytosis Read the full presentation

1677225722 February 24, 2023	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Development of a Clinical Scoring System to Identify Conditions of Mast Cell Proliferation in a Community Setting Read the full presentation

1670785551 December 11, 2022	Research - SM	American Society of Hematology (ASH) Annual Meeting
Patterns of Disease Progression in Patients with Systemic Mastocytosis: A U.S. Population-Level Analysis Using Health Claims-Based Dataset Read the full presentation

1670785529 December 11, 2022	BLU-263	American Society of Hematology (ASH) Annual Meeting
AZURE: A Phase 1/2 Study of BLU-263 as Monotherapy and in Combination with Azacitidine in Patients with Advanced Systemic Mastocytosis Read the full presentation

1670780114 December 11, 2022	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Avapritinib as First-Line Therapy in Patients with Advanced Systemic Mastocytosis: Efficacy and Safety from the PATHFINDER Clinical Study Read the full presentation

1670697336 December 10, 2022	Research - SM	American Society of Hematology (ASH) Annual Meeting
NGS Testing Practices and Molecular Profile Landscape of the KIT Gene in Systemic Mastocytosis: Real-World Insights from Selected European Countries Read the full presentation

1670697327 December 10, 2022	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
An Updated Analysis on Safety and Efficacy of Avapritinib in Patients with Advanced Systemic Mastocytosis from the EXPLORER Clinical Study: Long-term Efficacy and Safety Read the full presentation

1670573152 December 09, 2022	BLU-222	San Antonio Breast Cancer Symposium
CDK2 inhibition with BLU-222 in combination with ribociclib demonstrates robust antitumor activity in pre-clinical models of CDK4/6 inhibitor-naïve and -resistant HR+/HER2– breast cancer Read the full presentation

1666781274 October 26, 2022	BLU-701 & BLU-945	Molecular Targets and Cancer Therapeutics (AACR – NCI – EORTC)
BLU-945 or BLU-701 as single agents versus their combination with osimertinib in EGFR L858R-driven tumor models Read the full presentation

1662978722 September 12, 2022	BLU-222	European Society of Medical Oncology (ESMO) Congress
A first-in-human phase 1/2 study of BLU-222, a potent, selective CDK2 inhibitor in patients with CCNE1-amplified or CDK4/6 inhibitor-resistant advanced solid tumors Read the full presentation

1662951923 September 12, 2022	Pralsetinib	European Society of Medical Oncology (ESMO) Congress
Updated efficacy and safety data from the phase I/II ARROW study of pralsetinib in patients (pts) with advanced RET fusion+ non-small cell lung cancer (NSCLC) Read the full presentation

1662865517 September 11, 2022	Pralsetinib	European Society of Medical Oncology (ESMO) Congress
Updated ARROW data: pralsetinib in patients with advanced or metastatic RET-altered thyroid cancer Read the full presentation

1661266906 August 23, 2022	Research - SM	Cancer
Perceptions of Patient Disease Burden and Management Approaches in Systemic Mastocytosis: Results of the TouchStone Healthcare Provider Survey Read the full article

1661266754 August 23, 2022	Research - SM	Cancer
Patient-reported Outcomes Among Patients with Systemic Mastocytosis in Routine Clinical Practice: Results of the TouchStone SM Patient Survey Read the full article

1660403055 August 13, 2022	Pralsetinib	Annals of Oncology
Safety and efficacy of pralsetinib in RET fusion–positive non-small cell lung cancer including as first-line therapy: Update from the ARROW trial Read the full presentation

1659939924 August 08, 2022	Research - NSCLC	IASLC World Conference on Lung Cancer
Real-World Landscape of EGFR C797X Mutation as a Resistance Mechanism to Osimertinib in NSCLC Read the full presentation

1657892710 July 15, 2022	BLU-945	Journal of Medicinal Chemistry
Discovery of BLU-945, a Reversible, Potent, and Wild-Type-Sparing Next-Generation EGFR Mutant Inhibitor for Treatment-Resistant Non-Small-Cell Lung Cancer Read the full article

1657015894 July 05, 2022	Avapritinib - SM	Leukemia
Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis Read the full article

1656854125 July 03, 2022	Avapritinib - SM	European Academy of Allergy and Clinical Immunology (EAACI) Congress
Effect of avapritinib on skin disease burden in patients with advanced systemic mastocytosis (AdvSM): A novel, artificial intelligence (AI)-based technology Read the full presentation

1654830142 June 10, 2022	Avapritinib - SM	European Hematology Association (EHA) Congress
Overall Survival in Patients with Systemic Mastocytosis with Associated Hematologic Neoplasm Treated with Avapritinib versus Best Available Therapy Read the full presentation

1654830138 June 10, 2022	Avapritinib - SM	European Hematology Association (EHA) Congress
Duration of Treatment and Reduction in Serum Tryptase Levels in Patients with Advanced Systemic Mastocytosis Treated with Avapritinib versus Best Available Therapy Read the full presentation

1654830120 June 10, 2022	Avapritinib - SM	European Hematology Association (EHA) Congress
Overall Survival in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib Versus Midostaurin or Cladribine Read the full presentation

1654830108 June 10, 2022	Avapritinib - SM	European Hematology Association (EHA) Congress
Responses to Avapritinib in Patients With Advanced Systemic Mastocytosis: Histopathologic Analyses From EXPLORER and PATHFINDER Clinical Studies Read the full presentation

1654830080 June 10, 2022	BLU-263	European Hematology Association (EHA) Congress
HARBOR: A Phase 2/3 Study of BLU-263 in Patients With Indolent Systemic Mastocytosis and Monoclonal Mast Cell Activation Syndrome Read the full presentation

1654525003 June 06, 2022	Pralsetinib	American Society of Clinical Oncology (ASCO) Annual Meeting
Pralsetinib in patients with advanced or metastatic RET-altered thyroid cancer: updated data from the ARROW trial Read the full presentation

1654506117 June 06, 2022	BLU-701	American Society of Clinical Oncology (ASCO) Annual Meeting
A phase 1/2 study of the highly selective EGFR inhibitor, BLU-701, in patients with EGFR-mutant non-small cell lung cancer (NSCLC) Read the full presentation

1654506098 June 06, 2022	BLU-945	American Society of Clinical Oncology (ASCO) Annual Meeting
A phase 1/2 study of BLU-945 in patients with common activating EGFR-mutant non-small cell lung cancer (NSCLC) (SYMPHONY trial-in-progress) Read the full presentation

1654506077 June 06, 2022	BLU-451	American Society of Clinical Oncology (ASCO) Annual Meeting
A phase 1/2 study of BLU-451, a central nervous system (CNS) penetrant, small molecule inhibitor of EGFR, in incurable advanced cancers with EGFR exon 20 insertion (ex20ins) mutations Read the full presentation

1654506070 June 06, 2022	Pralsetinib	American Society of Clinical Oncology (ASCO) Annual Meeting
AcceleRET Lung: a phase 3 study of first-line pralsetinib in patients with RET fusion–positive advanced/metastatic NSCLC Read the full presentation

1653130260 May 21, 2022	Avapritinib - SM	American Initiative in Mast Cell Diseases (AIM) Conference
Overall Survival, Duration of Treatment, and Reduction in Serum Tryptase Levels in Patients with Advanced Systemic Mastocytosis Treated with Avapritinib versus Best Available Therapy Read the full presentation

1649422974 April 08, 2022	BLU-945	American Association for Cancer Research (AACR) Annual Meeting
Emerging evidence of activity of BLU-945 in patients with advanced EGFR-mutant NSCLC utilizing circulating tumor DNA in the phase 1/2 SYMPHONY study Read the full presentation

1649422912 April 08, 2022	BLU-701 & BLU-945	American Association for Cancer Research (AACR) Annual Meeting
Antitumor activity of BLU-945 and BLU-701 as single agents and in combination in EGFR L858R-driven models of NSCLC Read the full presentation

1649422898 April 08, 2022	BLU-451	American Association for Cancer Research (AACR) Annual Meeting
LNG-451 (BLU-451), a potent inhibitor of EGFR exon 20 insertion mutations with high CNS exposure Read the full presentation

1649422883 April 08, 2022	BLU-222	American Association for Cancer Research (AACR) Annual Meeting
BLU-222, an investigational, potent, and selective CDK2 inhibitor, demonstrated robust antitumor activity in CCNE1-amplified ovarian cancer models Read the full presentation

1649422872 April 08, 2022	Research	American Association for Cancer Research (AACR) Annual Meeting
Efficacy of a highly potent and selective KIT V654A inhibitor for treatment of imatinib-resistant GIST Read the full presentation

1649422870 April 08, 2022	BLU-451	American Association for Cancer Research (AACR) Annual Meeting
LNG-451 (BLU-451) is a potent, CNS-penetrant, wild-type EGFR sparing inhibitor of EGFR exon 20 insertion mutations Read the full presentation

1646052340 February 28, 2022	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Comorbidity and Disability in Medicare Beneficiaries Newly Diagnosed with Non-Advanced Systemic Mastocytosis Read the full presentation

1646052312 February 28, 2022	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Utility of a Validated Disease-Specific Measure to Assess Symptomology in Patients with Indolent Systemic Mastocytosis (ISM) Read the full presentation

1646052311 February 28, 2022	BLU-263	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
A Phase 2/3 Study of BLU-263 in Patients with Indolent Systemic Mastocytosis or Monoclonal Mast Cell Activation Syndrome (TiP) Read the full presentation

1645879535 February 26, 2022	Research - SM	American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting
Disease Symptomology, Quality of Life (QoL), and Employment Status Among Patients with Systemic Mastocytosis (SM) Read the full presentation

1643360826 January 28, 2022	BLU-701 & BLU-945	British Thoracic Oncology Group (BTOG) Annual Conference
BLU-701 tumour suppression and intracranial activity as a single agent and in combination with BLU-945 in models of non-small cell lung cancer driven by EGFR mutations Read the full presentation

1639299900 December 12, 2021	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Efficacy of Avapritinib in Patients With Advanced Systemic Mastocytosis: Hematologic and Bone Marrow Responses From the Phase 2 Open-Label, Single-Arm, PATHFINDER Study Read the full presentation

1639238702 December 11, 2021	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Effective Control of Advanced Systemic Mastocytosis with Avapritinib: Mutational Analysis from the EXPLORER Clinical Study Read the full presentation

1638806695 December 06, 2021	Avapritinib - SM	Nature Medicine
Efficacy and Safety of Avapritinib in Advanced Systemic Mastocytosis: Interim Analysis of the Phase 2 PATHFINDER trial Read the full article

1638806644 December 06, 2021	Avapritinib - SM	Nature Medicine
Safety and Efficacy of Avapritinib in Advanced Systemic Mastocytosis: the Phase 1 EXPLORER trial Read the full article

1637350218 November 19, 2021	Avapritinib - GIST	2021 Society for Neuro-Oncology Annual Meeting and Education Day
A phase 1/2 study of avapritinib in pediatric patients with solid tumors dependent on KITor PDGFRA signaling Read the full presentation

1625972401 July 11, 2021	Avapritinib - SM	European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Annual Congress 2021
Avapritinib improves overall symptoms, skin lesions and quality of life in patients with advanced systemic mastocytosis in the PATHFINDER study Read the full presentation

1623402340 June 11, 2021	Avapritinib - SM	European Hematology Association (EHA) Virtual Congress
Efficacy and safety of avapritinib in patients with advanced systemic mastocytosis: Interim results from the open-label, single-arm, phase 2 PATHFINDER study Read the full presentation

1623316840 June 10, 2021	Avapritinib	2021 Society for Neuro-Oncology (SNO) Pediatric Conference
A phase 1/2, single-arm study to evaluate the safety, pharmacokinetics, and antitumor activity of avapritinib in pediatric patients with solid tumors dependent on KIT or PDGFRA signaling Read the full presentation

1623229664 June 09, 2021	Pralsetinib	The Lancet Oncology
Pralsetinib for RET fusion-positive non-small-cell lung cancer (ARROW): a multi-cohort, open-label, phase 1/2 study Read the full article

1623229642 June 09, 2021	Pralsetinib	The Lancet Diabetes and Endocrinology
Pralsetinib for patients with advanced or metastatic RET-altered thyroid cancer (ARROW): a multi-cohort, open-label, registrational, phase 1/2 study Read the full article

1622797336 June 04, 2021	Pralsetinib	American Society for Clinical Oncology (ASCO) Annual Meeting
Safety and efficacy of pralsetinib in patients with advanced RET fusion-positive non-small cell lung cancer: update from the ARROW trial Read the full presentation

1622797320 June 04, 2021	Pralsetinib	American Society of Clinical Oncology (ASCO) Annual Meeting
Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion-positive solid tumors: update from the ARROW trial Read the full presentation

1618150109 April 11, 2021	Avapritinib - SM	American Association for Cancer Research (AACR) Annual Meeting
PATHFINDER: Interim analysis of avapritinib in patients with Advanced Systemic Mastocytosis (AdvSM) Read the full presentation

1618043459 April 10, 2021	BLU-222	American Association for Cancer Research (AACR) Annual Meeting
Development of a selective CDK2-E inhibitor in CCNE-aberrant cancers Read the full presentation

1618043457 April 10, 2021	BLU-852	American Association for Cancer Research (AACR) Annual Meeting
MAP4K1 inhibition enhances immune cell activation and anti-tumor immunity in preclinical tumor models Read the full presentation

1618043450 April 10, 2021	BLU-945	American Association for Cancer Research (AACR) Annual Meeting
BLU-945, a fourth-generation, potent and highly selective epidermal growth factor receptor tyrosine kinase inhibitor with intracranial activity, demonstrates robust in vivo anti-tumor activity in models of osimertinib-resistant non-small cell lung cancer Read the full presentation

1618043435 April 10, 2021	BLU-701	American Association for Cancer Research (AACR) Annual Meeting
BLU-701 is a highly potent, brain-penetrant and WT-sparing next-generation EGFR TKI for the treatment of sensitizing (ex19del, L858R) and C797S resistance mutations in metastatic NSCLC Read the full presentation

1618043429 April 10, 2021	BLU-263	American Association for Cancer Research (AACR) Annual Meeting
Safety and pharmacokinetics of BLU-263, a next-generation KIT inhibitor, in normal healthy volunteers Read the full presentation

1618043402 April 10, 2021	Avapritinib - SM	American Association for Cancer Research (AACR) Annual Meeting
Changes in mast cell numbers and phenotype in patients with indolent systemic mastocytosis treated with avapritinib Read the full presentation

1610790107 January 16, 2021	Avapritinib - GIST	European Journal of Cancer
Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: Long-term efficacy and safety data from the NAVIGATOR phase I trial Read the full article

1607335543 December 07, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
PIONEER Part 2: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib in Indolent Systemic Mastocytosis Read the full presentation

1607335517 December 07, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Perceptions of Patient Disease Burden and Management Approaches of Systemic Mastocytosis (SM) By Healthcare Providers (HCPs): Results from the TouchStone SM Survey Read the full presentation

1607335504 December 07, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Increased Detection of KIT D816V Mutation in Peripheral Blood Samples from Patients with Indolent Systemic Mastocytosis (ISM) in the Phase 2 PIONEER Study Using a High Sensitivity Droplet Digital (dd) PCR Assay Compared with Next Generation Sequencing (NGS) Read the full presentation

1607260818 December 06, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Pure Pathologic Response Is Associated with Improved Overall Survival in Patients with Advanced Systemic Mastocytosis Receiving Avapritinib in the Phase I EXPLORER Study Read the full presentation

1607162755 December 05, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Patient Reported Outcomes Among Systemic Mastocytosis (SM) Patients in Routine Clinical Practice: Results from the TouchStone Survey Read the full presentation

1607162751 December 05, 2020	Avapritinib - SM	American Society of Hematology (ASH) Annual Meeting
Results from PIONEER: A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Avapritinib in Patients with Indolent Systemic Mastocytosis Read the full presentation

1605104268 November 11, 2020	Avapritinib - GIST	Connective Tissue Oncology Society (CTOS) Annual Meeting
Avapritinib vs. Regorafenib in Patients With Locally Advanced Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST): Efficacy and Safety Data From Phase 3 VOYAGER Study Read the full presentation

1602924933 October 17, 2020	Pralsetinib	IASLC North America Conference on Lung Cancer
Analysis of Resistance Mechanisms to Pralsetinib (BLU-667) in Patients with RET Fusion-Positive Non-Small Cell Lung Cancer (NSCLC) from the ARROW Study Read the full presentation

1600591101 September 20, 2020	Pralsetinib	European Society for Medical Oncology (ESMO) Virtual Congress
Results from the registrational phase 1/2 ARROW trial of pralsetinib (BLU-667) in patients with advanced RET mutation-positive medullary thyroid cancer Read the full presentation

1600419622 September 18, 2020	Avapritinib - GIST	European Society for Medical Oncology (ESMO) Virtual Congress
Long-term efficacy, tolerability and overall survival in patients with unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumor treated with avapritinib: NAVIGATOR phase 1 trial update Read the full presentation

1600333219 September 17, 2020	Avapritinib - GIST	European Society for Medical Oncology (ESMO) Virtual Congress
Clinical Efficacy Comparison of Avapritinib With Other Tyrosine Kinase Inhibitors (TKIs) in Gastrointestinal Stromal Tumors (GIST) With PDGFRA D842V Mutation: A Retrospective Analysis of Clinical Trial and Real-World Data Read the full presentation

1600330034 September 17, 2020	BLU-945	European Society for Medical Oncology (ESMO) Virtual Congress
BLU-945, a highly potent and selective 4^th-generation EGFR TKI for the treatment of EGFR+/T790M/C797S resistant NSCLC Read the full presentation

1593456441 June 29, 2020	Avapritinib - GIST	The Lancet Oncology
Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial Read the full article

1591929312 June 12, 2020	Avapritinib - SM	Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress
Results from PIONEER: a randomized, double-blind, placebo-controlled, Phase 2 study of avapritinib in patients with indolent systemic mastocytosis (ISM) Read the full presentation

1591929310 June 12, 2020	Avapritinib - SM	Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress
Avapritinib induces responses in patients with advanced systemic mastocytosis (AdvSM), regardless of prior midostaurin therapy Read the full presentation

1591434304 June 06, 2020	Avapritinib - SM	European Academy of Allergy and Clinical Immunology (EAACI) 2020 Digital Congress
Avapritinib reduces cutaneous symptoms and mast cell burden in patients with indolent systemic mastocytosis in the PIONEER study Read the full presentation

1590912102 May 31, 2020	Pralsetinib	American Society of Clinical Oncology (ASCO) Virtual Scientific Program
Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RET fusion+ solid tumors Read the full presentation

1590739456 May 29, 2020	Pralsetinib	American Society of Clinical Oncology (ASCO) Virtual Scientific Program
AcceleRET Lung: A Phase 3 Study of First-Line Pralsetinib in Patients with RET Fusion+ Advanced/Metastatic Non-Small-Cell Lung Cancer (NSCLC) Read the full presentation

1590739434 May 29, 2020	Pralsetinib	American Society of Clinical Oncology (ASCO) Virtual Scientific Program
Registrational Dataset from the Phase 1/2 ARROW Trial of Pralsetinib (BLU-667) in Patients with Advanced RET Fusion+ Non-Small Cell Lung Cancer (NSCLC) Read the full presentation

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 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 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 ≥ 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 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 Read the full presentation

1573137287 November 07, 2019	Avapritinib - SM	ISPOR Europe
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 Read the full article

1569920650 October 01, 2019	Fisogatinib	Cancer Discovery
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 Read the full presentation

1569155420 September 22, 2019	BLU-782	American Society for Bone and Mineral Research (ASMBR) Annual Meeting
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 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) Read the full presentation

1559552740 June 03, 2019	Pralsetinib	American Society of Clinical Oncology (ASCO) Annual Meeting
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	American Society of Clinical Oncology (ASCO) Annual Meeting
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	American Society of Clinical Oncology (ASCO) Annual Meeting
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 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) 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) 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) 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) 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) 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 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 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) 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 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) 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) Read the full presentation

1523830353 April 15, 2018	Pralsetinib	Cancer Discovery
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) 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) Read the full presentation

1509574157 November 01, 2017	Avapritinib	Science Translational Medicine
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 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) 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) 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 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) 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) 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) 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) 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) 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) 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 Read the full presentation

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

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 Read the full article

1411162202 September 19, 2014	Research	Nature Communications
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 Read the full article

The importance of Rare Disease Day

Presentations and publications