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617.844.8205.
PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease. 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA CLINICAL ACTIVITY AND SAFETY OF THE RET INHIBITOR Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion‒positive solid tumors: update from the ARROW trial Vivek Subbiah,1 Philippe A. Cassier, 2 Salvatore Siena,3 Guzman Alonso, 4 Luis Paz-Ares,5 Pilar Garrido,6 Ernest Nadal,7 Giuseppe Curigliano,8 Jacqueline Vuky,9 Gilberto Lopes,10 Greg Kalemkerian,11 Daniel W. Bowles,12 Mahesh Seetharam,13 Jianhua Chang CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare OVERVIEW | BLUEPRINT MEDICINES CORP. Our approach combines world-leading expertise in protein kinases with sound execution and an intense focus on discovery. Investor Relations. Investor contact Kristin Hodous Senior Manager, Investor Relations ir@blueprintmedicines.com 617.714.6674 Media contact Andrew Law Director, Corporate Communications media@blueprintmedicines.com617.844.8205.
PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease. 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA CLINICAL ACTIVITY AND SAFETY OF THE RET INHIBITOR Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion‒positive solid tumors: update from the ARROW trial Vivek Subbiah,1 Philippe A. Cassier, 2 Salvatore Siena,3 Guzman Alonso, 4 Luis Paz-Ares,5 Pilar Garrido,6 Ernest Nadal,7 Giuseppe Curigliano,8 Jacqueline Vuky,9 Gilberto Lopes,10 Greg Kalemkerian,11 Daniel W. Bowles,12 Mahesh Seetharam,13 Jianhua Chang CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. OVERVIEW | BLUEPRINT MEDICINES CORP. Our approach combines world-leading expertise in protein kinases with sound execution and an intense focus on discovery. Investor Relations. Investor contact Kristin Hodous Senior Manager, Investor Relations ir@blueprintmedicines.com 617.714.6674 Media contact Andrew Law Director, Corporate Communications media@blueprintmedicines.com617.844.8205.
PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease.; 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan.; 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRAD842V mutations.
PRESENTATIONS & PUBLICATIONS 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. CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLUEPRINT MEDICINES PRESENTS FOUNDATIONAL PRECLINICAL DATA CAMBRIDGE, Mass., Sept. 17, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced preclinical proof-of-concept data for BLU-945, an investigational precision therapy for patients with epidermal growth factor receptor-mutated (EGFRm) non-small cell STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. BLUEPRINT MEDICINES SUBMITS SUPPLEMENTAL NEW DRUG Blueprint Medicines requested priority review for this application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine whether the sNDA is complete and acceptable for filing. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) BLUEPRINT MEDICINES' HIGHLY SELECTIVE RET INHIBITOR BLU The new results support Blueprint Medicines' plans to submit an initial New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for BLU-667 for the treatment of patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020, and an NDA to the FDA for the treatment of patients with RET-mutant MTC previously treated REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase AVAPRITINIB FOR THE TREATMENT OF GIST: 3258000 OBJECTIVES AND STUDY DESIGN This post-hoc analysis of the safety and tolerability of avapritinib at the recommended starting dose of 300 mgQD included data
AVAPRITINIB, A POTENT AND SELECTIVE INHIBITOR OF KIT D816V Biochemical binding by DiscoverRX at 3uM KIT D816V biochemical IC 50 avapritinib* imatinib* masitinib# midostaurin* ripretinib# 0.27 nM 8150 nM >1000 nM 2.9 nM 2.6 nM Avapritinib potently and selectively targets KIT D816V Kinome illustrations reproduced courtesy of Cell Signaling Technology, Inc. (CSTI) HIGHLY POTENT AND SELECTIVE RET INHIBITOR, BLU -667 5 RET is a rare driver of multiple, diverse tumor types 1. Drilon A et al. Nat Rev Clin Oncol. 2018;15:151-67 2.Kato S, et al. Clin Cancer Res 2017;23:1988-1997. CLINICAL ACTIVITY OF BLU-667, A HIGHLY SELECTIVE RET I have the following financial relationships to disclose: • Research support: Sanofi-Genzyme • Consultant: Blueprint Medicines Corporation • Advisory board: Loxo Oncology 2 Disclosures BLU-667 is an investigational agent discovered and currently in development by Blueprint Medicines Corporation (Blueprint Medicines) BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE Meet the Blue Crew Andrew Kim, International Launch Lead and General Manager, Nordics. As Blueprint Medicines’ International Launch Lead and General Manager, Nordics, my role is to strategically plan and lead outreach to clinical investigators, healthcare providers and laboratories outside the United States to support the company’s portfolio of clinical-stage programs. CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE Meet the Blue Crew Andrew Kim, International Launch Lead and General Manager, Nordics. As Blueprint Medicines’ International Launch Lead and General Manager, Nordics, my role is to strategically plan and lead outreach to clinical investigators, healthcare providers and laboratories outside the United States to support the company’s portfolio of clinical-stage programs. CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase BLUEPRINT MEDICINES' HIGHLY SELECTIVE RET INHIBITOR BLU The new results support Blueprint Medicines' plans to submit an initial New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for BLU-667 for the treatment of patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020, and an NDA to the FDA for the treatment of patients with RET-mutant MTC previously treated AVAPRITINIB FOR THE TREATMENT OF GIST: 3258000 OBJECTIVES AND STUDY DESIGN This post-hoc analysis of the safety and tolerability of avapritinib at the recommended starting dose of 300 mgQD included data
AVAPRITINIB, A POTENT AND SELECTIVE INHIBITOR OF KIT D816V Biochemical binding by DiscoverRX at 3uM KIT D816V biochemical IC 50 avapritinib* imatinib* masitinib# midostaurin* ripretinib# 0.27 nM 8150 nM >1000 nM 2.9 nM 2.6 nM Avapritinib potently and selectively targets KIT D816V Kinome illustrations reproduced courtesy of Cell Signaling Technology, Inc. (CSTI) HIGHLY POTENT AND SELECTIVE RET INHIBITOR, BLU -667 5 RET is a rare driver of multiple, diverse tumor types 1. Drilon A et al. Nat Rev Clin Oncol. 2018;15:151-67 2.Kato S, et al. Clin Cancer Res 2017;23:1988-1997. CLINICAL ACTIVITY OF BLU-667, A HIGHLY SELECTIVE RET I have the following financial relationships to disclose: • Research support: Sanofi-Genzyme • Consultant: Blueprint Medicines Corporation • Advisory board: Loxo Oncology 2 Disclosures BLU-667 is an investigational agent discovered and currently in development by Blueprint Medicines Corporation (Blueprint Medicines) BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced AVAPRITINIB, A POTENT AND SELECTIVE INHIBITOR OF KIT D816V Biochemical binding by DiscoverRX at 3uM KIT D816V biochemical IC 50 avapritinib* imatinib* masitinib# midostaurin* ripretinib# 0.27 nM 8150 nM >1000 nM 2.9 nM 2.6 nM Avapritinib potently and selectively targets KIT D816V Kinome illustrations reproduced courtesy of Cell Signaling Technology, Inc. (CSTI) BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
AVAPRITINIB REDUCES CUTANEOUS SYMPTOMS AND MAST CELL • Currently no approved therapies effectively reduce the burden of disease in indolent SM, including the skin lesions2 Systemic mastocytosis is a clonal mast cell neoplasm driven by KIT D816V • KIT D816V mutation drives mast cell hyperactivation and accumulation throughout various organs1 • This leads to debilitating skin, gastrointestinal, neurocognitive and systemic symptoms2 REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. DISCOVERY AND DEVELOPMENT OF BLU-554: A POTENT, HIGHLY Discovery and development of BLU-554: a potent, highly selective covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4) in development for the targeted treatment of advanced AVAPRITINIB, A POTENT AND SELECTIVE INHIBITOR OF KIT D816V Biochemical binding by DiscoverRX at 3uM KIT D816V biochemical IC 50 avapritinib* imatinib* masitinib# midostaurin* ripretinib# 0.27 nM 8150 nM >1000 nM 2.9 nM 2.6 nM Avapritinib potently and selectively targets KIT D816V Kinome illustrations reproduced courtesy of Cell Signaling Technology, Inc. (CSTI) CLINICAL ACTIVITY AND SAFETY OF THE RET INHIBITOR Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion‒positive solid tumors: update from the ARROW trial Vivek Subbiah,1 Philippe A. Cassier, 2 Salvatore Siena,3 Guzman Alonso, 4 Luis Paz-Ares,5 Pilar Garrido,6 Ernest Nadal,7 Giuseppe Curigliano,8 Jacqueline Vuky,9 Gilberto Lopes,10 Greg Kalemkerian,11 Daniel W. Bowles,12 Mahesh Seetharam,13 Jianhua Chang BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) BLUEPRINT MEDICINES ANNOUNCES FDA APPROVAL OF GAVRETO CAMBRIDGE, Mass., Dec. 1, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that the U.S. Food and Drug Administration (FDA) has approved GAVRETO™ (pralsetinib) for the treatment of patients with RET-altered thyroid cancers. REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase CLINICAL RESPONSE TO AVAPRITINIB BY RECIST AND CHOI Analysis of avapritinib starting dose 300/400 mg QD in ≥4th line (4L+) and PDGFRA exon 18 mutated GIST aEnrollment criteria specified that patients were required to have received only ≥2 prior lines of TKI therapy (ie, analysis population of 3L+), observed enrollment 5 reflected a more heavily pretreated population (ie, 4L+). bMutational analysis was performed locally and confirmed HIGHLY POTENT AND SELECTIVE RET INHIBITOR, BLU -667 5 RET is a rare driver of multiple, diverse tumor types 1. Drilon A et al. Nat Rev Clin Oncol. 2018;15:151-67 2.Kato S, et al. Clin Cancer Res 2017;23:1988-1997. CLINICAL ACTIVITY OF BLU-667, A HIGHLY SELECTIVE RET I have the following financial relationships to disclose: • Research support: Sanofi-Genzyme • Consultant: Blueprint Medicines Corporation • Advisory board: Loxo Oncology 2 Disclosures BLU-667 is an investigational agent discovered and currently in development by Blueprint Medicines Corporation (Blueprint Medicines) BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare OVERVIEW | BLUEPRINT MEDICINES CORP. Our approach combines world-leading expertise in protein kinases with sound execution and an intense focus on discovery. Investor Relations. Investor contact Kristin Hodous Senior Manager, Investor Relations ir@blueprintmedicines.com 617.714.6674 Media contact Andrew Law Director, Corporate Communications media@blueprintmedicines.com617.844.8205.
PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease. 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster PIONEER PART 2: A RANDOMIZED, DOUBLE-BLIND, PLACEBO Key eligibility criteria 6 aThe ISM-SAF TSS is composed of 11 individual 24-hour recall patient-reported symptoms (score 0–110 total) within the gastrointestinal, skin, and neurologic domains plus bone pain and fatigue, using a 14-day moving average. ECOG PS, Eastern Cooperative Oncology Group performance status; ISM-SAF, Indolent Systemic Mastocytosis-Symptom Assessment Form; BLU-285: A POTENT AND HIGHLY SELECTIVE INHIBITOR DESIGNED 7 BLU-285 is a potent, highly selective inhibitor of KIT and PDGFR activation loop mutants Compound PDGFR D842V IC 50 nM KIT D816V IC 50 nM KIT V560G/D816V IC 50 nM BLU-285 0.24 0.27 0.10 imatinib 759 81506145
ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. BLUEPRINT MEDICINESPATIENTSMEDICAL PROFESSIONALSINVESTORS & MEDIAABOUTSCIENCEPIPELINE 4 pending. marketing. applications. 6 clinical-stage. therapeutic. candidates. 8 research. programs. Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare OVERVIEW | BLUEPRINT MEDICINES CORP. Our approach combines world-leading expertise in protein kinases with sound execution and an intense focus on discovery. Investor Relations. Investor contact Kristin Hodous Senior Manager, Investor Relations ir@blueprintmedicines.com 617.714.6674 Media contact Andrew Law Director, Corporate Communications media@blueprintmedicines.com617.844.8205.
PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease. 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA CLINICALLY INDICATED. WITHHOLD, REDUCE DOSE, OR 4 . Pharmacology (12.3)]. Continue treatment until disease progression or until unacceptable toxicity. If a dose of GAVRETO is missed, it can be taken as soon as possible on the same day. BLU-945, A HIGHLY POTENT AND SELECTIVE 4TH-GENERATION EGFR 0 50 100 150 200 250 300 350 400 450 500 1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 Vehicle BID (n=6) Osimertinib, 25mg/kg QD (n=7) BLU-945, 100mg/kg BID (n=6) CLINICAL ACTIVITY OF THE RET INHIBITOR PRALSETINIB (BLU Clinical activity of the RET inhibitor pralsetinib (BLU-667) in patients with RETfusion+ solid tumors Vivek Subbiah1, Mimi I Hu1, Justin F. Gainor2, Aaron Scott Mansfield3, GuzmanAlonso4, Matthew H Taylor5, Viola Weijia Zhu6, Pilar Garrido López7, AlessioAmatu8, Robert C Doebele9, Philippe Alexandre Cassier10, Bhumsuk Keam11, Martin H. Schuler12, Hui Zhang13, Corinne Clifford13, MichaelPalmer13,
BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster PIONEER PART 2: A RANDOMIZED, DOUBLE-BLIND, PLACEBO Key eligibility criteria 6 aThe ISM-SAF TSS is composed of 11 individual 24-hour recall patient-reported symptoms (score 0–110 total) within the gastrointestinal, skin, and neurologic domains plus bone pain and fatigue, using a 14-day moving average. ECOG PS, Eastern Cooperative Oncology Group performance status; ISM-SAF, Indolent Systemic Mastocytosis-Symptom Assessment Form; BLU-285: A POTENT AND HIGHLY SELECTIVE INHIBITOR DESIGNED 7 BLU-285 is a potent, highly selective inhibitor of KIT and PDGFR activation loop mutants Compound PDGFR D842V IC 50 nM KIT D816V IC 50 nM KIT V560G/D816V IC 50 nM BLU-285 0.24 0.27 0.10 imatinib 759 81506145
ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. ABOUT | BLUEPRINT MEDICINES Striving to improvehuman health. We are working to advance a deep pipeline of precision therapies, designed to allow patients to live longer, healthier lives. Our approach combines our leading expertise in protein kinases with sound execution and an intense focus on discovery. We maintain a diverse and growing portfolio of researchprograms
CLINICAL ACTIVITY AND SAFETY OF THE RET INHIBITOR Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion‒positive solid tumors: update from the ARROW trial Vivek Subbiah,1 Philippe A. Cassier, 2 Salvatore Siena,3 Guzman Alonso, 4 Luis Paz-Ares,5 Pilar Garrido,6 Ernest Nadal,7 Giuseppe Curigliano,8 Jacqueline Vuky,9 Gilberto Lopes,10 Greg Kalemkerian,11 Daniel W. Bowles,12 Mahesh Seetharam,13 Jianhua Chang PIPELINE | BLUEPRINT MEDICINES 1 Unresectable or metastatic disease.; 2 CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan.; 3 Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRAD842V mutations.
CAREERS | BLUEPRINT MEDICINES Careers | Blueprint Medicines. Don’t wait for your next career: step forward and claim it. Our culture of transparency, curiosity and diversity empowers us to draw upon a wide range of backgrounds and perspectives to build a conscientious team dynamic. Ideas come from every member of the Blue Crew, from the C-suite to new hires. BLU-945, A FOURTH-GENERATION, POTENT AND HIGHLY SELECTIVE Blueprint-Medicines-AACR-2021-BLU-945-Lung-Cancer-Poster STAYING ONE STEP AHEAD OF DISEASE 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetiniband fisogatinib in
BLU-701 IS A HIGHLY POTENT, BRAIN-PENETRANT AND WT-SPARING 0 500 1000 1500 2000 0 7 14 21 28 35 42 49 56 63 Days after the start of treatment (mm 3), Last dose Vehicle PO QD BLU-701 1 mg/kg PO QD BLU-701 3 mg/kg PO QD REGISTRATIONAL DATASET FROM THE PHASE 1/2 ARROW TRIAL OF • The advent of targeted therapies for molecularly defined subtypes has revolutionized treatment of NSCLC1 • Oncogenic fusions in the proto-oncogene RET, which encodes a receptor tyrosine kinase, are present in 1%–2% of NSCLC2–5 • Pralsetinib is an investigational, highly potent, oral, selective RET kinase ACTIVITY AND TOLERABILITY OF BLU-667, A HIGHLY POTENT AND 0 2 4 6 8 10 12 14 16 Study Month aData for response-evaluable patients enrolled by 14 Nov 2018.Response-evaluable population includes patients with measurable disease and ≥1 evaluable post-treatment disease assessment. BLU-667 IS A POTENT AND HIGHLY SELECTIVE RET INHIBITOR IN BLU-667 is a potent and highly selective RET inhibitor in development for RET-driven thyroid cancers Rami Rahal, PhD Blueprint MedicinesJuly 30, 2017
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PRECISION THAT MOVES Our uniquely targeted, scalable approach empowers the rapid design and development of new precision therapies as we aim to stay one stepahead of disease.
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A BLUEPRINT FOR A HEALTHIER TOMORROW We design and develop precision therapies, with the goal of transforming patient care. These include multiple programs for genomically defined cancers, rare diseases and cancer immunotherapy.Our pipeline
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Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
AVAPRITINIB
DEVELOPMENT PROGRAMS * Advanced gastrointestinal stromal tumors * Advanced systemic mastocytosis * Non-advanced systemic mastocytosisLearn more
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
PRALSETINIB
DEVELOPMENT PROGRAMS * Advanced RET fusion-positive non-small cell lung cancer * Advanced RET-altered thyroid cancers * Other RET-altered solid tumorsLearn more
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
FISOGATINIB
DEVELOPMENT PROGRAM
* Advanced hepatocellular carcinomaLearn more
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
BLU-263
DEVELOPMENT PROGRAM
* Non-advanced systemic mastocytosisLearn more
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
BLU-782
DEVELOPMENT PROGRAM
* Fibrodysplasia ossificans progressivaLearn more
BLU-782 is an investigational ALK2 inhibitor being developed for the treatment of FOP. In October 2019, we entered into an exclusive, worldwide license agreement with Clementia Pharmaceuticals, a subsidiary of Ipsen, to develop and commercialize BLU-782. Kinome illustration reproduced courtesy of Cell Signaling Technology,Inc.
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MEET THE BLUE CREW
A corporate staff member at Blueprint Medicines.MEET THE BLUE CREW
ANDREW KIM, INTERNATIONAL LAUNCH LEAD AND GENERAL MANAGER, NORDICS As Blueprint Medicines’ International Launch Lead and General Manager, Nordics, my role is to strategically plan and lead outreach to clinical investigators, healthcare providers and laboratories outside the United States to support the company’s portfolio of clinical-stage programs.Read on
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We aspire to pioneer multiple new scientific advances in disease biology and the design of precision therapies. To learn more about our scientific leadership and perspectives, visit The Lens.Read on
UNLEASHING THE VAST POTENTIAL OF KINASE THERAPIES Our presentations at ASCO ------------------------- SEEKING TO DELIVER TRANSFORMATIVE MEDICINES Download Our latest corporate presentation > “We ignore traditional boundaries and let science drive us. It’s > the only way to revolutionize how we treat cancer.”Jeff Albers,
Chief Executive Officer > “We ignore traditional boundaries and let science drive us. It’s > the only way to revolutionize how we treat cancer.” Jeff Albers, Chief Executive Officer DARE TO BE DIFFERENT We believe diversity is the key to unlocking our full potential. We push boundaries and take well-calculated risks, committing courageously and wholeheartedly to what we believe in.Read on
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