Territories. Matthew Taylor, M.D., American Society of Clinical Oncology (ASCO) Annual Meeting, June 1, 2019. Justin Gainor, M.D., American Society of Clinical Oncology (ASCO) Virtual Scientific Program, May 29, 2020. Erica Evans, Ph.D., American Association for Cancer Research (AACR) Annual Meeting, April 2, 2017.
BLU-285: A potent and highly selective inhibitor designed to target malignancies driven by KIT and PDGFRα mutations. Vivek Subbiah, M.D., American Association for Cancer Research (AACR) Annual Meeting, April 15, 2018. Blueprint Medicines and Roche are developing pralsetinib globally for the treatment of patients with RET fusion-positive NSCLC. Clinical Activity of Avapritinib in ≥4th Line (4L+) and PDGFRA Exon 18 Gastrointestinal Stromal Tumors (GIST). The two primary mechanisms of oncogenic RET activation are fusions and mutations. Nearly half of the world’s new HCC cases are diagnosed in China annually. Karin Hartmann, M.D., European Academy of Allergy and Clinical Immunology (EAACI) 2020 Digital Congress, June 6, 2020. Across a wide range of cancers, oncogenic alterations in RET result in kinase activation, driving tumor formation and growth. Blueprint Medicines is developing avapritinib as a potential treatment for a broad population of patients with SM, including advanced, smoldering and indolent SM. As of a data cutoff date of March 31, 2020, updated data from part 1 of the PIONEER trial showed a deepening of symptom reductions in patients treated with avapritinib through 24 weeks of follow-up.
BLU-782 is designed to selectively target mutant ALK2, while sparing closely related anti-targets including ALK1, ALK3, and ALK6. SM is a rare disease driven by the KIT D816V mutation.
As of a data cutoff date of March 31, 2020, avapritinib 25 mg QD was well-tolerated and safety results were consistent with previously reported data, with no Grade ≥3 adverse events or discontinuations due to adverse events. Photo: R.T., living with gastrointestinal stromal tumor. 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). Psychometric Performance of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF). These data were presented today during the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress 2020. Avapritinib induces responses in patients with advanced systemic mastocytosis (AdvSM), regardless of prior midostaurin therapy. At Blueprint Medicines, we work together in pursuit of one common goal: to dramatically improve the lives of people with genomically defined diseases. Zofia Piotrowska, M.D., IASLC World Conference on Lung Cancer, September 26, 2018. Benjamin Besse, M.D., Ph.D., American Society of Clinical Oncology (ASCO) Virtual Scientific Program, May 29, 2020. A strong team embraces different perspectives, opinions and ideas. The presentation is available on-demand via the EAACI Digital Congress 2020 website at www.eaaci.org/eaaci-congresses/eaaci-2020. Blueprint Medicines estimates about 30% of patients with hepatocellular carcinoma (HCC) have tumors with aberrantly activated FGFR4 signaling and is working to develop fisogatinib for the treatment of patients with FGFR4-driven HCC. This website does not provide medical advice. Identifying these specific – and often rare – mutations is important, because drugs can then be developed to target them.
In October 2019, we entered into an exclusive, worldwide license agreement with Clementia Pharmaceuticals, a subsidiary of Ipsen, to develop and commercialize BLU-782. FOP is caused by a gain-of-function mutation in ACVR1, which encodes activin-like kinase 2 (ALK2). This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans and timelines for the development of its drug candidates, including the timing, design, implementation, enrollment, plans and announcement of results regarding Blueprint Medicines' ongoing and planned clinical trials for avapritinib and BLU-263; plans, timelines and expectations for initiating patient screening in part 2 of the PIONEER trial; expectations regarding the potential benefits of avapritinib and BLU-263 in treating patients with SM and mast cell disorders; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus.
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