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EX - 99.1 2 bpmc - 20210729xex99d1.htm EX - 99.1 Exhibit 99.1 -- Blueprint Medicines Reports Second Quarter 2021 Financial Results Obtained FDA approval and launched AYVAKITTM ( avapritinib ) for advanced systemic mastocytosis -- -- Expect topline data from Part 2 of registration - enabling Phase 2 PIONEER trial of AYVAKIT in non - advanced SM in mid - 2022 -- -- Initiated Phase 1/2 trial of BLU - 945 in EGFR - driven NSCLC and Phase 2/3 HARBOR trial of BLU - 263 in non - advanced SM -- -- Announced strategic research collaboration with MD Anderson to accelerate development of BLU - 222 -- CAMBRIDGE , Mass . , July 29 , 2021 -- Blueprint Medicines Corporation ( NASDAQ : BPMC ) today reported financial results and provided a business update for the second quarter ended June 30 , 2021 . " In the second quarter , we achieved one of our foundational corporate goals , securing U.S. approval of AYVAKIT for advanced systemic mastocytosis , and the launch of this therapy is off to a great start with broad prescriber demand , " said Jeff Albers , Chief Executive Officer of Blueprint Medicines . " In addition , we continued to progress our expanding clinical portfolio with the initiation of the Phase 1/2 trial of BLU - 945 , the initiation of the Phase 2/3 HARBOR trial of BLU - 263 and the announcement of a strategic research collaboration with MD Anderson to accelerate the development of BLU - 222 , all of which exemplify the potential impact of our next wave of therapeutic candidates . This commercial and clinical progress , combined with our financial strength , provides a robust foundation for future growth as we look to address the needs of many more patients with cancer and hematologic disorders . " Second Quarter 2021 Highlights and Recent Progress AYVAKIT ™ / AYVAKYTⓇ ( avapritinib ) : systemic mastocytosis ( SM ) and gastrointestinal stromal tumor ( GIST ) • Recorded $ 8.5 million in net product revenue during the second quarter of 2021 for AYVAKIT / AYVAKYT , which was approved by the U.S. Food and Drug Administration ( FDA ) in June 2021 for the treatment of adult patients with advanced systemic mastocytosis ( Advanced SM ) , including aggressive SM ( ASM ) , SM with an associated hematologic neoplasm ( SM - AHN ) and mast cell leukemia ( MCL ) . Read the press release announcing the expansion of the FDA label for AYVAKIT into advanced SM here . AYVAKIT received its initial approval from the FDA in 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation , including PDGFRA D842V mutations . Please click here to see the full Prescribing Information for AYVAKIT and visit www.AYVAKIT.com for more information on AYVAKIT . GAVRETOⓇ ( pralsetinib ) : RET - altered cancers Recorded $ 2.9 million in net product revenue during the second quarter of 2021 for GAVRETO , which was approved by the FDA in September 2020 for the treatment of adult patients with metastatic rearranged during transfection ( RET ) fusion - positive non- small cell lung cancer ( NSCLC ) as detected by an FDA approved test and in December 2020 for the treatment of patients 12 years of age and older with advanced or metastatic RET - mutant medullary thyroid cancer ( MTC ) and RET fusion - positive thyroid cancer . Blueprint Medicines is commercializing GAVRETO in the U.S. together with Genentech , Inc. , a member of the Roche Group . Please click here to see the full Prescribing Information for GAVRETO and visit www.GAVRETO.com for more information on GAVRETO . • Reported updated data from the Phase 1/2 ARROW clinical trial of GAVRETO in metastatic RET fusion - positive NSCLC and other advanced solid tumors at the 2021 American Society of Clinical Oncology ( ASCO ) Annual Meeting . The data showed high response rates to GAVRETO in treatment - naïve patients with RET fusion - positive NSCLC , clinical activity across a number of RET fusion - positive tumor types and a safety profile consistent with previously reported results . Read the press release here . BLU - 263 : SM