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EX - 99.1 2 bpmc - 20210429xex99d1.htm EX - 99.1 Exhibit 99.1 Blueprint Medicines Reports First Quarter 2021 Financial Results -- 7 research- and clinical - stage programs highlighted at AACR , including recently nominated development candidate with best - in - class potential targeting CDK2 -- -- GAVRETO® ( pralsetinib ) becomes the first selective RET inhibitor approved in China ; AYVAKIT ™ M / AYVAKYTⓇ ( avapritinib ) becomes the first precision therapy for patients with PDGFRA exon 18 mutant GIST in China -- -- Preparing to launch avapritinib in advanced SM in Q2 2021 -- On track to initiate Phase 1 trial of BLU - 945 in EGFR - driven NSCLC in Q2 2021 and Phase 2/3 HARBOR trial of BLU - 263 in non - advanced SM in mid - 2021 -- CAMBRIDGE , Mass . , April 29 , 2021 -- Blueprint Medicines Corporation ( NASDAQ : BPMC ) today reported financial results and provided a business update for the first quarter ended March 31 , 2021 . " In recent months , we made significant progress toward achieving our key 2021 portfolio goals , illustrating our clinical leadership in systemic mastocytosis and the rapid advancement of our next wave of therapeutic candidates with first- or best - in - class potential , ” said Jeff Albers , Chief Executive Officer of Blueprint Medicines . " With a potential U.S. approval for AYVAKIT in advanced systemic mastocytosis in the second quarter , we are looking forward to welcoming a new era of precision medicines care for people living with this debilitating disease . In parallel , we are initiating multiple clinical trials across our pipeline , beginning with the Phase 1 trial of BLU - 945 and Phase 2/3 HARBOR trial of BLU - 263 , as we continue to translate our research productivity and precision therapy expertise to address patient need across cancers and hematologic malignancies . Taken together , these accomplishments position Blueprint Medicines to help an increasing number of patients globally as we advance our robust portfolio . " First Quarter 2021 Highlights and Recent Progress AYVAKITTM / AYVAKYTⓇ ( avapritinib ) : systemic mastocytosis ( SM ) • Reported data at the virtual American Association for Cancer Research ( AACR ) Annual Meeting , including registrational PATHFINDER trial data in advanced systemic mastocytosis ( SM ) , which demonstrated an overall confirmed response rate of 75 percent , as well as PIONEER Part 1 data highlighting the impact of AYVAKIT on skin manifestations in non - advanced SM . Read the full data here . Received European Medicines Agency ( EMA ) validation of the Type II variation marketing authorization application ( MAA ) for AYVAKYT for the treatment of advanced SM . Validation of the MAA confirms that the submission is sufficiently complete to begin the formal review process . Read the press release here . AYVAKIT ™ M / AYVAKYT® ( avapritinib ) : gastrointestinal stromal tumor ( GIST ) • Recorded $ 7.1 million in net product revenue during the first quarter of 2021 for AYVAKIT / AYVAKYT , which was approved by the U.S. Food and Drug Administration ( FDA ) in January 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation , including PDGFRA D842V mutations , and by the European Commission in September 2020 as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation . Received National Medical Products Administration ( NMPA ) approval in China , via our collaboration with CStone Pharmaceuticals , for the treatment of adults with unresectable or metastatic PDGFRA exon 18 mutant GIST , the first approved precision therapy for this patient population in China .