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EX - 99.1 2 tm2131101d1_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 Blueprint Medicines Reports Third Quarter 2021 Financial Results -- Strong AYVAKIT launch in advanced SM with $ 17.3 million in net product revenues in the first full quarter following U.S. approval -- -- Raising guidance for 2021 total revenue to $ 170- $ 180 million , based on portfolio progress including anticipated achievement of multiple collaboration milestones in the fourth quarter Broad clinical pipeline advancing toward multiple key data readouts in 2022 -- CAMBRIDGE , Mass . , October 28 , 2021 -- Blueprint Medicines Corporation ( NASDAQ : BPMC ) today reported financial results and provided a business update for the third quarter ended September 30 , 2021 . " We successfully executed across our entire portfolio in the third quarter , expanding the commercial adoption of AYVAKIT and GAVRETO and further advancing our next wave of therapeutic candidates toward potentially transformative proof - of - concept datasets , " said Jeff Albers , Chief Executive Officer of Blueprint Medicines . " We are particularly encouraged by the launch momentum for AYVAKIT in advanced systemic mastocytosis , where we see broad prescriber demand across both academic and community centers and in patients regardless of prior therapy . In addition , we are pleased by the progress across our clinical portfolio , with topline data from the PIONEER trial of AYVAKIT in non - advanced systemic mastocytosis expected in mid - 2022 , strong patient interest fueling enrollment in the ongoing SYMPHONY trial of our EGFR inhibitor BLU - 945 , and multiple trial initiations on - track for the months ahead . Overall , this commercial and clinical progress across our portfolio , combined with the strength of our financial position and diversity of revenue , provides a robust foundation for growth and continuous innovation . " Third Quarter 2021 Highlights and Recent Progress AYVAKIT ™ M / AYVAKYT® ( avapritinib ) : systemic mastocytosis ( SM ) and gastrointestinal stromal tumor ( GIST ) Recorded $ 17.3 million in net product revenue during the third quarter of 2021 , an increase of 104 percent over the second quarter , for AYVAKIT / AYVAKYT . GAVRETO® ( pralsetinib ) : RET - altered cancers Transferred responsibilities associated with the booking of U.S. product sales of GAVRETO to our collaboration partner Roche on July 1 , 2021 and began to recognize the company's share of U.S. revenue in the GAVRETO profit and loss . As previously reported by Roche , net end user product revenues for GAVRETO were $ 5.5 million . Received , via our collaboration with Roche , a positive opinion from the European Medicines Agency's ( EMA ) Committee for Medicinal Products for Human Use ( CHMP ) for GAVRETO for the treatment of adults with RET fusion - positive advanced non- small cell lung cancer ( NSCLC ) . If approved , GAVRETO will be the first and only targeted treatment approved by the EMA that includes first - line treatment of people with RET fusion - positive advanced NSCLC . Key Upcoming Milestones The company expects to achieve the following near - term milestones : Initiate a Phase 1 trial of BLU - 701 in patients with EGFR - driven NSCLC in the fourth quarter of 2021 . Present preclinical data supporting combination of BLU - 945 and BLU - 701 in EGFR - driven NSCLC at a medical conference in early 2022 . Initiate a Phase 1 trial of BLU - 222 , a CDK2 inhibitor targeting cyclin - E aberrant cancers , in the first quarter of 2022 . Disclose topline data for the registration - enabling Part 2 of the PIONEER trial of AYVAKIT in non - advanced systemic mastocytosis in mid - 2022 .