Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of Regi ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q2 2020 Earnings Conference Call July 30, 2020 8:30 AM ET Company Participants Kristin Hodous - Senior Manager, IR Jeff Albers - CEO Christy Rossi - Chief Commercial Officer Mike Landsittel - CFO Andy Boral - Chief Medical Officer Conference Call Participants Peter Lawson - Barclays Marc Frahm - Cowen and Company David Lebowitz - Morgan Stanley Reni Benjamin - JMP Securities George Farmer - BMO Capital Markets Arlinda Lee - Canaccord Operator Ladies and gentleme ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q1 2020 Earnings Conference Call May 6, 2020 8:30 AM ET Company Participants Kristin Hodous - Senior Manager, IR Jeff Albers - CEO Christy Rossi - Chief Commercial Officer Mike Landsittel - CFO Andy Boral - Chief Medical Officer Conference Call Participants Dane Leone - Raymond James Joseph Thome - Cowen and Company Konstantinos Aprilakis - Deutsche Bank Reni Benjamin - JMP Securities Eun Yang - Jefferies Peter Lawson - Barclays Operator Ladies and gentlemen, th ...

Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of Reg ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37359 BLUEPRINT MEDICINES CORPORATION (Exact name of registrant as specified in its charter) Delaware 26-3632015 (St ...

Financial Data and Key Metrics Changes - The company ended 2019 with $548 million in cash and closed a public offering in January 2020, resulting in net proceeds of approximately $308 million, providing sufficient capital to fund operations into the second half of 2022 [32][33] - Collaboration revenues increased in Q4 to $66.5 million, primarily due to a license agreement with Clementia Pharmaceuticals, including a $25 million upfront payment and an additional $20 million payment due in Q3 2020 [33] - Total operating expenses increased in Q4 2019 compared to the prior quarter due to investments in global commercial infrastructure and acceleration of clinical development activities [34] Business Line Data and Key Metrics Changes - The company highlighted the initial launch of AYVAKIT for PDGFRalpha Exon 18 mutant GIST, with strong initial reception from healthcare providers and payers [21][26] - AYVAKIT is the first precision medicine for GIST and the only effective treatment for patients with PDGFRalpha mutation, with a well-prepared commercial launch strategy [8][25] - The company plans to continue avapritinib towards regulatory filings for advanced systemic mastocytosis in the second half of 2020 and indolent SM in 2021 [10][12] Market Data and Key Metrics Changes - The company is focusing on systemic mastocytosis and other KIT-driven mass cell disorders, with a potential patient population of up to 75,000 in major markets [9] - AYVAKIT has been added to the NCCN clinical practice guidelines for soft tissue sarcoma, indicating broad consensus for its use in treatment [28] Company Strategy and Development Direction - The company aims to create substantial therapeutic value through the commercialization of avapritinib and pralsetinib, with potential commercial launches occurring as frequently as once per quarter over the next year and a half [13] - The company is strengthening its early-stage research pipeline, with plans to nominate up to three new development candidates in 2020 [12] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the strong topline data from the Phase 1/2 ARROW trial of pralsetinib in patients with RET fusion-positive non-small cell lung cancer, indicating a sense of urgency to deliver pralsetinib to patients quickly [14][19] - The company anticipates a decision from the FDA on the NDA for avapritinib for fourth-line GIST in the second quarter, with a PDUFA date extension allowing for additional data submission [20][90] Other Important Information - The company is initiating a Phase 1 healthy volunteer trial for BLU-263, a next-generation KIT inhibitor, aiming to reach more patients with indolent SM and other KIT-driven disorders [11] - The company is focused on ensuring that PDGFRalpha mutations are reflex tested every time KIT is negative, aiming to improve diagnostic testing and treatment outcomes [78] Q&A Session Summary Question: Insights on AAAAI meeting data and AYVAKIT launch feedback - Management indicated that the purpose of Part 1 of the PIONEER study is to guide registration enabling Part 2, focusing on dose selection and sample size [38] - Initial feedback from prescribing physicians on AYVAKIT has been positive, with excitement about having an effective treatment available for the first time in years [41] Question: Update on BLU-263 study and pralsetinib filing - The healthy volunteer study for BLU-263 is set to start this year, with no specific timing provided yet [45] - The rolling submission for pralsetinib in RET-driven non-small cell lung cancer is on track to be completed this quarter [52] Question: Clarification on VOYAGER study and FDA review - The FDA is looking for topline results from the VOYAGER study, which includes a mix of third and fourth-line patients, primarily focusing on PFS assessment [90] Question: Discussion on avapritinib and accelerated approval scenarios - Management clarified that multiple accelerated approvals can occur simultaneously, and this should not affect the approval process for avapritinib [96]

Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of ...

Financial Data and Key Metrics Changes - The company ended Q2 2019 with $667.3 million in cash, cash equivalents, and investments, which is expected to fund operations into mid-2021 [23] - Operating expenses increased in Q2 due to manufacturing and supply activities, driven by accelerated clinical development and filing timelines for avapritinib and pralsetinib [24] Business Line Data and Key Metrics Changes - The company submitted two marketing applications for avapritinib to the FDA and EMA for adult patients with PDGFRalpha GIST, marking significant progress in their regulatory strategy [7][8] - The company plans to hold its first Research and Development Day on November 5, focusing on indication expansion opportunities and new targets [9] Market Data and Key Metrics Changes - The company aims to increase tumor mutation testing rates among GIST patients, currently at 30% to 40% in the U.S., to improve access to precision medicine [14][15] - The commercial strategy includes educating healthcare providers and patients about avapritinib, targeting a shift in treatment paradigms for GIST [16][17] Company Strategy and Development Direction - The company is focused on evolving the treatment paradigm for GIST and expanding its commercial infrastructure to support multiple product launches [13][19] - The collaboration with CStone Pharmaceuticals in China is progressing well, with multiple clinical trial protocols approved and patient enrollment underway [21][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming regulatory submissions and the potential for avapritinib to transform treatment for GIST patients [6][10] - The company is preparing for significant growth opportunities across its portfolio, with a focus on patient access and education [11][19] Other Important Information - The company plans to provide an update on the avapritinib program in systemic mastocytosis in Q4 2019, with promising clinical data reported [10][11] - The PIONEER trial for indolent systemic mastocytosis is designed to evaluate lower dose levels, with initial data expected by the end of the year [26][36] Q&A Session Summary Question: Can you discuss the side effects profile of the drug in the context of the SM indication particularly the indolent population? - Management indicated that early safety data from the PIONEER study showed low side effects at lower dose levels, which are expected to be well tolerated for patients with indolent systemic mastocytosis [26] Question: Any color around expected patient demographic distribution for VOYAGER now that you're enrolling Chinese patients? - Management noted that the VOYAGER study is open internationally and expects no regulatory impact from the inclusion of Chinese patients [29] Question: How much longer-term safety and efficacy follow-up will you need to demonstrate for chronic conditions? - Management stated that ongoing discussions with regulatory authorities will determine the specifics of safety follow-up for the PIONEER study [30] Question: How much synergy could you expect to capture between indolent and advanced SM? - Management anticipates leveraging learnings from advanced SM to inform strategies for indolent SM, emphasizing the need for precision therapy approaches [31][34] Question: Can you give us an update on the PIONEER trial's dose-ranging portion? - Management confirmed that the PIONEER trial is ongoing, with preliminary information expected by the end of the year [36] Question: What is the expected safety and efficacy if you exclude certain mutations in the GIST program? - Management explained that specific mutations would be excluded from the COMPASS study to ensure appropriate patient selection for avapritinib [49] Question: Will you be including any data on the PRO endpoint in the initial update for PIONEER? - Management indicated that PRO data will not be available in the initial update but is a critical aspect of the study [54]

Table of Contents _____________________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 _____________________________ FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number 001-37359 _____________________________ BLUEPRINT MEDICINES CORPORATION (Exact Name of Regi ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q1 2019 Earnings Conference Call May 9, 2019 8:30 AM ET Company Participants Kristin Hodous - Senior Manager, Investor Relations Jeff Albers - Chief Executive Officer Christy Rossi - Chief Commercial Officer Mike Landsittel - Chief Finance Officer Andy Boral - Chief Medical Officer Marion Dorsch - Chief Scientific Officer Conference Call Participants Terence Flynn - Goldman Sachs Joseph Thome - Cowen and Company Charles Zhu - Guggenheim Securities David Nierenga ...