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, 2021 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 ...

Financial Data and Key Metrics Changes - Total revenues for Q1 2021 were $21.6 million, which included $9 million in net product sales and $12.6 million in collaboration revenue [36] - Net product revenue consisted of $7.1 million from AYVAKIT and $1.8 million from GAVRETO [13] - The company ended Q1 2021 with $1.4 billion in cash, ensuring sufficient resources for pipeline investments [38] - Operating expenses increased moderately compared to Q4 2020, driven by costs associated with advancing early-stage pipeline programs [37] Business Line Data and Key Metrics Changes - AYVAKIT generated $7.1 million in sales, with consistent new patient starts in the US and progress in the EU launch [13][19] - GAVRETO sales reached $1.8 million, with a growing share of new patient starts exceeding 35% by the end of Q1 [15] - The company is preparing for the anticipated approval of AYVAKIT in advanced systemic mastocytosis, targeting approximately 2,000 to 3,000 patients in the US [20] Market Data and Key Metrics Changes - The markets targeted by new programs are forecasted to more than double in size over the next five years, indicating significant growth opportunities [11] - GAVRETO received NPA approval in China for RET fusion-positive non-small cell lung cancer, marking it as the first approved selective RET inhibitor in the market [18] Company Strategy and Development Direction - The company focuses on three strategic pillars: accelerating global adoption of AYVAKIT and GAVRETO, advancing new therapeutic candidates, and expanding precision therapy research [7] - The company aims to drive patient identification and education for AYVAKIT and GAVRETO, particularly in advanced systemic mastocytosis [21][23] - The company plans to initiate multiple clinical trials in the coming months, positioning itself for significant proof-of-concept data sets [12] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about growth in patient identification and treatment opportunities, particularly as effective therapies become available [43] - The company is confident in its financial strength and ability to invest in its commercial efforts and pipeline programs [13][40] - Management highlighted the importance of ongoing education for healthcare providers to ensure proper patient identification and treatment [23] Other Important Information - The company has nominated five new development candidates in the past 18 months, with two expected to begin clinical development soon [27] - The company is committed to providing best-in-class access and support for patients through its patient support program [24] Q&A Session Summary Question: Trends in RET patient identification and diagnostics - Management noted that while there has not been significant quarter-over-quarter growth in patient identification, they expect improvement as effective treatments become available [42][43] Question: Patient definitions between ISM and ASM - Management acknowledged the challenges in accurately subtyping patients and indicated that initial utilization will likely be driven by those already diagnosed [44][45] Question: Current treatment with midostaurin and crossover potential to AYVAKIT - Management estimated that around 30% of adjusted SM patients are treated with midostaurin, but actual numbers may be lower due to treatment duration issues [48] Question: Initial feedback from physicians on GAVRETO versus Retevmo - Management highlighted points of differentiation such as efficacy breadth and safety profiles, noting that many prescribers are new to the market [56][57] Question: Blood test for diagnosing mastocytosis patients - Management confirmed that while blood tests can aid in identifying potential patients, bone marrow biopsies remain important for advanced SM diagnosis [68][69]

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, 2021 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 ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q4 2020 Results Conference Call February 17, 2021 8:30 AM ET Company Participants Kristin Hodous - Senior Manager, IR Jeff Albers - CEO Christy Rossi - Chief Commercial Officer Fouad Namouni - President, Research and Development Mike Landsittel - CFO Conference Call Participants Marc Frahm - Cowen and Company Dane Leone - Raymond James Charles Zhu - Guggenheim David Lebowitz - Morgan Stanley Peter Lawson - Barclays Arlinda Lee - Canaccord Eun Yang - Jefferies Mi ...

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, 2020 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 ...

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, 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 ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q3 2020 Earnings Conference Call October 29, 2020 8:30 AM ET Company Participants Kristin Hodous - Senior Manager, IR Jeff Albers - CEO Christy Rossi - COO Mike Landsittel - CFO Conference Call Participants Marc Frahm - Cowen and Company Reni Benjamin - JMP Securities Andrew Berens - SVB Leerink Peter Lawson - Barclays David Lebowitz - Morgan Stanley Nicole Gabreski - Piper Sandler Dane Leone - Raymond James Arlinda Lee - Canaccord Genuity Inc Michael Schmidt - ...

```markdown PART I – FINANCIAL INFORMATION [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) This section presents the company's unaudited condensed consolidated financial statements for the three and six months ended June 30, 2020, along with notes detailing significant transactions and subsequent events [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2020 | December 31, 2019 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $288,678 | $113,938 | | Total current assets | $568,312 | $516,786 | | **Total assets** | **$817,457** | **$707,694** | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $105,086 | $106,482 | | **Total liabilities** | **$236,090** | **$243,335** | | **Total stockholders' equity** | **$581,367** | **$464,359** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2020 | Three Months Ended June 30, 2019 | Six Months Ended June 30, 2020 | Six Months Ended June 30, 2019 | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $5,680 | $— | $9,138 | $— | | Collaboration revenue | $2,663 | $5,110 | $5,372 | $5,840 | | **Total revenues** | **$8,343** | **$5,110** | **$14,510** | **$5,840** | | Research and development | $91,079 | $87,101 | $175,225 | $161,351 | | Selling, general and administrative | $42,174 | $21,923 | $77,829 | $38,476 | | **Net loss** | **$(123,474)** | **$(99,681)** | **$(234,428)** | **$(187,088)** | | **Net loss per share** | **$(2.28)** | **$(2.04)** | **$(4.39)** | **$(4.03)** | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for the Six Months Ended June 30 (in thousands) | Cash Flow Activity | 2020 | 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | $(210,492) | $(161,416) | | Net cash provided by (used in) investing activities | $71,421 | $(158,739) | | Net cash provided by financing activities | $313,325 | $334,309 | | **Net increase in cash, cash equivalents, and restricted cash** | **$174,254** | **$14,154** | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - The company's first commercial product, **AYVAKIT™ (avapritinib)**, was approved by the FDA for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation[34](index=34&type=chunk) - As of June 30, 2020, the company had **$650.3 million** in cash, cash equivalents, and investments, which management believes is sufficient to fund operations for at least the next twelve months[35](index=35&type=chunk) Product Revenue from AYVAKIT (in thousands) | Period | Net Product Revenue | | :--- | :--- | | Three months ended June 30, 2020 | $5,700 | | Six months ended June 30, 2020 | $9,100 | - Subsequent to the quarter end, in July 2020, the company entered into a major collaboration with Roche for pralsetinib, receiving an upfront cash payment of **$675.0 million**. Roche also made a related equity investment of approximately **$94.0 million**[138](index=138&type=chunk)[139](index=139&type=chunk)[143](index=143&type=chunk) - In July 2020, the company received a **$20.0 million** cash milestone payment under its license agreement with Clementia[146](index=146&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=43&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's business strategy, clinical pipeline progress, and financial performance, emphasizing the commercial launch of AYVAKIT, regulatory advancements, increased revenues, rising operating expenses, and enhanced liquidity [Overview](index=43&type=section&id=Overview) - The company is advancing multiple investigational medicines, including avapritinib for systemic mastocytosis (SM), with plans to submit a supplemental NDA in **Q4 2020**[149](index=149&type=chunk)[152](index=152&type=chunk) - For pralsetinib, an NDA for RET fusion-positive NSCLC was accepted by the FDA with a PDUFA action date of **November 23, 2020**. A separate NDA was submitted for RET-mutant MTC and RET fusion-positive thyroid cancers[158](index=158&type=chunk) - In July 2020, the company entered into a collaboration with Roche to co-develop and co-commercialize pralsetinib in the U.S. and for Roche to commercialize it exclusively outside the U.S. (excluding Greater China)[172](index=172&type=chunk) - The company acknowledges potential temporary delays or disruptions to clinical trials due to the COVID-19 pandemic but states it has sufficient supply to meet anticipated global commercial and clinical needs through **2021**[177](index=177&type=chunk) [Results of Operations](index=63&type=section&id=Results%20of%20Operations) Comparison of Three Months Ended June 30, 2020 and 2019 (in thousands) | Account | Q2 2020 | Q2 2019 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Total revenues | $8,343 | $5,110 | $3,233 | 63% | | Research and development | $91,079 | $87,101 | $3,978 | 5% | | Selling, general and administrative | $42,174 | $21,923 | $20,251 | 92% | | **Net loss** | **$(123,474)** | **$(99,681)** | **$(23,793)** | **24%** | - The increase in total revenues for Q2 2020 was driven by **$5.7 million** in net product revenue from the launch of AYVAKIT[225](index=225&type=chunk) - The **92% increase** in SG&A expense for Q2 2020 was primarily due to building the commercial infrastructure for AYVAKIT and preparing for the potential commercialization of pralsetinib[231](index=231&type=chunk) [Liquidity and Capital Resources](index=67&type=section&id=Liquidity%20and%20Capital%20Resources) - As of June 30, 2020, the company had cash, cash equivalents, and investments of **$650.3 million**[248](index=248&type=chunk) - In July 2020, the company's cash position was significantly enhanced by receiving **$769.0 million** from its pralsetinib collaboration with Roche and a **$20.0 million** milestone payment from Clementia[247](index=247&type=chunk) - Management anticipates that existing cash, combined with the Roche upfront payments and future product revenues, will be sufficient to achieve a self-sustainable financial profile[253](index=253&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=73&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk exposures are interest rate sensitivity on its cash and investment portfolio and foreign currency exchange rate fluctuations. Management believes a 10% change in interest rates would not materially affect the portfolio's fair value. The company does not currently hedge its foreign currency risk - The company's primary market risk is interest rate sensitivity related to its **$650.3 million** in cash, cash equivalents, and investments as of June 30, 2020[266](index=266&type=chunk) - Due to the short-term duration and low-risk profile of its investments, the company believes an immediate **10% change** in interest rates would not have a material effect on the portfolio's fair market value[267](index=267&type=chunk) [Item 4. Controls and Procedures](index=75&type=section&id=Item%204.%20Controls%20and%20Procedures) Based on an evaluation as of June 30, 2020, the company's Chief Executive Officer and Chief Financial Officer concluded that the disclosure controls and procedures were effective. There were no material changes in internal control over financial reporting during the quarter - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective as of **June 30, 2020**[272](index=272&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[273](index=273&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=76&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently a party to any material legal proceedings - As of the filing date, the company is not a party to any material legal proceedings[276](index=276&type=chunk) [Item 1A. Risk Factors](index=76&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant business risks, including historical operating losses, the impact of the COVID-19 pandemic, capital needs, drug development uncertainties, commercialization challenges, and reliance on third-party operations - The company has a limited operating history and has incurred significant operating losses since inception, with a net loss of **$234.4 million** for the six months ended June 30, 2020[278](index=278&type=chunk)[281](index=281&type=chunk) - The COVID-19 pandemic poses a risk to business operations, including potential delays or disruptions to clinical trials, supply chains, and commercial activities[287](index=287&type=chunk)[289](index=289&type=chunk) - The company faces substantial competition. For pralsetinib, it will compete with Eli Lilly's selpercatinib, which was approved by the FDA in **May 2020** for similar indications[393](index=393&type=chunk) - The company relies on single-source third-party suppliers for the API, drug substance, and drug product of avapritinib and pralsetinib, which poses a significant risk to supply continuity[476](index=476&type=chunk)[477](index=477&type=chunk) [Item 5. Other Information](index=166&type=section&id=Item%205.%20Other%20Information) This section discloses a key corporate action taken after the quarter's end. On July 30, 2020, the company established an "at-the-market" (ATM) offering agreement with Cowen and Company, LLC, enabling it to sell up to $250.0 million of its common stock - On **July 30, 2020**, the company entered into a sales agreement with Cowen and Company, LLC, for an "at-the-market" offering, allowing it to sell up to **$250.0 million** of its common stock from time to time[588](index=588&type=chunk) [Item 6. Exhibits](index=168&type=section&id=Item%206.%20Exhibits) This section provides an index of the exhibits filed with the Form 10-Q. Notable exhibits include the sales agreement for the at-the-market offering, the collaboration agreement with Roche, and certifications by the Principal Executive Officer and Principal Financial Officer - Key exhibits filed with this report include the Sales Agreement with Cowen and the Collaboration Agreement with F. Hoffmann-La Roche Ltd and Genentech, Inc[595](index=595&type=chunk) ```

Financial Data and Key Metrics Changes - Blueprint Medicines reported $5.7 million in net sales for AYVAKIT in Q2 2020, totaling $9.1 million since its launch, indicating strong execution against launch strategies [20][27] - Total operating expenses increased slightly compared to the prior quarter, driven by rising stock-based compensation expenses [28] - The company holds over $1.4 billion in cash, positioning it in the strongest financial state in its history [29] Business Line Data and Key Metrics Changes - AYVAKIT's launch for treating PDGFRA alpha exon 18 mutant GIST has been successful, with broad access and high refill rates, suggesting real-world duration may exceed clinical study results [21][22] - The systemic mastocytosis program is highlighted as the largest opportunity in the clinical stage portfolio, with significant medical need and a differentiated approach [12][13] Market Data and Key Metrics Changes - The company received a positive CHMP opinion for avapritinib for treating PDGFRA alpha D842V mutant GIST, setting the stage for a marketing authorization decision by the end of Q3 2020 [10][11] - The anticipated launch of pralsetinib in the U.S. and avapritinib in Europe is expected to enhance the company's market presence [19] Company Strategy and Development Direction - The company aims to establish a commercial foundation with AYVAKIT, prioritize systemic mastocytosis, and leverage its discovery platform for future pipeline opportunities [8][9] - The collaboration with Roche is expected to stabilize expenses while allowing for increased R&D investment in systemic mastocytosis and discovery portfolio [28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for systemic mastocytosis treatments and the ongoing enrollment of patients despite the pandemic [46][47] - The company is focused on building a best-in-class commercial organization to deliver multiple precision therapies to patients [25][84] Other Important Information - The company plans to report top-line data from the EXPLORER and PATHFINDER trials of avapritinib in advanced systemic mastocytosis later in the quarter [14] - The company is on track to nominate up to three new development candidates by the end of the year, with BLU-945 being the first [17][82] Q&A Session Summary Question: Timing for patient screening enrollment for Part 2 of the PIONEER study - Management indicated that enthusiasm from sites is high, but specific timing for readouts has not been guided due to variability in site operations during COVID [33] Question: Cadence of hiring for AYVAKIT launch in the U.S. and EU - The U.S. team is already in place and focused on launching pralsetinib, while a small team in Europe is prepared for the initial AYVAKIT launch [34][35] Question: Feedback from physicians on CR rates and safety profiles - Management noted encouraging feedback regarding deep and durable responses, with a predictable safety profile being a significant factor for physician comfort [39][41] Question: Status of the advanced SM pivotal data set - Management confirmed that the data set shows high response rates and that they are blending data from multiple trials for regulatory submission [66][70] Question: Patient demographics receiving AYVAKIT - Management indicated that patients are a mix of those treated in frontline settings and those receiving standard-of-care before AYVAKIT [75] Question: Update on BLU-263 - The first human health volunteer study for BLU-263 has started and is progressing as expected [55]

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 ...