4. Marketable Securities | --- | --- | --- | --- | --- | --- | --- | --- | |------------------------------------------------|-------|--------------------|-------------------|-------|-----------------------|-------|--------------| | thousands): \nMarch 31, 2023 | | Amortized \nCost | Unrealized \nGain | | Unrealized \nLosses | | Fair \nValue | | Marketable securities, available-for-sale: | | | | | | | | | U.S. government agency securities | $ | 314,891 | | 28 | (2,117) | $ | 312,802 | | U.S. treasury obligat ...

Financial Data and Key Metrics Changes - For the full year 2022, total revenues were $204 million, including $111 million in net product revenues from AYVAKIT and $93 million in collaborations and license revenues, exceeding the high end of the 2022 total revenue guidance of $200 million [46][73] - Total operating expenses were $723.7 million for the full year, with Q4 operating expenses showing a quarter-over-quarter decline from Q3 as the company continues to leverage operating efficiencies [47][48] - The company expects operating expenses to grow moderately in Q1 2023, driven by preparations for the ISM launch and planned manufacturing investments [48] Business Line Data and Key Metrics Changes - AYVAKIT net product revenue doubled year-over-year in 2022, achieving $111 million, with Q4 revenues at $30.1 million, of which $26.3 million came from the U.S. [18][46] - The company anticipates AYVAKIT net product revenues of $130 million to $140 million in 2023, representing a more than 20% increase year-over-year [20][73] Market Data and Key Metrics Changes - The U.S. accounted for the majority of AYVAKIT product sales, with nearly 500 patients on AYVAKIT by the end of 2022 [8][34] - The company is preparing for the ISM launch, targeting approximately 7,500 patients with moderate to severe ISM, who are actively seeking treatment [36][40] Company Strategy and Development Direction - The company aims to expand its leadership in systemic mastocytosis and advance its clinical stage pipeline focused on best-in-class investigational therapies [13][31] - The strategy includes engaging a broader provider base for ISM patients and maintaining strong patient access to therapy [22][40] - The company is focused on achieving a sustainable financial profile while executing on a range of opportunities [31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory for AYVAKIT and the anticipated launch in ISM, highlighting the strong medical need and motivated patient base [40][128] - The company is optimistic about capturing the significant ISM opportunity, supported by a strong cash position of over $1 billion [15][74] Other Important Information - The company has received EMA validation for its type 2 variation for AYVAKIT and ISM, bringing it closer to the important indication expansion and launch in Europe [69] - The FDA issued a partial clinical hold on the VELA trial due to visual adverse events, but the company is working closely with the FDA to resolve this issue [45][120] Q&A Session Summary Question: What is the overlap between current prescriber base of AYVAKIT and potential ISM patients? - The company indicated that among the top 350 prescribers, there are nearly 400 moderate to severe ISM patients who could rapidly start AYVAKIT upon approval [76][88] Question: What is the current thinking regarding vision events and the CDK2 program? - Management stated that the FDA is working cooperatively to modify the protocol and informed consent regarding the vision events observed [56][120] Question: How does the company view the early trajectory for ISM? - The company does not anticipate an initial launch bolus but expects gradual growth driven by increased disease awareness and advanced patient identification capabilities [126][128] Question: What is the current payer coverage for ISM? - The company reported strong payer coverage with virtually no access challenges for SM patients, and they do not expect a new code for ISM [153][183] Question: How is the sales force adapting to focus on allergists? - The company has expanded its field force to engage a broader provider base, including allergists, and has achieved over 40% awareness of AYVAKIT among target prescribers [149][150]

Brexit and the Regulatory Framework in the United Kingdom The UK formally left the EU on January 31, 2020, and the EU and the UK have concluded a trade and cooperation agreement, or TCA, which was provisionally applicable since January 1, 2021 and has been formally applicable since May 1, 2021. The TCA includes specific provisions concerning pharmaceuticals, which include the mutual recognition of GMP, inspections of manufacturing facilities for medicinal products and GMP documents issued, but does not fore ...

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, 2022 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) Q2 2022 Earnings Conference Call August 2, 2022 8:00 AM ET Company Participants Jenna Cohen - Senior Director and Head of Investor Relations Kate Haviland - Chief Executive Officer Philina Lee - Chief Commercial Officer Becker Hewes - MD, Chief Medical Officer Christy Rossi - Chief Operating Officer Mike Landsittel - Chief Financial Officer Conference Call Participants Ernie Rodriguez - Cowen and Company Dane Leone - Raymond James Andrea Tan - Goldman Sachs Pete ...

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, 2022 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 2022 Earnings Conference Call May 3, 2022 8:30 AM ET Company Participants Jenna Cohen - Senior Director and Head of Investor Relations Kate Haviland - Chief Executive Officer Christy Rossi - Chief Operating Officer Philina Lee - Chief Commercial Officer Becker Hewes - MD, Chief Medical Officer Mike Landsittel - Chief Financial Officer Conference Call Participants Marc Frahm - Cowen Dane Leone - Raymond James Elizabeth - Goldman Sachs Reni Benjamin - JMP Secur ...

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, 2022 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 2021 Earnings Conference Call February 16, 2022 8:30 AM ET Company Participants Kristin Hodous – Senior Manager-Investor Relations Jeff Albers – Chief Executive Officer Kate Haviland – Chief Operating Officer Christy Rossi – Chief Commercial Officer Becker Hewes – Chief Medical Officer Mike Landsittel – Chief Financial Officer Fouad Namouni – President-Research & Development Conference Call Participants Marc Frahm – Cowen Dane Leone – Raymond James Brad Canin ...

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, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to | --- | --- | --- | --- | |---------------------------------------------------------------------------|----------------------------------------- ...