Blueprint Medicines Corporation (NASDAQ:BPMC) Q2 2023 Earnings Conference Call August 2, 2023 8:00 AM ET Company Participants Jenna Cohen - VP, IR Kate Haviland - CEO Philina Lee - CCO Fouad Namouni - President, R&D Mike Landsittel - CFO Christy Rossi - COO Becker Hewes - CMO Conference Call Participants Brad Canino - Stifel Dane Leone - Raymond James Reni Benjamin - JMP Securities Eun Yang - Jefferies Marc Frahm - TD Cowen Salveen Richter - Goldman Sachs Michael Schmidt - Guggenheim Partners Ami Fadia - Ne ...

Table of Contents The Company evaluated the Zai Lab agreement to determine whether it is a collaborative arrangement in the scope of ASC 808. The Company concluded that the Zai Lab agreement is a collaborative agreement under ASC 808 as both parties are active participants in the clinical trials and are exposed to significant risks and rewards of those activities under the Zai Lab agreement. The Company determined that the Zai Lab agreement contained two material components: (i) licenses granted to Zai Lab ...

Blueprint Medicines Corporation (NASDAQ:BPMC) Q1 2023 Earnings Conference Call May 4, 2023 8:00 AM ET Company Participants Jenna Cohen - Senior Director and Head, Investor Relations Kate Haviland - Chief Executive Officer Philina Lee - Chief Commercial Officer Becker Hewes - Chief Medical Officer Mike Landsittel - Chief Financial Officer Fouad Namouni - President, Research & Development Christina Rossi - Chief Operating Officer Conference Call Participants Reni Benjamin - JMP Securities Brad Canino - Stifel ...

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 ...

Summary of Cogent Biosciences Conference Call Company Overview - **Company**: Cogent Biosciences - **Lead Product**: Bezuclasinib, a drug targeting KIT mutations, particularly D816V, associated with systemic mastocytosis and gastrointestinal stromal tumors (GIST) [4][6] Key Points and Arguments Product Development and Pipeline - **Bezuclasinib** is in development for two subgroups of mastocytosis and in a global Phase III trial for GIST [4][6] - The company has a strong cash position, expected to last until 2025, allowing for continued development of its pipeline [6][40] - The company is running three clinical studies: APeX, SUMMIT, and PEEK, which could serve as the basis for registration if successful [6][14] Competitive Landscape - In GIST, the current standard of care is **Sunitinib**, which has limitations against certain mutations [10][12] - Bezuclasinib is positioned to cover all secondary resistance mutations, potentially leading to better outcomes in combination with Sunitinib [12][30] - In systemic mastocytosis, Bezuclasinib competes with **Avapritinib** and **BLU-263**, with a focus on a better tolerability profile [13][61] Market Potential - The total available market in the U.S. for the targeted indications is estimated at **$2.5 billion**, with a global potential of **$3.5 billion** [14][65] - The systemic mastocytosis market is projected to be around **$300 million** in the U.S. and **$500 million** globally [65] Clinical Data and Efficacy - The APeX study has shown an objective response rate of **73%**, increasing to **89%** in treatment-naive patients [19][20] - The safety profile of Bezuclasinib is favorable, with no significant cognitive effects or bleeding events reported [21][22] - The company plans to initiate investigations of Bezuclasinib in combination with other therapies to enhance its market position [24] Future Catalysts - Upcoming milestones include updates from the APeX study, results from the Phase III PEEK study, and progress on early-stage programs targeting FGFR2 and ErbB2 [38][39][40] Important but Overlooked Content - The company emphasizes the unmet need for effective therapies in systemic mastocytosis, particularly for the indolent form of the disease, where current options are limited [15][17] - The potential for Bezuclasinib to be used alongside other hematologic therapies could unlock significant market opportunities that competitors may not address effectively [65] Conclusion Cogent Biosciences is strategically positioned in the precision medicine space with its lead product, Bezuclasinib, targeting rare diseases driven by genetic mutations. The company is focused on demonstrating clinical efficacy and safety to capture a significant share of the market, while navigating a competitive landscape with limited alternatives for patients.

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 ...

Financial Data and Key Metrics Changes - Blueprint Medicines reported total revenues of $36.5 million for Q2 2022, including $28.5 million in net product revenues from AYVAKIT, reflecting a 20% quarter-over-quarter growth in AYVAKIT revenues [7][41]. - The company reiterated its revenue guidance for 2022, expecting total revenues between $180 million and $200 million, and AYVAKIT net product revenues between $115 million and $130 million [41][42]. - R&D expenses for Q2 were $128.3 million, an increase of approximately $25 million from Q1, driven by the startup and supply activities of new clinical trials [42][43]. Business Line Data and Key Metrics Changes - AYVAKIT has established itself as the standard of care for advanced systemic mastocytosis (SM), with over 50% market share in the U.S. and treatment of choice for more than 70% of new therapy patients [14][60]. - The company has seen four consecutive quarters of double-digit revenue growth for AYVAKIT, with $24.7 million generated in the U.S. and $3.8 million ex-U.S. in Q2 [13][41]. - The launch in Germany is progressing well, with early adoption at mastocytosis centers and ongoing reimbursement submissions in other key markets [13]. Market Data and Key Metrics Changes - The number of diagnosed SM patients in the U.S. has grown by 63% since AYVAKIT's launch in January 2020, with over 16,000 unique diagnosed patients identified [20]. - The addressable market opportunity for AYVAKIT in non-advanced SM is estimated at approximately 7,500 patients with moderate to severe disease, indicating significant growth potential [22][23]. Company Strategy and Development Direction - The company plans to leverage its strong cash position and operational flexibility from a $1.25 billion non-dilutive financing to accelerate its pipeline and explore strategic business development opportunities [9][45]. - Blueprint Medicines aims to capture the non-advanced SM market, which currently lacks approved therapies, and anticipates a multibillion-dollar opportunity for AYVAKIT in this space [23][24]. - The company is focused on advancing its EGFR and CDK2 programs, with multiple data catalysts expected in the second half of 2022 [33][38]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in AYVAKIT's potential to transform treatment for patients with non-advanced SM, highlighting the urgency to treat this patient population [17][19]. - The management team emphasized the importance of upcoming data from the PIONEER Part 2 study, which is expected to provide robust evidence for AYVAKIT's efficacy and safety [25][31]. - The company remains optimistic about its growth trajectory, supported by a strong cash position and a diverse pipeline of programs [47]. Other Important Information - Blueprint Medicines out-licensed its internally discovered KIT exon 13 inhibitor to IDRx, receiving a 15% equity stake and potential milestone payments totaling up to $217 million [39]. - The company plans to provide further insights into its growth drivers at an Investor Day scheduled for November 1, 2022 [11]. Q&A Session Summary Question: What level of granularity should be expected for the safety portion of the PIONEER study? - Management plans to present adverse events and serious adverse events comparing the two arms, with expectations for a safety profile similar to Part 1 [51][52]. Question: What are the expectations for market share in advanced SM? - The company confirmed over 50% market share in the U.S. and is encouraged by the growth potential in both treatment-naïve and previously treated patients [60][62]. Question: How will the new endpoint in PIONEER impact physician utilization? - The change in endpoint is expected to provide clinically meaningful benefits, which could significantly influence physician prescribing behavior [92][96]. Question: What is the anticipated approval process for PIONEER results? - The company expects a full approval from the FDA with a rapid review of the dossier following the submission of PIONEER results [81][82]. Question: How does the company view the competitive landscape post-PIONEER? - Management believes that the high bar set by AYVAKIT's data will make it challenging for follow-on compounds to gain traction in the market [82][101].

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 ...