To access the live conference call, please dial 833-470-1428 (domestic) or 404-975-4839 (international) and refer to conference ID 299779. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at http://ir.blueprintmedicines.com/. The archived webcast will be available on Blueprint Medicines' website approximately two hours after the conference call and will be available for 30 days following the call. Blueprint Medi ...

Core Insights - Blueprint Medicines Corporation is advancing the understanding and treatment of systemic mastocytosis (SM) through multiple upcoming datasets presented at key conferences, emphasizing the real-world burden of the disease and the clinical outcomes of AYVAKIT/AYVAKYT (avapritinib) [1][2][5] Group 1: Clinical Efficacy and Safety of AYVAKIT - AYVAKIT demonstrated durable efficacy and a favorable safety profile in patients with indolent systemic mastocytosis (ISM) over a median follow-up of more than two years, with safety data consistent for approximately 10% of patients who escalated to a 50 mg daily dose [1][2] - The median overall survival (OS) for AYVAKIT in advanced SM has not been reached, with a historical median OS ranging from less than six months to about 3.5 years, and an overall response rate of 73% [3][4] - AYVAKIT showed sustained improvements in total symptom scores and quality of life, with the most common treatment-related adverse event being edema, primarily Grade 1, and a low discontinuation rate of 3% due to treatment-related adverse events [2][4] Group 2: Patient Perspectives and Disease Burden - The PRISM Survey highlighted the significant burden of systemic mastocytosis on patients' quality of life, with many reporting debilitating symptoms and impaired physical and mental health functioning [5][6] - Healthcare providers acknowledged the impact of SM, with 65% indicating it affected patients' lives "quite a bit" or "a great deal," and 58% noting that patients lost employment opportunities due to the disease [6] Group 3: Upcoming Presentations and Research - Data presentations will be made available at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024 and the European Hematology Association 2024 (EHA24) Hybrid Congress, covering various aspects of AYVAKIT's safety, efficacy, and the burden of SM [7][8]

AHN, or MCL, after at least one systemic therapy. In December 2023, the European Commission approved AYVAKYT for the treatment of adult patients with indolent SM with moderate to severe symptoms inadequately controlled on symptomatic treatment. These approvals in advanced SM were supported by our ongoing Phase 1 clinical trial in advanced SM, which we refer to as our EXPLORER trial, and our ongoing Phase 2 clinical trial in advanced SM, which we refer to as our PATHFINDER trial. The FDA's approval of AYVAKI ...

Financial Data and Key Metrics Changes - The company reported total revenues of $96.1 million for Q1 2024, including $92.5 million from AYVAKIT net product revenue, which represents a year-over-year growth of over 135% [34][44][92] - AYVAKIT revenue guidance for the year has been increased to $390 million to $410 million, reflecting a strong revenue trajectory towards peak sales of over $2 billion globally [32][67][93] - The company maintains a strong financial position with $735.6 million in cash on hand and aims to reduce cash burn in 2024 [46][54] Business Line Data and Key Metrics Changes - AYVAKIT's net product revenue for Q1 2024 was $92.5 million, with $83.1 million from the US and $9.4 million from ex-US sales [34][44] - The company is seeing strong and steady growth in patients on AYVAKIT, with a strong pace of new patient starts and low discontinuation rates [34][92] - The mix of commercial versus free goods has skewed favorably, with commercial patients now comprising the majority of those on therapy [56][67] Market Data and Key Metrics Changes - The company is expanding its prescriber base across various specialties, with a notable increase in allergists prescribing AYVAKIT [113] - The international launch of AYVAKIT is off to a robust start, particularly in Germany, contributing to the overall revenue growth [56][108] - The unaided awareness of AYVAKIT among patients has grown nearly eightfold since approval, indicating significant market penetration potential [61][125] Company Strategy and Development Direction - The company is focused on expanding its pipeline in mast cell disorders, with BLU-808 being a key program aimed at addressing various indications beyond chronic urticaria [33][90] - The strategic partnership discussions are ongoing, particularly for the CDK2 program, which is expected to advance in the second half of the year [134] - The company aims to redefine disease control for providers and patients, enhancing awareness and education initiatives to drive growth [37][39] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining growth due to strong patient engagement and educational initiatives [39][92] - The company anticipates continued growth in net patient adds, driven by a strong cadence of new patient starts and favorable compliance rates [72][92] - Management highlighted the importance of building a sustainable business model while focusing on long-term value creation [119] Other Important Information - The company is preparing to file the IND for BLU-808 to initiate a Phase 1 study in healthy volunteers [33][122] - The company is also advancing its oncology portfolio, with plans to present data on BLU-222 at the upcoming ASCO meeting [43][112] Q&A Session Summary Question: What is the split between allergists and hematologists prescribing AYVAKIT? - Management noted a strong foundation in prescribing across both hematology and allergy immunology, with a balanced mix of prescribers [12][13] Question: What are the assumptions behind the updated guidance? - Management clarified that the guidance reflects strong growth expectations, with no anticipated slowdown in net patient adds [16][72] Question: How is the company addressing the potential patient population not currently being prescribed AYVAKIT? - The company is focused on identifying patients who are not well-controlled and increasing awareness through direct-to-consumer efforts [14][37] Question: What is the strategy for expanding into new indications for BLU-808? - The company plans to establish proof of concept in chronic urticaria before exploring additional indications based on the drug's profile [24][122] Question: How is the company measuring the success of its direct-to-consumer campaign? - Management highlighted the importance of patient storytelling and educational initiatives to enhance awareness and engagement [150][151]

First Quarter 2024 Financial Results Agenda AYVAKIT PERFORMANCE | --- | --- | --- | --- | --- | |-------|-------|-------------------------|-------|-------| | | | | | | | | | | | | | | | | | | | | | Q1 2024 FINANCIAL | | | | | | PERFORMANCE | | | | | | | | | | | | | | | | | | Mike Landsittel | | | | | | Chief Financial Officer | | | 2 3 INTRODUCTION Kate Haviland Chief Executive Officer THE POWERFUL MAST CELL Not for promotional use Forward-looking statements This presentation contains forward-looking statem ...

Blueprint Medicines (BPMC) came out with a quarterly loss of $1.32 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to loss of $2.15 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 19.51%. A quarter ago, it was expected that this cancer drug developer would post a loss of $2.04 per share when it actually produced a loss of $1.82, delivering a surprise of 10.78%.Over the last four quarters, the co ...

Blueprint Medicines Reports Strong First Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance -- Raising guidance to $390 million to $410 million in full year AYVAKIT net product revenues -- -- Strengthening presence in allergy and inflammation with the IND for wild-type KIT inhibitor BLU-808 on track for filing in Q2 -- "We delivered another very strong quarter in our launch of AYVAKIT in indolent systemic mastocytosis (ISM) and have entered 2024 in a position of stren ...

-- Achieved $92.5 million in AYVAKIT net product revenues in the first quarter 2024 -- -- Raising guidance to $390 million to $410 million in full year AYVAKIT net product revenues -- -- Strengthening presence in allergy and inflammation with the IND for wild-type KIT inhibitor BLU-808 on track for filing in Q2 -- CAMBRIDGE, Mass., May 2, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today reported financial results, provided a business update for the first quarter ended March 31, 202 ...

­-- Dr. Mariana Castells, M.D., Ph.D., a renowned researcher and clinical expert in mast cell diseases, will join members of Blueprint Medicines' management team to provide a perspective on the mast cell as a promising target for treating allergic and inflammatory disease on April 25, 2024, at 10:00 a.m. ET -- -- First in a series of planned webcasts hosted by Blueprint to highlight evolving science around R&D and pipeline strategy -- CAMBRIDGE, Mass., April 11, 2024 /PRNewswire/ -- Blueprint Medicines Corp ...

-- AYVAKIT data in patients with indolent systemic mastocytosis show durable efficacy and favorable safety supporting long-term treatment, consistent with real-world experience observed in commercial setting -- -- Preclinical data for BLU-808, a highly selective and potent oral wild-type KIT inhibitor with best-in-class potential, supports development in chronic urticaria and other mast cell diseases; on track to submit IND to FDA in Q2 2024 -- -- Breadth of data across 9 presentations, including 2 oral, hi ...