On May 1, 2025 Blueprint Medicines Corporation (Nasdaq: BPMC) reported financial results, provided a business update for the first quarter ended March 31, 2025, and provided corporate updates (Press release, Blueprint Medicines, MAY 1, 2025, View Source [SID1234652428]).

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"Blueprint Medicines strives to be a top-tier standout in biotech, with a core focus on innovation, commercial excellence, and a disciplined approach to global investment across our portfolio. Following strong performance in 2024, we have continued our executional momentum in 2025," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "AYVAKIT is well on its way to meeting our goal of $2 billion in revenue by 2030, as we continue to capture the substantial and growing multi-billion-dollar systemic mastocytosis opportunity that we anticipate will drive topline revenue growth into the next decade. We also advanced our prioritized pipeline programs, achieving significant portfolio milestones, including the initiation of two proof-of-concept studies for wild-type KIT inhibitor BLU-808 and advancing the HARBOR study of elenestinib in indolent systemic mastocytosis. The combination of our durable and growing commercial revenue, our strong cash position, and our disciplined capital allocation strategy enables us to focus on executing our business to plan and insulates us from broader market volatility."

First Quarter 2025 Highlights and Recent Progress

● Achieved AYVAKIT net product revenues of $149.4 million for the first quarter of 2025, including $129.4 million in the US and $20 million ex-US, representing 61% percent growth year-over-year.
● Initiated two clinical proof-of-concept studies of BLU-808, a highly selective and potent investigational oral wild-type KIT inhibitor for the treatment of mast cell disorders, including:
o A randomized, double-blind, placebo-controlled Phase 2a challenge study of BLU-808 in patients with allergic rhinoconjunctivitis; and
o A Phase 2a proof-of-concept study in chronic urticaria. This study comprises an open-label portion in chronic inducible urticaria and a randomized, double-blind, placebo-controlled portion in chronic spontaneous urticaria.
● Presented 12 poster and two oral data presentations at the American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) conference. The breadth of data included three-year long-term follow-up data from the PIONEER study of AYVAKIT in patients with indolent systemic mastocytosis (ISM), data showing AYVAKIT-treated patients with ISM achieved improvements in bone health, and data from the healthy volunteer study of BLU-808. Read the presentations here.
● Strengthened cash balance with $78.7 million in connection with the sale of the company’s equity investment in IDRx, Inc. following its acquisition by GSK plc.
● Hosting scientific seminar on mast cell activation syndrome (MCAS) with expert physician Dr. Matt Giannetti on Wednesday, June 4, 2025 at 1:00 p.m.

2025 Financial Guidance

Blueprint Medicines is raising guidance and now anticipates approximately $700 million to $720 million in global AYVAKIT net product revenues in 2025, on the path to achieving $2 billion in global AYVAKIT net product revenues

by 2030. This guidance increase reflects favorability observed in the free versus commercial mix of AYVAKIT sales in the first quarter and continued strength in underlying fundamentals of growth. Blueprint continues to expect a year-over-year reduction in cash burn in 2025, as it continues to invest in advancing its prioritized programs, balancing investments in innovation with financial discipline. Blueprint continues to anticipate that its existing cash, cash equivalents and investments, together with anticipated product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following remaining milestones in 2025:

Mast cell disorders

● Deliver continued strong and steady AYVAKIT revenue growth.
● Achieve reimbursement of AYVAKYT in ≥ 20 countries overall.
● Activate sites and drive enrollment in HARBOR trial of elenestinib.
● Initiate proof of concept studies of BLU-808 in allergic asthma and MCAS.

Discovery

● Nominate two development candidates, including the company’s first protein degrader.

First Quarter 2025 Results

● Revenues: Revenues were $149.4 million for the first quarter of 2025, generated by net product sales of AYVAKIT/AYVAKYT. Revenues were $96.1 million in the first quarter of 2024, including $92.5 million of net product revenues from sales of AYVAKIT/AYVAKYT and $3.6 million in collaboration revenues.
● Cost of Sales: Cost of sales was $2.8 million for the first quarter of 2025, as compared to $3.2 million for the first quarter of 2024. The decrease was primarily due to lower sales to our collaboration partner offset by an increase in product sales volume.
● R&D Expenses: Research and development expenses were $91.9 million for the first quarter of 2025, as compared to $88.2 million for the first quarter of 2024. This increase was primarily due to the increased investment in our priority programs to advance the associated clinical trials. Research and development expenses included $12.1 million in stock-based compensation expenses for the first quarter of 2025.
● SG&A Expenses: Selling, general and administrative expenses were $95.8 million for the first quarter of 2025, as compared to $83.6 million for the first quarter of 2024. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $16.9 million in stock-based compensation expenses for the first quarter of 2025.
● Net Income: Net income was $0.5 million for the first quarter of 2025, as compared to a net income of $89.1 million for the first quarter of 2024. The net income for the first quarter of 2025 was primarily driven by a one-time net gain of $50.0 million recorded in connection with the sale of the company’s equity investment in IDRx, Inc. following its acquisition by GSK plc. The net income for the first quarter of 2024 was primarily driven by a one-time non-cash debt extinguishment gain of $173.7 million recorded in connection with the Royalty Pharma termination agreement.
● Cash Position: As of March 31, 2025, cash, cash equivalents and investments were $899.8 million, as compared to $863.9 million as of December 31, 2024.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss first quarter 2025 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international) and referring conference ID 082088. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source 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.

Upcoming Investor Conferences

Blueprint Medicines will participate in two upcoming investor conferences:

● Citizens JMP Life Science Conference on Wednesday, May 7, 2025 at 12:00 p.m. ET.
● Goldman Sachs 46th Annual Global Healthcare Conference on Wednesday, June 11, 2025 at 10:40 a.m. ET.

Scientific Webinar Series

● Blueprint Medicines will host the third event in its scientific seminar series, focused on mast cell activation syndrome (MCAS), on Wednesday, June 4, 2025 at 1:00 p.m. ET.

A live webcast of the above presentations and any related slides will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source A replay of the webcasts will be archived on the Blueprint Medicines website following the events.