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Safety: BLU-808 was well-tolerated at all doses tested. All treatment-emergent adverse events (AEs) in the MAD cohorts [1-12 mg once daily (QD)] in those who received BLU-808 were Grade 1. There were no serious AEs, no discontinuations or dose modifications due to AEs, and no significant changes in laboratory measures.

Pharmacokinetics: BLU-808 showed a half-life of approximately 40 hours, enabling once-daily dosing, and consistent, dose-dependent increases in drug exposure. In the MAD cohorts, all BLU-808 doses led to sustained target coverage, with mean plasma concentrations exceeding predicted KIT IC₅₀ levels at ≥1 mg QD and IC₉₀ levels at ≥3 mg QD.

Pharmacodynamics: BLU-808 showed dose-dependent serum tryptase responses, reflecting evidence of mast cell target engagement across multiple dose levels. In the SAD cohorts, reductions in tryptase were observed after a single dose of BLU-808. In the MAD cohorts, rapid, robust and sustained reductions in tryptase were observed, with reductions below the lower limit of quantification (LLOQ) at multiple dose levels.

^a One participant in the 12 mg cohort had undetectable tryptase levels at baseline and was not included in the tryptase analysis.

"These Phase 1 data support the best-in-class potential of BLU-808, which was designed to achieve unique potency and selectivity enabling a tunable treatment approach and optimization of benefit-risk across a diverse set of mast cell-driven diseases," said Percy Carter, Ph.D., Chief Scientific Officer at Blueprint Medicines. "The results show that BLU-808 performed consistently, with dose-dependent outcomes, including rapid, robust and sustained reductions in serum tryptase across a range of doses, which reinforce the potential for tunable treatment. Based on these positive data, we are initiating proof-of-concept studies in chronic urticaria, allergic asthma, allergic rhinitis, allergic conjunctivitis and mast cell activation syndrome to characterize BLU-808's broad therapeutic potential across multiple diseases where mast cells play a core role in the disease biology."

Additional Pipeline Updates

Blueprint continues to evaluate programs across the company's diverse pipeline and prioritize investments in the most compelling programs with first- or best-in-class potential. Blueprint today announced the following updates:

• With a focus driving continued innovation and extending the longer-term lifecycle of the company's SM franchise, Blueprint has initiated the registration-enabling Phase 3 HARBOR trial of elenestinib, a next-generation KIT D816V inhibitor, in patients with indolent systemic mastocytosis (ISM).

• Blueprint is advancing CDK2 and CDK4 targeted protein degraders, which have progressed faster than expected in preclinical development toward potentially best-in-class profiles and is prioritizing further investment in its CDK franchise for breast cancer and other solid tumors to these programs. Blueprint is completing the Phase 1 dose escalation study of its CDK2 inhibitor BLU-222 and plans to de-prioritize further investment in this program. The company continues to engage strategic partners on potential opportunities to broadly advance its franchise of CDK programs.

2025 Corporate Goals

Grow franchise leadership in SM

• Deliver continued strong and steady AYVAKIT revenue growth in 2025

• Present additional long-term data from the PIONEER trial of AYVAKIT in ISM in the first half of 2025

• Achieve reimbursement of AYVAKYT in ≥20 countries overall by the end of 2025

• Activate sites and drive patient enrollment of the Phase 3 HARBOR trial of elenestinib in ISM throughout 2025

Achieve BLU-808 clinical proof-of-concept in allergic and inflammatory diseases

• Present topline results from the Phase 1 healthy volunteer trial at the J.P. Morgan conference on January 13, 2025

• Initiate proof-of-concept trials in chronic spontaneous urticaria, chronic inducible urticaria, allergic rhinitis and allergic conjunctivitis in the first half of 2025

• Initiate proof-of-concept trials in allergic asthma and mast cell activation syndrome in the second half of 2025

Drive research innovation in allergy/inflammation and oncology/hematology

• Nominate two development candidates, including the company's first targeted protein degrader, in the second half of 2025

J.P. Morgan Healthcare Conference Presentation Information

Kate Haviland, Chief Executive Officer of Blueprint Medicines, will present a company overview and 2025 outlook at the 43^rd Annual J.P. Morgan Healthcare Conference on Monday, January 13 at 9:00 a.m. PT (12:00 p.m. ET). A live webcast of the presentation and Q&A session will be available by visiting the "Events and Presentations" section of Blueprint Medicines' website at http://ir.blueprintmedicines.com. A replay of the webcast will be archived on Blueprint Medicines' website for 30 days following the presentation.

About Blueprint Medicines

Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit www.BlueprintMedicines.com and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Blueprint Medicines' views with respect to the peak systemic mastocytosis franchise and AYVAKIT revenue opportunities; the continued growth of the AYVAKIT launch in the U.S. and in other countries; the expansion of Blueprint Medicines' mast cell disease franchise with the development of elenestinib and BLU-808; the advancement of BLU-808 and targeted protein degrader research programs for CDK2; the potential for BLU-808 to address allergic and inflammatory diseases;  plans to continue the Phase 3 portion of the HARBOR trial of elenestinib; plans, strategies, timelines, expectations and potential benefits for Blueprint Medicines' current or future approved drugs and drug candidates and in treating patients; and Blueprint Medicines' financial performance, strategy, including the prioritization of its capital resources, goals and anticipated 2025 corporate milestones, business plans, outlook and focus. The words "aim," "goals" "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, the risk that the market opportunities for systemic mastocytosis, including AYVAKIT (avapritinib), or other Blueprint Medicines drug candidates are smaller than the company estimates or that any approval it obtains may be based on a narrower definition of the patient population than it anticipates; the marketing and sale of AYVAKIT (avapritinib) or any future approved drugs may be unsuccessful or less successful than anticipated, or that AYVAKIT (avapritinib) may not gain market acceptance by physicians, patients, third-party payors and others in the medical community; the risk of delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; risks related to Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; preclinical and clinical results for Blueprint Medicines' drug candidates may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates may be delayed or slower than anticipated; actions of regulatory agencies may affect its approved drugs or its current or future drug candidates, including affecting the initiation, timing and progress of clinical trials; risks related to Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for its products and current or future drug candidates it is developing; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing and other arrangements; risks related to its liquidity and financial position, including the risk that Blueprint Medicines may be unable to generate sufficient future product revenues to achieve and maintain a self-sustainable financial profile; and the accuracy of its estimates of revenues, expenses and capital requirements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.

Trademarks

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.

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SOURCE Blueprint Medicines Corporation