Blueprint Medicines Corporation is a global, fully-integrated biopharmaceutical company that invents life-changing medicines.
The company seeks to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. The company’s approach targets the root causes of disease, using deep scientific knowledge in the company’s core focus areas and drug discovery expertise across multiple therapeutic modalities.
The company has a trac...
Blueprint Medicines Corporation is a global, fully-integrated biopharmaceutical company that invents life-changing medicines.
The company seeks to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. The company’s approach targets the root causes of disease, using deep scientific knowledge in the company’s core focus areas and drug discovery expertise across multiple therapeutic modalities.
The company has a track record of success with two approved medicines, including AYVAKIT/AYVAKYT (avapritinib), which the company is bringing to patients with systemic mastocytosis (SM) and PDGFRA Exon 18 mutant GIST in the U.S. and Europe. Leveraging the company’s established research, development, and commercial capability and infrastructure, the company intends to significantly scale its 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.
Since 2011, the company has advanced a drug discovery approach that combines evolving biological insights with the company’s proprietary research platform and drug design capabilities, which focuses on small molecule inhibitors and targeted protein degraders. The company intends to rapidly and reproducibly translate science into durable clinical benefit for broad populations of patients with significant medical needs, including patients with mast cell diseases, breast cancer and other solid tumors. The company’s focused business model integrates its research engine with robust clinical development and commercial capabilities in allergy/inflammation and solid tumors to create a sustainable cycle of innovation.
Mast Cell Diseases — AYVAKIT/AYVAKYT (avapritinib), Elenestinib (BLU-263), and BLU-808
The company continues to build a mast cell disease franchise, based on its deep understanding of mast cell biology and the KIT pathway. The company is commercializing its first KIT D816V inhibitor, AYVAKIT/AYVAKYT globally for the treatment of advanced SM and indolent SM. The company is developing elenestinib, or BLU-263, an investigational, orally available, potent and highly selective KIT D816V inhibitor, for the treatment of indolent SM. Additionally, the company is advancing BLU-808, a potent and selective wild-type KIT inhibitor, for mast cell diseases, including chronic urticaria allergic rhinitis/allergic conjunctivitis, allergic asthma, and mast cell activation syndrome (MCAS). With AYVAKIT, the company was able to demonstrate that successful inhibition of mutated KIT with a highly potent and specific molecule can result in the first and only approved disease modifying therapy for SM. Through AYVAKIT development, the company has amassed considerable data on mast cell biology and a strong clinical understanding of disease areas connected to mast cell activation, which is helping to drive the company’s efforts to extend its position in SM and expand into other mast cell disorders. The company is doing this by leveraging its deep understanding of mast cell biology to drive scientific innovation; bringing that innovation to patients with the company’s clinical and regulatory know-how; and driving compelling top-line revenue growth through commercial execution.
Systemic Mastocytosis (SM)
SM comprises a spectrum of disease, including indolent and advanced subtypes, with nearly all patients (approximately 95 percent) having a KIT D816V mutation as the underlying driver of the disease. Indolent SM, which is the most common form of SM, is characterized by often severe, unpredictable and debilitating symptoms due to mast cell activation. Symptoms may include unpredictable hypersensitivity reactions, including anaphylaxis, gastrointestinal distress including severe nausea, vomiting and diarrhea, and extensive skin rashes that cause pain, discomfort and social isolation. Advanced SM is a rarer form of SM associated with mast cell infiltration of organ systems resulting in an increasingly severe impact on life expectancy, and includes three subsets: aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). These advanced forms of SM have historically had a median overall survival (OS) of less than six months to 3.5 years and are characterized by prominent organopathy and dysfunction, as well as the debilitating symptoms of mast cell activation.
The current treatment paradigm for SM varies by disease subtype. There are no approved therapies other than AYVAKIT designed to potently and selectively inhibit the KIT D816V mutation. There are two approved therapies for advanced SM: midostaurin and imatinib. Midostaurin is a multi-kinase inhibitor with limited KIT D816V inhibitory activity. Imatinib is approved only for patients with the ASM subtype who do not harbor the KIT D816V mutation, or who have an unknown mutation status. Other treatments used in advanced SM include interferon alpha or cytoreductive agents to reduce mast cell burden, or treatments aimed at addressing the associated blood disorder.
For patients with indolent SM, AYVAKIT/AYVAKYT is the only approved therapy in the U.S. and EU. Beyond AYVAKIT, management is symptom-directed and includes avoidance of triggers of mast cell activation (such as insect stings). Common off-label treatments for indolent SM include histamine blockers, cromolyn, epinephrine, corticosteroids, and, in cases of refractory patients, cytoreductive agents. Patients often take multiple symptom-directed treatments to manage their disease, and a reduction in polypharmacy burden is an important treatment goal.
AYVAKIT/AYVAKYT (avapritinib)
The company is commercializing avapritinib for the treatment of advanced SM and indolent SM. The FDA approved avapritinib under the brand name AYVAKIT for the treatment of adult patients with advanced SM, including ASM, SM-AHN, and MCL in June 2021, and for adult patients with indolent SM in May 2023. In March 2022, the European Commission approved the marketing authorization for AYVAKYT for the treatment of adult patients with ASM, SM-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 the company’s Phase 1 clinical trial in advanced SM, which the company refers to as its EXPLORER trial, and the company’s ongoing registrational clinical trial in advanced SM, which the company refers to as its PATHFINDER trial. The approvals of AYVAKIT for the treatment of patients with indolent SM, were supported by data from the company’s ongoing Phase 2/3 clinical trial in indolent SM, which the company refers to as the PIONEER trial. At the European Academy of Allergy and Clinical Immunology (EAACI) Congress May/June 2024, the company presented long-term data from PIONEER, demonstrating that with a median follow-up of more than two years, AYVAKIT showed durable efficacy and a favorable safety profile in patients with indolent SM, and that safety data were consistent for the small number of patients who doses escalated to 50 mg once daily.
The FDA has granted breakthrough therapy designation to avapritinib for (i) the treatment of advanced SM, including the subtypes of ASM, SM-AHN and MCL, and (ii) the treatment of moderate to severe indolent SM. In addition, the FDA has granted orphan drug designation to avapritinib for the treatment of mastocytosis, and the European Commission has granted orphan medicinal product designation to avapritinib for the treatment of mastocytosis.
Avapritinib is also approved in the U.S. under the brand name AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and is approved in the EU, the U.K. and Switzerland with conditional marketing authorization under the brand name AYVAKYT as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring a PDGFRA D842V mutation. AYVAKIT is the only FDA-approved treatment for patients with D842V mutant PDGFRA-driven GIST. Through the company’s collaboration with CStone, China’s National Medicinal Products Administration approved AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT also received accelerated approval from the Taiwan Food and Drug Administration and approval in Hong Kong, both for adults with unresectable or metastatic GIST harboring PDGFRA D842V mutations. The company also has distributor arrangements to commercialize AYVAKIT in global jurisdictions, including Israel and Canada, where approvals have been received. As of December 31, 2024, AYVAKIT/AYVAKYT was approved and reimbursed for one or more indications in 15 countries, where the company or its distribution partners are commercializing it. The FDA has granted breakthrough therapy designation for avapritinib for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation. In addition, the FDA has granted orphan drug designation to avapritinib for the treatment of GIST, and the European Commission has granted orphan medicinal product designation to avapritinib for the treatment of GIST.
Avapritinib Clinical Data in SM
Registrational PIONEER Trial in Indolent SM
PIONEER is a randomized, double-blind, placebo-controlled, registration-enabling trial evaluating avapritinib in patients with indolent SM. The trial includes three parts: dose-finding Part 1, registration-enabling Part 2 and long-term treatment Part 3. All patients who completed Parts 1 or 2 had an opportunity to receive treatment with avapritinib in Part 3. Key trial endpoints include the change in patient-reported disease symptoms as measured by the Indolent SM Symptom Assessment Form Total Symptom Score, or ISM-SAF TSS, quantitative measures of mast cell burden and safety.
In June 2024, the company presented data at EAACI and in February 2024, the company presented data at American Academy of Allergy, Asthma, and Immunology (AAAAI) Annual Meeting. In February 2023 the company presented fulsome results from PIONEER Part 2 at the AAAAI Annual Meeting and in August 2022 the company presented top line data from PIONEER Part 2.
Data Presented at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Meeting in June 2024
The company presented long-term data from PIONEER, demonstrating that with a median follow-up of more than two years, AYVAKIT showed durable efficacy and a favorable safety profile in patients with indolent SM, and that safety data were consistent for the small number of patients who dose escalated to 50 mg once daily.
Data Presented at the AAAAI Annual Meeting in February 2024
The company presented long-term data from PIONEER, demonstrating that AYVAKIT had a durable symptom impact and a well-tolerated safety profile, supporting long-term treatment and consistent with real-world experience observed in the commercial setting.
Data Presented at the AAAAI Annual Meeting in February 2023
In the randomized, double-blind, placebo-controlled part of the PIONEER trial, 141 patients received AYVAKIT 25 mg once daily plus best supportive care and 71 patients received placebo plus best supportive care (placebo) at 49 sites in 13 countries. The study included adults with an indolent SM diagnosis confirmed by central pathology review, and moderate-to-severe symptom burden despite an optimized regimen of best supportive care. All patients were able to continue symptom-directed therapy throughout the trial and, following completion of the 24-week treatment period, had the option to receive AYVAKIT in an open-label extension study. Baseline patient demographics were balanced between treatment arms and reflected significant disease burden. Disease symptoms were assessed using the Indolent SM Symptom Assessment Form (ISM-SAF). Results were reported as of a data cutoff date of June 23, 2022.
Clinical Activity Data. Part 2 of the PIONEER study showed clinically meaningful and highly significant improvements across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of disease burden. For the avapritinib arm relative to the control arm, the trial achieved the primary endpoint with a highly significant difference in the mean change in TSS at 24 weeks (p=0.003). The avapritinib arm had a reduction of 15.6 points in mean TSS at 24 weeks, which continued to deepen to 20.2 points at 48 weeks in patients who rolled over to the Part 3 open-label extension study. At 24 weeks, the control arm had a reduction of 9.2 points in mean TSS. In addition, the trial met all key secondary endpoints, including significant improvements across measures of mast cell burden. More than half of AYVAKIT-treated patients had a greater than or equal to 50 percent reduction of serum tryptase, compared to no patients in the control arm (53.9% vs. 0%; p<0.0001).
Safety Data. Avapritinib had a favorable safety profile compared to the control arm. The rate of adverse events (AEs) was 90.8 percent in the avapritinib arm and 93.0 percent in the control arm. Serious AEs occurred in 5.0 percent of avapritinib-treated patients, compared to 11.3 percent of patients in the control arm. Discontinuations due to treatment-related AEs occurred in 0.7 percent of avapritinib-treated patients and 0 percent of patients in the placebo arm. The avapritinib arm had a lower rate of cognitive AEs than the control arm - 2.8% avapritinib vs. 4.2% control – and there were no intracranial bleeding events. Treatment-related AEs reported in at least three patients in either arm and at least 5 percent of avapritinib-treated patients included headache, nausea, peripheral edema and periorbital edema.
Elenestinib (BLU-263)
The company is developing elenestinib an investigational, orally available, potent and highly selective KIT inhibitor, for the treatment of indolent SM and other mast cell disorders. Elenestinib is designed to have equivalent potency as avapritinib, with low off-target activity and minimal penetration of the central nervous system relative to avapritinib based on preclinical data.
The company is evaluating elenestinib in an ongoing Phase 2/3 clinical trial in indolent SM, which the company refers to as its HARBOR trial. In December 2022, the company announced top-line, 12-week data from the dose-finding Part 1 of the HARBOR trial. In December 2023, the company presented HARBOR Part 1 trial data for elenestinib in indolent SM at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition. The company initiated the registration-enabling Phase 3 HARBOR trial of elenestinib in indolent SM in the fourth quarter of 2024.
Elenestinib Clinical Data in Indolent SM
Phase 2/3 HARBOR Trial in Indolent SM
HARBOR is a randomized, double-blind, placebo-controlled trial evaluating elenestinib in patients with indolent SM. The trial includes multiple parts, including dose-finding Part 1, registration-enabling Part 2 and long-term treatment Part 3. All patients who complete Parts 1 or 2 will have an opportunity to receive treatment with elenestinib in Part 3. Key trial endpoints include the change in patient-reported disease symptoms as measured by the ISM-SAF TSS, quantitative measures of mast cell burden and safety.
Data Presented at the ASH Annual Meeting in December 2023
Part 1 of the HARBOR trial enrolled 29 patients who received elenestinib plus best available care, including 10 patients at 25 mg once daily, 10 patients at 50 mg once daily and nine patients at 100 mg once daily, and 10 patients who received placebo plus best available care. Baseline patient and disease characteristics were similar to those reported for the general ISM population. Results were reported as of a data cutoff date of October 17, 2022.
Clinical Activity Data. 12-week results from Part 1 of the HARBOR study showed elenestinib treatment led to rapid improvements in patient-reported symptoms, or TSS as measured by the ISM-SAF, as well as objective measures of mast cell burden. The elenestinib arms saw a mean percent reduction from baseline TSS of 28.5% at 25 mg, 31.8% at 50 mg, and 33.6% at 100 mg at 12 weeks, compared to a mean percent reduction from baseline TSS of 22.2% in the control arm. In addition, patients receiving elenestinib at doses of 25 mg, 50 mg, and 100 mg demonstrated dose-dependent mean percent reductions from baseline in serum tryptase levels, KIT D816V variant allele fraction (VAF), and bone marrow mast cells versus placebo.
Safety Data. Elenestinib safety results were consistent with its preclinical profile and a completed Phase 1 healthy volunteer trial. Elenestinib was generally well-tolerated at all dose levels with most AEs reported as Grade 1–2, and there were no discontinuations due to AEs. At the time of data cut-off, median treatment duration was 22 weeks and there were no grade 4 or 5 AEs, no treatment-related SAEs, no AEs that led to drug discontinuation, and all patients were still on treatment.
BLU-808
In the first half of 2023, the company nominated the development candidate BLU-808 from its discovery programs, an oral, highly potent and selective wild-type KIT inhibitor. The company is developing BLU-808 as a potential first- and best-in-class treatment for mast cell disorders, including chronic urticaria, a debilitating inflammatory skin disorder characterized by wheals (hives), and sleep disruption, stress and anxiety due to severe itching are major contributors to disease burden. Wild-type KIT inhibition has an established proof-of-concept in chronic urticaria, and BLU-808 represents a small molecule approach with the opportunity to drive market expansion with an oral regimen. Beyond chronic urticaria, the company plans to initiate proof of concept studies in other related allergic-inflammatory indications, including but not limited to allergic rhinitis/allergic conjunctivitis, allergic asthma, and MCAS.
In February 2024, at the AAAAI Annual Meeting, the company presented the preclinical attributes of BLU-808 that demonstrate its potency, selectivity, low potential for drug-drug interactions, and peripheral restriction. BLU-808 treatment led to dose-dependent inhibition and depletion of mast cells in multiple in vivo studies, and also improved lung function in an ovalbumin-induced asthma model. In June 2024, the company submitted an Investigational New Drug (IND) application to FDA for BLU-808. The company received FDA clearance to proceed with a Phase 1 study in healthy volunteers and initiated that study in the third quarter of 2024.
Data Presented at the J.P. Morgan Conference in January 2025
In January 2025, the company presented results from the Phase 1 single-ascending dose (SAD; n=56) and multiple-ascending dose (MAD; n=31, 14-day dosing) trial of BLU-808, a highly potent and selective oral KIT inhibitor, in healthy volunteers at the J.P. Morgan Healthcare Conference.
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 clinically significant changes in laboratory measures.
Pharmacokinetics: BLU-808 showed a half-life of approximately 40 hours, enabling once-daily dosing, and consistent, dose-proportional 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 IC88 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, exceeding 60 percent at 42 mg. In the MAD cohorts, rapid, robust and sustained reductions in tryptase were observed, with reductions exceeding 80 percent at 12mg and below the lower limit of quantification (LLOQ) at multiple dose levels.
Oncology/Hematology
The company’s oncology research program has delivered a number of innovative therapies and continues to be an active area of discovery. Based on early clinical success, the company plans to further advance discovery research in oncology in 2025.
Cell Cycle Inhibition Programs
The company is advancing multiple therapeutic candidates and research programs targeting the cell cycle as potential treatments for patients with hormone-receptor-positive/human epidermal growth receptor 2 negative (HR+/HER2-) breast cancer and other solid tumors. These include CDK2 and CDK4 targeted protein degraders, which have progressed rapidly in preclinical development toward potentially best-in-class development candidate profiles supporting the company’s prioritization of these programs. The company is completing the Phase 1 dose escalation study of its CDK2 inhibitor BLU-222 and are de-prioritizing any further investment in this program. The company continues to engage strategic partners on potential opportunities to broadly advance the company’s franchise of CDK programs.
Discovery Platform
The company continues to drive organic growth with its highly productive research platform, which has nominated 17 development candidates to date. With drug design capabilities spanning small molecule inhibitors and targeted protein degraders, the company’s approach begins by choosing the best modality for the targets the company is pursuing and designing highly potent and selective therapeutic candidates. Within the company’s focus areas of allergy/inflammation and oncology/hematology, the company pursue targets where the biology is clear and there is opportunity to impact large patient populations. The company consistently intends to achieve first- or best-in-class profiles with the potential to disrupt the current standard of care and dramatically improve patient outcomes. In addition, the company prioritizes opportunities where early data can de-risk future investment and the company deeply integrates the insights and capabilities of the company’s R&D and commercial functions to create significant and sustainable growth opportunities.
Collaborations, Licenses and Other Agreements Summary
Roche—Pralsetinib Collaboration. In July 2020, the company entered into a collaboration agreement with Roche, which the company refers to as the Roche pralsetinib collaboration, to develop and commercialize pralsetinib for the treatment of RET-altered cancers. Under the Roche pralsetinib collaboration, the company and Genentech co-commercialized GAVRETO in the U.S., and Roche was granted exclusive commercialization rights for pralsetinib outside of the U.S., excluding Mainland China, Hong Kong, Macau and Taiwan (each a CStone region and, collectively, the CStone Territory). In February 2023, the company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination became effective on February 22, 2024, at which time the company entered into a transition agreement with Roche (the Roche transition agreement) and sold the U.S. rights to research, develop, manufacture and commercialize pralsetinib to Rigel Pharmaceuticals (Rigel). In January 2024, the company decided to discontinue global development and marketing of GAVRETO in territories excluding the U.S. and CStone Territory, due to a lack of an alternate partner in these regions. The company continues to work with Roche on the transition and wind-down activities contemplated in the Roche transition agreement.
CStone. In June 2018, the company entered into a collaboration with CStone to develop and commercialize avapritinib, pralsetinib and fisogatinib, as well as any back-up and other forms thereof, in the CStone Territory either as a monotherapy or as part of a combination therapy.
Clementia. In October 2019, the company entered into a license agreement with Clementia, which the company refers to as the Clementia license agreement. Pursuant to the Clementia license agreement, the company granted Clementia an exclusive, worldwide, royalty-bearing license to develop and commercialize BLU-782, as well as specified other compounds related to the BLU-782 program. BLU-782 is an investigational, orally available, potent and highly selective inhibitor that targets mutant activin-like kinase 2 (ALK2) in development for the treatment of fibrodysplasia ossificans progressiva (FOP). The FDA has granted a rare pediatric disease designation, orphan drug designation and fast track designation to BLU-782, each for the treatment of FOP. Clementia has an ongoing Phase 2 clinical trial of BLU-782, now referred to as fidrisertib.
Zai Lab. In November 2021, the company entered into a license and collaboration agreement with Zai Lab, which the company refers to as the Zai Lab agreement, to develop and commercialize certain licensed products for the treatment of EGFR-driven NSCLC in Greater China, including Mainland China, Hong Kong, Macau and Taiwan, which includes BLU-945 and BLU-525. In January 2024 at the J.P. Morgan Healthcare Conference, the company announced that the company is discontinuing further investment in early clinical-stage therapies for EGFR-mutant NSCLC globally; however, Zai Lab retains its rights to BLU-945 and BLU-525 under the Zai Lab agreement.
IDRx. In August 2022, the company entered into a license agreement with IDRx, Inc., or IDRx, which the company refers to as the IDRx License Agreement. Pursuant to the IDRx License Agreement, the company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the company’s internally discovered development candidate-stage KIT exon 13 inhibitor, IDRX-73.
In January 2025, IDRx announced that they had entered into an agreement under which GSK plc will acquire IDRx. The transaction is expected to close in the first quarter 2025.
Strategy
As a global, fully-integrated biopharmaceutical company that invents life-changing medicines, the company seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. The company’s approach targets the root causes of disease, using deep scientific knowledge in the company’s core focus areas and drug discovery expertise across multiple therapeutic modalities. The key elements of the company’s strategy are to:
Accelerate the adoption of the company’s approved medicines, including AYVAKIT/AYVAKYT in the U.S. and Europe, continue to strengthen and expand the company’s global commercial capabilities, and prepare for future potential commercial launches of new investigational medicines.
Deepen the company’s strategic focus on mast cell diseases, including systemic mastocytosis, chronic urticaria, and other diseases implicated by KIT-mediated signaling, by continuing the development of elenestinib and BLU-808.
Advance the company’s innovative research programs, including the company’s CDK2 and CDK4 targeted protein degraders, and the company’s other preclinical programs, rapidly through development as the company continues ongoing discussions for potential strategic partnership.
Expand the company’s focused, differentiated pipeline, with prioritization towards mast cell diseases within allergy/inflammation and solid tumors within oncology/hematology, and continued internal discovery research and innovation, inclusive of targeted protein degradation, as well as opportunities to acquire or in-license complementary technologies or therapies.
Evaluate potential additional collaborations, partnerships and licenses that could maximize the value of the company’s existing programs and allow the company to leverage the expertise of strategic collaborators, partners and licensors, including in additional geographies where the company may not have current operations or expertise.
Maintain a commitment to building a corporate culture centered by the company’s focus on patient needs, science-driven approach to drug development, and organizational strength through the diversity of experience and perspective across the company’s workforce.
Collaborations, Licenses and Other Agreements
Roche
Pralsetinib Collaboration. In July 2020, the company entered into a collaboration agreement with Roche, which the company refers to as the Roche pralsetinib collaboration, to develop and commercialize pralsetinib for the treatment of RET-altered cancers. Under the Roche pralsetinib collaboration, the company and Genentech co-commercialized GAVRETO in the U.S., and Roche was granted exclusive commercialization rights for pralsetinib outside of the U.S., excluding Mainland China, Hong Kong, Macau and Taiwan (each a CStone region and, collectively, the CStone Territory). In February 2023, the company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination became effective on February 22, 2024, at which time the company entered into the Roche transition agreement and sold the U.S. rights to research, develop, manufacture and commercialize pralsetinib to Rigel pursuant to an Asset Purchase Agreement and certain supporting agreements, including a customary transition agreement (such agreements collectively referred to as the Rigel Agreement). In January 2024, the company decided to discontinue global development and marketing of GAVRETO in territories excluding the U.S. and CStone Territory, due to a lack of an alternate partner in these regions. The company continues to work with Roche on the transition and wind-down activities contemplated in the Roche transition agreement.
CStone
On June 1, 2018, the company entered into a collaboration and license agreement, or the CStone agreement, with CStone pursuant to which the company granted CStone exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib, as well as any back-up and other forms thereof, which the company refers to collectively as the licensed products, in the CStone Territory, either as a monotherapy or as part of a combination therapy. The company will retain exclusive rights to the licensed products outside the CStone Territory.
Pursuant to the terms of the CStone agreement, CStone is responsible for conducting all development and commercialization activities in the CStone Territory related to the licensed products. Subject to specified exceptions, during the term of the CStone agreement, each party has agreed that neither it nor its affiliates will conduct specified development and commercialization activities in the CStone Territory related to selective inhibitors of FGFR4, KIT, PDGFRA and RET. In addition, under the CStone agreement, each party has granted the other party specified intellectual property licenses to enable the other party to perform its obligations and exercise its rights under the CStone agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the CStone agreement.
Clementia
On October 15, 2019, the company entered into a license agreement, or the Clementia agreement, with Clementia. Under the Clementia agreement, the company granted an exclusive, worldwide, royalty-bearing license to Clementia to develop and commercialize BLU-782, an oral, highly selective investigational ALK2 inhibitor in clinical development for the treatment of FOP, as well as specified other compounds related to the BLU-782 program, which the company refers to as the Clementia licensed products.
Under the terms of the Clementia agreement, the company was responsible for specified activities during a transition period, which has been completed, and Clementia is responsible for conducting all development and commercialization activities related to the Clementia licensed products, including the design, timing and conduct of any Phase 2 clinical trial evaluating BLU-782 for the treatment of FOP.
Zai Lab
On November 8, 2021, the company entered into a license and collaboration agreement, or the Zai Lab agreement, with Zai Lab. Under the Zai Lab agreement, the company granted an exclusive license for the development and commercialization of BLU-945 and BLU-525, including any back-up and other forms thereof, for the treatment of EGFR-driven NSCLC in Greater China, including Mainland China, Hong Kong, Macau and Taiwan, either as a monotherapy or as part of a combination therapy. The company retains exclusive rights to the licensed products outside the Zai Lab territory.
IDRx
In August 2022, the company entered into the IDRx License Agreement, pursuant to which the company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the company’s internally discovered KIT exon 13 inhibitor, IDRX-73. IDRx is a clinical-stage biopharmaceutical company.
Intellectual Property
The company has patent rights that are the subject of the U.S. and foreign patents and patent applications in the U.S. and a number of other jurisdictions, including Australia, Canada, certain Central and South American countries, certain Asian countries (including Greater China), the EU, certain Eurasian countries, certain Middle Eastern countries, New Zealand, and certain African countries. The company’s issued patents and patent applications of the company’s most advanced programs pertain to the company’s approved medicine AYVAKIT and clinical candidates.
The company files trademarks to protect its products. Typically, the company files trademark applications in the U.S., Europe, and elsewhere in the world as appropriate. In addition to multiple pending trademark applications in the U.S. and other major countries, the company has registered trademarks, including but not limited to AYVAKIT in the U.S. and to AYVAKYT in the EU.
The patent portfolios relating to the company’s approved medicines and most advanced programs as of December 31, 2024 are summarized below.
Mast Cell Diseases - AYVAKIT/AYVAKYT (avapritinib), Elenestinib (BLU-263), and BLU-808
The patent portfolios relating to the company’s approved medicines and most advanced programs as of December 31, 2024 are summarized below.
KIT D816V Mutant Program - AYVAKIT/AYVAKYT (avapritinib) and elenestinib
The patent portfolio for the company’s mutant KIT programs contain patents and patent applications directed to compositions of matter for AYVAKIT/AYVAKYT, elenestinib, and other compound families, including solid forms and methods of use and manufacture. As of December 31, 2024, the company owned 16 U.S. patents, 4 European patents, validated in multiple states and 34 other foreign patents and multiple pending patent applications in the U.S., Europe and in various foreign jurisdictions. The patents that have issued or will issue covering AYVAKIT/AYVAKYT and elenestinib will have a statutory expiration date between 2034 and 2043. Patent term adjustments, patent term extensions, and supplementary protection certificates could result in later expiration dates.
In addition, in connection with the company’s FDA approval on January 9, 2020, the FDA granted AYVAKIT new chemical entity, or NCE, exclusivity until January 9, 2025 and Orphan Drug Exclusivity, or ODE, until January 9, 2027. In connection with the company’s FDA approval on June 16, 2021, the FDA granted AYVAKIT new clinical indication exclusivity for two indications until June 16, 2024 and ODE until June 16, 2028. In connection with the company’s FDA approval on May 22, 2023, the FDA granted AYVAKIT new clinical indication exclusivity until May 22, 2026 and ODE until May 22, 2030.
In connection with notification of the company’s EMA approval on September 25, 2020, the EMA granted AYVAKYT Orphan marketing exclusivity until September 25, 2030. Additionally, 1 year of market protection was granted after authorization of new therapeutic indication representing a significant clinical benefit in comparison with existing therapies until September 25, 2031. In connection with notification of the company’s EMA approval on March 25, 2022, the EMA granted AYVAKYT Orphan marketing exclusivity until March 25, 2032. In connection with notification of the company’s EMA approval on December 12, 2023, the EMA granted AYVAKYT Orphan marketing exclusivity December 12, 2033.
Wild-type KIT Program — BLU-808
The patent portfolio for the company’s wild-type KIT program contains patents and patent applications directed to compositions of matter for BLU-808 and other compound families, as well as solid forms and methods of use and manufacture. As of December 31, 2024, the company owned 8 pending patent applications in the U.S., Argentina, and Taiwan. The patents that will issue covering the company’s wild-type KIT program will have a statutory expiration date between 2043 and 2045. Patent term adjustments, patent term extensions, and supplementary protection certificates could result in later expiration dates.
Cell Cycle Inhibition Program - CDK2 Inhibitors (BLU-222, BLU-956) and CDK Protein Degraders
The patent portfolio for the company’s Cell Cycle Inhibition program contains patent applications directed to CDK2 inhibitors and CDK protein degraders, including compositions of matter for BLU-222, BLU-956 and other compound families, as well as solid forms and methods of use and manufacture. As of December 31, 2024, the company owned 2 issued U.S. patents, 1 issued foreign patent and 88 patent applications pending in the U.S., PCT and in various foreign jurisdictions. The patents that will issue covering the company’s Cell Cycle Inhibition program will have a statutory expiration date between 2042 and 2045. Patent term adjustments, patent term extensions, and supplementary protection certificates could result in later expiration dates.
Competition
SM
AYVAKIT/AYVAKYT and elenestinib (BLU-263) face competition for advanced SM from Novartis AG’s midostaurin and imatinib.
Government Regulation
The company’s drug candidates must be approved by the FDA through the NDA process before they may be legally marketed in the U.S.
The company relies on third parties for the production of clinical and commercial quantities of the company’s drugs in accordance with cGMP regulations.
History
The company was founded in 2008. It was incorporated in Delaware in 2008 under the name ImmunoCo, Inc. and changed its name to Hoyle Pharmaceuticals, Inc. in 2010. Further, the company changed its name to Blueprint Medicines Corporation in 2011.
