RegulationFast Track (United States), Orphan Drug (United States), Priority Review (China), Conditional marketing approval (China), Priority Review (United States), Orphan Drug (United Kingdom), Breakthrough Therapy (United States)

Structure/Sequence

Molecular FormulaC26H27FN10

InChIKeyDWYRIWUZIJHQKQ-SANMLTNESA-N

CAS Registry1703793-34-3

Clinical Trials associated with Avapritinib

NCT06765915

/ Not yet recruitingPhase 2IIT

Avapritinib Maintenance Following Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia With KIT Mutations

A multicenter, single-arm clinical study of evaluate the efficacy and safety of avapritinib as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients with KIT mutation.

Start Date01 Feb 2025

Sponsor / Collaborator

Ruijin Hospital

NCT06783790

/ RecruitingPhase 2IIT

An Exploratory Clinical Study of the Safety and Efficacy of Avapritinib Combined With Azacitidine and Venetoclax in the Treatment of Relapsed Acute Myeloid Leukemia After Allogeneic Hematopoietic Stem Cell Transplantation

This is a single-center, prospective, single-arm, exploratory clinical study. To explore the efficacy and safety of avapritinib in patients with recurrent acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation with C-KIT mutation RUNX1::RUNX1T1 or CBFB::MYH11.

Start Date20 Jan 2025

Sponsor / Collaborator

Hematology Hospital of Chinese Academy of Medical Sciences

NCT06748001

/ RecruitingPhase 4

An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study

The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.

Start Date28 Nov 2024

Sponsor / Collaborator

Blueprint Medicines Corp.

100 Clinical Results associated with Avapritinib

100 Translational Medicine associated with Avapritinib

100 Patents (Medical) associated with Avapritinib

261

Literatures (Medical) associated with Avapritinib

01 Apr 2025·JOURNAL OF PATHOLOGY

A deep‐learning model for predicting tyrosine kinase inhibitor response from histology in gastrointestinal stromal tumor

Article

Author: Wu, Can ; Wu, Haibo ; Shi, Jun ; Wang, Wei ; Cheng, Lanqing ; Jiang, Zhiguo ; Sun, Dongdong ; Kong, Xue ; Zheng, Yushan

Abstract:

Over 90% of gastrointestinal stromal tumors (GISTs) harbor mutations in KIT or PDGFRA that can predict response to tyrosine kinase inhibitor (TKI) therapies, as recommended by NCCN (National Comprehensive Cancer Network) guidelines. However, gene sequencing for mutation testing is expensive and time‐consuming and is susceptible to a variety of preanalytical factors. To overcome the challenges associated with genetic screening by sequencing, in the current study we developed an artificial intelligence‐based deep‐learning （DL） model that uses convolutional neural networks (CNN) to analyze digitized hematoxylin and eosin staining in tumor histological sections to predict potential response to imatinib or avapritinib treatment in GIST patients. Assessment with an independent testing set showed that our DL model could predict imatinib sensitivity with an area under the curve (AUC) of 0.902 in case‐wise analysis and 0.807 in slide‐wise analysis. Case‐level AUCs for predicting imatinib‐dose‐adjustment cases, avapritinib‐sensitive cases, and wildtype GISTs were 0.920, 0.958, and 0.776, respectively, while slide‐level AUCs for these respective groups were 0.714, 0.922, and 0.886, respectively. Our model showed comparable or better prediction of actual response to TKI than sequencing‐based screening (accuracy 0.9286 versus 0.8929; DL model versus sequencing), while predictions of nonresponse to imatinib/avapritinib showed markedly higher accuracy than sequencing (0.7143 versus 0.4286). These results demonstrate the potential of a DL model to improve predictions of treatment response to TKI therapy from histology in GIST patients. © 2025 The Pathological Society of Great Britain and Ireland.

01 Apr 2025·MOLECULAR DIVERSITY

Targeting AFP-RARβ complex formation: a potential strategy for treating AFP-positive hepatocellular carcinoma

Article

Author: Dev, Radul R ; Soman, Sowmya ; Banjan, Bhavya ; Raju, Rajesh ; Vishwakarma, Riya ; Rehman, Niyas ; Kalath, Haritha ; Kumar, Pankaj ; Revikumar, Amjesh ; Abhinand, Chandran S ; Ramakrishnan, Krishnapriya

Alpha-fetoprotein (AFP) is a glycoprotein primarily expressed during embryogenesis, with declining levels postnatally. Elevated AFP levels correlate with pathological conditions such as liver fibrosis, cirrhosis, and hepatocellular carcinoma (HCC). Recent investigations underscore AFP's intracellular role in HCC progression, wherein it forms complexes with proteins like Phosphatase and tensin homolog (PTEN), Caspase 3 (CASP3), and Retinoic acid receptors and Retinoid X receptors (RAR/RXR). RAR and RXR regulate gene expression linked to cell death and tumorigenesis in normal physiology. AFP impedes RAR/RXR dimerization, nuclear translocation, and function, promoting gene expression favoring cancer progression in HCC that provoked us to target AFP as a drug candidate. Despite extensive studies, inhibitors targeting AFP to disrupt complex formation and activities remain scarce. In this study, employing protein-protein docking, amino acid residues involved in AFP-RARβ interaction were identified, guiding the definition of AFP's active site for potential inhibitor screening. Currently, kinase inhibitors play a significant role in cancer treatment and, the present study explores the potential of repurposing FDA-approved protein kinase inhibitors to target AFP. Molecular docking with kinase inhibitors revealed Lapatinib as a candidate drug of the AFP-RARβ complex. Molecular dynamics simulations and binding energy calculations, employing Mechanic/Poisson-Boltzmann Surface Area (MM-PBSA), confirmed Lapatinib's stability with AFP. The study suggests Lapatinib's potential in disrupting the AFP-RARβ complex, providing a promising avenue for treating molecularly stratified AFP-positive HCC or its early stages.

02 Jan 2025·Bioanalysis

A green bioanalytical spectrofluorimetric approach for estimation of Avapritinib anti-tumor drug; application to quality control and clinical studies

Article

Author: Hassan, Yasser F. ; Saraya, Roshdy E. ; Al-Harrasi, Ahmed ; Ibrahim, Adel Ehab ; Hassan, Ahmed I. ; Batakoushy, Hany A. ; Abdel-Aal, Mohamed A.A. ; Salman, Baher I.

AIMS:

Gastrointestinal stromal tumors (GISTs) account for about 80% of the mesenchymal tumors of the GI tract. About 5000-6000 patients are diagnosed in the United States (US) alone, and up to 14.5 cases per million discovered in Europe annually. Avapritinib (AVP) is a potent selective targeted medication that has been recently approved, by the US Food and Drug Administration, in 2020 for treatment of GISTs. AVP is currently considered the first-line treatment for mutant GIST, which is resistant to other medications. This in turn stimulates the need for fast, green, and efficient methods for routine AVP estimation in quality control and clinical studies.

MATERIALS AND METHODS:

The proposed approach designs a spectrofluorimetric tool to estimate AVP in different matrices, based on a nucleophilic substitution reaction. A highly fluorescent product was measured at 535 nm following excitation at 470 nm. The research procedure was bioanalytically validated within AVP range between 80 and 900 ng mL^-1, where the limit of quantitation (LOQ) was 15.78 ng mL^-1.

CONCLUSION:

The developed approach was successfully applied to investigate AVP in content uniformity testing of tablet dosage forms, and biological plasma in AVP pharmacokinetic study. The proposed approach could be recommended for AVP therapeutic drug monitoring.

107

News (Medical) associated with Avapritinib

28 Feb 2025

·www.prnewswire.com

Blueprint Medicines Highlights Leading Portfolio of Mast Cell-Targeted Therapies at 2025 AAAAI / WAO Joint Congress

-- Updated data show AYVAKIT® (avapritinib) was safe and effective for long-term treatment of ISM, with sustained symptom and quality-of-life benefit and no new safety signals with multiple years of therapy -- -- AYVAKIT-treated patients achieved improvements in bone health based on an analysis of bone mineral density scans, underscoring the importance of treating patients early in the ISM disease course -- -- BLU-808 healthy volunteer trial results demonstrate wide therapeutic window for modulating mast cell activity, affirming potential as best- and first-in-class oral wild-type KIT inhibitor -- CAMBRIDGE, Mass., Feb. 28, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting its ongoing efforts to innovate the treatment of mast cell-driven diseases. The presentations include three-year median follow-up data from the registrational PIONEER trial demonstrating the strong long-term efficacy and safety of AYVAKIT® (avapritinib) in patients with indolent systemic mastocytosis (ISM), and real-world evidence further characterizing the substantial burden of the disease, highlighting the urgency to treat. In addition, the company will report positive data from the Phase 1 healthy volunteer trial evaluating BLU-808, an investigational, highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line data previously announced in January 2025. A total of 14 data presentations, including two oral presentations, will be reported at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress being held February 28-March 3 in San Diego. "We are incredibly proud to showcase our mast cell therapy portfolio at AAAAI / WAO, with 14 presentations highlighting more than a decade of leadership and collaboration with the mast cell disease community, including renowned medical institutions pioneering innovative research and multi-disciplinary patient care," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "In ISM, new data have reinforced that AYVAKIT was very well-tolerated after multiple years of treatment, with a low discontinuation rate and safety results that have remained consistent with the safety profile from the placebo-controlled study as summarized in the FDA-approval label. This updated AYVAKIT data is particularly important for clinicians considering long-term treatment options for their ISM patients. Further, we are building on our foundational expertise in ISM with our oral wild-type KIT inhibitor BLU-808, with healthy volunteer data showing a differentiated clinical profile and wide therapeutic index that supports broad development across a range of mast cell diseases. Based on these data, we are initiating multiple proof-of-concept trials, with initial data expected later this year." AYVAKIT: PIONEER Three-Year Efficacy and Safety Data in ISM AYVAKIT 25 mg once daily (QD) was well-tolerated and no new safety signals were identified with a median of 3 years of exposure, with some patients out to five years of treatment. The most common treatment-related adverse events (AEs; ≥5 percent) were low-grade peripheral edema, periorbital edema, headache and nausea. AYVAKIT 25 mg QD led to robust and durable improvements in overall symptoms, individual symptom domains (skin, gastrointestinal, neurocognitive) and quality of life through 144 weeks of treatment. In patients who had high disease burden and escalated to AYVAKIT 50 mg QD (median follow-up: 10.6 months since escalation), treatment was well-tolerated, with side effects consistent with those reported at AYVAKIT 25 mg QD and no patients discontinuing due to AEs. Nearly all patients (93 percent) had improved or stable benefits in overall symptoms. Low Bone Density: Positive Clinical Impact of AYVAKIT and Real-World Prevalence in ISM In a single-site case series from the PIONEER trial, AYVAKIT showed clinically meaningful and durable improvements in bone density, including in the lumbar spine, femoral neck and total proximal femur. In real-world data generated from Mayo Clinic electronic health records, 67 percent of ISM patients had osteoporosis/osteopenia compared to 34 percent in a matched control cohort (p<0.0001). AYVAKIT: Clinical Benefits in Patients with Undetected KIT D816V by ddPCR Assay In the PIONEER trial, 15 percent of patients with ISM did not have KIT D816V mutations detected by the commercially available Droplet Digital Polymerase Chain Reaction (ddPCR) assay. Within this group, most patients had KIT mutations identified by the more sensitive duplex sequencing method, and these patients achieved similar reductions in serum tryptase and improvements in symptoms as those with detectable KIT mutations by the ddPCR assay. BLU-808: Positive Healthy Volunteer Data In the Phase 1 single-ascending dose (SAD) and multiple-ascending dose (MAD, 14-day dosing) trial, BLU-808 was well-tolerated at all doses tested. There were no serious AEs, no discontinuations or dose modifications due to AEs, and no significant changes in laboratory measures. BLU-808 achieved dose-dependent serum tryptase reductions exceeding 80 percent, reflecting evidence of mast cell target engagement. BLU-808 had a half-life supporting once-daily oral dosing and showed sustained target coverage across a range of doses. Blueprint Medicines' AAAAI / WAO data presentations are listed below. Copies of the data presentations will be available in the "Science—Publications and Presentations" section of the company's website at . Oral Presentation: Assessing Real-World Natural History of Indolent Systemic Mastocytosis: Retrospective Matched Cohort Study from Mayo Clinic Electronic Health Records (Abstract 955 – Monday, March 3) Oral Presentation: Responses to Avapritinib in Patients without Detectable KIT Mutations by ddPCR in Peripheral Blood Highlight Diagnostic Challenges and Opportunities in Indolent Systemic Mastocytosis (Abstract 965 – Monday, March 3) Poster Presentation: Patient Reported Burden of Indolent Systemic Mastocytosis in White vs. Non-White Patients (Abstract 220 – Friday, February 28) Poster Presentation: Identification of Clonal Mast Cell Disease in Patients with Anaphylaxis or Evidence of Systemic Mast Cell Activation: A Post Hoc Analysis from PROSPECTOR (Abstract 301 – Saturday, March 1) Poster Presentation: Avapritinib Impacts the Plasma Inflammatory Proteome in Patients with Indolent Systemic Mastocytosis (Abstract 517 – Saturday, March 1) Poster Presentation: Multiple Proteins Correlate with Tryptase Levels in Patients with Indolent Systemic Mastocytosis (ISM): Preliminary Results of Plasma Proteomic Analysis in PIONEER (Abstract 518 – Saturday, March 1) Poster Presentation: Comprehensive Analysis of Immunoglobulin E Levels in Healthy Donors and Patients with Indolent Systemic Mastocytosis Enrolled on the PIONEER Trial of Avapritinib (Abstract 521 – Saturday, March 1) Poster Presentation: Avapritinib and Bone Health in Indolent Systemic Mastocytosis: Learnings from the PIONEER Trial (Abstract 527– Saturday, March 1) Poster Presentation: PREDICT-SM: Development of Machine Learning Models to Support Screening for Undiagnosed Systemic Mastocytosis (Abstract 530 – Saturday, March 1) Poster Presentation (Trial in Progress): HARBOR: An Ongoing Phase 2/3 Study of Elenestinib in Patients with Indolent Systemic Mastocytosis (Abstract 533 – Saturday, March 1) Poster Presentation: Long-Term Quality of Life and Safety in Patients with Indolent Systemic Mastocytosis Treated with Avapritinib in the PIONEER Study (Abstract 534 – Saturday, March 1) Poster Presentation: BLU-808: A Potent and Selective Oral Small Molecule Wild-Type KIT Tyrosine Kinase Inhibitor for Allergic Conditions (Abstract 535 – Saturday, March 1) Poster Presentation: Utility of Large Language Models to Quantify Diagnostic Delays in Systemic Mastocytosis: A Multi-Center Real World Study (Abstract 544 – Saturday, March 1) Poster Presentation: Safety and Pharmacokinetics (PK) of BLU-808 Following Oral Dosing in Healthy Volunteers (Abstract 698 – Sunday, March 2) About Systemic Mastocytosis Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have indolent systemic mastocytosis (ISM). A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Until 2023, there were no approved therapies for the treatment of ISM. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. About AYVAKIT (avapritinib) AYVAKIT (avapritinib) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Under the brand name AYVAKYT® (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN and MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL). Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. Important Safety Information Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC; <1% were Grade 3. Depending on the severity, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT. Photosensitivity — AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment. Embryo-Fetal Toxicity — AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose. Adverse Reactions — The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing. Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors or inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or visit . AYVAKIT is available in 25-mg, 50-mg, 100-mg and 200-mg tablets. Please click here to see the full Prescribing Information for AYVAKIT. 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 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 expectations for the potential benefits of AYVAKIT in treating patients with ISM; Blueprint Medicines' ability to transform treatment across the spectrum of SM; the development and potential benefits of BLU-808; statements regarding plans and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; and Blueprint Medicines' strategy, goals, business plans and focus. The words "aim," "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, without limitation, risks and uncertainties related to Blueprint Medicines' ability and plans in continuing to build out and expand a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT/AYVAKYT or obtain marketing approval for AYVAKIT/AYVAKYT in additional geographies in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; Blueprint Medicines' advancement of multiple early-stage efforts; 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; the preclinical and clinical results for Blueprint Medicines' drug candidates, which 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; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for AYVAKIT/AYVAKYT or any drug candidates it is developing; Blueprint Medicines' ability to successfully expand its operations, research platform and portfolio of therapeutic candidates, and the timing and costs thereof; and the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing arrangements. 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 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. SOURCE Blueprint Medicines Corporation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 1Drug Approval

13 Feb 2025

·www.prnewswire.com

Blueprint Medicines Reports Fourth Quarter and Full Year 2024 Results

-- Achieved $479.0 million in AYVAKIT® (avapritinib) global net product revenues in 2024, including $144.1 million in the fourth quarter -- -- Anticipate global AYVAKIT net product revenue of approximately $680 million to $710 million in 2025, representing 45% percent year-over-year growth at the midpoint -- -- Peak systemic mastocytosis franchise revenue opportunity updated to $4 billion, with AYVAKIT expected to achieve $2 billion in revenue by 2030 -- -- AYVAKIT 3-year safety and efficacy results in ISM and BLU-808 healthy volunteer data among 14 total abstracts accepted for presentation at 2025 AAAAI / WAO Joint Congress -- CAMBRIDGE, Mass., Feb. 13, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC) today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2024 and provided financial guidance. "We are entering 2025 in the strongest position we have ever been in as a company with a focus on driving growth and innovation through operational excellence. AYVAKIT is a medicine that is changing patients' lives, and the SM market is larger and growing faster than we originally expected. Our 2025 guidance of $680 to $710 million represents robust growth, putting AYVAKIT firmly on track to achieve $2 billion in revenue by 2030. With the larger potential SM franchise peak opportunity of $4 billion, we have a plan to drive innovation in the treatment of SM with elenestinib as we move beyond symptom control to disease modification in the HARBOR registration study, building a durable SM franchise that we anticipate will grow throughout the next decade," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our next potential blockbuster opportunity, BLU-808, is also coming into focus with the healthy volunteer data reported last month. BLU-808 demonstrated a best-in-class profile for an oral wild-type KIT inhibitor enabling our differentiated approach to modulating mast cell activity across thousands of patients with mast-cell-mediated allergic and inflammatory diseases. BLU-808 offers us the opportunity to find the right balance of efficacy and tolerability to realize true pipeline-in-a-medicine potential. Altogether, our portfolio represents a unique set of assets, targeting fundamental mast cell biology, that will drive durable and diversified growth over the next decade with significant operating leverage." Fourth Quarter and Full Year 2024 Highlights and Recent Progress Mast cell disorders For the full year, achieved AYVAKIT net product revenues of $479.0 million, including $421.8 million in the US and $57.1 million ex-US, representing 135 percent growth year-over-year. In the fourth quarter of 2024, AYVAKIT achieved $144.1 million in net product revenues, including $124.1 million in the US and $20.0 million ex-US. Blueprint estimates the peak revenue opportunity for the company's SM franchise is $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030. Presented AYVAKIT data at the 2024 American Society of Hematology Annual Meeting, showing a significant survival benefit in treatment-naïve patients with advanced SM, and sustained improvement in bone density for advanced SM patients with low bone mass at baseline. View the presentations here. Reported in January 2025 results from the Phase 1 healthy volunteer study of BLU-808, showing a differentiated profile that enables the evaluation of tunable dosing strategies. BLU-808 was well-tolerated at all doses tested, showed consistent pharmacokinetics supporting once daily oral dosing, and achieved dose-dependent reductions in tryptase exceeding 80 percent. These positive data support initiation of multiple proof-of-concept studies, which are planned in 2025. Corporate Presented the company's 2025 corporate outlook and strategy to drive continued growth at the 43rd Annual J.P. Morgan Healthcare Conference. View the press release here. Following closing of the IDRx acquisition by GSK, Blueprint anticipates approximately $80 million in gross proceeds from an equity stake in IDRx. Announced the appointment of Sherwin Sattarzadeh to Chief Business Officer, overseeing business development, portfolio leadership and project management, and alliance management. His previous roles over a decade of service at Blueprint include Head of Regulatory Affairs, Chief of Staff to the Chief Executive Officer, and Senior Vice President of Strategic Operations. He succeeds Helen Ho, Ph.D. who is departing the company to pursue another career opportunity. 2025 Financial Guidance Blueprint today announced it anticipates approximately $680 million to $710 million in global AYVAKIT net product revenues for all approved indications in 2025. The midpoint of this range represents 45 percent year-over-year revenue growth. In 2024, Blueprint reduced cash burn by more than 50 percent and expects further reduction in 2025 as the company continues to invest in advancing prioritized programs, balancing investing in innovation with financial discipline. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will maintain a durable capital position to enable the company to achieve a self-sustainable financial profile. Key Upcoming Milestones The company plans to achieve the following milestones in the first half of 2025: Present 14 abstracts at the 2025 American Academy of Allergy, Asthma and Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress, including: Three-year safety and efficacy data from the PIONEER trial of AYVAKIT. Bone density improvements shown by AYVAKIT in the PIONEER trial. Phase 1 healthy volunteer data for BLU-808, a highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line results reported in January 2025. Initiate two proof-of-concept studies of BLU-808 in patients with chronic spontaneous urticaria/chronic inducible urticaria, and allergic rhinitis/allergic conjunctivitis. Fourth Quarter and Year End 2024 Results Revenues: Revenues were $146.4 million for the fourth quarter of 2024, including $144.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.2 million in collaboration revenues. Revenues for the year ended December 31, 2024 were $508.8 million, including $479.0 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $29.9 million in collaboration and license revenues. Blueprint Medicines recorded $72.0 million and $249.4 million in revenues in the fourth quarter and year ended December 31, 2023, respectively. Cost of Sales: Cost of sales was $7.4 million for the fourth quarter of 2024 and $20.2 million for the year ended December 31, 2024, as compared to $0.3 million and $8.5 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to higher sales to our collaboration and other partners and an increase in product sales volume. R&D Expenses: Research and development expenses were $83.7 million for the fourth quarter of 2024 and $341.4 million for the year ended December 31, 2024, as compared to $97.5 million and $427.7 million for the fourth quarter and year ended December 31, 2023, respectively. This decrease was primarily due to operational efficiency across our portfolio as we executed across our top priority programs, and the timing of manufacturing of clinical trial materials. Research and development expenses also included $11.7 million in stock-based compensation expenses for the fourth quarter of 2024 and $47.5 million in stock-based compensation for the year ended December 31, 2024. SG&A Expenses: Selling, general and administrative expenses were $96.5 million for the fourth quarter of 2024 and $359.3 million for the year ended December 31, 2024, as compared to $79.3 million and $295.1 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $16.6 million in stock-based compensation expenses for the fourth quarter of 2024 and $61.5 million in stock-based compensation for the year ended December 31, 2024. Net Loss: Net loss was $(50.0) million for the fourth quarter of 2024 and $(67.1) million for the year ended December 31, 2024, or a diluted net loss per share of $(0.79) and diluted net loss per share of $(1.07), respectively, as compared to a net loss of $(110.9) million for the fourth quarter of 2023 and a net loss of $(507.0) million for the year ended December 31, 2023, or a diluted net loss per share of $(1.82) and a diluted net loss per share of $(8.37), respectively. Cash Position: As of December 31, 2024, cash, cash equivalents and investments were $863.9 million, as compared to $767.2 million as of December 31, 2023. Conference Call Information Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss fourth quarter and full year 2024 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 to conference ID 349846. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at . 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: 45th Annual Cowen Health Care Conference on Monday, March 3, 2025 at 1:10 p.m. ET. H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference on Thursday, March 27, 2025 at 12:00 p.m. ET. Live webcasts of the presentations will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at . Replays of the webcasts will be archived on the Blueprint Medicines website for 30 days following the presentations. About Blueprint Medicines Blueprint Medicines is a fully integrated, commercial-stage, global biopharmaceutical company that invents life-changing medicines in two core, strategic areas of allergy/inflammation and oncology/hematology. We pursue discovery, development, and commercialization of therapies that potently and selectively target known drivers of disease, with focused investment in therapeutic areas where we can leverage our core expertise and business infrastructure to bring scale to our science. We are bringing AYVAKIT®/AYVAKYT® (avapritinib) to people living with systemic mastocytosis (SM) in the U.S. and Europe. Additionally, we have a pipeline of research and development programs that range from early science to advanced clinical trials in mast cell-mediated diseases, including SM and chronic urticaria, breast cancer, and other solid tumors vulnerable to CDK2 inhibition. For more information, visit 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; its planned commercial investments and its future business growth; the advancement of BLU-808 and its potential to benefit patients with allergic and inflammatory diseases; the potential benefits of any of the Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; the timing of its pre-clinical and clinical trials; and Blueprint Medicines' financial performance, strategy, including its planned investment allocation; projected cash burn, the anticipated proceeds upon the IDRx acquisition, goals and anticipated milestones, business plans and focus. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate,", "achieve", "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, without limitation: the uncertainty of Blueprint's ability to execute on its strategic plan for sustainable growth; 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 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; and the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing arrangements. 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. SOURCE Blueprint Medicines Corporation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Clinical ResultFinancial StatementExecutive ChangeASH

12 Jan 2025

·ir.blueprintmedicines.com

Blueprint Medicines Provides 2025 Outlook and Highlights Strategy for Continued Growth

-- Updating peak systemic mastocytosis franchise revenue opportunity to $4 billion , catalyzed by strong AYVAKIT® (avapritinib) launch and evolving SM prevalence estimates -- -- Expect to achieve $2 billion in AYVAKIT revenue by 2030 -- -- BLU-808 demonstrates wide therapeutic window with rapid, robust and sustained tryptase reductions exceeding 80% in Phase 1 healthy volunteer study -- -- Kate Haviland , CEO, to present at J.P. Morgan conference on Monday, January 13 at 9:00 a.m. PT ( 12:00 p.m. ET ) -- CAMBRIDGE, Mass. , Jan. 12, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today provided a 2025 corporate outlook and strategy for continued growth leveraging the company's proven R&D and commercial capabilities. "With AYVAKIT growing towards a multibillion-dollar opportunity, anchoring our SM franchise, and with BLU-808, our next program with blockbuster potential coming into focus, we enter 2025 in the strongest position we have ever been in as a company. We have a number of commercial and clinical catalysts that we expect to deliver significant near- and long-term value to both patients and shareholders," said Kate Haviland , Chief Executive Officer of Blueprint Medicines . "Driven by scalable innovation and operational excellence, we aspire to fundamentally shift the way many allergic and inflammatory diseases are treated by targeting the mast cell. We have built a high-performing commercial engine that enables a truly integrated approach from research to development to commercialization, allowing us to realize operational efficiencies and resulting in a durable financial profile." Systemic Mastocytosis (SM): A Large and Growing Opportunity Based on the strength of the global AYVAKIT launch to date, significant sustained growth in diagnosed SM patients, and new epidemiology data suggesting SM prevalence is greater than previously thought, Blueprint now estimates the peak revenue opportunity for the company's SM franchise is $4 billion , with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030. Blueprint plans to report financial results for the fourth quarter and full-year 2024 in February 2025 . The company previously provided guidance in October 2024 for AYVAKIT product revenue of $475 to $480 million for full-year 2024, representing an increase of more than 130 percent over 2023. Positive Data from BLU-808 Healthy Volunteer Trial Blueprint today announced 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 wild type KIT inhibitor, in healthy volunteers. Detailed data will be presented at the J.P. Morgan 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 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. Change in Serum Tryptase (MAD) Dose Tryptase reduction at Day 15 Participants reaching LLOQ Placebo (n=8) -4 % 0 1 mg (n=6) -23 % 1/6 3 mg (n=6) -41 % 1/6 6 mg (n=6) -66 % 3/6 12 mg (n=4) a -87 % 3/4 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 43rd 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/blueprint-medicines-provides-2025-outlook-and-highlights-strategy-for-continued-growth-302348688.html SOURCE Blueprint Medicines Corporation Media Contact: Andrew Law, +1 (617) 844-8205, media@blueprintmedicines.com Investor Contact: Cassie Saitow, +1 (617) 909-3127, ir@blueprintmedicines.com

Phase 1Clinical ResultFinancial Statement

100 Deals associated with Avapritinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11279	Avapritinib	L01XE	DB15233

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Gastrointestinal Stromal Tumors	Canada	Blueprint Medicines Corp.	18 Sep 2024
Indolent systemic mastocytosis	United States	Blueprint Medicines Corp.	22 May 2023
Aggressive Systemic Mastocytosis	European Union	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Aggressive Systemic Mastocytosis	Iceland	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Aggressive Systemic Mastocytosis	Liechtenstein	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Aggressive Systemic Mastocytosis	Norway	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Mast-Cell Leukemia	European Union	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Mast-Cell Leukemia	Iceland	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Mast-Cell Leukemia	Liechtenstein	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Mast-Cell Leukemia	Norway	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Metastatic Gastrointestinal Stromal Tumor	European Union	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Metastatic Gastrointestinal Stromal Tumor	Iceland	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Metastatic Gastrointestinal Stromal Tumor	Liechtenstein	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Metastatic Gastrointestinal Stromal Tumor	Norway	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Unresectable Gastrointestinal Stromal Tumor	European Union	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Unresectable Gastrointestinal Stromal Tumor	Iceland	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Unresectable Gastrointestinal Stromal Tumor	Liechtenstein	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Unresectable Gastrointestinal Stromal Tumor	Norway	Blueprint Medicines (Netherlands) BV	14 Mar 2023
Systemic Mastocytosis	United States	Blueprint Medicines Corp.	16 Jun 2021
PDGFRA D842V mutation Gastrointestinal Stromal Tumors	European Union	Blueprint Medicines (Netherlands) BV	24 Sep 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Central Nervous System Neoplasms	Phase 2	United States	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	Australia	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	Austria	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	Canada	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	France	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	Germany	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	Italy	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	South Korea	Blueprint Medicines Corp.	24 Feb 2022
Central Nervous System Neoplasms	Phase 2	United Kingdom	Blueprint Medicines Corp.	24 Feb 2022
Relapsed Solid Neoplasm	Phase 2	United States	Blueprint Medicines Corp.	24 Feb 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


VOYAGER trial (ESMO_SRC2025)	Phase 3	Toxicity KIT D816V \| PDGFRA D842V	31	Avapritinib	bsoxlgnptx(cfdpzbmxmr) = Cognitive impairment was more common and more severe than previously reported. Symptoms are persistent and may worsen after discontinuation. We assume that cognitive impairment might be linked to PDGFRβ innhibition, thereby compromising pericyte function and vascular integrity causing brain accumulation of blood-derived neurotoxic molecules. fudrmyltxv (roppnmdfyh ) View more	Positive	21 Mar 2025
NCT03580655 \| NCT04695431 (PATHFINDER, ASH2024) Manual	Not Applicable	Systemic Mastocytosis	-	AvapritinibAvapritinib 200mg/day (1L)	rpatdebslt(bkhnocwblo) = ksyitggckk wdcqmzlgdf (xjfuyowegx ) View more	Positive	07 Dec 2024
				Midostaurin (1L)	rpatdebslt(bkhnocwblo) = kvgvbdiqgi wdcqmzlgdf (xjfuyowegx, 20.0 - 44.6) View more
NCT04825574 (CTgov)	Phase 4	Gastrointestinal Stromal Tumors	2	Avapritinib	ledeprtmwe = dticgtgzrb sbahbjqfah (prknpkxzqe, ipyltbebgs - arrbtfusxb) View more	-	26 Nov 2024
NCT03580655 (PATHFINDER, EHA 12024 \| EHA 22024) Manual	Phase 2	Systemic Mastocytosis KIT D816V	107	Avapritinib	ripzlpbyry(lohxaohkpz) = vphpevkepk xkoeaxywlc (mlpcrcthqg, 63 - 83) View more	Positive	14 May 2024
NCT03731260 (PIONEER, AAAAI2024)	Phase 2	Indolent systemic mastocytosis D816V-mutant KIT	-	Avapritinib	tpsuxnkxdg(latzlnbdkh) = sxypzrcccv fkkzfdmfvq (gaiebgfmpw, 0 - 11) View more	Positive	23 Feb 2024
				Placebo	tpsuxnkxdg(latzlnbdkh) = ijgfbdjcli fkkzfdmfvq (gaiebgfmpw, 1 - 8)
NCT03580655 (PATHFINDER, SOHO2023)	Phase 2	Systemic Mastocytosis KIT D816V mutation	107	Avapritinib 200 mg	ecbcnwgaph(ahpwbneepn) = mwvntyrwnf dixphnhkqe (nyhlzqkrex, 63 - 83) View more	Positive	01 Sep 2023
NCT03731260 (PIONEER, SOHO2023)	Phase 2	Indolent systemic mastocytosis KIT D816V mutation \| serum tryptase \| bone marrow (BM) MC burden ... View more	-	Avapritinib 25 mg	usukhwmuip(mbyggnbisf) = zgxypmedag cmfmutigkz (wqngmuchmp ) View more	Positive	01 Sep 2023
				Placebo	usukhwmuip(mbyggnbisf) = zxxrymljew cmfmutigkz (wqngmuchmp ) View more
NCT03731260 (PIONEER, EHA2023)	Phase 2	Indolent systemic mastocytosis	251	Avapritinib 25 mg QD	rjufjgolgv(tjzqjxdeho) = xdubhlkluw dpxqknarur (fdiexuuzto ) View more	-	08 Jun 2023
				Placebo	suwospasoc(whrcxzuzip) = nqrypnpvee vejcbjolyl (ddkrwqlcuq ) View more
NCT02508532 \| NCT04254939 (CS3007-101, ASCO2023) Manual	Phase 1/2	Gastrointestinal Stromal Tumors KIT-AL-mutant \| AL exon 18 mutations	160	Avapritinib 300 mg once daily (ALposABPneg group)	dwwhrfrmws(ajhtmgvalj) = bfpfjbtkyc jjwxtwiszu (wphbarmsxb ) View more	Positive	31 May 2023
				Avapritinib 300 mg once daily (KIT OTHERS group)	dwwhrfrmws(ajhtmgvalj) = fllhjdlkmr jjwxtwiszu (wphbarmsxb ) View more
NCT03731260 (PIONEER, FDA) Manual	Phase 2	Indolent systemic mastocytosis	212	AYVAKIT 25 mg+BSC	ohibcmpulb(ywxjzjyzyz) = The most common adverse reactions (≥ 10%) in the AYVAKIT group were eye edema, dizziness, peripheral edema and flushing. Of all adverse reactions, 55% were Grade 1, 38% were Grade 2 and 7% were Grade 3. viajfrughn (dlgugkdhjl ) View more	Positive	22 May 2023
				Placebo + BSC

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