Niagen Bioscience Announces First-Ever Peer-Reviewed Study Highlighting the Potential of Nicotinamide Riboside (NR) for Werner Syndrome, a Rare Genetic Disorder
Results demonstrated that nicotinamide riboside (NR) significantly elevated NAD+ levels and improved multiple clinical markers in people with Werner Syndrome
Niagen Bioscience expands rare disease research portfolio, supporting further investigation of NAD+ augmentation with NR as a therapeutic strategy in rare progeroid diseases
LOS ANGELES, June 09, 2025--(BUSINESS WIRE)--Niagen Bioscience, Inc. (NASDAQ: NAGE) (formerly ChromaDex Corp.), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, shares positive results from a clinical study published in the peer-reviewed journal Aging Cell, by a team led by Masaya Koshizaka, M.D., Ph.D., Associate Professor, and Koutaro Yokote, M.D., Ph.D., MBA, President of Chiba University, both of the Center for Preventive Medical Sciences, Chiba University, and the Department of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Japan. This is the first study to demonstrate the safety and efficacy of Niagen Bioscience's patented nicotinamide riboside (NR) ingredient, Niagen®, in individuals with Werner syndrome (WS), a rare genetic disorder marked by rapid aging and premature mortality.
The newly published double-blind, placebo-controlled study found that daily supplementation with Niagen significantly elevated blood NAD+ levels by approximately 140% and improved multiple clinical markers of cardiovascular and skin health in individuals with WS. Affecting approximately 1 in 380,000 to 1 in 1,000,000 people globally (GeneReviews), Werner syndrome is caused by mutations that impair DNA repair, leading to cellular aging decades ahead of normal progression.
Dr. Koshizaka noted, "We hope our work will accelerate studies on not only WS but also other premature aging disorders and common age-related diseases—ultimately helping to extend health span and improve quality of life in both patients and the broader population."
Developing NAD+ Therapies for Rare, Age-Related Diseases
This study builds on the growing body of clinical research demonstrating Niagen's potential in rare, age-related diseases (see Table 1 below). In Ataxia Telangiectasia (AT), Niagen has shown improvements in neurological function, coordination, and immune markers, including in pediatric populations.
Rob Fried, CEO of Niagen Bioscience, stated, "Rare disease research is a priority for Niagen Bioscience, particularly those indications associated with accelerated aging, mitochondrial dysfunction, or NAD+ deficiency."
The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation to NR for the treatment of AT. These designations underscore the urgent unmet need and potential therapeutic value of Niagen in rare disease populations.
Why NAD+ Matters in Rare DiseaseScientific research has shown that declining NAD+ levels can contribute to age-related decline, mitochondrial dysfunction, and impaired DNA repair. In both Werner syndrome and AT, NAD+ deficiency appears to play a central role in disease progression (Fang et al., 2019). By restoring NAD+ levels, Niagen may help activate protective enzymes like SIRT1 and PARP1, reduce oxidative stress, and improve tissue function (Fang et al., 2016; Veenhuis et al., 2021; Presterud et al., 2023; Shoji et al., 2025; Lautrup et al., 2025).
Vilhelm Bohr, M.D., Ph.D., D.Sc., former National Institute on Aging (NIA) Chief of the Laboratory of Molecular Genetics, and current Affiliate Professor in Genome Instability and Neurodegeneration at the University of Copenhagen and Scientific Advisor to Niagen Bioscience and coauthor on the study, commented, "Werner syndrome is a rare genetic disorder that has long served as a valuable model for understanding the mechanisms of human aging. This study marks the first clinical trial using Niagen in those with Werner syndrome, and the findings—particularly improvements in cardiovascular markers—are promising. As Werner syndrome is a well-established model for normal aging, these results also suggest that NAD+ supplementation may support healthier aging in the broader population."
Study Highlights
This 52-week randomized, double-blind, placebo-controlled crossover trial evaluated the safety and efficacy of oral Niagen supplementation in individuals with WS. The study randomized nine individuals (mean age: 47) who received 1,000 mg/day of Niagen or placebo for 26 weeks before crossing over to the alternate treatment for an additional 26 weeks.
Key findings include:
Robust increase in NAD+ levels: Niagen supplementation led to a ~140% increase in plasma NAD+ levels, compared to a ~4% decrease in the placebo group.
Improved arterial stiffness: Niagen significantly improved cardio-ankle vascular index (CAVI), a measure of arterial stiffness.
Cardioprotective lipid shift: Niagen increased the number of large HDL particles, indicating potential cardiovascular benefits.
Wound healing support: Niagen reduced skin ulcer size and heel pad thinning, while ulcers worsened in the placebo group.
Clinical safety profile: No moderate or severe adverse events were reported. Mild adverse events were fewer in individuals who received Niagen (7) compared to those who received placebo (12).
Tolerability in complex individuals: Although mild liver enzyme elevations were noted, they were deemed manageable and consistent with underlying liver sensitivities common in Werner syndrome.
This study represents the first clinical evaluation of Niagen in WS and supports further investigation of NAD+ augmentation as a therapeutic strategy in rare progeroid diseases.
For additional information on the science supporting Niagen®, visit www.niagenbioscience.com.
Table 1The table below provides an overview of the clinical research published to date on Niagen in rare age-related conditions.
Publication
Dose
Duration
Health Area
Study Design
Key Outcomes
Shoji et al., 2025
1000 mg
52 weeks
Werner syndrome
Randomized, double-blind, placebo-controlled crossover study in 11 individuals with Werner syndrome
~140% increase in plasma NAD+ levels; improved arterial stiffness and reduced skin ulcer size.
No moderate or severe adverse events reported.
Presterud et al., 2023
500 mg
2 years
Ataxia Telangiectasia (AT)
Open-label, single-arm observational study in 10 AT individuals
Significant improvements in motor coordination and eye movements. No serious adverse events. Longest NR supplementation trial to date.
Veenhuis et al., 2021
25 mg/kg
4 months
Ataxia Telangiectasia (AT)
Open-label proof-of-concept study in 24 AT individuals
Improved ataxia scores (SARA, ICARS); effects reversed after withdrawal. Increased IgG levels in immunodeficient individuals.
Tinnevelt et al., 2020
25 mg/kg
4 months
Ataxia Telangiectasia (AT)
Comparative study in 14 individuals with AT
NR-related pathways and metabolites significantly increased following NR supplementation.
About Niagen Bioscience:
Niagen Bioscience, Inc. (NASDAQ: NAGE), formerly ChromaDex Corp., is the global leader in NAD+ (nicotinamide adenine dinucleotide) science and healthy-aging research. As a trusted pioneer of NAD+ discoveries, Niagen Bioscience™ is dedicated to advancing healthspan through precision science and innovative NAD+-boosting solutions.
The Niagen Bioscience team, composed of world-renowned scientists, works with independent investigators from esteemed universities and research institutions around the globe to uncover the full potential of NAD+. A vital coenzyme found in every cell of the human body, NAD+ declines with age and exposure to everyday lifestyle stressors. NAD+ depletion is a key contributor to age-related changes in health and vitality.
Distinguished by state-of-the-art laboratories, rigorous scientific and quality protocols, and collaborations with leading research institutions worldwide, Niagen Bioscience sets the gold standard for research, quality, and innovation. There's a better way to age.
At the heart of its clinically proven product portfolio is Niagen® (patented nicotinamide riboside, or NR), the most efficient, well-researched, high-quality, and legal NAD+ booster available.
Niagen Bioscience's robust patent portfolio protects NR and other NAD+ precursors. Niagen Bioscience maintains a website at www.niagenbioscience.com, where copies of press releases, news, and financial information are regularly published.
Forward Looking Statements:
This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended, including statements related to infringement or non-infringement of intellectual property rights. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as "expects," "anticipates," "intends," "estimates," "plans," "potential," "possible," "probable," "believes," "seeks," "may," "will," "should," "could" or the negative of such terms or other similar expressions. Risks that contribute to the uncertain nature of these forward-looking statements include the impact of the COVID-19 pandemic on our business and the global economy; our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. Xx Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Niagen Bioscience undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250609224907/en/
Contacts
Niagen Bioscience Media Contact: Kendall Knysch, Senior Director of Media Relations & Partnerships310.405.5227kendall.knysch@niagenbio.com
Niagen Bioscience Investor Relations Contact: ICR, LLCReed Anderson(646) 277-1260Stephanie Carrington(646) 277-1282niagenir@icrinc.com
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These are not all of the signs and symptoms of immune system problems that can happen with Zynyz. Call or see your doctor right away for any new or worsening signs or symptoms, which may include: chest pain, irregular heartbeat, shortness of breath, or swelling of ankles confusion, sleepiness, memory problems, changes in mood or behavior, stiff neck, balance problems, tingling or numbness of the arms or legs double vision, blurry vision, sensitivity to light, eye pain, changes in eyesight persistent or severe muscle pain or weakness, muscle cramps low red blood cells, bruising Infusion reactions that can sometimes be severe. Signs and symptoms of infusion reactions may include: chills or shaking, itching or rash, flushing, shortness of breath or wheezing, dizziness, feel like passing out, fever, back or neck pain. Rejection of a transplanted organ or tissue. Your doctor should tell you what signs and symptoms you should report and monitor you, depending on the type of organ or tissue transplant that you have had. Complications, including graft-versus-host disease, in people who have received a bone marrow (stem cell) transplant that uses donor stem cells (allogeneic). These complications can be serious and can lead to death. These complications may happen if you underwent transplantation either before or after being treated with Zynyz. Your doctor will monitor you for these complications. Getting medical treatment right away may help keep these problems from becoming more serious. Your doctor will check you for these problems during your treatment. Your doctor may treat you with corticosteroid or hormone replacement medicines and may also need to delay or completely stop treatment if you have severe side effects. Before you receive Zynyz, tell your doctor about all of your medical conditions, including if you: have immune system problems such as Crohn's disease, ulcerative colitis, or lupus have received an organ transplant or tissue transplant, including corneal transplant have received or plan to receive a stem cell transplant that uses donor stem cells (allogeneic) have received radiation treatment to your chest area have a condition that affects your nervous system, such as myasthenia gravis or Guillain-Barré syndrome are pregnant or plan to become pregnant. Zynyz can harm your unborn baby Females who are able to become pregnant: Your doctor should do a pregnancy test before you start treatment. You should use an effective method of birth control during your treatment and for 4 months after your last dose. Talk to your doctor about birth control methods that you can use during this time. Tell your doctor right away if you become pregnant or think you may be pregnant during treatment. are breastfeeding or plan to breastfeed. It is not known if Zynyz passes into your breast milk. Do not breastfeed during treatment and for 4 months after your last dose Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. The most common side effects of Zynyz when given with the chemotherapy medicines carboplatin and paclitaxel in people with SCAC include tiredness, numbness, pain, tingling, or burning in your hands or feet; nausea; hair loss; diarrhea; muscle and bone pain; constipation; bleeding; rash; vomiting; decreased appetite; itching; stomach-area pain. The most common side effects of Zynyz when used alone in people with SCAC include tiredness, muscle and bone pain, diarrhea, infection, rectal or genital-area pain, bleeding, urinary tract infection (UTI), rash, nausea, loss of appetite, constipation, stomach-area pain, shortness of breath, fever, vomiting, cough, itching, low levels of thyroid hormone, headache, decreased weight. The most common side effects of Zynyz when used alone in people with MCC include tiredness, muscle and bone pain, itching, diarrhea, rash, fever, nausea. These are not all the possible side effects of Zynyz. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. You may also report side effects to Incyte Corporation at 1-855-463-3463. General information about the safe and effective use of Zynyz Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. If you would like more information about Zynyz, talk with your doctor. You can ask your doctor for information about Zynyz that is written for health professionals. Please see the full Prescribing Information, including the Medication Guide, for Zynyz. You may also report side effects to the FDA or to Incyte Corporation at 1-855-463-3463. About Incyte A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia. For additional information on Incyte, please visit or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube. Incyte Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether and when Zynyz might provide a successful treatment option for patients with SCAC, contain predictions, estimates and other forward-looking statements. These forward-looking statements are based on Incyte's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by FDA or other regulatory authorities; Incyte's dependence on its relationships with its collaboration partners; the efficacy or safety of Incyte's products and the products of Incyte's collaboration partners; the acceptance of Incyte's products and the products of Incyte's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte's reports filed with the Securities and Exchange Commission, including its annual report and its quarterly report on Form 10-Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.
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Blueprint Medicines Announces Data Reinforcing Sustained Clinical Efficacy and Well-Tolerated Safety Profile of Long-Term AYVAKIT®/AYVAKYT® (avapritinib) Treatment at 2025 EHA and EAACI Congresses
-- Breadth of presentations, including one oral and two flash talks, showcase Blueprint Medicines' leadership role in advancing care for patients with systemic mastocytosis -- CAMBRIDGE, Mass., June 12, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM). Key results continue to position AYVAKIT®/AYVAKYT® (avapritinib) as the durable standard of care across indolent and advanced SM, and highlight the real-world burden of the disease, reinforcing the importance of treating with a therapy that addresses the root cause of SM. These data will be reported at the 2025 European Hematology Association (EHA2025) Hybrid Congress, being held June 12 to 15 in Milan, Italy, and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, being held June 13 to 16 in Glasgow, United Kingdom. "Our presentations feature large patient populations from the PIONEER, PATHFINDER and EXPLORER trials, with follow-up reaching up to five years in ISM and up to 6.5 years in advanced SM, reflecting both the favorable long-term benefits of AYVAKIT and the unprecedented datasets we have amassed over time," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT has shown transformative clinical outcomes for patients across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM. These compelling results have translated into real-world practice, with clinicians expanding their view of who is an appropriate candidate for disease-modifying therapy after positive AYVAKIT experiences, and treatment durations trending toward multiple years." PIONEER Three-Year Data: Durable Clinical Benefits and Consistent Safety Profile with Long-Term AYVAKIT Use in ISM As previously presented,1 AYVAKIT demonstrated robust improvements through 144 weeks in overall symptom and symptom domain measures (skin, gastrointestinal, neurocognitive) representative of real-world patient impacts. AYVAKIT showed a well-tolerated safety profile and a low discontinuation rate due to treatment-related adverse events (TRAEs; 3 percent) with a median of three years of exposure, and some patients out to five years on therapy. Common TRAEs included low-grade edemas, headache and nausea. In newly reported data, AYVAKIT showed sustained clinical benefits across quality-of-life measures that reflect general health status and are broadly recognized by allergists/immunologists, validating previously presented results from the disease-specific, Mastocytosis Quality of Life (MC-QoL) questionnaire. This data presentation follows the May 2025 online publication of PIONEER two-year efficacy and safety data in The Journal of Allergy and Clinical Immunology: In Practice. PATHFINDER/EXPLORER Multi-Year Data: Long-Term Survival Benefits of AYVAKIT in Advanced SM AYVAKIT showed prolonged overall survival (OS) in PATHFINDER and EXPLORER, when indirectly compared to real-world data for midostaurin from the German Registry on Disorders of Eosinophils and Mast Cells (GREM). AYVAKIT led to meaningful survival benefits in patients across all prognostic categories (low, intermediate and high risk), per the Revised Mutation-Adjusted Risk Score (MARS-R) – a new OS risk assessment tool for advanced SM. Conducted in collaboration with University Hospital Mannheim, the analyses validate the MARS-R tool's ability to assess OS risks in advanced SM patients treated with AYVAKIT or midostaurin, using clinical and genetic parameters. The MARS-R was developed to inform physician care decisions based on individual patient needs. PRISM Data: Substantial Disease Burden Across Broad Population of Patients with ISM PRISM is one of the largest studies characterizing the impact of SM from both patient and clinical perspectives. Across the spectrum of disease severity, patients with ISM experienced physical, social and emotional challenges that caused meaningful disruption to their daily lives. Patients reported a broad constellation of disease-related impacts, including limitations to physical activities, work/college and relationships; problems with pain/discomfort and anxiety/depression; and adjustments in their daily lives to avoid certain foods, extended sun exposure and smells. Data Presentations EHA2025 Congress Oral Presentation: The Revised Mutation-Adjusted Risk Score (MARS-R) for Predicting Overall Survival in Patients with Advanced Systemic Mastocytosis Treated with Midostaurin or Avapritinib (Abstract S216) Poster Presentation: Blood-Based Proteomics for Deeper Insights Into Indolent Systemic Mastocytosis: The PIONEER Trial Experience (Abstract PS1838) Poster Presentation: High Accuracy of Peripheral Blood Testing Using Machine Learning–Derived Predictive Models to Distinguish Advanced from Indolent Systemic Mastocytosis: Analysis of Avapritinib and Elenestinib Trial Data (Abstract PF1310) Publication-Only Abstract: Phase 2/3 HARBOR Study of Elenestinib in ISM: A Trial-in-Progress Update of Novel Endpoints and Biomarkers Aimed at Evaluating Disease Modification (Abstract PB3108) EAACI Congress 2025 Flash Talk Presentation: Favorable Benefit-Risk Profile of Avapritinib in Indolent Systemic Mastocytosis Is Maintained After 3 Years of Therapy: Longer-Term Analysis of the PIONEER Study (Abstract 000621) Flash Talk Presentation: The Socio-Emotional Impact of Indolent Systemic Mastocytosis: Insights from the PRISM Survey (Abstract 000488) Poster Presentation: The Phase 2/3 Study of Elenestinib, a Highly Potent and Selective Tyrosine Kinase Inhibitor, in Patients with Indolent Systemic Mastocytosis (Abstract 001121) Data presentations are being made available in the "Science―Publications and Presentations" section of the company's website at 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, pruritus, 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 AYVAKIT (avapritinib) is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) to treat the root cause of SM. It was FDA approved for the treatment of advanced SM in June 2021 and ISM in May 2023. It now is indicated in 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. The medicine is approved in the EU as AYVAKYT for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN or 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). Globally, the medicine is approved for one or more indications in 16 countries, including China where it is marketed by CStone Pharmaceuticals, paying tiered percentage royalties on sales. 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 Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In Advanced SM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study. Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs. In Advanced SM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in Advanced SM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction. Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 28% of 148 Advanced SM patients (3% were Grade ≥3), and 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 and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue. 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 method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose. Adverse Reactions — The most common adverse reactions (≥20%) in patients with Advanced SM were edema, diarrhea, nausea, and fatigue/asthenia. 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. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with Advanced SM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A 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 AYVAKIT is available in 25-mg, 50-mg, 100-mg and 200-mg tablets. Please click here to see the full U.S. 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 SM in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we 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 and 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 Blueprint Medicines' leadership role and its ability to transform treatment across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM; AYVAKIT/AYVAKYT's position as the durable standard of care and clinicians' view of appropriate candidates and treatment duration; 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; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing arrangements; and the ability of the parties to consummate the proposed merger between Blueprint Medicines and Sanofi on the timeline anticipated or at all, including the occurrence of any event, change or other circumstance that could give rise to the termination of the merger. 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. Footnote 1 Reported at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. 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