Elenestinib

-- Real-world study of AYVAKIT highlights improvement across ISM symptoms -- -- 12 data presentations at 2026 AAAAI Annual Meeting reflect company's leadership in elevating SM care -- CAMBRIDGE, Mass., Feb. 28, 2026 /PRNewswire/ -- Blueprint Medicines, a Sanofi company, today announced AYVAKIT® (avapritinib) data showing clinically meaningful outcomes and a well-tolerated long-term safety profile in patients with indolent systemic mastocytosis (ISM). Key presentations include four-year PIONEER clinical study results highlighting sustained symptom control and quality of life improvement in AYVAKIT-treated patients, and real-world data showing meaningful symptom benefit in patients receiving the therapy in a community practice setting. In addition, a study conducted with the Advanced Practitioner Society for Hematology and Oncology (APSHO) and The Mast Cell Disease Society (TMS) reinforces the severe symptoms and impaired quality of life experienced by patients with ISM. In total, 12 data presentations are being reported at the 2026 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting, February 27 – March 2 in Philadelphia. "Patients with indolent systemic mastocytosis and healthcare providers have expressed the need for a therapy that meaningfully improves quality of life through durable symptom benefit and a safety profile enabling long-term treatment, and AYVAKIT is delivering this significant impact to a wide range of people living with the disease," said Mik Rinne, M.D., Ph.D., Head of Development at Blueprint Medicines. "Across clinical and real-world settings, AYVAKIT has shown robust efficacy and a well-tolerated safety profile, helping patients realize the sustained benefit of KIT D816V-targeted therapy. In addition, emerging evidence continues to underscore the substantial burden of SM, highlighting the urgency to treat the underlying cause of the disease." PIONEER: Long-Term Data Reinforce AYVAKIT as Durable Standard of Care in ISM Abstracts 509, 518, 530 Patients showed continued improvement through four years in overall symptoms, all symptom domains (skin, gastrointestinal [GI], neurocognitive) and most severe symptom per the ISM Symptom Assessment Form (ISM-SAF), and quality of life per the Mastocytosis Quality of Life Questionnaire (MC-QoL). In patients with frequent episodes of diarrhea at baseline and at least four years of AYVAKIT (n=44), the median reduction in diarrhea frequency was 65.6 percent. In the overall clinical study population, individual GI symptoms meaningfully improved, allowing for decreased use of cromolyn sodium and other supportive care medicines. Patients had increased bone mineral density – a clinical measure of disease-modifying effects – in the lumbar spine, left total hip and left femoral neck after three years of AYVAKIT. In PIONEER, the change in bone mineral density was assessed in patients who received dual-energy X-ray absorptiometry (DXA) scans (n=79). With a median patient follow-up of about four years (46.5 months; N=226), the safety of AYVAKIT was consistent with the 24-week placebo-controlled portion of the clinical study, and the discontinuation rate due to treatment-related adverse events (TRAEs) was 3 percent (n=6). The majority of edema events, the most common TRAEs, were mild (Grade 1). Real-World Evidence Further Validates Clinical Benefit of AYVAKIT in ISM Abstracts 516, 529 A retrospective review of electronic medical records was conducted at ONCare Alliance, a network of community oncology practices, to assess the impact of AYVAKIT in the real-world setting. Median follow-up was 14.2 months. In AYVAKIT-treated patients (N=18), the median time to symptom improvement was rapid (36.5 days). Benefits were frequently reported across a range of symptoms, including GI, skin, abdominal pain and headache symptoms. Among patients who initiated the 25 mg daily dose of AYVAKIT (n=16), 87.5 percent have remained on therapy, reflecting a well-tolerated safety profile. An additional presentation features a systematic literature review of AYVAKIT data, including results from the real-world AVATAR study in patients with uncontrolled ISM (N=17). In this study, the median MC-QoL score decreased from 61 (baseline) to 17 (24 weeks), representing a 76 percent improvement. Lack of Disease Control and Substantial Burden in Patients with ISM Abstract 514 In collaboration with APSHO and TMS, a survey was developed to identify factors affecting perceptions of disease control in patients with ISM (N=53). Three-quarters of patients (75 percent; n=40) reported that their disease was not optimally controlled. Patients reported severe symptoms regardless of their perceived disease control, underscoring the significant burden across the spectrum of ISM. Copies of the AAAAI poster presentations are available in the "Science—Publications and Presentations" section of Blueprint Medicines' website, . Avapritinib Continues to Demonstrate Durable Symptom Control with a Well-Tolerated Safety Profile: Long-Term Outcomes from PIONEER (Abstract 509 – Saturday, February 28) Real-World Experience of Patients with Indolent Systemic Mastocytosis Treated with Avapritinib in a Community Oncology Setting (Abstract 516 – Saturday, February 28) Avapritinib Durably Improves Gastrointestinal Symptoms of Indolent Systemic Mastocytosis: Long-Term Outcomes from the PIONEER Study (Abstract 518 – Saturday, February 28) Long-Term Bone Health Outcomes in Patients Treated with Avapritinib for Indolent Systemic Mastocytosis: Findings from the PIONEER Study (Abstract 530 – Saturday, February 28) A Systematic Literature Review of Avapritinib Outcomes in Indolent Systemic Mastocytosis (ISM) Patients (Abstract 529 – Saturday, February 28) Understanding Disease Control: What Shapes Patient Experience in Indolent Systemic Mastocytosis (Abstract 514 – Saturday, February 28) When One Is Diagnosed, Two Are Changed: The Caregiver Experience in Indolent Systemic Mastocytosis (Abstract 513 – Saturday, February 28) Systemic Mastocytosis: Five-Year Mortality Comparison with Matched Reference Cohorts (Abstract 533 – Saturday, February 28) Mortality Risk by Subtype in Systemic Mastocytosis: A Retrospective Cohort Analysis (Abstract 522 – Saturday, February 28) Ultra-Sensitive SuperRCA Assay Enhances the Ability to Detect KIT D816V Mutations in Peripheral Blood with a Sensitivity Comparable to That of Bone Marrow Testing (Abstract 524 – Saturday, February 28) Are Tryptase Thresholds for Systemic Mastocytosis Adequate? Electronic Health Record (EHR) Data Weigh In (Abstract 527 – Saturday, February 28) Project BEACON: Developing a Predictive Model to Accelerate Diagnosis of Indolent Systemic Mastocytosis (Abstract 517 – Saturday, February 28) 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). Despite treatment with multiple symptom-directed therapies, patients with ISM frequently experience persistent symptoms including anaphylaxis, maculopapular rash, pruritus, diarrhea, brain fog, fatigue and bone pain, as well as long-term health complications such as osteoporosis. 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. There were no approved therapies for ISM until 2023, when AYVAKIT received U.S. Food and Drug Administration (FDA) approval for this indication. 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 – including bone lesions, malabsorption, liver dysfunction and bone marrow failure – as well as poor overall survival. About Blueprint Medicines' Clinical Studies in SM Blueprint Medicines has pursued a broad development program for patients living with SM, including three registrational clinical studies for AYVAKIT and one for elenestinib. PIONEER is a randomized, double-blind, placebo-controlled clinical study designed to assess the safety and efficacy of AYVAKIT in patients with ISM, and PATHFINDER and EXPLORER are open-label, single-arm clinical studies designed to evaluate the safety and efficacy of AYVAKIT in patients with advanced SM. The HARBOR clinical study (NCT04910685) of elenestinib, an investigational next-generation KIT D816V inhibitor, is currently enrolling patients with ISM. HARBOR is a randomized, double-blind, placebo-controlled clinical study that includes dose-finding Part 1, registrational Part 2 and long-term treatment Part 3. All patients who complete Parts 1 or 2 have an opportunity to receive treatment with elenestinib in Part 3. Key endpoints include changes in patient-reported symptoms, biomarkers and bone mineral density, annualized rate of anaphylaxis events, and safety. Patients and clinicians interested in the ongoing HARBOR clinical study can contact Blueprint Medicines at [email protected] or +1 (617) 714-6707. Additional details are available at or . About AYVAKIT AYVAKIT (avapritinib) is the first and only medicine approved by the 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 more than 35 countries worldwide, including China where it has been developed and commercialized by CStone Pharmaceuticals, which pays 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 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 . Please click here to see the full U.S. Prescribing Information for AYVAKIT. About Blueprint Medicines, a Sanofi Company Blueprint Medicines, a Sanofi company, seeks to alleviate human suffering by solving important medical problems in allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, leveraging deep expertise in our core focus areas. 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, and we aim to scale our impact by advancing programs in mast cell diseases and solid tumors. For more information, visit and follow us on X (formerly Twitter; @BlueprintMeds) and LinkedIn. Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. SOURCE Blueprint Medicines, a Sanofi company 21% more press release views with Request a Demo

iStock, SvetaZi Four of this year’s biggest acquisitions topped 11-figure figures. One was 2025’s messiest bidding war. After a post-pandemic correction—marked by a nosedive in dealmaking activity , a spike in bankruptcies and dried-up financing —M&A activity finally seemed to pick back up for biopharma in 2025. The year opened with the largest deal that would come: Johnson & Johnson’s $14.6 billion play for Intra-Cellular Therapeutics, putting the pharma in a commanding position in the neuroscience space. This deal also icked off a trend we would see throughout the year. In an industry with an underrepresented by women, many of the big ticket transactions this year invilved companies with a woman CEO. Besides Intra-Cellular, there was plenty of other exciting activity, especially later in the year, which witnessed two big-ticket bidding wars. One, between Novo Nordisk and Pfizer over obesity star Metsera, landed itself in this list by driving Pfizer to double its initial offer. The ordeal was also among the most dramatic public spats between two pharma powerhouses. The other bidding war was between Alkermes and Lundbeck over Avadel . While it got messy and hectic times, the dealmaking activity last year suggests that Big Pharmas have the money to fund hefty purchases—and are willing to spend it. Indeed, as of October 2025, the industry had hit an aggregate takeover value of $70 billion , according to a report from Oppenheimer at the time. This sum already eclipsed the total M&A spend in 2024, 2022 and 2021. Here, BioSpace looks back at the biggest acquisition deals of 2025. J&J Strikes Year’s Largest Deal in January Target: Intra-Cellular Therapies Acquisition date: January 13 Final bid: $14.6 billion 2025 got off to an explosive start when J&J announced in January the acquisition of neurology specialist Intra-Cellular Therapies for an eye-watering $14.6 billion . The deal, which formally closed in April , gave the healthcare giant Caplyta, a daily antipsychotic pill approved for the treatment of schizophrenia and depressive episodes in patients with bipolar I or bipolar II disorder. In November, the FDA approved an expansion for Caplyta as an adjunctive therapy with antidepressants for adults with major depressive disorder (MDD). M&A 5 Women Biotech Leaders Bagging Billions in M&A Dollars Some of the most high-pro in recent years have involved women-fronted biotechs. BioSpace reviews five of the most notable here. July 16, 2025 · 8 min read · Tristan Manalac Aside from Caplyta, J&J also got Intra-Cellular’s clinical pipeline, including the mid-stage ITI-1284, a deuterated form of Caplyta being tested for generalized anxiety disorder, as well as Alzheimer’s disease agitation and psychosis. These assets will complement J&J’s own neuro portfolio, led by the antidepressant nasal spray Spravato and the antipsychotic franchise Invega. Reacting to J&J’s takeover of Intra-Cellular, Stifel analysts wrote in a Jan. 15 note that the pharma’s $14.6-billion vote of confidence “is a much-needed win for the neuroscience space,” breathing life into a field that has notoriously been difficult to succeed in . The deal also set the tone for women-led biotechs securing exits in the double-digit billions, with CEO Sharon Mates, who co-founded Intra-Cellular back in 2002 and served as its chair, president and CEO. Months of Negotiations Land RNA Pipeline for Novartis Target: Avidity Biosciences Acquisition date: October 25 Final bid: $12 billion Joining J&J in giving neuro a nudge is Novartis, which in October put $12 billion on the line to swallow Avidity Biosciences and its portfolio of investigational RNA therapies, landing the second spot on this list. Novartis’ proposal wasn’t always that hefty, however. Regulatory documents released a month later showed that the pharma’s initial offer was $7.4 billion —way too low for the biotech’s CEO Sarah Boyce. Months of negotiation followed, with Novartis upping its bid to $8.5 billion then $10 billion and, lastly, to the final price of $12 billion. Avidity’s pipeline is led by three antibody-oligonucleotide conjugates (AOCs), all of which are in registrational development . These include del-zota for Duchenne muscular dystrophy (DMD), del-desiran for myotonic dystrophy type 1 and del-brax for facioscapulohumeral muscular dystrophy. AOCs consist of a monoclonal antibody to target a specific disease-related protein attached to a short string of nucleotides that produces therapeutic effects. Leveraging this mechanism, del-zota elicited a 25% increase in dystrophin expression in DMD patients, according to a Phase I/II readout in August last year. A week before being acquired by Novartis, Avidity announced that it expects to submit a biologics license application (BLA) for del-zota in the first quarter of 2026. Similarly, del-desiran and del-brax are moving steadily toward registration. The former is in Phase III development with first enrollment in July , while the company opened an accelerated pathway for the latter in June. Aside from del-zota, del-desiran and del-brax, Novartis also picked up other preclinical DMD and rare neuromuscular programs from the acquisition. Avidity also has two early precision cardiology programs that will spin out into a new public company. Mergers & acquisitions Avidity Played Hardball To Land Favorable Terms in $12B Novartis Deal At one point in merger negotiations with Novartis, Avidity CEO Sarah Boyce and her team walked, cutting off access to a data room and moving on to a capital raise. November 25, 2025 · 3 min read · Annalee Armstrong Merck Nabs Approved COPD Drug With $5B Potential Target: Verona Pharma Acquisition date: July 9 Final bid: $10 billion Also breaking the 11-figure mark this year is Verona Pharma, which in July commanded a $10 billion buyout offer from Merck. The centerpiece of the acquisition was Ohtuvayre, which won the FDA’s approval in June 2024 for chronic obstructive pulmonary disease (COPD), opening up a new mechanism of action for the indication for the first time in more than two decades. In the first quarter of this year, before Merck’s takeover, Verona reported around 25,000 prescriptions for Ohtuvayre, bringing in $71.3 million in sales. Jefferies has previously forecasted peak annual sales for Ohtuvayre between $3 billion and $5 billion. Regulatory documents released in August 2025 revealed that Merck was the sole bidder for Verona—despite the biotech starting its search for suitors in 2023. Still, during the duration of its courtship with Merck, which began in February, a handful of other unidentified companies approached Verona to explore the possibility of Ohtuvayre-centered agreements, though none of these worked out. The Merck transaction officially closed in October 2025 . At the time the acquisition was announced, analysts at BMO Capital Markets said that Merck’s $10-billion bet is part of its effort to “to replace commercial revenues now,” before mega-blockbuster cancer drug Keytruda loses patent protections in 2028 . Indeed, the pharma in recent years has gone on a shopping spree, buying Prometheus Biosciences for $10.8 billion in April 2023. M&A Merck Makes Big Respiratory Play With $10B Acquisition of Verona In the second biggest acquisition of the year, Merck gains the commercial COPD drug Ohtuvayre, which could help offset the loss of revenue when Keytruda’s patent expires later this decade. July 9, 2025 · 2 min read · Tristan Manalac Pfizer Battles Novo for the Hottest Obesity Biotech Target: Metsera Acquisition date: November 7 Final bid: $10 billion In November, Pfizer acquired one of the brightest rising stars in the weight-loss game with a $10 billion play for Metsera . The takeover represented Pfizer’s comeback in the obesity arena after scrapping a string of disappointing assets over the years, and the pharma had to work extra hard for its prize. Pfizer’s first bid for Metsera was a relatively modest $4.9 billion, which the biotech accepted in September. A month later, however, Novo Nordisk filed an unsolicited rival bid of $8.5 billion to snatch Metsera out from under Pfizer. Metsera dubbed the proposal “superior” to Pfizer’s offer and set off a clock for negotiations. Pfizer bristled at these developments and called Novo’s rival proposal “reckless and unprecedented.” Later, the pharma also claimed that Metsera’s controlling stockholders—including the investors ARCH Venture Fund and Validae Health—“conspired” with Metsera and Novo to promote “anti-competitive activities.” Pfizer sued both Novo and Metsera to stop what it called the “tortious interference” from Novo “to suppress competition.” At one point, the U.S. Federal Trade Commission, joined the fray and wrote to both Novo and Metsera, warning them that the proposed deal structure may be in violation of U.S. merger guidelines. All the while, Novo stood by its decision to try to steal Metsera away—no matter how unpopular it was—upping the bid to $10 billion. Novo’s new CEO, Maziar Mike Doustdar , even goaded Pfizer from the White House. “Our message to Pfizer is that if they would like to buy the company, then put your hand in the pocket and bid higher,” Doustdar said during an event for a drug pricing deal with the government for its GLP-1 drugs. Ultimately, that’s what Pfizer did. The pharma eventually matched Novo’s offer, finally convincing its rival to back down and Metsera to accept the proposal. Obesity Is Metsera Worth the Fuss? Pfizer and Novo Nordisk seem to want Metsera bad. Analysts are wondering, though: Is the obesity biotech really worth this much effort? November 5, 2025 · 4 min read · Annalee Armstrong Sanofi Takes Over Rising Cancer Star Target: Blueprint Medicines Acquisition date: June 2 Final bid: $9.5 billion Closing out this list is the mid-year merger between Sanofi and rare disease specialist Blueprint Medicines, led by CEO Kate Haviland, the third female leader on this list. The companies in June agreed to a $9.5 billion acquisition deal , giving Sanofi the FDA-approved systemic mastocytosis drug Ayvakit and a pipeline of investigational immunology and cancer therapies. In 2024, Ayvakit made $479 million . Because of the Sanofi takeover, Blueprint hasn’t reported sales figures for Ayvakit for the latter half of the year, but the pharma in its Q3 business report credited the drug as being partly responsible for a 57.1% increase in sales. Speaking to analysts in a call after the acquisition, Sanofi’s head of specialty care Brian Foard said that Ayvakit’s launch was “just getting started” and that the pharma sees a lot of long-term potential for the drug. Beyond Ayvakit, Sanofi also got elenestinib, a KIT blocker for systemic mastocytosis. Blueprint’s pipeline also includes BLU-808, in early-stage development for chronic urticaria, allergic rhinoconjunctivities, allergic asthma and mast cell activation syndrome. Blueprint is also bringing to the Sanofi fold several discovery-phase assets for various cancers, including breast cancer and other solid tumors. All told, Leerink analysts in a June 2 note estimated that Blueprint’s revenues could hit $2.1 billion by 2030—though to hit this bar, Sanofi “will need to deliver on its plans to find and treat more patients.” And, for the acquisition to pay off, “it is critical, in our view, that BPMC’s pipeline delivers blockbuster revenue over the long term,” Leerink added. 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