On June 12, 2025 Sumitomo Pharma America, Inc. (SMPA) reported that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to nuvisertib (TP-3654) for the treatment of patients with intermediate or high-risk myelofibrosis (MF) (Press release, Sumitomo Pharmaceuticals, JUN 12, 2025, View Source [SID1234653857]). The FDA Fast Track Designation is granted to investigational therapies being developed to treat serious or life-threatening conditions that demonstrate the potential to address unmet medical needs. Nuvisertib is an oral, investigational, highly selective inhibitor of PIM1 kinase, which demonstrated clinical activity including symptom and spleen responses correlating with cytokine modulation in the updated preliminary Phase 1/2 data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress in Milan, Italy.

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MF, a serious and rare type of blood cancer, is characterized by the buildup of fibrous tissues in the bone marrow which is caused by dysregulation in the Janus-associated kinase (JAK) signaling pathway. The clinical manifestations of MF include an enlarged spleen, debilitating symptoms and reduction in hemoglobin and/or platelets. MF affects 1 in 500,000 people worldwide.1

"This positive momentum for nuvisertib signals strong promise in our pipeline and reflects our dedication to addressing unmet medical needs on behalf of patients with myelofibrosis and their families," said Tsutomu Nakagawa, Ph.D, President and Chief Executive Officer of SMPA. "Receiving FDA Fast Track Designation for nuvisertib in the treatment of myelofibrosis reinforces our confidence in its potential as a treatment option for patients facing a poor prognosis with limited treatment options. We are committed to working closely with the FDA to progress the clinical development of nuvisertib and bring an alternative treatment option to patients with myelofibrosis."

Updated data from the ongoing Phase 1/2 study of nuvisertib in patients with relapsed/refractory MF were presented at the EHA (Free EHA Whitepaper) Congress on June 12, 2025. Preliminary data showed that nuvisertib monotherapy appears to be well tolerated with no dose-limiting toxicities (DLTs). Evaluable patients showed clinical activity including a ≥25% spleen volume reduction (SVR25) in 22.2% of patients and a ≥50% reduction in total symptom score (TSS50) of 44.4% of patients, as well as improvement of bone marrow fibrosis (42.9% patients), hemoglobin (24% patients) and platelet count (26.7% patients). Data also showed that nuvisertib treatment led to significant cytokine modulation [reduction of pro-inflammatory cytokines (e.g. EN-RAGE, MIP-1β) and increase of anti-inflammatory cytokines (e.g. adiponectin)], which demonstrated significant (p<0.001) correlation with symptom and spleen responses. Preclinical2 and emerging clinical data support the development of nuvisertib in combination with JAK inhibitors for the treatment of patients with MF.

"The data observed to date demonstrate promising clinical activity for nuvisertib and the strong potential for selective PIM1 inhibition to slow the progression of myelofibrosis," said Jatin Shah, MD, Chief Medical Officer, Oncology. "Patients with myelofibrosis are in need of new therapeutic approaches, including combination treatment options, that can provide increased and durable response rates with limited hematologic adverse events. The FDA Fast Track Designation reinforces the potential of nuvisertib to provide clinical benefits for patients with myelofibrosis, an unmet medical need."

About Nuvisertib (TP-3654)
Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.2,3 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.3 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.2 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024.

On June 12, 2025 Specialised Therapeutics (ST) reported the expansion of its existing supply and distribution agreement with Incyte Biosciences International Sàrl, the Swiss-based affiliate of Incyte (NASDAQ:INCY), to launch and distribute two additional medicines from its oncology portfolio in Australia, New Zealand and Singapore, with an option to add further countries in the Asia-Pacific region (Press release, Specialised Therapeutics Australia, JUN 12, 2025, https://www.prnewswire.com/news-releases/specialised-therapeutics-expands-partnership-with-incyte-to-include-two-additional-therapies-for-hard-to-treat-conditions-302479723.html [SID1234653856]). The expanded agreement will see new therapies axatilimab (registered as Niktimvo in the United States) and retifanlimab (registered as Zynyz in the U.S. and European Union) added to the current partnered portfolio of Minjuvi (tafasitamab) and Pemazyre (pemigatinib).

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Under the terms of the expanded agreement, Incyte will be responsible for the development, manufacture and supply of both axatilimab and retifanlimab to the region, while ST will have responsibility for regulatory, distribution and local marketing and medical affairs related activities.

ST Chief Executive Officer, Mr Carlo Montagner, welcomed the expansion of the partnership agreement with Incyte, a leading global biopharmaceutical company, and the opportunity to bring these important medicines to eligible patients in the local region.

"Specialised Therapeutics has partnered with Incyte since 2021 and we are delighted to be extending this successful partnership," he said. "As part of our initial agreement, our teams have worked collaboratively to commercialise Minjuvi, which has been approved for use in Australia, and Pemazyre, which has been approved in Australia and Singapore, and we look forward to expanding the portfolio to help more patients with rare and hard-to-treat conditions."

"The addition of axatilimab and retifanlimab to the partnership agreement with Incyte recognises our strong track record of working with local stakeholders to bring innovative medicines to patients where unmet medical needs persist, despite the availability of existing treatments," said Mr Montagner. "Our focus will be on seeking regulatory and reimbursement approval for all four medicines in the region, to ensure equitable access to these treatments are available for patients as soon as practicable."

ST anticipates submitting axatilimab and retifanlimab for local regulatory and reimbursement approval in 2025.

Incyte CEO, Mr Hervé Hoppenot said the expanded partnership agreement reflected the synergies between the two companies, with a shared goal of improving outcomes for patients with unmet medical needs.

"We are pleased to extend our partnership with ST to include axatilimab and retifanlimab," he said. "ST has already demonstrated its ability to navigate complex regional regulatory pathways for Minjuvi and Pemazyre, and we look forward to continuing our work together to make these new therapies available for the oncology community in the Asia-Pacific region."

Axatilimab is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody. It was approved by the US Food and Drug Administration (FDA) in August 2024 as a treatment for adults and children with chronic graft-versus-host disease (GVHD) who have received at least two prior treatments (systemic therapy) and require additional treatment.1 Chronic GVHD usually occurs 3 months after a transplant – typically haematopoietic stem cell or bone marrow transplantation, but occasionally also solid organ transplants – where the donor cells ("graft") attack the graft recipient’s cells ("host").2 Chronic GVHD can affect all organs, but commonly impacts the skin, mouth, eyes, lungs, stomach, bowel, and liver.2,3 The Phase II clinical trial for axatilimab involved 79 patients from 13 countries, including Australia and Singapore.4

Retifanlimab is an intravenous immune checkpoint (PD-1) inhibitor that has been approved in the US in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy.5 Retifanlimab is also approved in the US6 and Europe7 for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). MCC is a rare and aggressive type of skin cancer, that is often difficult to diagnose due to the lack of specific features to distinguish it from other common skin cancers.8 Australia has the highest incidence of MCC in the world.9 The pivotal Phase II clinical trial of retifanlimab in MCC enrolled 101 chemotherapy-naïve patients from 12 countries, including Australia.10 In addition to MCC, retifanlimab, in combination with chemotherapy, is currently being investigated as a potential therapeutic option in other hard-to-treat cancers, including metastatic non-small cell lung cancer (NSCLC).

On June 12, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported updated long term data (up to approximately three years of follow up) from the FELIX study of obecabtagene autoleucel (obe-cel) in adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL), to be presented in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress between June 12-15, 2025, in Milan, Italy (Press release, Autolus, JUN 12, 2025, View Source [SID1234653855]). Autolus will also present an additional oral and poster presentation, the details of which are included below.

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"Obe-cel’s durability of response without any subsequent therapy in two out of every five responders is a key factor leading the transformation of therapy for adult r/r B-ALL patients. At a median follow up of 33 months, we are encouraged to see a continuation of the long-term plateau we observed at the last data cut," said Dr. Christian Itin, Chief Executive Officer of Autolus. "A well-tolerated, effective, durable treatment option for ALL patients who often have a poor prognosis and have had multiple prior treatments is of significant clinical benefit."

Oral S113:
Title: Can CAR T-cell therapy be a definitive treatment for adult r/r B-ALL without transplant? Long-term findings and predictors of sustained remission for obecabtagene autoleucel
Session Name: s447 Immunotherapy and CAR-T cells for ALL
Session room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Jae H Park, MD

Summary: At the updated median follow up of 32.8 months, 38.4% of responders were in ongoing remission without consolidative SCT or other therapies (versus the previously reported 40% at a median follow up of 21.5 months). The 24-month probability of Event Free Survival was 43%, and for Overall Survival was 46%, with an emerging long-term plateau observed. A substantial subset of patients benefit from standalone treatment with obe-cel, achieving long-term remission. No new safety signals or Grade ≥3 secondary malignancies were observed at the extended follow-up. These results suggest that obe-cel may be a definitive treatment for some patients with r/r B-ALL – specific analysis will be needed to determine which patients may need additional treatments.

The multivariate analysis demonstrated that Philadelphia chromosome-positive disease, earlier obe-cel use, and relapsed disease correlated with achieving higher remission rates. Lower disease burden at lymphodepletion and ongoing CAR T-cell persistence were independent factors associated with long-term remission and survival.

Oral S114:
Title: Efficacy and Safety Outcomes of Obecabtagene Autoleucel (obe-cel) Stratified by Age in Patients with r/r B-ALL
Session Name: Immunotherapy and CAR-T cells for ALL
Session Room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Bijal D. Shah, MD

Summary: Obe-cel treatment was associated with deep and durable remissions resulting in favorable overall remission rate, event free survival, and overall survival with low incidence of Grade ≥3 CRS and ICANS in both age groups (<55 years and ≥55 years). These findings indicate that obe-cel is effective and has a positive benefit and risk profile regardless of patient age, including in older adults with R/R B-ALL.

Poster PF378:
Title: Predicting Hematotoxicity Risk and Outcomes in Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-All): Should Hematotox Models be CAR Specific Rather than Disease Specific
Session Title: Poster Session 1
Session date and time: Friday, June 13; 18:30 – 19:30 CEST.
Presenting Author: Claire Roddie, MD

Summary: Although both the CAR-Hematotox (CAR-HT) model, and the ALL-Hematotox (ALL-HT) model show potential, ALL-HT appears to improve risk stratification and may be a better predictor of response, survival and safety outcomes in adult patients with r/r B-ALL treated with obe-cel, than CAR-HT. Taken together with other published reports, our data suggest that the strength of HT-model predictions may be CAR T-cell product specific. Further analyses are needed.

On June 12, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that it will present new clinical data from three cornerstone hematology assets at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (Press release, BeOne Medicines, JUN 12, 2025, View Sourcenews/beone-medicines-showcases-breakthrough-data-in-cll-and-mcl-at-eha-2025/b755b055-956b-40e5-8a80-763881ca3f12" target="_blank" title="View Sourcenews/beone-medicines-showcases-breakthrough-data-in-cll-and-mcl-at-eha-2025/b755b055-956b-40e5-8a80-763881ca3f12" rel="nofollow">View Source [SID1234653854]). Four oral presentations highlight the promising clinical activity of BeOne’s next-generation BCL2 inhibitor sonrotoclax, BTK protein degrader BGB-16673, and the backbone of our hematology franchise, BTK inhibitor BRUKINSA (zanubrutinib), which has the broadest label globally of any approved BTK inhibitor. These data reinforce the company’s strategic vision to redefine the standard of care for B-cell malignancies.

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"The data presented at EHA (Free EHA Whitepaper) 2025 underscore the strength of BeOne’s comprehensive hematology pipeline, built on the success of BRUKINSA, the only BTK inhibitor to demonstrate superior progression-free survival over ibrutinib in a Phase 3 trial,1" said Lai Wang, Ph.D. Global Head of R&D at BeOne. "With our potentially best-in-class BCL2 inhibitor, sonrotoclax, and first-in-class BTK degrader, BGB-16673, we are advancing innovative therapies aimed at addressing resistance mechanisms and improving outcomes for patients with B-cell malignancies."

The data presented at EHA (Free EHA Whitepaper) 2025 support the ongoing advancement of sonrotoclax and BGB-16673 into Phase 3 studies and lay the groundwork for BeOne’s first regulatory submissions for these programs. The company’s integrated development approach—anchored in differentiated mechanisms and translational science—positions its programs to address key areas of unmet need in hematologic oncology.

"While existing therapies have improved outcomes in CLL and related malignancies, many patients still relapse or develop resistance and continue to face limited options," said Stephan Stilgenbauer, Professor of Medicine and Medical Director of the Comprehensive Cancer Center Ulm (CCCU), Head of the Early Clinical Trials Unit (ECTU), and Head of the Division of CLL Dept. of Internal Medicine III at Ulm University. "The updated data presented at EHA (Free EHA Whitepaper) underscore the potential of novel approaches, including BTK degradation and BCL2-based combinations, to overcome known mechanisms of resistance and expand treatment options for patients."

Sonrotoclax + BRUKINSA Demonstrates Deep Responses in CLL and MCL

BeOne’s data will highlight the emerging potential of its next-generation assets to address the unmet needs of patients with B-cell malignancies.

Updated results from Phase 1 studies evaluating sonrotoclax in combination with BRUKINSA in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and with R/R mantle cell lymphoma (MCL) demonstrated consistent, deep responses and a manageable safety profile. Sonrotoclax is well-positioned to improve key aspects of the BCL2 inhibitor class and has demonstrated robust and durable antitumor activity and a tolerable safety profile across all dose levels.

R/R CLL/SLL (Oral Presentation S159):
Overall response rate (ORR): 96%; complete response (CR): 52% across all dose levels
Undetectable minimal residual disease (uMRD) was achieved in 82% of patients overall, with responses deepening over time
No tumor lysis syndrome (TLS) or febrile neutropenia observed
R/R MCL (Oral Presentation S234):
ORR: 79%; CR rate: 66%, with 84% of responders in ongoing response at data cutoff
No TLS or atrial fibrillation/flutter reported; the most common grade ≥3 adverse event was neutropenia (19.6%)
Robust Early Results in CLL and WM with Potentially First-in-Class BTK Degrader

As the most clinically advanced BTK degrader, BGB-16673 continues to show potential in patients with various hematological malignancies. Updated data from the ongoing Phase 1 CaDAnCe-101 study of BGB-16673 in R/R CLL/SLL and R/R Waldenström macroglobulinemia (WM) showed substantial antitumor activity and a tolerable safety profile across heavily pretreated populations.

R/R CLL/SLL (Oral Presentation S158):
ORR: 84.8% across all dose levels and 93.8% at the recommended phase 2 dose of 200mg
Patients were heavily pretreated, with most previously treated with BTK and BCL2 inhibitors. The median number of prior therapies was four. Responses deepened over time
Grade ≥3 adverse events included neutropenia (24%) and pneumonia (11%); no reported deaths were attributed to study drug
R/R Waldenström Macroglobulinemia (Oral Presentation S231):
ORR: 84.4%; major response rate: 75.0%; very good partial response rate (VGPR): 31.3%
Rapid onset of response (median: 1.0 month), with continued deepening over time
Efficacy observed across genetic backgrounds and prior BTKi exposure
Most common grade ≥3 TEAE was neutropenia/neutrophil count decreased (31%); no reported atrial fibrillation or febrile neutropenia.
BRUKINSA Monotherapy Showed Sustained OS and PFS Benefit

Data from Arm D of the SEQUOIA Phase 3 trial will also be presented at the meeting (Abstract PS1566), demonstrating that treatment with BRUKINSA plus venetoclax has the potential to drive progression-free survival and overall deep and durable responses across the frontline CLL patient spectrum, including patients with high-risk mutational status. SEQUOIA Arm D investigated BRUKINSA plus venetoclax in 114 patients with treatment-naïve (TN) CLL / SLL with or without del(17p) and/or TP53 high-risk mutations. At a median follow-up of 31.2 months, the combination induced a high 24-month progression-free survival (PFS) rate of 92% (95% CI, 85-96%) and an impressive overall response rate (ORR) of 97%. The 24-month overall survival (OS) rate was 96% (95% CI, 90%-98%). Notably, these benefits were observed regardless of del(17p)/TP53 mutational status. The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified.

Arm C of the SEQUOIA study investigated BRUKINSA monotherapy in patients with TN CLL / SLL and del(17p) mutations, representing the largest prospective cohort of CLL/SLL patients with del(17p), will be presented at EHA (Free EHA Whitepaper) (Abstract: PS1565). At a median follow-up of over 5.5 years (65.8 months), most patients remained progression-free. Notably, at 60 months, 72.2% of patients who received BRUKINSA remained progression-free (95% CI, 62.4, 79.8). When adjusted for the impact of the COVID-19 pandemic, 73.0% of patients in the cohort remained progression-free (95% CI, 63.3, 80.6) at 60 months. The 60-month OS rate was 85.1% (95% CI, 76.9, 90.6) and 87.0% (95% CI, 79.0, 92.1) when adjusted for COVID-19. At the time of data cut-off, the ORR was 97.3%, and 62.2% of patients were still receiving treatment with BRUKINSA. The safety profile of BRUKINSA was consistent with the results of prior studies with no new safety signals identified.

BeOne will host an investor R&D Day on June 26 at 8:30 am ET covering our deep and broad global innovation pipeline and platforms, as well as the Company’s vision, differentiated capabilities, and value creation drivers. The live webcast can be accessed from the investors section of BeOne’s website at View Source, View Source, or View Source An archived replay will be available to investors for 90 days following the event.

About Sonrotoclax (BGB-11417)

Sonrotoclax is designed to block the B-cell lymphoma 2 (BCL2) protein, which is one of several proteins that help cancer cells survive. It is part of a group of drugs called BH3 mimetics, which mimic natural cell death signals. Studies in the lab and during early drug development have shown that sonrotoclax is a potent and specific inhibitor of BCL2 with a short half-life and no accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, and more than 1,900 patients have been enrolled to date across the global development program. The U.S. Food and Drug Administration (FDA) granted sonrotoclax Fast Track Designation for the treatment of adult patients with mantle cell lymphoma (MCL) and Waldenström macroglobulinemia (WM).

About BGB-16673

BGB-16673 is an orally available Bruton’s tyrosine kinase (BTK) targeting protein degrader from BeOne’s chimeric degradation activation compound (CDAC) platform. BGB-16673 is designed to promote the degradation, or breakdown, of both wildtype and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease. BGB-16673 is the most advanced BTK protein degrader in the clinic, with an extensive global clinical development program. The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to BGB-16673 for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL).

About BRUKINSA (zanubrutinib)

BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. Additionally, BRUKINSA is also the only BTK inhibitor to demonstrate PFS superiority to a first-generation BTK inhibitor in a Phase 3 study.

The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved for at least one indication in more than 75 markets, and more than 200,000 patients have been treated globally.

Select Important Safety Information

Serious adverse reactions, including fatal events, have occurred with BRUKINSA, including hemorrhage, infections, cytopenias, second primary malignancies, cardiac arrhythmias, and hepatotoxicity (including drug-induced liver injury).

In the pooled safety population (N=1729), the most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA were neutrophil count decreased (51%), platelet count decreased (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%).

Please see full U.S. Prescribing Information including U.S. Patient Information.

The information provided in this press release is intended for a global audience. Product indications vary by region.

On June 12, 2025 Blueprint Medicines Corporation (Nasdaq: BPMC) reported data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM) (Press release, Blueprint Medicines, JUN 12, 2025, View Source [SID1234653853]). 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 (EHA) (Free EHA Whitepaper) (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.

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"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 www.blueprintmedicines.com.