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

Structure/Sequence

Molecular FormulaC26H27FN10

InChIKeyDWYRIWUZIJHQKQ-SANMLTNESA-N

CAS Registry1703793-34-3

Clinical Trials associated with Avapritinib

ChiCTR2600129807

/ Not yet recruitingPhase 4IIT

Luvometinib Monotherapy or Combined With Cytarabine for Langerhans Cell Histiocytosis: A Response-adapted, Interventional, Prospective Study

Start Date31 Aug 2026

Sponsor / Collaborator

The Ninth People's Hospital of Shanghai Jiaotong University Medical College

NCT07255638

/ RecruitingNot Applicable

A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings

This is a non-interventional study assessing the effectiveness of avapritinib (BLU-285) in the management of ISM in real-world settings in Germany. The study also seeks to address the existing data gap in the natural history and management of participants with ISM.
The study is designed to follow each participant up to a maximum of 24 months.

Start Date09 Dec 2025

Sponsor / Collaborator

Blueprint Medicines Corp.

NCT07028073

/ RecruitingPhase 1/2IIT

Avapritinib Combined With Standard Therapy for the Treatment of Newly Diagnosed KIT Mutation Acute Myeloid Leukemia With t(8;21)(q22;q22.1); Inv(16)(p13.1q22) or t(16;16)(p13.1;q22): a Prospective, Multi-center, Single-arm, Two-cohorts Study

The goal of this clinical trial is to learn if avapritinib combined with standard induction therapy works to treat newly diagnosed adult acute myeloid leukemia (AML) patients with KIT mutations and t(8;21)(q22;q22.1); inv(16)(p13.1q22) or t(16;16)(p13.1;q22). It will also investigate the safety and tolerability of this combination therapy. The main questions it aims to answer are:
To determine the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D) of avapritinib combined with chemotherapy by Dose-limiting toxicity (DLT).
Does this combination therapy improve the rates of minimal residual disease (MRD) negativity and long-term survival outcomes?

Start Date15 May 2025

Sponsor / Collaborator

The First Affiliated Hospital of Soochow University

100 Clinical Results associated with Avapritinib

100 Translational Medicine associated with Avapritinib

100 Patents (Medical) associated with Avapritinib

274

Literatures (Medical) associated with Avapritinib

31 Dec 2026·Future Science OA

A comprehensive analysis of the adverse drug events of metastatic and unresectable gastrointestinal stromal tumor treatment options: avapritinib, imatinib, regorafenib, ripretinib, and sunitinib safety insights and implications

Article

Author: Andrews, Michael B. ; Thompson, Cameron M. ; Adler, Douglas G.

BACKGROUND:

Treatments for metastatic, unresectable, or treatment-resistant gastrointestinal stromal tumors (GIST) include avapritinib, imatinib, regorafenib, ripretinib, and sunitinib. The FDA Adverse Event Reporting System (FAERS) database collects medication-related adverse events (AEs).

DESIGN AND METHODS:

Reports in the FAERS database from each medication's FDA approval to June 30, 2025 were analyzed. Reports containing other medications or indications outside of GIST treatment were excluded.

RESULTS:

Avapritinib was associated with fatigue (n = 826, 10.2%), nausea/vomiting (n = 574, 7.1%), eye/facial swelling (n = 631, 7.8%), diarrhea n = 361, 4.5%), peripheral edema (n = 231, 2.5%). Imatinib was associated with nausea/vomiting (n = 677, 4.4%), fatigue (n = 549, 3.6%), anemia (n = 404, 2.6%), abdominal pain (n = 400, 2.6%), and rash (n = 340, 2.2%), hemorrhage (317 AEs, 2.1%). Regorafenib was associated with fatigue (n = 104, 8.6%), palmar-plantar erythrodyesthesia syndrome (n = 66, 5.4%), diarrhea (n = 60, 4.9%). Ripretinib was associated with fatigue (n = 534, 8.4%), alopecia (n = 355, 5.6%), nausea/vomiting (n = 273, 4.3%). Sunitinib was associated with fatigue (n = 420, 6.1%%), nausea/vomiting (n = 284, 4.2%), diarrhea (n = 262, 3.8 %), hemorrhage (n = 149, 2.2%).

CONCLUSIONS:

The FAERS database revealed multiorgan AEs associated with five GIST treatment medications. Novel findings include eye/facial and peripheral edema with avapritinib, palmar-plantar erythrodyesthesia syndrome, headache/migraine, and gait abnormalities across all five medications, pancytopenia with imatinib, and hemorrhage with imatinib and sunitinib.

01 Jul 2026·JOURNAL OF GENE MEDICINE

Identification and Experimental Verification of Key Genes Related to the Ras Signaling Pathway and the Hippo Signaling Pathway in Osteoarthritis Based on Transcriptome Data

Article

Author: Lu, Ying ; Sheng, Bin ; Liu, Diefeng ; Zhang, Lina

ABSTRACT:

Background:

Osteoarthritis (OA) is a chronic degenerative joint disease characterized by the progressive deterioration of articular cartilage, significantly impacting the quality of life in middle‐aged and elderly populations. The Ras and Hippo signaling pathways play critical roles in regulating cell proliferation, differentiation, and stress responses; however, their interactive mechanisms in OA remain unclear. This study aimed to identify key genes associated with these two pathways using bioinformatic approaches and to elucidate their potential mechanisms in OA.

Methods:

Transcriptomic data of OA along with Ras signaling pathway‐related genes (RSPRGs) and Hippo signaling pathway‐related genes (HSPRGs) were obtained from public databases. Differentially expressed genes (DEGs) were identified, and key genes were screened through machine learning, expression validation, and receiver operating characteristic (ROC) curve analysis. Functional insights were further explored via gene set enrichment analysis (GSEA), subcellular localization, immune infiltration analysis, regulatory network construction, and drug prediction. Finally, the expression of key genes was validated in clinical samples.

Results:

KIT and CSF1R were identified as key genes. GSEA indicated their involvement in pathways such as the lysosome pathway. Subcellular localization predicted that KIT and CSF1R are distributed in the nucleus, extracellular region, and plasma membrane. Immune infiltration analysis revealed that KIT showed a positive correlation with eosinophils and a negative correlation with immature dendritic cells, whereas CSF1R was positively correlated with macrophages and negatively correlated with CD56 bright natural killer cells. Drug prediction suggested interactions between the key genes and several therapeutic agents, including avapritinib and IMC‐CS4. Subsequently, we validated our findings in cartilage tissue samples and discovered that compared to the control group, both CSF1R mRNA and protein expression were significantly upregulated in OA tissue, while KIT expression was significantly downregulated. The same were also validated in immunofluorescence staining of chondrocytes.

Conclusion:

This study identified KIT and CSF1R as key genes in OA, providing new theoretical insights and potential targets for mechanistic research and targeted therapy.

01 Jul 2026·Cancer research communications

Predicting Clinical Sensitivities of PDGFRA Exon 18 Mutations to Imatinib and Avapritinib to Optimize Gastrointestinal Stromal Tumor Treatment

Article

Author: Bauer, Sebastian ; Khosroyani, Homma M. ; Thirasastr, Prapassorn ; Rutkowski, Piotr ; Heinrich, Michael C. ; Town, Ajia ; Rothschild, Sara ; Liu, Mengyuan ; Hohenberger, Peter ; Falkenhorst, Johanna ; Call, Jerry ; Evans, Denisse ; Killian, Jonathan K. ; Teuber, Alina ; Beadling, Carol ; Schöffski, Patrick ; Klug, Lillian R. ; Chi, Ping ; Frampton, Garrett M. ; Somaiah, Neeta ; Brahmi, Mehdi ; Agaimy, Abbas

Abstract:

The most common platelet-derived growth factor receptor α (PDGFRA) alteration in gastrointestinal stromal tumors (GIST) is the exon 18 activation loop mutation D842V, which is resistant to imatinib and other type II tyrosine kinase inhibitors (TKI) but sensitive to type I TKI avapritinib. Avapritinib is FDA-approved for first-line treatment of all PDGFRA exon 18–mutant GIST cases but is only available for D842V-mutant cases outside the United States. Non-D842V exon 18–mutant GIST cases are understudied and lack evidence-based treatment guidelines. However, there are a few previous reports describing non-D842V exon 18–mutant patients with GIST who responded well to imatinib therapy. Given that imatinib is more tolerable, globally accessible, and significantly less expensive than avapritinib, we sought to define which patients could be treated with imatinib rather than avapritinib. We assembled a cohort of more than 1,000 PDGFRA exon 18–mutant GIST cases and identified that 78% of these mutations involved a key autoinhibitory aspartic acid residue at position 842. Using cell-based models, we demonstrated that imatinib sensitivity was dependent on the amino acid class of the 842-position residue, with all hydrophobic amino acids except alanine conferring resistance. In contrast, all 842-position mutations were avapritinib-sensitive. Structural modeling supported our biochemical results and revealed how 842-position mutations induce changes that can interfere with imatinib binding. Lastly, our biochemical data were validated using imatinib response data for first-line metastatic disease; patients with predicted exon 18–sensitive mutations had longer progression-free survival than patients with predicted imatinib-resistant mutations. These results provide key evidence that should be used to guide therapy selection for PDGFRA-mutant GIST.

Significance::

Biochemical and structural modeling of PDGFRA exon 18 842-position mutations allows for the prediction of clinical TKI responses. These data can be used to generate new treatment guidelines for PDGFRA exon 18–mutant GISTs and select optimal therapies for patients.

164

News (Medical) associated with Avapritinib

10 Aug 2026

·www.biospace.com

Cogent Biosciences Reports Recent Business Highlights and Second Quarter 2026 Financial Results

On track for potential approval of bezuclastinib for GIST patients (PDUFA November 30) and NonAdvSM patients (PDUFA December 30) later this year; AdvSM New Drug Application submitted to FDA on June 30 Completed onboarding of expert cross-functional Cogent customer-facing team, including all field-based commercial and medical team members Strong pro forma cash balance of $865.9 million, sufficient to fund operations into late 2028 WALTHAM, Mass. and BOULDER, Colo., Aug. 10, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today reported financial results for the second quarter ended June 30, 2026. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); “The first half of 2026 has been marked by transformative milestones toward our vision of creating best-in-class therapies for patients fighting rare, mutationally driven diseases as we submitted three New Drug Applications following positive results from each of the bezuclastinib pivotal trials,” said Andrew Robbins, the Company’s President and Chief Executive Officer. “We are excited to welcome the new Cogent customer facing team to the company, and supported by our strong balance sheet, we are well prepared to launch bezuclastinib and advance the standard of care for patients with GIST and Systemic Mastocytosis while continuing to invest in our broader pipeline of precision therapies for genetically defined diseases.” Recent Business Highlights Cogent Biosciences Integrated Business Team Successfully hired and onboarded all Integrated Business Team members, spanning clinical account managers, patient access navigators and patient educators. Selected for their expertise in commercializing Oncology and Rare Disease products, Cogent now has an exceptional commercial field team in place across the country. Together with our existing medical affairs team, Cogent is well prepared for potential launch with a broad, expert, cross-functional customer-facing organization. Bezuclastinib in GIST Presented detailed clinical data from the Phase 3 PEAK trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with imatinib-resistant or intolerant Gastrointestinal Stromal Tumors (GIST) at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. As of the cutoff date, September 30, 2025, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of PFS, reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant/intolerant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. Data for overall survival remains immature. Based on the ongoing patients receiving treatment on the bezuclastinib arm as of March 31, 2026, the mean duration of treatment for the bezuclastinib combination is estimated to be 21.4 months. Announced the initiation of a single-arm, 40 patient extension cohort of the PEAK trial investigating the safety and efficacy of the bezuclastinib combination in first-line GIST patients with KIT exon 9 primary mutations who have received limited or no imatinib treatment. This cohort is designed to prospectively measure ORR and PFS in this patient population, building upon the 25.1 month mPFS reported in a subgroup of 32 patients with detectable exon 9 mutations treated with the bezuclastinib combination in the Phase 3 PEAK trial. Announced FDA acceptance of the New Drug Application (NDA) with priority review for bezuclastinib in combination with sunitinib in patients with GIST who have received prior treatment with imatinib. Bezuclastinib in Systemic Mastocytosis Announced submission of the NDA for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM). Presented detailed data from the pivotal APEX trial at the 2026 European Hematology Association (EHA) Congress. As of the March 31, 2026 data cutoff, 81 AdvSM patients were treated with 150 mg of bezuclastinib, including 57 patients with SM-AHN, 11 patients with ASM and 13 patients with MCL. The primary endpoint of response per mIWG-MRT-ECNM was assessed on 68 evaluable patients and showed 65% ORR (CR+CRh+PR+CI), including 57% of patients who achieved CR, CRh or PR as best response. Announced completion of enrollment in the “avapritinib switch” SUMMIT extension trial with preliminary results expected by end of 2026. Presented preclinical data from the novel JAK2 V617F program at EHA. The poster highlighted CGT1145, a potent inhibitor of the JAK2 V617F mutation with >100x selectivity over JAK2 WT and the JAK1/3 isoforms, along with high oral bioavailability and low clearance across species. CGT1145 has the potential to eradicate JAK2 V617F myeloproliferative neoplasm propagating cells and induce molecular remission with improved hematologic tolerability. Anticipated Upcoming Milestones Potential FDA approval of bezuclastinib in GIST – PDUFA date of November 30, 2026 Potential FDA approval of bezuclastinib in NonAdvSM – PDUFA date of December 30, 2026 Submit Investigational New Drug (IND) applications for CGT1815, Cogent’s novel, selective pan-KRAS(ON) inhibitor, and CGT1145, Cogent’s novel, selective JAK2 V617F inhibitor Complete dose escalation for CGT4255, Cogent’s CNS-penetrant, selective mutant ErbB2 inhibitor Second Quarter 2026 Financial Results Cash Position: As of June 30, 2026, Cogent had cash, cash equivalents and marketable securities of $792.3 million. The company expects its existing cash, cash equivalents and marketable securities, with the $73.6 million gross proceeds from shares sold through the Company’s at-the-market (ATM) facility after the end of the quarter, will be sufficient to fund its operating expenses and capital expenditure requirements into late 2028, including through potential FDA approvals of bezuclastinib for GIST, NonAdvSM and AdvSM and early commercial launch activities. R&D Expenses: Research and development expenses were $70.8 million for the second quarter of 2026 as compared to $62.2 million for the second quarter of 2025. The increase was primarily driven by costs to support the SUMMIT, PEAK and APEX clinical programs, regulatory activities associated with potential approvals of bezuclastinib, continued investment in the company's early-stage research pipeline, and pre-approval manufacturing costs that will be capitalized following anticipated FDA approval. R&D expenses include non-cash stock compensation expense of $8.6 million for the second quarter of 2026 as compared to $5.0 million for the second quarter of 2025. G&A Expenses: General and administrative expenses were $31.8 million for the second quarter of 2026 as compared to $13.4 million for the second quarter of 2025. The increase was primarily driven by continued investments in commercial readiness, including personnel and infrastructure to support the anticipated launch of bezuclastinib, as well as overall organizational growth. G&A expenses include non-cash stock compensation expense of $8.5 million for the second quarter of 2026 as compared to $4.8 million for the second quarter of 2025. Net Loss: Net loss was $96.4 million for the second quarter of 2026 as compared to a net loss of $73.5 million for the same period of 2025. Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) Cogent also announced today that, on August 4, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to 59 new employees under the company’s 2020 Inducement Plan with grant dates of August 4, 2026 and August 10, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 226,700 shares of Cogent common stock and (ii) 189,700 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date. About Cogent Biosciences, Inc. Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Kα, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at . Follow Cogent Biosciences on social media: X and LinkedIn . Information that may be important to investors will be routinely posted on our website and X . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the company’s expectation to receive regulatory approval of bezuclastinib later this year for both GIST patients and NonAdvSM patients; the company’s anticipated cash runway into late 2028 and the expectation that it will be sufficient to fund operations through anticipated FDA approvals of bezuclastinib for GIST, NonAdvSM and AdvSM and early commercial launch activities; the company’s level of preparation to launch bezuclastinib and advance the standard of care for patients with GIST and Systemic Mastocytosis while also continuing to invest in its broader pipeline of precision therapies for genetically defined diseases; the company’s expectation that it will present preliminary results from its “avapritinib switch” SUMMIT extension trial by the end of 2026; the potential of CGT1145 to eradicate JAK2 V617F myeloproliferative neoplasm propagating cells and induce molecular remission with improved hematologic tolerability; the company’s plans to submit INDs for both CGT1815, its novel, selective pan-KRAS(ON) inhibitor, and CGT1145, its novel, selective JAK2 V617F inhibitor; and the company’s plans to complete dose escalation for CGT4255, its CNS-penetrant, selective mutant ErbB2 inhibitor. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words or expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Annual Report on Form 10-K filed with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. COGENT BIOSCIENCES, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands) (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Operating expenses: Research and development $ 70,811 $ 62,203 $ 146,176 $ 125,232 General and administrative 31,827 13,379 60,069 25,283 Total operating expenses 102,638 75,582 206,245 150,515 Loss from operations (102,638 ) (75,582 ) (206,245 ) (150,515 ) Other income: Interest income 7,461 2,373 15,069 5,325 Interest expense (1,217 ) (314 ) (2,430 ) (314 ) Other income (expense), net (11 ) (6 ) (151 ) (11 ) Total other income, net 6,233 2,053 12,488 5,000 Net loss $ (96,405 ) $ (73,529 ) $ (193,757 ) $ (145,515 ) COGENT BIOSCIENCES, INC. SELECTED CONDENSED CONSOLIDATED BALANCE SHEET DATA (in thousands) (unaudited) June 30, December 31, 2026 2025 Cash, cash equivalents and marketable securities $ 792,300 $ 900,765 Working capital $ 745,524 $ 846,402 Total assets $ 834,546 $ 937,607 Total liabilities $ 302,158 $ 301,236 Total stockholders’ equity $ 532,388 $ 636,371 CONTACT: Contact: Christi Waarich Sr. Director, Investor Relations christi.waarich@cogentbio.com 617-830-1653

Clinical ResultPhase 3Financial StatementDrug ApprovalASCO

30 Jul 2026

·www.sanofi.com

Press Release: Q2 2026: double-digit sales growth and strong business EPS growth; 2026 guidance upgraded

Share Q2 2026: double-digit sales growth and strong business EPS growth; 2026 guidance upgraded July 30, 2026 Q2 sales growth of 17.8% at CER1 and business earnings per share (EPS)2 of €2.09 Pharma launches sales increased by 48.3%, reaching €1.3 billion, driven mostly by Ayvakit, ALTUVIIIO, and Sarclisa Dupixent sales increased by 37.6% to €5.2 billion, above €5 billion per quarter for the first time Vaccines sales decreased by 4.7% to €1.1 billion, impacted by a high basis of comparison in influenza vaccines Research and Development expenses reached €2.2 billion, up by 17.9%, including cost from pipeline prioritizations Selling and general expenses reached €2.5 billion, up by 9.0%, mainly because of recent acquisitions and one-offs Business EPS was €2.09, up by 33.3% at CER; 31.4% at actual exchange rates; IFRS EPS €0.29 Pipeline update Seven regulatory approvals; in immunology, rare diseases, oncology, and neurology Two positive phase 3 readouts: Nexviazyme in infantile-onset Pompe disease; amlitelimab maintenance in atopic dermatitis Four regulatory submission acceptances, four regulatory designations (priority review, fast-track, breakthrough) Strategic pipeline decisions: amlitelimab will not progress to global regulatory submission. Itepekimab and balinatunfib clinical development programs discontinued Capital allocation Completion of the €1 billion share buyback program Sustainability The Impact® medicines portfolio is now available in 30 underserved countries, supporting the Global Health Unit’s goal of reaching two million patients with non-communicable disease treatments by 2030 Guidance upgraded In 2026, sales are now expected to grow by around 10% at CER. Business EPS at CER is expected to grow slightly faster than sales.3 Belén Garijo, Chief Executive Officer: “We delivered double-digit sales growth and strong business EPS growth in Q2. Sales increased by 17.8%, driven by Pharma launches, including recent acquisitions, and by Dupixent, up by 37.6%. Business EPS increased by 33.3%, supported by disciplined cost management and one-offs. Pipeline milestones included seven regulatory approvals, two positive phase 3 studies, and four additional regulatory designations. Based on our strong performance in the first half and anticipating normalization of growth in the second half, we are upgrading our 2026 guidance. Sales are now expected to grow by around 10% with business EPS growing slightly faster than sales at constant exchange rates — delivering profitable, sustainable growth. My first months as CEO have focused on assessing the challenges facing Sanofi and working on an accelerated transformation roadmap that leverages our commercial strengths and focuses on the need for R&D and pipeline improvements. We have taken the first decisive steps on pipeline prioritization and recently, we appointed a focused Executive Committee aligned with the strategic priorities. Looking ahead, we're confident in our trajectory of profitable growth: Dupixent sales are now expected to reach around €25 billion in 2030, complemented by approximately €10 billion from Pharma launches — both at constant exchange rates. This strong financial foundation is expected to support continued pipeline enhancement and external growth opportunities in the years ahead. We are committed to acting and reporting on our progress to shareholders and other stakeholders in the quarters to come.” 1 Changes in net sales are at constant exchange rates (CER) unless stated otherwise (definition in Appendix 8).2 To facilitate an understanding of operational performance, Sanofi comments on the business net income, a non-IFRS financial measure (definition inAppendix 8). The income statement is in Appendix 3 and a reconciliation of IFRS net income to business net income is in Appendix 4.3 Applying July 2026 average currency exchange rates, the currency impacts are estimated at c.-1% on sales and at c.-2% on business EPS.4 Free cash flow is a non-IFRS financial measure (definition in Appendix 8). Attachment Press release Share

Phase 3VaccineClinical ResultBreakthrough TherapyFinancial Statement

28 May 2026

·allsci.com

Cogent files KIT-targeted bezuclastinib + Sutent for post-imatinib GIST with FDA priority review

Cogent Biosciences (Nasdaq: COGT), based in Waltham, Massachusetts, has submitted a New Drug Application (NDA) to the US FDA for bezuclastinib in combination with sunitinib (Sutent) in patients with gastrointestinal stromal tumors (GIST) who have received prior treatment with imatinib. The FDA has accepted the application and granted Priority Review, with a PDUFA target action date of November 30, 2026. The NDA seeks approval for bezuclastinib used in combination with sunitinib in the second-line GIST setting, specifically in patients with imatinib-resistant or intolerant disease. The FDA has indicated it does not plan to convene an advisory committee and has not identified any potential review issues at this stage, the company said. The filing builds on prior regulatory designations, including Breakthrough Therapy Designation and eligibility for Real-Time Oncology Review, both granted following Phase III PEAK trial data. The combination of Priority Review and these earlier designations reflects the FDA’s assessment of the unmet need in this patient population, where sunitinib monotherapy has remained the standard of care since its approval in 2006. The NDA is supported by data from the Phase III PEAK trial (NCT05208047), a global, randomized, open-label study evaluating bezuclastinib in combination with sunitinib versus sunitinib monotherapy in patients with imatinib-resistant or intolerant advanced GIST. As of the data cutoff of September 30, 2025, the combination arm met the primary endpoint of progression-free survival (PFS) with a hazard ratio of 0.50 (95% CI: 0.39–0.65; p \< 0.0001), corresponding to a 50% reduction in the risk of disease progression or death compared with sunitinib alone. Median PFS, assessed by blinded independent central review, was 16.5 months for the combination versus 9.2 months for sunitinib monotherapy. In imatinib-resistant patients, the objective response rate (ORR) was 46% in the combination arm compared with 26% in the sunitinib arm. Overall survival data remain immature. On safety, Grade 3 or higher treatment-emergent adverse events occurring in either arm included hypertension (29.4% combination vs 27.4% sunitinib), neutropenia (15.2% vs 15.4%), elevated ALT/AST (10.8% vs 1.4%), anemia (9.3% vs 4.8%), and diarrhea (7.8% vs 7.2%). Treatment discontinuation due to treatment-related adverse events occurred in 7.4% of patients in the combination arm versus 3.8% in the sunitinib arm. Hepatic laboratory abnormalities were predominantly low grade, transient, and reversible; all Grade 3 ALT/AST elevations resolved and no Grade 4 elevations were reported. Bezuclastinib dose reductions due to ALT/AST elevations occurred in 12.7% of patients, with 1.5% discontinuing bezuclastinib for this reason. Bezuclastinib is a selective KIT tyrosine kinase inhibitor designed to inhibit KIT exon 17 activation-loop mutations alongside primary KIT mutations — a resistance mechanism that limits the efficacy of earlier-generation TKIs including sunitinib. In the GIST setting, acquired resistance to imatinib is driven predominantly by secondary KIT kinase domain mutations, many of which fall in the activation loop and are poorly covered by sunitinib. The filing adds to a second-line GIST landscape that has seen no new approved therapy since sunitinib’s approval nearly two decades ago. Approved agents across lines of therapy include regorafenib (Stivarga) in the third-line setting, avapritinib (Ayvakit) for PDGFRA exon 18-mutant disease, and ripretinib (Qinlock) for patients who have received three or more prior kinase inhibitors. None of these approvals addressed the second-line population with a combination approach or demonstrated superiority over an active comparator in a randomized Phase III trial in this setting, according to the available clinical record. The PEAK data, if reflected in an eventual approval, would position bezuclastinib plus sunitinib as the first regimen to demonstrate a statistically significant PFS advantage over sunitinib monotherapy in a randomized trial in post-imatinib GIST. Full results from PEAK are scheduled for oral presentation at the American Society of Clinical Oncology annual meeting on May 30, 2026. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

Phase 3Priority ReviewBreakthrough TherapyNDAClinical Result

100 Deals associated with Avapritinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11279	Avapritinib	L01XE	DB15233

R&D Status

Approved

10 top approved records.

to view more data

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

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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


NCT03731260 (PIONEER, Pubmed \| J Am Acad Dermatol)	Phase 2	Indolent systemic mastocytosis KIT D816V mutation	212	Avapritinib 25 mg once daily	jbmfesiacl(pppxkganfb) = kajgsltxup odkpralepj (plhflfshjk ) View more	Positive	01 Jun 2026
NCT03731260 (PIONEER, Pubmed \| J Am Acad Dermatol)	Phase 2	Indolent systemic mastocytosis KIT D816V mutation	212	Placebo	jbmfesiacl(pppxkganfb) = kbegyzvduo odkpralepj (plhflfshjk ) View more	Positive	01 Jun 2026
NCT03580655 (PATHFINDER, Pubmed \| Blood Adv)	Phase 1	Aggressive Systemic Mastocytosis D816V-mutant KIT	107	Avapritinib 200 mg once daily	wcjrevngdf(skbgmcokaw) = sqfieytztb ansybxnwvl (zxespesdqn, 63 - 83) View more	Positive	26 May 2026
DDW2026	Not Applicable	Gastrointestinal Stromal Tumors KIT gene mutation \| PDGFRA gene mutation \| BRAF kinase mutation	14	Avapritinib	rfnolfllns(eavmpltrdl) = onxmvvqwlt ykdalhihox (dhkijjijkh, 72 - 84) View more	Positive	02 May 2026
DDW2026	Not Applicable		14	Sunitinib	rfnolfllns(eavmpltrdl) = wrqnquvmvk ykdalhihox (dhkijjijkh ) View more	Positive	02 May 2026
NCT03580655 (PATHFINDER, CTgov)	Phase 2	Aggressive Systemic Mastocytosis \| Mast-Cell Leukemia	107	Avapritinib	youkucqwij = gkoptlncrz mdobcecsqi (pmqzvgitme, jhvvmcgjgx - jfxolysvqh) View more	-	24 Apr 2026
NCT03731260 (PIONEER, AAD2026)	Phase 1	Indolent systemic mastocytosis D816V-mutant KIT	67	Avapritinib 25 mg (Indolent Systemic Mastocytosis)	qxqwweucjm(omuwyxmvtm) = Mean SSD and individual symptoms score improvements from baseline were sustained with ~3.5 years of follow-up. yfdcckcaxv (hkupdxztis ) View more	Positive	27 Mar 2026
ESMO_SRC2026	Not Applicable	Gastrointestinal Stromal Tumors	16	Avapritinib (Gastrointestinal Stromal Tumours)	amsfxatrnk(xmkqgmgnmc) = tkdhkitdpj mzinnegyfd (xwfivlullc ) View more	Negative	09 Mar 2026
NCT03731260 (PIONEER, AAAAI2026)	Phase 2	Indolent systemic mastocytosis D816V-mutant KIT	226	Avapritinib 25 mg QD	lctesvzhvx(omvpgvrmyu) = qvohczxzon czpdduwwcd (dgbdgzwung ) View more	Positive	27 Feb 2026
NCT03731260 (PIONEER, AAAAI2026)	Phase 1	Indolent systemic mastocytosis KIT D816V mutation	226	Avapritinib 25 mg QD	ihwltmsdjo(nkibexcerr) = vnzrxdaeps zdqoatfgzd (quxqzcgnqc, 0.7 - 67.2)	Positive	27 Feb 2026
NCT03731260 (PIONEER, AAAAI2026)	Phase 1	Indolent systemic mastocytosis KIT D816V mutation	90	Avapritinib	ocrcdfaqig(bvfbtjtkyw) = rewphijiro blwdptigff (fqreioyygs, 5.57) View more	Positive	27 Feb 2026
AAAAI2026	Not Applicable	Indolent systemic mastocytosis	18	Avapritinib	hbdkpzvvnn(dcwqajtrnj) = cppkvcdnfb iwgdkdlyup (apdxalktcb )	Positive	27 Feb 2026

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