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

Structure/Sequence

Molecular FormulaC24H21BrFN5O2

InChIKeyCEFJVGZHQAGLHS-UHFFFAOYSA-N

CAS Registry1442472-39-0

Clinical Trials associated with Ripretinib

NCT07618377

/ RecruitingPhase 1

A Phase 1 Study to Evaluate the Safety of DCC-2618 in Patients With Unresectable, Metastatic, or Recurrent Gastrointestinal Stromal Tumor (GIST) Who Are Refractory or Intolerant to at Least 3 Prior Therapies

To evaluate the safety of DCC-2618 in patients with unresectable, metastatic, or recurrent gastrointestinal stromal tumor (GIST) who have received three or more prior therapies

Start Date07 Jul 2026

Sponsor / Collaborator

Ono Pharmaceutical Co., Ltd.

NCT06619275

/ CompletedNot Applicable

Ripretinib in Patients With Previously Treated Advanced Gastrointestinal Stromal Tumor (GIST): a Prospective, Longitudinal, Multicenter, Observational Study in Germany

The goal of this prospective, observational study INTEREST is to collect real-world data on ripretinib treatment in a broad patient population in Germany. Ripretinib will be administered according to the current SmPC. Thus, INTEREST will evaluate for the first time ripretinib in GIST patients in a real-world setting in Germany.
The main questions the study aims to answer are:
* Evaluate quality of live (QoL) using the questionnaire EQ-5D-5L to gain knowledge about how ripretinib treatment affects GIST patients' well-being (change compared to Baseline, Time to Deterioration)
* Assessment of effectiveness in routine treatment (Progression-Free Survival, Overall Survival, Best Response, Overall Response Rate, Disease Control Rate
* Assessment of drug safety
* Assessment of parameters of physicians' treatment decision making
* Description of treatment reality in detail

Start Date17 Oct 2024

Sponsor / Collaborator

IOMEDICO AG

CTIS2022-501477-40-00

/ Not yet recruitingPhase 1

A study to learn the effects of ripretinib (study drug) on how midazolam behaves in the body of people with advanced gastrointestinal stromal tumor

Start Date25 Jun 2024

Sponsor / Collaborator

Deciphera Pharmaceuticals, Inc.

100 Clinical Results associated with Ripretinib

100 Translational Medicine associated with Ripretinib

100 Patents (Medical) associated with Ripretinib

188

Literatures (Medical) associated with Ripretinib

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.

15 Dec 2026·RAPID COMMUNICATIONS IN MASS SPECTROMETRY

LC–HRMS/MS Characterization of Hydrolytic and Photolytic Degradation Pathways of Ripretinib Following Forced Stress Studies

Article

Author: Golla, Vijaya Madhyanapu ; Chaganti, Sowmya ; Pilli, Pushpa ; Kushwah, Bhoopendra Singh ; Khemchandani, Rahul ; Samanthula, Gananadhamu

ABSTRACT:

Introduction:

Ripretinib is an innovative multitargeted kinase inhibitor introduced for the treatment of advanced gastrointestinal stromal tumors, was investigated to elucidate its degradation behavior and structural transformation pathways under forced stress conditions. Although degradation studies are essential for evaluating the intrinsic stability of pharmaceutical compounds, the degradation products of ripretinib have not been comprehensively characterized. The present work focuses on LC–HRMS/MS‐based structural elucidation of degradation products arising from photolytic and hydrolytic stress conditions.

Methods:

In accordance with International Council for Harmonisation guidelines, ripretinib was subjected to acidic, alkaline, oxidative, thermal, and photolytic stress conditions. Liquid chromatography was employed to separate the degradation products, which were subsequently characterized by high‐resolution tandem mass spectrometry (LC–HRMS/MS) operated in positive electrospray ionization mode. Structural assignments were established using accurate mass measurements, isotopic pattern analysis, elemental composition determination, and collision‐induced dissociation pathways. Degradation kinetics under photolytic conditions were also evaluated. An in silico toxicity assessment of degradation products was performed using DEREK and SARAH prediction platforms.

Results:

Ripretinib exhibited degradation predominantly under photolytic and hydrolytic stress conditions, leading to the formation of five degradation products. The protonated molecule of ripretinib at m/z 510 displayed a characteristic bromine isotopic distribution that facilitated differentiation between brominated and debrominated products. Hydrolytic degradation generated two products through cleavage of the urea linkage, whereas photolytic degradation produced three products involving intramolecular cyclization with concomitant debromination, hydroxylation, and N–C bond cleavage pathways. Collision‐induced dissociation of protonated molecules generated diagnostic product ions that enabled structural elucidation of all degradation products. Photodegradation followed apparent first‐order kinetics with a half‐life of 4.2 min. One hydrolytic degradation product containing a primary aromatic amine moiety was predicted to possess mutagenic potential.

Conclusion:

This investigation elucidates the degradation pathways of ripretinib and highlights the utility of LC–HRMS/MS in the structural characterization of degradation products generated under stress conditions.

25 Aug 2026·Zhonghua wei chang wai ke za zhi = Chinese journal of gastrointestinal surgery

[Efficacy of ripretinib in advanced gastrointestinal stromal tumor and treatment options after resistance: a retrospective real-world study].

Article

Author: Sun, L Y ; Han, Y H ; Yin, Y ; Yang, H X ; Yin, X N ; Mao, J ; Zhang, B

Objective: To investigate the clinical efficacy, safety, and post-resistance treatment strategies of ripretinib in the treatment of advanced gastrointestinal stromal tumors (GIST), and to provide evidence-based references for optimizing individualized treatment of advanced GIST. Methods: This is a single-center retrospective real-world study. Inclusion criteria: (1) GIST confirmed by histopathology; (2) disease progression after previous treatment with other targeted drugs including imatinib; (3) continuous administration of ripretinib for more than one month with at least one efficacy evaluation. Exclusion criteria: (1) Clinical data were seriously missing, and data such as patient dosage, efficacy evaluation results, and adverse reactions could not be obtained; (2) complicated with other serious underlying diseases, such as uncontrolled severe cardiovascular and cerebrovascular diseases, liver and kidney failure, etc.; (3) Allergic or severe drug intolerance to ripretinib. According to the above criteria, a total of 93 patients with advanced GIST treated in West China Hospital of Sichuan University from July 2021 to March 2025 were collected. In this study, evaluable patients were defined as those who met any of the following criteria: (1) baseline and follow-up imaging examinations were completed in our center with complete imaging data, and the long diameter of target lesions could be accurately measured and the efficacy could be evaluated; (2) The efficacy evaluation was completed in other hospitals, but the image data and report were complete, and the exact measurement data of target lesions could be obtained after review by the radiology department of our hospital. All patients with advanced GIST enrolled in the study initially received the standard dose of ripretinib (150 mg once daily). After disease progression (PD), the following treatment strategies could be selected after multi-disciplinary team (MDT) discussion: (1) the dose of ripretinib was increased from 150 mg once daily to 150 mg twice daily (ripretinib dose-escalation group); (2) The standard-dose ripretinib (150 mg, once daily) was combined with local treatment, including surgery, interventional therapy and radiotherapy (ripretinib combined with local treatment group); (3) standard-dose ripretinib combined with frontline TKI, including imatinib, sunitinib and regorafenib (ripretinib combined with frontline TKI group); (4) other TKI treatment, including imatinib, sunitinib, regorafenib and avapritinib (other TKI treatment group). The primary outcome was median progression-free survival (mPFS). Secondary outcomes included objective response rate [ORR, the proportion of patients with complete response (CR) or partial response (PR)], disease control rate [DCR, the proportion of patients with CR, PR, or stable disease (SD)], overall survival (OS), and adverse events. Results: A total of 93 patients with advanced GIST were enrolled. Among 59 evaluable patients, 6 had PR, 30 had SD, and 23 had PD, with ORR of 10.2% and DCR of 61.0%. With a median follow-up time of 13 (2-47) months, the median mPFS was 7.0 (95%CI: 4.9-9.1) months, and the 1-year OS rate was 86.1%. Genotyping analysis showed that the mPFS in the KIT exon 11 mutation group was significantly longer than that in the exon 9 mutation group (12.0 months vs. 4.0 months, P=0.007). No statistically significant difference in mPFS was observed among different recurrence/metastasis sites (liver, peritoneum, or liver + peritoneum, P = 0.149). A total of 60 patients experienced progression on standard-dose ripretinib. According to the subsequent treatment strategy, the patients were divided into 4 groups: ripretinib dose-escalation group (16 patients), ripretinib combined with local therapy group (9 patients), ripretinib combined with frontline TKI group (6 patients), and other TKI group (9 patients). The mPFS in these groups was 4.0 months (95% CI: 1.5-6.5), not reached, 4.0 (95%CI: could not be estimated) and 12.0 months (95%CI: 1.0-25.5), respectively. There was no significant difference in mPFS among the four groups (P=0.345). In terms of safety, no grade 3-4 serious adverse reactions were observed in the whole group, and dose-escalation or combination therapy did not increase toxicity. Conclusion: Ripretinib can serve as an effective treatment option for Chinese patients with advanced GIST after failure of first-line or multiple prior TKI therapies, and it demonstrates a favorable safety profile. Although no standard treatment is currently available for patients with resistance to ripretinib, dose escalation of ripretinib, combination with local therapy, combination with prior TKIs, or switching to alternative TKIs can be considered, and all regimens exhibit a favorable safety profile.

114

News (Medical) associated with Ripretinib

17 Jun 2026

·www.businesswire.com

Deciphera Pharmaceuticals Announces First Patient Dosed in Pivotal Phase 3 INTREPID Study of Sapablursen in Polycythemia Vera

WALTHAM, Mass.--(BUSINESS WIRE)--Deciphera Pharmaceuticals, LLC, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), announced the first patient was dosed in the global pivotal Phase 3 INTREPID study evaluating sapablursen for the treatment of polycythemia vera (PV). Sapablursen is an investigational drug that has the potential to offer a once-monthly treatment option for patients with PV, a rare and potentially life-threatening hematologic disease. “We look forward to building upon the positive efficacy and safety results from the Phase 2a IMPRSSION study, which demonstrated the ability of sapablursen to reduce the frequency of phlebotomy, control hematocrit, and improve PV symptoms in patients treated with phlebotomy alone and those on cytoreductive therapies,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “Sapablursen has the potential to be an important new treatment option for patients with PV, and we are excited to begin our Phase 3 INTREPID study, which brings us one step closer to addressing the unmet needs of these patients.” INTREPID is a pivotal global Phase 3 study in patients with phlebotomy-dependent PV designed to compare the efficacy and safety of sapablursen to placebo over a 32-week double-blind treatment period followed by up to 124 weeks of open label treatment. The primary endpoint is response, defined by the absence of phlebotomy eligibility. Key secondary endpoints are number of phlebotomies, which is the primary endpoint for the European Medicines Agency for potential regulatory approval, hematocrit control, and improvement in the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score and the Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score. Durability of response will be evaluated in patients randomized to sapablursen over 52 weeks of study treatment, including 32 weeks of blinded treatment and 20 weeks of open-label treatment. The INTREPID clinical trial has initiated in the United States and is planned in additional regions including North America, Latin America, Asia Pacific and Europe. For more information on the INTREPID study, please visit https://clinicaltrials.gov/study/NCT07429266. Sapablursen received Fast Track designation and Orphan Drug Designation in 2024 and Breakthrough Therapy Designation in 2025 by the U.S. Food and Drug Administration (FDA). Sapablursen was discovered and advanced through Phase 2 clinical development by Ionis Pharmaceuticals. Ono obtained exclusive global rights for the development and commercialization of sapablursen after entering into a license agreement with Ionis in March 2025. About Sapablursen Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, the key regulator of iron homeostasis. By increasing the production of hepcidin, sapablursen has the potential to positively impact PV by decreasing hematocrit, reducing the need for phlebotomy, and improving quality of life. About Polycythemia Vera Polycythemia vera (PV) is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, heart attack, stroke, and death. The primary treatment goal in PV is to maintain blood hematocrit levels <45% to prevent thrombotic events and alleviate burdensome symptoms, such as severe fatigue, difficulty concentrating, night sweats, and pruritus. Current treatment options often worsen symptoms and inadequately maintain hematocrit control. About Deciphera Pharmaceuticals, LLC Deciphera, a member of Ono Pharmaceutical Co., Ltd., is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines and providing hope to people living with cancer, neurologic, and autoimmune disease. Deciphera is leveraging its proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from Deciphera’s platform in clinical studies, QINLOCK® (ripretinib) is Deciphera’s switch-control kinase inhibitor approved in many countries including the European Union and the United States for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. ROMVIMZATM (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability. For more information, visit www.deciphera.com and follow us on LinkedIn and X (@Deciphera). About Ono Pharmaceutical Co., Ltd. Ono Pharmaceutical Co., Ltd. delivers innovative medicines for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology & inflammation, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at https://www.ono-pharma.com/en. Cautionary Note Regarding Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice.

Phase 3Orphan DrugClinical ResultPhase 2Fast Track

01 Jun 2026

·www.fiercebiotech.com

At ASCO 2026, a ‘paradigm shift’ for a rare gut cancer looms

For GSK, ASCO was the perfect setting to share its plans to revolutionize treatment for a rare gut cancer that hasn\'t seen new innovation in decades.\n For patients with a rare cancer that arises in the gut wall, standard treatment hasn’t changed in the last 20 years. At the American Society of Clinical Oncology meeting in Chicago, GSK shared a first look at its attempt to change that.GSK bought the precision medicine biotech IDRx in January 2025 to secure a small molecule that targets a type of enzyme called a tyrosine kinase inhibitor (TKI). By specifically homing in on multiple mutations of the tyrosine kinase c-KIT, GSK hopes that the molecule, called velzatinib, can improve on the earlier TKIs Gleevec (imatinib) and Sutent (sunitinib).Novartis first garnered approval for Gleevec in metastatic gastrointestinal stromal tumors (GISTs) in 2002, and Pfizer followed with Sutent in 2006. Mutations in KIT cause GIST to develop in rare, specialized cells called interstitial cells of Cajal, which serve as the gut’s pacemaker.Non-metastatic GISTs can be removed surgically, but when the disease metastasizes, medicine must enter the conversation.“Imatinib is a very potent inhibitor of KIT,” Suzanne George, M.D., a medical oncologist at Dana-Farber Cancer Institute, told Fierce at ASCO. “But we\'ve learned over time that the primary mechanism of resistance to imatinib is the development of secondary mutations in KIT that imatinib does not work against.”For these secondary mutations, Sutent is deployed, with Bayer’s Stivarga (regorafenib) and Deciphera’s Qinlock (ripretinib) following in the third and fourth lines. Velzatinib is designed to not only attack the main mutations in KIT that drive the formation of GISTs, but also the subsequent mutations that the tumors develop in order to combat Gleevec and Sutent, Hesham Abdullah, M.D., GSK’s head of oncology R&D, explained to Fierce at the pharma’s conference booth.In its ASCO debut, velzatinib was confirmed to shrink tumors in 61% of 24 first-line patients, GSK presented, and 38% of tumors among 49 second-line patients.While acknowledging that the first-line data come from a “limited cohort of patients” from IDRx’s phase 1 trial, Abdullah said the results provide “the first look of what this drug can do.” “The preliminary data from the phase 1 is very promising in terms of response rate and initial duration,” George agreed. In the second-line setting, velzatinib’s response rates trump those of Sutent, Abdullah added. “You would typically expect response rates with Sutent to be in the teens,” Abdullah said, “and we\'re looking at a median progression-free survival of about 17 months in second-line-plus patients, which is quite impressive.”In addition to cancer being able to evolve resistance to it, Sutent is also hampered by side effects caused by its broad activity—in addition to KIT, the drug also hits other targets, most notably the signaling protein VEGF. By homing in just on KIT, velzatinib should be gentler on patients, George said.GSK also used the poster session at ASCO to share details of a phase 3 trial for velzatinib in second-line patients who have already developed resistance to Gleevec. That trial, StrateGIST 3, is testing GSK’s upstart inhibitor against Sutent, and the British pharma is also planning a first-line phase 3 trial pitting velzatinib against Gleevec.The aim is to “redefine what the potential standard of care is,” Abdullah said, much as Gleevec did for GIST patients two decades ago.George, who contributed heavily to sunitinib’s development, is now on the steering committee for GKS’s phase 3 trials of velzatinib and presented the poster outlining the pharma’s StrateGIST 3 plans.GSK is not the only company looking to modernize GIST care. Cogent Biosciences presented full data from the phase 3 trial of its KIT inhibitor bezuclastinib at ASCO, too, demonstrating an average progression-free survival of 16.5 months in Gleevec-resistant patients when paired with Sutent compared to 9.2 months for Sutent alone.“Importantly, there was a clear benefit across all mutational patient subgroups,” Cogent president and CEO Andrew Robbins said in a May 30 release. Bezuclastinib is already sitting with the FDA for an approval decision, with a verdict expected by November 30.“We plan to launch bezuclastinib later this year and are well prepared to ensure bezuclastinib combination access for GIST patients in need,” Robbins said.Bezuclastinib targets a specific KIT mutation and thus needs to be used in combination with other KIT inhibitors to hit the full panoply. So, while it may become a new standard of care in second-line treatment, should it be approved, it has a less clear path to replacing Gleevec in newly diagnosed patients.“The rationale behind the combination of sunitinib and bezuclastinib is that you\'re hitting all the mutations,” Dana-Farber’s George explained. “The hope with drugs like velzatinib is that with a single drug, you will hit that broad range of mutations.”Deciphera Pharmaceuticals, a subsidiary of Ono Pharma, also previously tried to take its late-line GIST medicine Qinlock into second-line treatment, but the molecule failed to beat Sutent. A later analysis revealed some promising patient subgroups who may see stronger efficacy from Qinlock than Sutent, however, raising a potential opportunity that Deciphera is now pursuing.As the next generation of KIT inhibitors rises, George is excited by the prospect of combining them with other up-and-coming GIST targets that have yet to be revealed.“There is absolutely a lot of interest in thinking about combinations of KIT,” she told Fierce. “GIST is ripe for a really strong paradigm shift.”

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 Ripretinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11353	Ripretinib	L01XE L01EX19 L01E	DB14840

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Gastrointestinal Stromal Tumors	United States	Deciphera Pharmaceuticals, Inc.	15 May 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Unresectable Gastrointestinal Stromal Tumor	Phase 1	Japan	Ono Pharmaceutical Co., Ltd.	07 Jul 2026
Melanoma	Phase 1	United States	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Melanoma	Phase 1	Canada	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Melanoma	Phase 1	Germany	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Melanoma	Phase 1	Italy	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Melanoma	Phase 1	Netherlands	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Melanoma	Phase 1	United Kingdom	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Systemic Mastocytosis	Phase 1	United States	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Systemic Mastocytosis	Phase 1	Canada	Deciphera Pharmaceuticals, Inc.	01 Nov 2015
Systemic Mastocytosis	Phase 1	Germany	Deciphera Pharmaceuticals, Inc.	01 Nov 2015

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


CSCO2026	Not Applicable	Gastrointestinal Stromal Tumors	127	Ripretinib (Surgical group)	ozclvignfp(oeomwwwugl) = qadhscbnid vllbktzhkk (emrtzhtrok ) View more	Positive	11 Sep 2026
				Ripretinib (Non-surgical group)	ozclvignfp(oeomwwwugl) = czktpsnrfy vllbktzhkk (emrtzhtrok ) View more
NCT05132738 (ASCO2026)	Phase 2	Gastrointestinal Stromal Tumors	20	Ripretinib 150 mg QD	huxoqyrmmh(wppzxmyqkf) = vfhlljqiwa cahgtcmjbn (gkwiizddyp ) View more	Positive	29 May 2026
ASCO2026	Not Applicable	Gastrointestinal Stromal Tumors	30	Ripretinib dose escalation group (150 mg twice daily)	sdglpxbhvz(onbtkjwhlg) = no grade ≥3 adverse events reported fihroxkmzy (spbvocheyu )	Positive	29 May 2026
				Combination therapy group (ripretinib 150 mg once daily plus sunitinib 25 mg once daily)
ESMO_SRC2026	Not Applicable	SDH-deficient Gastrointestinal Stromal Tumors	19	imatinib	xbubvnsnka(wgmqhhdppr) = jaihyiwxaz fqczzomkqf (kiofgkkqho, 50.7 - NR)	Positive	09 Mar 2026
				sunitinib	aqxtujdmep(byqfmbsish) = naagqbctul taeuxaoamj (wdnyuzirui )
NCT04282980 (CTgov)	Phase 2	Gastrointestinal Stromal Tumors	39	DCC-2618	vdrbzdbafn(xqrqpjnnsx) = cowdrweqge rwkwguloon (wudypmtdvf, camcxqbwii - ykcgyywwjz) View more	-	03 Mar 2025
NCT04633122 (INTRIGUE study, CTgov)	Phase 2	Gastrointestinal Stromal Tumors	108	Ripretinib (Ripretinib)	ovnauczijs(sngetpjcxx) = lkwnbfhuyr xqkskfqgao (tqejcnmyto, tgjotbjmpt - ixrgctyeuc) View more	-	15 Jan 2025
				sunitinib (Sunitinib)	ovnauczijs(sngetpjcxx) = fqrkaoeobo xqkskfqgao (tqejcnmyto, rzbrppuxsv - muidqtimsi) View more
NCT05132738 (ESMO_ASIA2024) Manual	Not Applicable	Metastatic Gastrointestinal Stromal Tumor	12	Ripretinib 150 mg	aqnqxefote(qalivyepcd) = thhgqhdjnt qpmtmnsrdw (gemvebxoum ) Met View more	Positive	07 Dec 2024
NCT04633122 (ESMO2024) Manual	Phase 2	Gastrointestinal Stromal Tumors Second line \| Third line KIT exon 11 mutation	108	Ripretinib 150 mgsunitinib	hkwouputyi(nvdhnrtdkq) = khyufywknv ycntzoqhpk (dwetzfekza ) View more	Positive	14 Sep 2024
				Sunitinib 50 mg	hkwouputyi(nvdhnrtdkq) = sjenhaicmn ycntzoqhpk (dwetzfekza ) View more
NCT03673501 (INTRIGUE, ESMO_SRC2024) Manual	Phase 3	KIT exon 11 and 18 mutation Gastrointestinal Stromal Tumor \| KIT exon 11 and 17 mutation Gastrointestinal Stromal Tumor KIT Exon 17 Mutation \| KIT Exon 18 Mutation \| KIT Exon 11 Mutation	52	Ripretinib	elfdlloobg(oyeztklhxd) = pomtmwgfyd mdzdjxglvc (zlpmccwdue ) View more	Positive	15 Mar 2024
NCT03673501 (INTRIGUE, Biospace) Manual	Phase 3	Gastrointestinal Stromal Tumors Second line	453	QINLOCKQINLOCK 150 mg	gltudfwauy(dqqyvipivk) = qguxtxksln jtveeziyes (qeywrzquhf ) View more	Positive	18 Jan 2024
				SunitinibSunitinib 50 mg	gltudfwauy(dqqyvipivk) = ieaqewbwfb jtveeziyes (qeywrzquhf ) View more

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