CSF-1R antagonists(Colony stimulating factor 1 receptor antagonists), FLT3 inhibitors(Tyrosine-protein kinase receptor FLT3 inhibitors), RIPK1 inhibitors(Receptor-interacting serine/threonine-protein kinase 1 inhibitors)

Therapeutic Areas

NeoplasmsNervous System DiseasesSkin and Musculoskeletal Diseases+ [4]

Active Indication

Giant Cell Tumor of Tendon SheathTesticular Germ Cell TumorAdenocarcinoma of Lung

Inactive Indication

Adenocarcinoma of large intestineAdvanced cancerAdvanced Malignant Solid Neoplasm+ [23]

Originator Organization

Daiichi Sankyo Co., Ltd.

Active Organization

Catalent CTS LLC

Istanbul University

Daiichi Sankyo Co., Ltd.

+ [2]

Inactive Organization

AstraZeneca PLC

Plexxikon, Inc.

Daiichi Sankyo Europe GmbH

License Organization-

Drug Highest PhaseApproved

First Approval Date

United States (02 Aug 2019),

Giant Cell Tumor of Tendon Sheath

RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (South Korea), Priority Review (China)

Structure/Sequence

Molecular FormulaC20H16Cl2F3N5

InChIKeyCJLUYLRKLUYCEK-UHFFFAOYSA-N

CAS Registry2040295-03-0

Clinical Trials associated with Pexidartinib Hydrochloride

NCT04703322

/ Active, not recruitingPhase 2

A Phase 2, Multicenter, Two-Part, Open-Label Study of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor in Japan

This phase 2, multicenter, two-part, open-label, single-arm study will be conducted in Japan and will evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of pexidartinib in adult participants with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitation and not amenable to improvement with surgery.

Start Date15 Mar 2021

Sponsor / Collaborator

Daiichi Sankyo Co., Ltd.

NCT04635111

/ RecruitingNot Applicable

A Long-term Study to Further Evaluate the Risk of Hepatotoxicity Associated With TURALIO™ (Pexidartinib) Treatment

A study to evaluate the long-term risk of hepatic failure with TURALIO™ (pexidartinib) and the mechanism of liver injury based upon liver biopsy information among patients who received or are receiving TURALIO™ (pexidartinib) and experience hepatotoxicity.

Start Date07 Jan 2021

Sponsor / Collaborator

Daiichi Sankyo Co., Ltd.

NCT04526704

/ CompletedPhase 4

A Phase 4, Multicenter Study to Evaluate Discontinuation and Re-Treatment in Subjects With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

This study is designed to evaluate the discontinuation/re-treatment of pexidartinib therapy in previously treated participants with tenosynovial giant cell tumor (TGCT).

Start Date20 Oct 2020

Sponsor / Collaborator

Daiichi Sankyo Co., Ltd.

100 Clinical Results associated with Pexidartinib Hydrochloride

100 Translational Medicine associated with Pexidartinib Hydrochloride

100 Patents (Medical) associated with Pexidartinib Hydrochloride

520

Literatures (Medical) associated with Pexidartinib Hydrochloride

01 Sep 2025·LIFE SCIENCES

Emerging strategies for targeting tumor-associated macrophages in glioblastoma: A focus on chemotaxis blockade

Review

Author: Rhee, Inmoo ; Nam, Dayoung ; Jeong, Moongyu ; Lee, Jaehyun ; Lee, Chaelin

Glioblastoma (GBM) remains one of the most aggressive and treatment-resistant brain tumors, with poor prognosis for affected patients. A key player in the GBM tumor microenvironment is the tumor-associated macrophage (TAM), which promotes tumor progression, immune evasion, and therapeutic resistance. The recruitment of TAMs to the tumor site is driven by specific chemotactic signals, including CSF-1/CSF-1R, CXCR4/CXCL12, and HGF/MET pathways. This review explores the current understanding of these chemotaxis mechanisms and their role in GBM progression. It highlights the potential therapeutic benefits of targeting TAM chemotaxis pathways to disrupt TAM infiltration, reduce immunosuppression, and enhance the efficacy of conventional treatments. Additionally, we discuss the preclinical and clinical evidence surrounding key inhibitors, such as PLX3397, AMD3100, and Crizotinib, which have shown promise in reprogramming TAMs and improving treatment outcomes in GBM. While these strategies offer hope for overcoming some of the challenges of GBM therapy, the review also addresses the limitations and obstacles in clinical translation, emphasizing the need for further research and the development of combination therapies to achieve sustained therapeutic benefit.

01 Aug 2025·BRAIN BEHAVIOR AND IMMUNITY

Microglia specific Csf1r haploinsufficiency induces depressive-like behaviors by promoting NLRP6/caspase-1 signaling in mice

Article

Author: Li, Xiao-Xiao ; Pang, Rui-Kang ; Su, Shan ; Zhang, Shi-Jie ; Zheng, Jia-Yi ; Cai, Ye-Feng ; Le, Kai ; Zhao, Yuan-Qi ; Xu, Hao-You

Depression is an early clinical manifestation of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), although the underlying molecular mechanisms remain poorly elucidated. The objective of this study was to investigate the mechanisms underpinning depressive behavior in the context of ALSP, utilizing microglial-specific Csf1r haploinsufficient mice. Our findings indicate that these mice exhibited depressive-like behaviors, as well as microglial hyper-ramification and aberrant synaptic pruning capacity. Blockade of CSF1R signaling with PLX3397 resulted in significant amelioration of depressive symptoms and restoration of normal microglial morphology and function. RNA sequencing analysis of microglia isolated from the medial prefrontal cortex (mPFC) of the brain indicated that NLRPs signaling pathways may play a significant role in the observed alterations in microglial Csf1r haploinsufficient mice. Notably, NLRP6, rather than NLRP3, was found to be upregulated, and the expression of caspase-1 exhibited colocalization with the microglial marker Iba1. Pharmacological inhibition of caspase-1 using VX-765 improved depressive-like behaviors, as well as microglial function. Taken together, our findings delineate a causal relationship between microglial Csf1r haploinsufficiency-induced activation of the NLRP6/caspase-1 signaling pathway and the manifestation of depressive-like behaviors in ALSP mice.

01 Aug 2025·INTERNATIONAL DENTAL JOURNAL

Pexidartinib Inhibits Macrophage Senescence Through Glycolysis in Periodontitis Microenvironment

Article

Author: Fang, Hui ; Zhang, Yongchun ; Kang, Jiabing ; Wei, Yan ; Yuan, Yiting ; Xiao, Tianjiao ; Li, Li ; Zhang, Jie ; Tian, Ai ; Zhan, Jifan

OBJECTIVES:

Periodontitis, a chronic inflammatory condition, is caused by complex interactions between periodontopathic bacteria and the local innate immune response. Macrophage senescence, a pivotal contributor to immune dysfunction, has been implicated in periodontitis progression. This research was conducted to clarify how macrophage senescence and glycolysis interact within the periodontal inflammatory microenvironment. Specifically, we investigated whether pexidartinib (PLX3397), known for inhibiting the colony-stimulating factor-1 receptor (CSF-1R), could mitigate macrophage senescence by modulating glycolytic activity, thereby attenuating periodontal inflammation.

METHODS:

We first constructed the experiential periodontitis mouse model. The alveolar bone volume was quantified using Micro-CT, while the periodontal ligament width and the distance from the cementoenamel junction (CEJ) to the alveolar bone crest (ABC) were evaluated using HE staining. The expression levels of macrophage senescence markers, glycolysis-related indicators, and CSF-1R in gingival tissues were assessed by immunofluorescence staining. For in vitro studies, Senescence was induced in RAW264.7 cells by stimulating them with Porphyromonas gingivalis lipopolysaccharide (Pg-LPS) with or without pretreatment of PLX3397 and glycolysis modulators. Cellular senescence rates were evaluated using Senescence-associated β-Galactosidase (SA-β-Gal) staining. The presence of markers indicating senescence, CSF-1R, and glycolysis-related indicators was further analysed by RT-qPCR and Western blotting.

RESULTS:

The gingival tissues of mice with periodontitis showed elevated senescent macrophages, which correlated with higher CSF-1R expression and glycolytic activity. Similarly, in Pg-LPS-treated RAW264.7 macrophages, senescence markers were upregulated alongside CSF-1R and glycolysis-related indicators. Meanwhile, modulating glycolysis in vitro directly influences senescence indicators. And PLX3397 treatment reduced glycolytic activity, leading to an improvement in macrophage senescence.

CONCLUSION:

Our findings indicate that PLX3397 alleviates periodontal tissue inflammation by inhibiting macrophage senescence via glycolytic modulation, offering potential for immune-regulatory therapies in periodontitis management.

News (Medical) associated with Pexidartinib Hydrochloride

28 May 2025

·www.fiercebiotech.com

Merck KGaA shares phase 3 rare tumor data ahead of showdown with Daiichi and Ono

Merck plans to start regulatory filings for the CSF-1R inhibitor pimicotinib this year.\n Merck KGaA has shown off data that fueled its $85 million bet on a late-phase treatment for a rare, locally aggressive tumor. The update provides a closer look at the hand Merck is holding as it gears up to fight Daiichi Sankyo and Ono Pharmaceutical for the tenosynovial giant cell tumor (TGCT) market.The German drugmaker shared top-line phase 3 data on the CSF-1R inhibitor pimicotinib in TGCT late last year. After seeing the data, Merck paid Abbisko Therapeutics $85 million to take up its option on a global license to the drug candidate. Merck paid Abbisko $70 million for rights to pimicotinib in greater China in 2023. May 28, Merck shared more phase 3 data ahead of its presentation at the 2025 American Society of Clinical Oncology (ASCO) annual meeting that starts later this week. The drugmaker included data on the primary response rate endpoint and secondary goals that looked at stiffness and pain in its top-line readout last year.Merck’s latest update sheds light on the speed and durability of the response. The drug worked quickly, with 26 patients responding to therapy after 13 weeks. The primary endpoint, which was assessed after 25 weeks, included 33 partial responses and one complete response. The median duration of response wasn’t reached by the data cutoff. Merck said 61.9% of patients on pimicotinib had a reduction in tumor volume on an endpoint designed for TGCT, compared to 3.2% of people on placebo. People on the study drug also experienced significant changes in their range of motion and physical function. The data complete a clean sweep of secondary endpoints that included the earlier readout of assessments of stiffness and pain. These types of noncancerous tumors don\'t spread to other parts of the body but can be locally aggressive and cause the affected synovium, bursae or tendon sheaths to thicken and overgrow. This, in turn, can cause damage to the surrounding tissue and structures of the affected limb. Symptoms can include pain and swelling while also limiting movement at the joint. Daiichi’s phase 3 TGCT trial of Turalio, which won FDA approval in 2019, linked the CSF-1R inhibitor to significant improvements in motion, stiffness and physical function. The Japanese drugmaker also had a higher proportion of complete responders than Merck. Yet, Daiichi’s trial had a lower overall response rate and missed its secondary pain endpoint. Merck also has a potential safety and tolerability advantage. Turalio carries a boxed warning for liver toxicity. Merck saw no evidence of cholestatic hepatotoxicity or drug-induced liver injury in its phase 3 trial. Ono’s Romvimza, which won FDA approval in February, is free from the boxed safety warning and risk-mitigation measures that apply to Turalio.Danny Bar-Zohar, who is set to become CEO of Merck’s healthcare unit next week, said in a statement that the company will start regulatory filings this year. The FDA’s reaction to the geographic mix is one outstanding question. Chinese sites enrolled almost half the patients. U.S. and Canadian sites collectively enrolled 22% of the patients. The FDA’s cancer unit recently made increased U.S enrollment a focus.

Phase 3Clinical ResultDrug ApprovalASCOLicense out/in

01 Apr 2025

·www.fiercebiotech.com

Merck KGaA pays $85M to nab global rights for Abbisko\'s rare tumor drug after phase 3 win

“Pimicotinib represents a key advancement within the emerging class of CSF-1R inhibitors,” Abbisko Therapeutics CEO Yao-Chang Xu said.\n With the CSF-1R inhibitor space heating up, Merck KGaA has gone all-in on pimicotinib, paying Abbisko Therapeutics $85 million to secure the global license to the drug.The German pharma originally paid $70 million for the greater China commercialization rights back in 2023, while retaining an option to expand its interest to cover the rest of the world. Japanese pharma Daiichi Sankyo has already shown that inhibiting the colony-stimulating factor 1 receptor can work in tenosynovial giant cell tumors (TGCTs) after winning FDA approval for Turalio in 2019, but Merck identified a gap for another drug in the class.That $70 million investment looked like a good bet last November when a phase 3 trial in patients with TGCTs showed that 54% of people on pimicotinib had responded at 25 weeks, compared to just 3.2% of the placebo cohort.Now, Merck said it has made the decision to pay the $85 million option fee to secure the rights to commercialize pimicotinib in the rest of the world. The pharma also has the option to codevelop the drug for “additional indications under certain conditions,” according to the April 1 press release.The expanded deal puts Shanghai-based Abbisko in line for up to $605.5 million in total payments—including the upfront fees as well as development and commercial milestones—alongside a share of royalties should pimicotinib make it to market. “Pimicotinib represents a key advancement within the emerging class of CSF-1R inhibitors, demonstrating a meaningful clinical efficacy and safety profile that positions it as an innovative treatment option for worldwide TGCT patients,” Abbisko CEO Yao-Chang Xu said in the release.“We look forward to deepening our collaboration with the Merck team to expedite the registration process of pimicotinib to bring benefits to patients as quickly as possible,” the CEO added.Daiichi’s CSF-1R inhibitor Turalio generated 5.1 billion Japanese yen ($33 million) of sales during the last nine months of 2024, up 25% year over year. But pimicotinib could also find itself in competition with Ono Pharmaceutical’s CSF-1R inhibitor vimseltinib, which bagged FDA approval for TGCTs last month.

$Merck KGaA pays $85M to nab global rights for Abbisko\'s rare tumor drug after phase 3 win$

Drug ApprovalPhase 3License out/inClinical ResultPhase 1

04 Mar 2025

·www.prnewswire.com

Ono's ROMVIMZA Approval Shakes Up Tenosynovial Giant Cell Tumors Market, Challenging Daiichi Sankyo's TURALIO Dominance | DelveInsight

On February 14, 2025, Ono Pharmaceuticals secured FDA approval for vimseltinib, a therapy for tenosynovial giant cell tumor (TGCT), which will be marketed as ROMVIMZA. This approval places Ono in direct competition with another Japanese pharmaceutical company, Daiichi Sankyo, in the TGCT market. LAS VEGAS, March 4, 2025 /PRNewswire/ -- Tenosynovial giant cell tumor, also known as pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), is primarily treated through surgical resection. For localized TGCT, the typical approach involves direct removal of the tumor nodule. In contrast, diffused-type TGCT (DTGCT) tends to present as larger tumors affecting major joints, making treatment more complex. Management options for DTGCT often include total synovectomy, joint replacement, or, in extremely rare cases, amputation. Among the 7MM, the United States had the highest prevalence of tenosynovial giant cell tumor, accounting for approximately 45% of total cases. In 2023, localized TGCT in the U.S. was most commonly found in the digits, representing around 75% of cases. Depending on whether the tumor is localized or diffuse, giant cell tumors can also occur in the knee, ankle, hip, and other areas. According to DelveInsight analysts, diffuse TGCT was most frequently observed in the knee, with an estimated 30,000 cases in the US in 2023. The primary approach to treating TGCT is surgery, often supplemented by alternative options like Radiation Therapy, Radiosynovectomy, and Systemic Therapies, including CSF1 inhibitors and Tyrosine Kinase Inhibitors. TURALIO (pexidartinib, previously known as PLX3397) is a targeted therapy developed by Daiichi Sankyo for adult patients with symptomatic tenosynovial giant cell tumor that leads to significant morbidity or functional limitations and cannot be effectively treated with surgery. This orally administered small-molecule tyrosine kinase inhibitor acts on colony-stimulating factor 1 receptor (CSF1R), KIT proto-oncogene receptor tyrosine kinase (KIT), and FMS-like tyrosine kinase 3 (FLT3) with an internal tandem duplication (ITD) mutation. Overexpression of the CSF1R ligand contributes to excessive cell proliferation and accumulation in the synovium. The US FDA approved TURALIO in August 2019 for the treatment of TGCT in adults with severe symptoms and limited surgical options. Before this, in October 2015, pexidartinib received Breakthrough Therapy Designation (BTD) from the FDA for TGCT treatment. TURALIO carries a risk of serious and potentially life-threatening liver toxicity and is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program. Learn more about the FDA-approved TGCT drugs @ Drugs for Tenosynovial Giant Cell Tumors Treatment Recently, in February 2025, Ono Pharmaceuticals secured FDA approval for vimseltinib for the treatment of tenosynovial giant cell tumor. Marketed under the brand name ROMVIMZA, the drug places Ono in direct competition with fellow Japanese pharmaceutical company Daiichi Sankyo in the TGCT space. Like TURALIO, which became the first FDA-approved TGCT treatment in 2019, ROMVIMZA is a colony-stimulating factor 1 receptor (CSF1R) inhibitor. As a kinase inhibitor, it blocks the CSF1 receptor to slow the growth of cells expressing this protein, potentially limiting tumor progression in TGCT. Ono Pharmaceuticals acquired ROMVIMZA through its $2.4 billion purchase of Deciphera Pharmaceuticals last year, making the U.S.-based firm a subsidiary. At the time, Ono's CEO, Gyo Sagara, described the acquisition as a strategic effort to expand the company's targeted oncology pipeline, strengthen its presence in the U.S. and European markets, and enhance its kinase drug discovery expertise. To compete with TURALIO's early market entry, Ono is positioning ROMVIMZA as a more convenient TGCT treatment. Unlike Daiichi Sankyo's drug, which requires twice-daily dosing, ROMVIMZA is administered only twice a week, with at least 72 hours between doses. Additionally, while TURALIO carries a boxed warning for hepatotoxicity due to the risk of severe liver damage, ROMVIMZA does not have this safety warning. To know more about TGCT treatment options, visit @ New Treatment for Tenosynovial Giant Cell Tumors TGCT that is not amenable to improvement from surgery. Several companies, such as SynOx Therapeutics (Emactuzumab), AmMax Bio (AMB-05X), Merck KGaA (pimicotinib), and others, are involved in developing therapies for TGCT. Discover which therapies are expected to grab major TGCT market share @ Tenosynovial Giant Cell Tumors Market Report Pimicotinib is an investigational, orally administered small-molecule inhibitor designed to selectively and effectively target the colony-stimulating factor-1 receptor. Developed by Abbisko, it remains under clinical evaluation. In December 2023, Abbisko Therapeutics agreed with Merck, granting Merck exclusive rights to commercialize pimicotinib-based products for all indications in mainland China, Hong Kong, Macau, and Taiwan, with the possibility of expanding to global markets, including the US. Pimicotinib has been granted Breakthrough Therapy Designation (BTD) by both the China National Medical Products Administration (NMPA) and the FDA, as well as Priority Medicine (PRIME) designation by the European Medicines Agency (EMA) for the treatment of patients with TGCT who are not candidates for surgery. In November 2024, Merck reported that the Phase III MANEUVER trial, conducted by Abbisko Therapeutics, met its primary endpoint. The study showed a significant improvement in the objective response rate (ORR) for patients with tenosynovial giant cell tumor. By week 25, pimicotinib achieved an ORR of 54.0%, compared to 3.2% in the placebo group (p<0.0001). Emactuzumab is a monoclonal antibody that serves as a potent and selective inhibitor of CSF-1R. By interacting with two key cytokines, CSF-1 and IL-34, the CSF-1 receptor plays a crucial role in regulating macrophages and related cells across various biological processes and organ systems. This makes it a compelling target with wide-ranging therapeutic potential. SynOx Therapeutics is currently assessing emactuzumab in the Phase III TANGENT clinical trial. In April 2024, the company secured a $35 million loan facility from Hercules Capital, bolstering its financial position as it advances the trial. Additionally, SynOx successfully completed a $75 million Series B funding round. Earlier, in August 2022, the European Medicines Agency (EMA) granted orphan medicinal product designation to emactuzumab for both localized and diffuse TGCT. AMB-05X is a powerful monoclonal antibody targeting CSF1R, designed as a therapeutic platform for severe macrophage-driven inflammatory, fibrotic, and neovascular conditions. It has received Fast Track designation from the FDA and PRIME designation from the EMA for TGCT. Currently, it is being evaluated in a Phase II clinical trial to assess its safety, tolerability, efficacy, and pharmacokinetics when administered via intra-articular injection in TGCT patients. Discover more about drugs for TGCT in development @ Tenosynovial Giant Cell Tumors Clinical Trials The anticipated launch of these emerging therapies for TGCT are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the TGCT market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for TGCT is expected to grow from USD 300 million in 2023, with a significant CAGR by 2034. This growth can be attributed to the approval of new therapies with improved safety and tolerability compared to pexidartinib, which will significantly impact the treatment landscape in the coming years. This will lead to increased adoption of these new therapies, with patients transitioning from off-label treatments to these newer options during the forecast period (2023‒2034). DelveInsight's latest published market report, titled as Tenosynovial Giant Cell Tumors Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the TGCT country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The TGCT market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Prevalent Cases of TGCT Growth Pattern-specific Prevalent Cases of TGCT Gender-specific Prevalent Cases of Localized TGCT Gender-specific Prevalent Cases of Diffuse TGCT Tumor Location-specific of Localized TGCT Tumor Location-specific Cases of Diffuse TGCT Total Treated Cases of TGCT The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM TGCT market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this TGCT market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the TGCT market. Also, stay abreast of the mitigating factors to improve your market position in the TGCT therapeutic space. Related Reports Tenosynovial Giant Cell Tumors Epidemiology Tenosynovial Giant Cell Tumors Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted TGCT epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan. Tenosynovial Giant Cell Tumors Pipeline Tenosynovial Giant Cell Tumors Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key TGCT companies, including AmMax Bio, Inc., Deciphera Pharmaceuticals SynOx Therapeutics, Abbisko Therapeutics Co, Ltd., Hutchison Medipharma Limited, Elixiron Immunotherapeutics, among others. Rheumatoid Arthritis Market Rheumatoid Arthritis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key rheumatoid arthritis companies, including Taisho Pharmaceuticals, R-Pharm, GlaxoSmithKline, Aclaris Therapeutics, Pfizer, Abivax, Bristol Myers Squibb, Oscotec, Genosco, Mesoblast, Pfizer Akros Pharma, Japan Tobacco, Abbvie, Horizon Therapeutics, Eli Lilly and Company, Taiho Pharmaceutical, Gilead Sciences, Kiniksa Pharmaceuticals, Istesso, SynAct Pharma, Cyxone, among others. Rheumatoid Arthritis Pipeline Rheumatoid Arthritis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key rheumatoid arthritis companies, including Taisho Pharmaceutical, Zhejiang Hisun Pharmaceutical, SinoMab, Jiangsu Hengrui Medicine, RemeGen, GlaxoSmithKline, Modern Biosciences, Jiangsu Alphamab Biopharmaceuticals, Akros Pharma, Hanlim Pharm, Galapagos NV, Chong Kun Dang Pharmaceutical, Bristol Myers Squibb, AstraZeneca, Aclaris Therapeutics, Abivax, AbbVie, Synact Pharma, Gilead sciences, Mesoblast, Rottapharm Biotech, Eli Lilly and Company, Kiniksa Pharmaceuticals, Abcentra, Cyxone, Yungjin Pharm. Co., Ltd. Taiho Pharmaceutical, Oscotec Inc., Pfizer, Janssen Research & Development, Cipher Pharmaceuticals Inc., KLUS Pharma Inc., Revolo Biotherapeutics, Lynk Pharmaceuticals, Beijing Baylx Biotech, Hope Biosciences, Kangstem Biotech, Celltex Therapeutics, I-Mab Biopharma Co. Ltd., Synermore Biologics Co., Ltd, Oryn Therapeutics, Arthrogen, Bayer, Amgen, Draconis Pharma, Biozeus, Landos Biopharma, Imcyse, Kymera Therapeutics, RedHill Biopharma, 4D Pharma, CV6 Therapeutics, VYNE Therapeutics, MyMD Pharmaceuticals, Brickell Biotech Inc, Worg Pharmaceuticals, Almirall, Standigm, Metrion Biosciences, Jnana Therapeutics, Silo Pharma, SFA Therapeutics, Neutrolis Therapeutics, Ahead Therapeutics, Fresenius Kabi, Kidswell Bio, BioXpress Therapeutics, CinnaGen, Alvotech, Hetero Biopharma, Shanghai Henlius Biotech, Cadila Pharmaceuticals, Innovent Biologics, Torrent Pharmaceuticals, Emcure Pharmaceuticals, mAbxience, Shanghai Junshi Biosciences, Enosi Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Phase 3Drug ApprovalFast Track

100 Deals associated with Pexidartinib Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D11271	Pexidartinib Hydrochloride	L01XE	DB12978

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Giant Cell Tumor of Tendon Sheath	United States	Daiichi Sankyo, Inc.	02 Aug 2019

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Pigmented Villonodular Synovitis	NDA/BLA	European Union	Daiichi Sankyo Europe GmbH	-
Testicular Germ Cell Tumor	Phase 3	China	Daiichi Sankyo Co., Ltd.	02 Nov 2020
Testicular Germ Cell Tumor	Phase 3	Taiwan Province	Daiichi Sankyo Co., Ltd.	02 Nov 2020
Melanoma	Phase 2	China	Plexxikon, Inc.	22 Feb 2017
Melanoma	Phase 2	South Korea	Plexxikon, Inc.	22 Feb 2017
Neurofibrosarcoma	Phase 2	United States	Daiichi Sankyo Co., Ltd.	04 Nov 2015
Unresectable Bone Sarcoma	Phase 2	United States	Daiichi Sankyo Co., Ltd.	04 Nov 2015
Gastrointestinal Stromal Tumors	Phase 2	United States	Daiichi Sankyo Co., Ltd.	02 Jul 2015
Locally Advanced Melanoma	Phase 2	United States	Daiichi Sankyo Co., Ltd.	02 Jul 2015
Non-Small Cell Lung Cancer	Phase 2	United States	Daiichi Sankyo Co., Ltd.	02 Jul 2015

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02584647 (PLX3397 \| phase II study, CTgov)	Phase 1/2	Neurofibrosarcoma \| Unresectable Bone Sarcoma	39	PLX3397+Sirolimus (Phase 1)	nylvhidvbi = yxxyugbnrf rgfazlhurz (ddqidxanht, qnwijmbdwz - oqcblmlyuz) View more	-	21 Mar 2025
NCT02584647 (PLX3397 \| phase II study, CTgov)	Phase 1/2	Neurofibrosarcoma \| Unresectable Bone Sarcoma	39	pexidartinib+sirolimus (Phase 2)	wleyyednxb(qqehrxbrqv) = qzctzasjkq gdwbqqgoag (elxhhbhpnt, inpnzrtbrb - cuawhatkdl) View more	-	21 Mar 2025
NCT04703322 (CTgov)	Phase 2	Giant Cell Tumor of Tendon Sheath	9	Pexidartinib	wzoagxnphn = jvqboxzyss lljtpcsgsi (dphlzgsxvz, fnwpyzkefd - qyeotebpta) View more	-	03 Mar 2025
NCT04526704 (PLX3397, CTgov)	Phase 4	Giant Cell Tumor of Tendon Sheath	32	Pexidartinib (Treatment-Free/Re-Treatment Cohort)	mlknbqlijk = ntwaumjvcx zbgifxvjou (ysylvsqiti, bthkfoourj - webopjloyd) View more	-	30 Dec 2024
NCT04526704 (PLX3397, CTgov)	Phase 4	Giant Cell Tumor of Tendon Sheath	32	Pexidartinib (Treatment Continuation Cohort)	qlmynyjwvg(jfxsnqxlsr) = bkfmqbyjrk uzdlnhyows (dtduwdsgtx, 6.42) View more	-	30 Dec 2024
NCT02584647 (phase II study, ASCO2024)	Phase 2	Neurofibrosarcoma	-	Pexidartinib 1000mg + Sirolimus 2mg	nwolmumyjt(qpxerndfxd) = leukopenia and rash gxvmpmysxe (uxhdozrgwi )	Negative	24 May 2024
ASCO2023	Not Applicable	Giant Cell Tumor of Tendon Sheath	167	Pexidartinib	rfchosrush(rrxiplscgi) = lwxwxzrdqj vlxrajvcdh (ovfrafyciy ) View more	-	31 May 2023
NCT04488822 (PLX3397, CTgov)	Phase 3	Giant Cell Tumor of Tendon Sheath	40	Pexidartinib	vxzrzitupn = fqesfiphug wgklxcqhhn (svdoagpacm, rukdqtiywl - enpagkfvyk) View more	-	27 Apr 2023
NCT01004861 (Pubmed) Manual	Phase 1	Solid tumor \| Giant Cell Tumor of Tendon Sheath	91	Pexidartinib (TGCT pts)	ixmyjdyoqp(nclwuqjmpx) = ztmnabxanz cpdeoqskpi (swezmopmad ) View more	Positive	15 Jan 2022
NCT01004861 (Pubmed) Manual	Phase 1	Solid tumor \| Giant Cell Tumor of Tendon Sheath	91	Pexidartinib (other solid tumor pts)	ypnvremvlt(abzbxlchut) = bcxlczrsgg xsuwjejoye (xgwmnluzmp )	Positive	15 Jan 2022
NCT02584647 (Pubmed \| Clin Cancer Res) Manual	Phase 1	Sarcoma \| Neurofibrosarcoma	18	Pexidartinib+Sirolimus	kvnthjzpvj(pnqlmydofo) = 2 mg of sirolimus combined with 1,000 mg of pexidartinib daily bbqimnajzm (ukdzxjcjky ) View more	Positive	15 Oct 2021
NCT04223635 (CTgov)	Early Phase 1	Hepatic Insufficiency	16	pexidartinib (Normal HF)	dkvdngjkxl(kejagabiuq) = ezaekcfryq tcaklyialm (zaihibvnoz, 738) View more	-	07 Jun 2021
NCT04223635 (CTgov)	Early Phase 1	Hepatic Insufficiency	16	pexidartinib (Moderate HI)	dkvdngjkxl(kejagabiuq) = wucejheqaa tcaklyialm (zaihibvnoz, 556) View more	-	07 Jun 2021
NCT01525602 (Pubmed \| Clin Cancer Res) Manual	Phase 1	Solid tumor \| Leukemia	16	Pexidartinib Hydrochloride	qetvtxaxci(rmfusimygu) = vlzgcktfcf algdaqvzfi (rjjuuisjqd ) View more	Positive	01 Dec 2020

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