A Phase III, Multicentre, Randomised, Double-Blind Study to Assess the Safety and Efficacy of Emactuzumab Vs. Placebo in Subjects with Tenosynovial Giant Cell Tumour

This is a multicenter, Phase 3, randomised, double-blind, placebo-controlled study, which aims to evaluate the efficacy and safety of the investigational drug emactuzumab for the treatment of patients with localized or diffuse TGCT where surgical removal of the tumor is not viewed as an option.
The study consists of two parts. In Part 1, eligible subjects will be assigned in a 2:1 ratio to receive either emactuzumab or matching placebo in a double-blind fashion, that will be administered in total 5 times as an intravenous (i.v.) infusion once every 2 weeks. This will be followed by an observation period of 3 months leading to a total duration of 24 weeks in Part 1. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. Part 2 is a long-term double-blind follow-up phase of the subjects on emactuzumab or placebo. Subjects assigned to placebo in Part 1 have the option, subject to eligibility, to crossover to receive open-label emactuzumab in Part 2. Subjects assigned to active drug in Part 1 have the option to receive open-label retreatment under certain circumstances.

Start Date30 Apr 2024

Sponsor / Collaborator

Synox Therapeutics Ltd.Startup

EUCTR2021-001716-29-AT

/ Not yet recruitingPhase 3

A Phase III, Multicentre, Randomised, Double-Blind Study to Assess the Safety and Efficacy ofEmactuzumab vs. Placebo in Subjects with Tenosynovial Giant Cell Tumour. - TANGENT

Start Date06 Sep 2022

Sponsor / Collaborator

Synox Therapeutics Ltd.Startup

NCT03768063

/ RecruitingPhase 3

An Open Label, Multicenter Extension Study in Patients Previously Enrolled in a Genentech and/or F. Hoffmann-La Roche Ltd Sponsored Atezolizumab Study (IMbrella B)

This is an open-label, multicenter, extension study. Patients who are receiving clinical benefit from atezolizumab monotherapy or atezolizumab in combination with other agent(s) or combination/comparator agent(s) during participation in a Genentech or Roche-sponsored study (the parent study), who are eligible to continue treatment and who do not have access to the study treatment locally, may continue to receive study treatment in this extension study following roll-over from the parent study.

Start Date28 Feb 2019

Sponsor / Collaborator

Hoffmann-La Roche, Inc.

100 Clinical Results associated with Emactuzumab

100 Translational Medicine associated with Emactuzumab

100 Patents (Medical) associated with Emactuzumab

Literatures (Medical) associated with Emactuzumab

01 Oct 2023·Hematology/oncology and stem cell therapy

Updates on the Treatment of Tenosynovial Giant Cell Tumor

Review

Author: Chan, Abigail S. ; Singh, Vikas ; Dy, Paul ; Katiyar, Vatsala

Tenosynovial giant cell tumor (TGCT) is a rare inflammatory disorder affecting the joint synovium, bursae, and tendon sheaths that causes non-specific and often insidious joint discomfort. The application of systemic chemotherapy has been limited due to poor and unsustained disease responses. Surgery with or without adjuvant radiation is the primary treatment modality for TGCT. With its locally destructive nature and increased recurrence, multiple surgical interventions become necessary throughout the course of the disease, leading to disfigurement, decreased quality of life, and increased mortality. However, owing to recent evidence demonstrating the overexpression of colony-stimulating factor 1 (CSF-1) in TGCT, selective tyrosine kinase inhibitors targeting CSF-1 receptors are being developed. Pex- idartinib is the first CSF-1 receptor inhibitor approved for the treatment of TGCT. Here, we discuss various available treatment strategies and ongoing investigations and trials targeting diffuse TGCT, which include nilotinib, lacnotuzu- mab, cabiralizumab, vimseltinib, and emactuzumab.

01 May 2022·Journal for immunotherapy of cancerQ2 · MEDICINE

Anti-CSF-1R emactuzumab in combination with anti-PD-L1 atezolizumab in advanced solid tumor patients naïve or experienced for immune checkpoint blockade

Q2 · MEDICINE

ArticleOA

Author: Taus, Alvaro ; Zamarin, Dmitriy ; Machiels, Jean-Pascal ; Mcdermott, David ; Korski, Konstanty ; Jegg, Anna-Maria ; Jacob, Wolfgang ; Terret, Catherine ; Gomez-Roca, Carlos ; Martinez Garcia, Maria ; Eder, Joseph P ; Cannarile, Michael A ; Delord, Jean-Pierre ; Babitzki, Galina ; Christen, Randolph ; Boni, Valentina ; Stephen Hodi, F ; Hafez, Navid ; Aspeslagh, Sandrine ; Sullivan, Ryan ; Meneses-Lorente, Georgina ; Watson, Carl ; Weisser, Martin ; Ries, Carola ; Michielin, Francesca ; Rüttinger, Dominik ; Marabelle, Aurelien ; Cassier, Philippe ; Champiat, Stephane ; Perez Gracia, Jose Luis

Background:

This phase 1b study (NCT02323191) evaluated the safety, antitumor activity, pharmacokinetics, and pharmacodynamics of colony-stimulating factor-1 receptor-blocking monoclonal antibody (mAb) emactuzumab in combination with the programmed cell death-1 ligand (PD-L1)-blocking mAb atezolizumab in patients with advanced solid tumors naïve or experienced for immune checkpoint blockers (ICBs).

Methods:

Emactuzumab (500–1350 mg flat) and atezolizumab (1200 mg flat) were administered intravenously every 3 weeks. Dose escalation of emactuzumab was conducted using the 3+3 design up to the maximum tolerated dose (MTD) or optimal biological dose (OBD). Extension cohorts to evaluate pharmacodynamics and clinical activity were conducted in metastatic ICB-naive urothelial bladder cancer (UBC) and ICB-pretreated melanoma (MEL), non-small cell lung cancer (NSCLC) and UBC patients.

Results:

Overall, 221 patients were treated. No MTD was reached and the OBD was determined at 1000 mg of emactuzumab in combination with 1200 mg of atezolizumab. Grade ≥3 treatment-related adverse events occurred in 25 (11.3%) patients of which fatigue and rash were the most common (14 patients (6.3%) each). The confirmed objective response rate (ORR) was 9.8% for ICB-naïve UBC, 12.5% for ICB-experienced NSCLC, 8.3% for ICB-experienced UBC and 5.6% for ICB-experienced MEL patients, respectively. Tumor biopsy analyses demonstrated increased activated CD8 +tumor infiltrating T lymphocytes (TILs) associated with clinical benefit in ICB-naïve UBC patients and less tumor-associated macrophage (TAM) reduction in ICB-experienced compared with ICB-naïve patients.

Conclusion:

Emactuzumab in combination with atezolizumab demonstrated a manageable safety profile with increased fatigue and skin rash over usual atezolizumab monotherapy. A considerable ORR was particularly seen in ICB-experienced NSCLC patients. Increase ofCD8 +TILs under therapy appeared to be associated with persistence of a TAM subpopulation.

01 Dec 2020·European journal of cancer (Oxford, England : 1990)Q1 · MEDICINE

Long-term clinical activity, safety and patient-reported quality of life for emactuzumab-treated patients with diffuse-type tenosynovial giant-cell tumour

Q1 · MEDICINE

Article

Author: Jegg, Anna-Maria ; Klaman, Irina ; Gomez-Roca, Carlos ; Blay, Jean-Yves ; Cassier, Philippe A ; Toulmonde, Maud ; Watson, Carl ; Jacob, Wolfgang ; D'Angelo, Sandra P ; Weisser, Martin ; Weber, Kristy ; Michielin, Francesca ; Le Tourneau, Christophe ; Delord, Jean-Pierre ; Ries, Carola H ; Abiraj, Keelara ; Italiano, Antoine ; Loirat, Delphine ; Cannarile, Michael ; Christen, Randolph ; Rüttinger, Dominik

OBJECTIVES:

This study investigated the safety, clinical activity and patient-reported outcomes of patients with diffuse-type tenosynovial giant-cell tumour (dTGCT) of the soft tissue who were treated with emactuzumab, a humanised anti-colony stimulating factor 1 receptor (CSF1R) monoclonal antibody and were followed up for up to 2 years after the start of treatment.

METHODS:

In this open-label phase 1 study (ClinicalTrials.govNCT01494688), patients received intravenous (IV) emactuzumab from 900 to 2000 mg every two weeks in the dose-escalation phase and at the optimal biological dose of 1000 mg with different schedules in the dose-expansion phase. Adverse event (AE) rates and biomarker assessments from tumour biopsies were analysed. Quality of life was assessed using a standard questionnaire (EuroQol-5D-3L) and the WOMAC® 3.1 Osteoarthritis Index. Tumour responses were determined with magnetic resonance imaging.

RESULTS:

Altogether, 63 patients were enrolled into the study. The most frequently reported AEs were pruritus, asthenia and oedema. In 36 patients for whom biopsy tissue was available a substantial decrease of CSF1R-positive and CD68/CD163-positive macrophages was detected. The independently reviewed best overall objective response rate (ORR) (Response Evaluation Criteria in Solid Tumors version 1.1) was 71%. Responses were durable, and an ORR of 70% and 64% was determined after one or two years after enrolment into the study. Clinical activity was accompanied by an improvement in EuroQol-5D-3L and particularly the joint disorder-specific WOMAC score.

CONCLUSIONS:

Systemic therapy of dTGCT patients with emactuzumab resulted in pronounced and durable responses associated with symptomatic improvement and a manageable safety profile.

News (Medical) associated with Emactuzumab

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

18 Feb 2025

·endpts.com

FDA approves Ono’s rare joint tumor drug, setting up battle with Daiichi

The FDA approval of Romvimza in a rare cancer means Ono Pharmaceutical has a fight on its hands. The company must work to displace Daiichi Sankyo’s Turalio, and fortunately, Romvimza appears to have better safety than the incumbent. On Friday , the FDA approved Romvimza for adults with symptomatic tenosynovial giant cell tumor (TGCT), in whom surgical resection could worsen functional limitation or cause severe morbidity. The pill inhibits a kinase called colony-stimulating factor 1 (CSF-1), overexpression, which is thought to be the primary cause of TGCTs. Ono obtained the drug when it bought Deciphera for $2.4 billion last year . The drug was approved based on the 40% overall response rate it achieved in the Phase 3 MOTION trial, significantly better than the 0% ORR with placebo. Turalio, which was approved in tenosynovial giant cell tumor in August 2019 , generated an ORR of 39% vs 0% for placebo in its pivotal ENLIVEN trial, so the two medicines’ efficacy is much the same. Their mechanisms are also similar — as well as inhibiting CSF-1, Turalio also hits the kinases KIT and FLT3. It is safety and convenience where Romvimza might have the edge. Turalio’s label carries a black box warning of potentially fatal hepatotoxicity; Romvimza’s does not. Only specially trained healthcare providers can prescribe Daiichi’s drug, and Turalio patients must be closely monitored for side effects. Furthermore, Daiichi’s pill must be taken twice a day; Romvimza is twice weekly. But Daiichi has the advantages of a five-year head start and much greater marketing might than Ono. Turalio’s sales came in at 5.1 billion yen ($34 million) in the first nine months of the company’s fiscal 2024. TGCTs are usually benign but can cause damage to the joints (mainly the knees, fingers, wrists and elbows) and surrounding tissues. Only around 3,700 new cases of TGCT are diagnosed in the US each year, so this is a tiny market, and every patient Ono can wrest away from Daiichi will count. Another player is preparing to enter the game. Merck KGaA’s once-daily pill pimicotinib, also a CSF-1 inhibitor, hit its Phase 3 trial in November , with an ORR of 54%, versus 3% for placebo. Crucially, there was no sign of liver toxicity in the trial, though one pimicotinib-treated patient discontinued the trial because of treatment-emergent adverse events, and five had their doses reduced for that reason. However, pimicotinib’s trial was conducted in China by Merck KGaA’s Shanghai-based partner Abbisko, the originator of the asset, and as such is unlikely to form the basis of a US filing. The other potential competitor here is SynOx, whose CSF-1R inhibitor emactuzumab is in a pivotal trial that could report data next year. Emactuzumab is intravenous, though, which might dull its appeal.

Phase 3Drug ApprovalClinical ResultAcquisition

18 Feb 2025

·medcitynews.com

Ono Pharma Drug Acquired in $2.4B Deal Wins FDA Approval for Treating Rare Type of Tumor - MedCity News

An Ono Pharma drug for a type of tumor affecting tissues of the joints is now FDA approved, giving the Japanese company a product with dosing and safety advantages over the Daiichi Sankyo product that was the first systemic therapy for this rare condition. The approval of the Ono drug, vimseltinib, covers the treatment of adults whose tenosynovial giant cell tumor (TGCT) cannot be surgically removed. The twice-weekly capsule will be marketed under the brand name Romvimza. TGCT stems from a genetic abnormality that results in overexpression of a protein called colony-stimulating factor receptor. This protein recruits other tumor-promoting cells, leading to the growth of tumors in and around joints. While these tumors are not malignant, they damage surrounding tissues and cause pain and swelling. These tumors also limit movement of the joint. Surgery is the standard treatment, but these tumors can come back and for some patients, surgery can lead to more complications. presented by Artificial Intelligence What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations When it comes to managing inbound phone calls, underperformance has devastating cost implications. By Stephanie Baum Romvimza inhibits CSF-1. The small molecule was initially developed by Waltham, Massachusetts-based Deciphera Pharmaceuticals, whose drugs are based on a technology that targets a region of an enzyme that activates it or inactivates it like an on-off switch. Last year, Ono acquired Deciphera in a $2.4 billion deal. In addition to Romvimza, Ono gained Qinlock, a cancer drug that Deciphera steered to a 2020 FDA approval in gastrointestinal stromal tumor. Inhibiting CSF-1 to treat TGCT was validated by Turalio, a twice-daily Daiichi Sankyo drug that won its FDA approval in this indication in 2019. But Turalio’s label carries a black box warning for the risk of liver toxicity. This complication was observed in some patients treated with the drug in clinical trials; there was one fatality. Daiichi Sankyo must provide a Risk Evaluation and Mitigation Strategy, a plan that informs clinicians and patients about the safety risks. In 2020, European regulators refused marketing authorization for Turalio. Clinical tests of Ono’s Romvimza showed no reports of liver toxicity. But due to the serious liver injury observed in Daichii’s CSF-1 inhibitor, Romvimza’s label advises avoiding use of the drug by patients who already have high levels of liver enzymes in the blood. The FDA also recommends liver tests before and during treatment with Romvinmza. In its 123-patient Phase 3 study, twice-weekly Romvimza led to a 40% overall response rate measured at week 25 compared to 0% in the placebo arm. Results also showed statistically significant improvement in active range of motion as well as patient reported measures of physical functioning and pain. Romvimza is still under regulatory review in Europe. Sponsored Post Tips for Greater Healthcare Practice Success in 2025 Join us on February 26 at 3 pm ET, and we’ll dive into the key areas where practices are losing time and money and provide solutions to overcome them. By Elation Health and MedCity News “The MOTION Phase 3 study demonstrated ROMVIMZA’s ability to shrink tumors along with being the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment,” Dr. Hans Gelderblom, chair of the department of medical oncology at Leiden University Medical Center, said in Ono’s approval announcement. “Romvimza is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.” There are other companies vying to treat TGCT. Merck KGaA could compete with pimicotinib, a CSF-1 inhibiting small molecule being developed under a commercialization agreement with Abbisko Therapeutics. Last November, the two companies announced this drug met the main goal of its pivotal study. Meanwhile, SynOx Therapeutics has reached Phase 3 testing with emactuzumab, an antibody designed to block the CSF-1 receptor. Last year, the privately held company raised $75 million in Series B financing for the pivotal test of this molecule.

Clinical ResultPhase 3Drug ApprovalAcquisition

100 Deals associated with Emactuzumab

External Link

KEGG	Wiki	ATC	Drug Bank
D11234	Emactuzumab	-	DB14905

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Giant Cell Tumor of Tendon Sheath	Phase 3	United States	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Belgium	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Canada	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	France	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Italy	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Netherlands	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Spain	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Sweden	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Switzerland	Synox Therapeutics Ltd.Startup	30 Apr 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Taiwan Province	Synox Therapeutics Ltd.Startup	30 Apr 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01494688 (Pubmed \| Eur J Cancer Care (Engl) \| Eur J Cancer) Manual	Phase 1	Pigmented Villonodular Synovitis	63	Emactuzumab	jgtrllgarl(iopuqmjrdk) = pruritus, asthenia and oedema shjdurvhca (wnlrygxbbp )	Positive	01 Dec 2020
NCT01494688 (Pubmed \| Ann Oncol) Manual	Phase 1	Solid tumor	99	paclitaxel+emactuzumab	nccgtmrgaz(zydqhrvcgd) = No maximum tolerated dose was reached in either study arm hztbqikqnu (fnsnlrawsc )	Negative	01 Aug 2019
NCT01494688 (Pubmed \| Ann Oncol) Manual	Phase 1	Solid tumor	99	emactuzumab		Negative	01 Aug 2019
NCT01494688 (Pubmed \| Lancet Oncol) Manual	Phase 1	Giant Cell Tumors	25	emactuzumab	ilrpnghluu(rubvozmrpe) = 5 events in 5 pts [lupus erythematosus（2 pts）, periorbital oedema（1 pt）, erythema（1 pt）, dermohypodermitis（1 pt）] vxstujlkgp (voktshhirz ) View more	Positive	01 Aug 2015

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis