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 Date09 Oct 2024

Sponsor / Collaborator

Synox Therapeutics Ltd.

NCT03768063

/ Active, not 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.

NCT03369964

/ WithdrawnPhase 1

A Phase Ib Study of The Safety and Pharmacology of Atezolizumab in Combination With an Immunotherapy Agent Investigated With or Without Anti-Cd20 Therapy in Patients With Relapsed or Refractory Non-Hodgkin Lymphoma

This study will investigate the safety, pharmacology, and activity of atezolizumab in combination with immunotherapy agents with or without an anti-CD20 agent (i.e., obinutuzumab) in patients with relapsed or refractory (R/R) follicular lymphoma (FL). The first immunotherapy molecule investigated will be emactuzumab (Arm A) in two combinations.

Start Date14 Mar 2018

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

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

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

13 Apr 2026

·endpoints.news

Neomorph raises $100M for molecular glues; Spyre's ulcerative colitis data

Plus, news about AbbVie, SynOx, Eli Lilly’s Jaypirca, ImageneBio, Nxera Pharma, Neurocrine and Novartis: Neomorph raises $100M more for molecular glues : San Diego-based Neomorph has raised a $100 million Series B round led by its founding investor, Deerfield Management. The startup dosed its first patient in February in a trial of NEO-811, its lead molecular glue degrader that targets a hard-to-drug driver of a type of kidney cancer. Neomorph has also signed research deals with Biogen, AbbVie and Novo Nordisk since emerging from stealth in December 2020 with a $109 million Series A round. — Andrew Dunn Spyre’s positive ulcerative colitis results: Spyre Therapeutics’ experimental antibody SPY001 met the primary endpoint in the first part of a Phase 2 trial, showing a statistically significant reduction from baseline on a scale used to measure ulcerative colitis disease activity. The first part of the trial was an open-label induction period testing three of Spyre’s antibodies as monotherapies. Induction data from the other two candidates, SPY002 and SPY003, are expected later this year. The next phase of the trial will test the antibodies alone and in combinations against a placebo. SPY001 is designed to improve on Takeda’s blockbuster Entyvio, which has been approved for ulcerative colitis since 2014. Spyre’s stock $SYRE climbed 26% Monday morning. — Nicole DeFeudis AbbVie licenses non-opioid pain drugs from China: The US drugmaker is paying $30 million upfront for the ex-China rights to two analgesic candidates developed by Haisco Pharmaceutical Group. Haisco is also eligible for up to $705 million in milestones and tiered royalties on sales. The two assets, known as HSK55718 and HSK51155, have been engineered to target the Nav1.8 sodium channel. — Ayisha Sharma SynOx plans FDA filing for rare tumor type: The biotech announced on Monday that its drug emactuzumab passed a Phase 3 trial in tenosynovial giant cell tumor, or TGCT. The non-malignant growths typically occur in the knee, hip or ankle joints, and cause loss of function, pain or stiffness. Emactuzumab met the primary endpoint of objective response rate, as well as key secondary endpoints such as improvement in pain and physical function. The drug was designed to be a “short-course” alternative to chronic treatment, and patients in the Phase 3 trial were given five doses over an eight-week period. SynOx said it will bring the results to the FDA in the second half of this year. — Nicole DeFeudis Lilly touts new Jaypirca data in CLL: Adding Eli Lilly’s BTK inhibitor Jaypirca to Venclexta (venetoclax) and rituximab significantly improved patients’ progression-free survival compared to the venetoclax and rituximab combination alone, Lilly said on Monday. The open-label trial enrolled patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma. Treatment in both arms was “time-limited,” meaning it had an end date. Patients received treatment for up to two years, then took no treatment until their disease progressed. Lilly didn’t spell out the full data, but its oncology president Jacob Van Naarden said in a news release that Jaypirca “outperformed our expectations.” Lilly said it plans to submit the data to regulators later this year in hopes of a label expansion. — Nicole DeFeudis ImageneBio raises $30M: The private placement will fund the development of Imagene’s lead drug IMG-007 in alopecia areata and atopic dermatitis. The monoclonal antibody is currently in a Phase 2b trial for atopic dermatitis. Coastlands Capital led the financing, with participation from other investors including Trails Edge Capital Partners, Omega Funds and OrbiMed. — Nicole DeFeudis Nxera secures $22.5M milestone from Neurocrine: The payment was triggered by Neurocrine Biosciences dosing the first patient in a Phase 2 schizophrenia trial testing a drug called NBI-570, which was discovered using Nxera’s NxWave platform. The two companies entered a collaboration in 2021 that could be worth up to $2.7 billion. — Ayisha Sharma Novartis to lay off 114 staffers in New Jersey: The Swiss drugmaker disclosed the staff cuts in East Hanover, which will take effect from June to November, in a WARN notice for the state. Novartis did not immediately respond to a request for comment. — Ayisha Sharma

13 Apr 2026

·www.fiercebiotech.com

BioNTech, SynOx plot paths to FDA as trials hit goals in competitive tumor types

BioNTech and SynOx plan to file for FDA approvals this year.\n The fight for two tumor types is poised to intensify, with BioNTech lining up a challenge to AstraZeneca, while SynOx Therapeutics is planning to enter a market served by companies including Daiichi Sankyo.BioNTech’s data come from a phase 2 trial of its Duality Biologics-partnered antibody-drug conjugate (ADC) trastuzumab pamirtecan. The biotech reported a 49.3% response rate to the HER2-targeted ADC in a phase 2 cohort of 73 patients with HER2-expressing, advanced endometrial cancer who progressed on or after first-line chemotherapy and checkpoint inhibitor therapy, hitting its primary endpoint.The primary analysis cohort was part of a broader study that included people without prior exposure to checkpoint inhibitor therapy. Across all 96 centrally tested patients, BioNTech reported a response rate of 47.9% and median progression-free survival of 8.1 months. The response rate fell to 44.1% in the full, 143-patient cohort, which included patients tested for HER2 at their trial site or local laboratory. BioNTech said the safety profile was manageable and as expected for HER2-targeted ADCs. Almost 47% of patients had grade 3 or greater treatment-related adverse events. Almost 5% of patients had grade 3 or greater interstitial lung disease (ILD) or pneumonitis. AstraZeneca and Daiichi’s HER2-directed ADC Enhertu carries a boxed warning for ILD and pneumonitis. The FDA approved Enhertu in adults with unresectable or metastatic HER2-positive solid tumors in 2024. The tumor-agnostic authorization was supported (PDF) by data including a 56.3% response rate in 16 endometrial cancer patients. BioNTech, which hailed its trial as the largest in the indication, plans to file for FDA approval this year.Separately, SynOx aims to file for FDA approval of emactuzumab in tenosynovial giant cell tumor (TGCT) patients in the second half of the year. SynOx outlined the plan as part of the release of phase 3 data on the CSF1R inhibitor, which achieved a significantly better response rate than placebo at Month 6 to hit the primary endpoint. The biotech also reported hits on secondary endpoints.Daiichi won FDA approval for a CSF1R inhibitor, Turalio, in TGCT in 2019. Last year, Ono Pharmaceutical got the green light to sell a rival drug, Romvimza, that it acquired in its $2.4 billion takeover of Deciphera. Merck KGaA filed for FDA approval of another CSF1R inhibitor, pimicotinib, in the indication in January. Having ceded a head start to rivals, SynOx is positioning the former Roche antibody emactuzumab as a differentiated, short-course treatment. Phase 3 participants received emactuzumab intravenously every two weeks for eight weeks. Turalio (PDF) and Romvimza (PDF) are given orally, respectively twice daily and twice weekly. Merck tested once-daily pimicotinib in its phase 3 trial.

Clinical ResultDrug ApprovalPhase 3ADCPhase 2

13 Apr 2026

·firstwordpharma.com

Study results set up FDA filing for SynOx's emactuzumab in giant cell tumour

SynOx Therapeutics is heading to the FDA with a filing for emactuzumab after the experimental anti-CSF-1 receptor monoclonal antibody met the primary and secondary endpoints of a Phase III study for adults with tenosynovial giant cell tumour (TGCT). A US submission planned for the second half of the year is expected to be followed by an application in the EU.The filings will be based on the TANGENT trial, which investigated emactuzumab at a dose of 1000 mg administered every two weeks, for a total of five doses over an eight-week period, versus placebo in patients with TGCT. The study's main goal is objective response rate (ORR) at six months, with secondary endpoints measuring the effect of emactuzumab on physical function, range of motion, stiffness, pain and duration of response.SynOx said Monday that top-line results across the primary and key secondary goals demonstrated clinically meaningful and statistically significant benefit for emactuzumab. The company noted that the drug's benefits were achieved rapidly in the short-course treatment cycle, were durable and were observed across clinically relevant patient segments."Emactuzumab's combination of rapid onset, response rate, meaningful functional improvement and a defined short-course regimen positions it as a potential alternative to chronic therapy," said CEO Ray Barlow. "We believe this approach directly addresses key limitations of existing treatments."According to SynOx, emactuzumab demonstrated a manageable safety profile in TANGENT, consistent with prior clinical experience. Full data from the trial will be presented at an upcoming medical meeting.SynOx was formed in 2020 as a spin-out from Celleron Therapeutics to develop emactuzumab, which the latter in-licensed from Roche earlier the same year.

Clinical ResultPhase 3Cell Therapy

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.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Belgium	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Canada	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	France	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Italy	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Netherlands	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	South Korea	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Spain	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Sweden	Synox Therapeutics Ltd.	09 Oct 2024
Giant Cell Tumor of Tendon Sheath	Phase 3	Switzerland	Synox Therapeutics Ltd.	09 Oct 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02323191 (Pubmed \| J Immunother Cancer)	Phase 1	Advanced Malignant Solid Neoplasm	221	Emactuzumab + Atezolizumab	wgwuszongs(mfunhrmyso) = 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) xvsjvokcwp (dsbxaxiguk )	Positive	01 May 2022
NCT01494688 (Pubmed \| Eur J Cancer Care (Engl) \| Eur J Cancer) Manual	Phase 1	Pigmented Villonodular Synovitis	63	Emactuzumab	faorppgpwp(rxkssxbdyi) = pruritus, asthenia and oedema rnynnirtnk (mivcexlywy )	Positive	01 Dec 2020
NCT01494688 (Pubmed \| Ann Oncol) Manual	Phase 1	Solid tumor	99	paclitaxel+emactuzumab	mebnqzyjbn(wqucmoklem) = No maximum tolerated dose was reached in either study arm eevvhnfxrb (axrvjxqrqt )	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	ufzpzpwfqd(stkamdcggq) = 5 events in 5 pts [lupus erythematosus（2 pts）, periorbital oedema（1 pt）, erythema（1 pt）, dermohypodermitis（1 pt）] upjbvkcimf (ptpmwelkhu ) 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

Hiro LS Researcher

The AI Search Engine Built to Accelerate Biopharma Decisions

Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.

Ask any biopharma research question→

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.

Discover Synapse Data Servers

Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis