Romvimza (vimseltinib) / Ono Pharma - LARVOL DELTA

Characterizing Improvements in Physical Functioning with PROMIS-PF and In-Trial Exit Interviews: A Mixed-Methods Analysis to Understand the Meaningful Changes Experienced by TGCT Patients on Vimseltinib in the MOTION Trial (ISPOR 2025) - P3 | "Many TGCT patients on vimseltinib experienced clinically meaningful changes in physical functioning, and through the approach presented here, the nature and extent of these improvements can now be more clearly communicated with physicians and patients. It is often challenging to characterize the specific improvements contributing to total scores that patients have experienced using multi-item measures such as the PROMIS-PF. The method presented here to characterize changes on multi-item and/or concept COA measures may be applicable across disease areas and provides clinically useful information for physicians and patients considering effective treatment options."

Clinical • Interview • Giant Cell Tumor of Bone • Oncology • Pain • Tenosynovial Giant Cell Tumor

Study of DCC-3014 in Combination With Avelumab in Patients With Advanced or Metastatic Sarcomas (clinicaltrials.gov) - P1 | N=32 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Jan 2025 ➔ Jan 2026 | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date • Leiomyosarcoma • Liposarcoma • Oncology • Sarcoma • Solid Tumor • Undifferentiated Pleomorphic Sarcoma

Vimseltinib (Romvimza) for tenosynovial giant cell tumor. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

NCCN has published updates to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines), and the NCCN Drugs & Biologics Compendium (NCCN Compendium) for Soft Tissue Sarcoma, Version 5.2024. (NCCN)

NCCN guideline • Soft Tissue Sarcoma

An evaluation of vimseltinib for treatment of tenosynovial giant cell tumors. (PubMed, Expert Rev Anticancer Ther) - "In the MOTION study, the use of vimseltinib in patients with advanced TGCT resulted in a high objective response rate, substantial benefit in reducing clinical symptoms (such as pain and stiffness), and a favorable safety profile. Vimseltinib represents a promising new therapeutic option for patients with unresectable TGCT and is currently awaiting regulatory review by the FDA and EMA."

Journal • Review • Colorectal Cancer • Giant Cell Tumor of Bone • Oncology • Pain • Solid Tumor • Tenosynovial Giant Cell Tumor

FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor (FDA) - "On February 14, 2025, the Food and Drug Administration approved vimseltinib (Romvimza, Deciphera Pharmaceuticals, LLC), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity....Efficacy was evaluated in MOTION (NCT05059262), a double-blind, multicenter, randomized (2:1), placebo-controlled trial in patients with TGCT for whom surgical resection may cause worsening functional limitation or severe morbidity....The recommended vimseltinib dose is 30 mg orally twice weekly, with a minimum of 72 hours between doses."

FDA approval • Tenosynovial Giant Cell Tumor

Emerging treatments for sarcoma: from 2024 onward. (PubMed, Expert Opin Emerg Drugs) - "While these strategies have demonstrated some success, the overall improvements in survival have often been modest, irrespective of the therapeutic modality. This underscores critical concerns regarding the true cost-benefit balance and the potential for adverse effects, particularly when evaluated over extended follow-up periods."

Journal • Review • Oncology • Osteosarcoma • Sarcoma • Solid Tumor

Safety, efficacy, and patientreported outcomes with vimseltinib in patients with tenosynovial giant cell tumor who received no prior anti–colony-stimulating factor 1 therapy: ongoing phase 2 study (AIOM 2024) - P1/2 | "Material and Pts with TGCT not amenable to surgery and with no prior anti-CSF1/CSF1R therapy (previous treatment with imatinib or nilotinib allowed) received vimseltinib 30 mg twice weekly (recommended phase 2 dose). These results demonstrate that vimseltinib can provide antitumor activity and pain relief in pts with TGCT not amenable to surgery with no prior anti-CSF1/ CSF1R therapy."

P2 data • Patient reported outcomes • Cardiovascular • Giant Cell Tumor of Bone • Hepatology • Hypertension • Oncology • Pain • Solid Tumor • Tenosynovial Giant Cell Tumor • CSF1 • CSF1R

SAFETY AND EFFICACY WITH VIMSELTINIB IN PATIENTS WITH TENOSYNOVIAL GIANT CELL TUMOR WHO RECEIVED NO PRIOR ANTI-COLONY-STIMULATING FACTOR 1 THERAPY: ONGOING PHASE 2 STUDY (CTOS 2024) - P1/2 | "Here, we will report long-term safety and efficacy from cohort A of the phase 2 part (expansion; enrollment complete) of an ongoing phase 1/2 study (NCT03069469) for patients with tenosynovial giant cell tumor (TGCT) treated with vimseltinib. Patients with symptomatic TGCT not amenable to surgery and no prior anti-CSF1/CSF1R therapy (previous imatinib or nilotinib allowed) received vimseltinib 30 mg twice weekly (recommended phase 2 dose). Vimseltinib is well tolerated and demonstrates robust antitumor activity in patients with TGCT. Updated results are expected to support long-term safety and efficacy of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy. Previously presented in part at CTOS 2023 and intended update submitted for consideration to ESMO 2024."

P2 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1

SAFETY, EFFICACY, AND PATIENT-REPORTED OUTCOMES WITH VIMSELTINIB IN PATIENTS WITH TENOSYNOVIAL GIANT CELL TUMOR WHO RECEIVED PRIOR ANTI-COLONY-STIMULATING FACTOR 1 THERAPY: ONGOING PHASE 2 STUDY (CTOS 2024) - No abstract available

P2 data • Patient reported outcomes • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1

EFFICACY, SAFETY, AND PATIENT-REPORTED OUTCOMES OF VIMSELTINIB IN PATIENTS WITH TENOSYNOVIAL GIANT CELL TUMOR: RESULTS FROM THE MOTION PHASE 3 TRIAL (CTOS 2024) - No abstract available

Clinical • P3 data • Patient reported outcomes • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

Efficacy, safety, and patientreported outcomes of vimseltinib in patients with tenosynovial giant cell tumor: Results from the MOTION phase 3 trial (AIOM 2024) - P3 | "Patients receiving vimseltinib experienced statistically significant and clinically meaningful improvements in the primary and all key secondary endpoints. These results demonstrate vimseltinib is an effective, welltolerated CSF1R-targeted therapy that improves functional health and quality of life in patients with TGCT. Previously presented at ASCO 2024."

Clinical • P3 data • Patient reported outcomes • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Pain • Solid Tumor • Tenosynovial Giant Cell Tumor • CSF1R

A Study to Evaluate Vimseltinib in Adults with Active Chronic Graft-Versus-Host Disease (cGVHD) (clinicaltrials.gov) - P2 | N=36 | Recruiting | Sponsor: Deciphera Pharmaceuticals, LLC | Not yet recruiting ➔ Recruiting

Enrollment open • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

A Mixed-Methods Analysis to Define Minimum Clinically Importance Differences (MCIDs) in Range of Motion, Physical Function, and Worst Stiffness in Patients With Tenosynovial Giant Cell Tumor (TGCT) (ISPOR-EU 2024) - P3 | " Qualitative embedded exit interviews were conducted with patients participating in the double-blind MOTION Phase 3 trial (NCT05059262) of vimseltinib vs placebo for TGCT... Combined evidence from anchor and distribution-based methods supported by patient insights from qualitative interviews informed the final selection of MCIDs of +3-points for PROMIS-PF, +10% for active ROM, and -2-points for Worst Stiffness NRS."

Clinical • Giant Cell Tumor of Bone • Oncology • Pain • Tenosynovial Giant Cell Tumor

Efficacy, Safety and Patient-Reported Outcomes of Vimseltinib in Patients with Tenosynovial Giant Cell Tumor: Results from the Phase 3 MOTION Trial (DGHO 2024) - P3 | "Pts receiving vimseltinib experienced statistically significant and clinically meaningful improvements in the primary and all key secondary endpoints. These results demonstrate vimseltinib is an effective, well-tolerated CSF1R-targeted therapy that improves functional health and quality of life in pts with symptomatic TGCT not amenable to surgery. Abstract to be first presented at ASCO 2024."

Clinical • P3 data • Patient reported outcomes • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Pain • Solid Tumor • Tenosynovial Giant Cell Tumor • CSF1R

Vimseltinib versus a placebo in patients with tenosynovial giant cell tumor: a plain language summary of the MOTION phase 3 trial. (PubMed, Future Oncol) - "Vimseltinib was better at shrinking tumors and improving active range of motion, stiffness, pain, and other health measures than the placebo for participants with TGCT. Vimseltinib has the potential to become a new treatment option for patients with TGCT for whom surgery may not provide benefit."

Journal • P3 data • Giant Cell Tumor of Bone • Oncology • Pain • Tenosynovial Giant Cell Tumor

A Phase 2 Study to Evaluate Vimseltinib in Adults with Active Chronic Graft-Versus-Host Disease (cGVHD) (clinicaltrials.gov) - P2 | N=36 | Not yet recruiting | Sponsor: Deciphera Pharmaceuticals, LLC

New P2 trial • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Safety, efficacy, and patient-reported outcomes (PROs) with vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT) who received prior anti–colony-stimulating factor 1 (CSF1) therapy: Ongoing phase II study (ESMO 2024) - P1/2 | "Other assessments included active range of motion and PROs: worst pain, physical function, worst stiffness, and health status. At a prior data cutoff (June 2023), 19 pts with TGCT who received prior anti-CSF1/CSF1R therapy were enrolled in cohort B. Most pts (79%) received prior pexidartinib. Vimseltinib is well tolerated and demonstrates robust antitumor activity in previously treated pts with TGCT. Updated results are expected to support long-term safety and efficacy and symptomatic improvements in pts with TGCT who received prior anti-CSF1/CSF1R therapy."

P2 data • Patient reported outcomes • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1 • CSF1R

Safety and efficacy with vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT) who received no prior anti–colony-stimulating factor 1 (CSF1) therapy: Ongoing phase II study (ESMO 2024) - P1/2 | "Here, we will report long-term safety and efficacy from cohort A of the phase II part (expansion; enrollment complete) of an ongoing phase I/II study (NCT03069469) for pts with TGCT treated with vimseltinib. Pts with symptomatic TGCT not amenable to surgery and no prior anti-CSF1/CSF1R therapy (previous imatinib or nilotinib allowed) received vimseltinib 30 mg twice weekly (recommended phase II dose). Vimseltinib is well tolerated and demonstrates robust antitumor activity in pts with TGCT. Updated results are expected to support long-term safety and efficacy of vimseltinib in pts with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy."

P2 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1 • CSF1R

Updated efficacy and safety of vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT): One-year follow-up from the MOTION phase III trial (ESMO 2024) - P3 | "In this first report of long-term results (≥1 year) from MOTION, vimseltinib continued to be well tolerated and provided long-term antitumor responses in pts with TGCT not amenable to surgery."

Clinical • Late-breaking abstract • P3 data • Giant Cell Tumor of Bone • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor

U.S. Food and Drug Administration Accepts for Priority Review Deciphera’s New Drug Application for Vimseltinib for the Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT) (Businesswire) - "Ono Pharmaceutical...announced that the U.S. Food and Drug Administration (FDA) accepted a priority review for the New Drug Application (NDA) on August 14 US time for vimseltinib, a colony stimulating factor 1 receptor (CSF1R), for the treatment of patients with tenosynovial giant cell tumor (TGCT), which is under development by Deciphera Pharmaceuticals, Inc. ('Deciphera'), a wholly-owned subsidiary of Ono. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of February 17, 2025....The submission is supported by the data from the pivotal Phase 3 MOTION study, evaluating the efficacy and safety of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo."

FDA filing • PDUFA • Priority review • Tenosynovial Giant Cell Tumor

CSF1R Inhibition in Patients With Advanced Solid Tumors or Tenosynovial Giant Cell Tumor: A Phase 1 Study of Vimseltinib. (PubMed, Clin Cancer Res) - P1/2 | "Vimseltinib demonstrated long-term tolerability, manageable safety, dose-dependent exposure, and robust antitumor activity in patients with TGCT whose disease is not amenable to surgery."

Journal • Metastases • P1 data • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor • CSF1R

European Medicines Agency Accepts Deciphera’s Marketing Authorization Application for Vimseltinib for Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT) (Businesswire) - "Ono Pharmaceutical...announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for vimseltinib, a colony stimulating factor 1 receptor (CSF1R), for the treatment of patients with tenosynovial giant cell tumor (TGCT), which is under development by Deciphera Pharmaceuticals....The review of the MAA begins under the EMA’s centralized review process for all 27 member states of the European Union (EU), as well as Iceland, Liechtenstein and Norway....The submission is supported by the data from the pivotal Phase 3 MOTION study, evaluating the efficacy and safety of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo."

EMA filing • Tenosynovial Giant Cell Tumor

Vimseltinib improves outcomes in tenosynovial giant cell tumour. (PubMed, Nat Rev Clin Oncol) - No abstract available

Journal • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

Ono Announces Results of Tender Offer to Acquire Deciphera Pharmaceuticals and Completion of Acquisition of Deciphera (a Wholly Owned Subsidiary of Ono) (PRNewswire) - "Ono Pharmaceutical...today announced that it has successfully completed the tender offer, previously announced on April 30, 2024 to acquire all outstanding shares of common stock of a US biopharmaceutical company, Deciphera Pharmaceuticals, Inc...for US$25.60 per share (total amount of approximately US$2.4 billion) net to the seller in cash, without interest thereon and less any applicable withholding taxes, through its wholly owned subsidiary, Topaz Merger Sub, Inc...established in the State of Delaware, United States, solely for the purpose of engaging in the transactions contemplated in the Merger Agreement....'Through this acquisition, we will leverage Deciphera's excellent research and development capabilities in the oncology field and its sales power in Europe and the United States, and work to further accelerate the expansion of our pipeline and global expansion, which are part of our growth strategies.'"

M&A • Gastrointestinal Stromal Tumor • Graft versus Host Disease • Hematological Malignancies • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • Tenosynovial Giant Cell Tumor