pimicotinib (ABSK021) / Abbisko, EMD Serono - LARVOL DELTA

Management of tenosynovial giant cell tumor: approved and investigational therapies. (PubMed, Expert Rev Anticancer Ther) - "Pexidartinib and vimseltinib demonstrate comparable efficacy in TGCT with objective response rates (ORR) of 39% and 40%, respectively, at week 25 of treatment; however, vimseltinib offers improved hepatic safety and tolerability, supporting its use as the preferred first-line systemic therapy. Emerging agents, including pimicotinib and emactuzumab, show potential for higher response rates and favorable safety profiles and may further reshape the TGCT treatment paradigm pending phase 3 trial results."

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

FDA Accepts New Drug Application for Pimicotinib for the Treatment of Tenosynovial Giant Cell Tumor (Businesswire) - "The application is based on the primary results and longer-term follow-up of the global Phase 3 MANEUVER study, which demonstrated deep and durable tumor responses and meaningful improvements in clinical outcomes with pimicotinib."

FDA filing • Tenosynovial Giant Cell Tumor

Pimicotinib Approved as Systemic Treatment in China for Tenosynovial Giant Cell Tumor (Businesswire) - "First regulatory approval in the world for pimicotinib based on positive data from global Phase 3 MANEUVER study."

China approval • Tenosynovial Giant Cell Tumor

A Phase II Study to Evaluate the Efficacy and Safety of Pimicotinib (ABSK021) in Chronic Graft Versus Host Disease (cGvHD) after 1 or More Lines of Systemic Treatment (ASH 2024) - P2 | "Pts had received a median of 4 prior systemic treatment (range, 1-9), including ruxolitinib (67.9%, n=19), ibrutinib (10.7%, n=3), and belumosudil (3.6%, n=1); with a median of 4 organs involved at baseline (range, 2-7). Conclusions The efficacy and safety results are in favor of Pimi as a promising Tx option for cGvHD, addressing significant unmet medical needs, especially in heavily pre-treated pts. Further explorations are warranted to confirm these findings with long-term outcomes."

Clinical • P2 data • Cardiovascular • Chronic Graft versus Host Disease • Fibrosis • Giant Cell Tumor of Bone • Graft versus Host Disease • Hypertension • Immunology • Infectious Disease • Oncology • Pneumonia • Respiratory Diseases • Scleroderma • Systemic Sclerosis • Tenosynovial Giant Cell Tumor

A Study to Evaluate the Relative Bioavailability of Pimicotinib Capsules Containing Free Base in Different Proportions in Healthy Subjects (clinicaltrials.gov) - P1 | N=20 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Active, not recruiting ➔ Completed

Trial completion

MEDICAL MANAGEMENT OF TENOSYNOVIAL GIANT CELL TUMOR (CTOS 2025) - "Multiple colony-stimulating factor 1 receptor inhibitors (CSF1Ris) have emerged for symptomatic pts, with pexidartinib (pexi) and vimseltinib (vim) approved. Here, we review phase 3 reports for pexi, vim, and pimicotinib (pimi). PubMed was searched for publications associated with TGCT, CSF-1Ri, therapies for treatment of TGCT, and known guidelines, with a focus on phase 3 trials and postmarket analyses... CSF1Ris show efficacy in TGCT. Pts should be monitored for potential side effects that are different for each presented drug. Further research to understand long-term side effects and duration of response will be paramount to optimize therapeutic strategies for pts with TGCT."

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

PIMICOTINIB IN TENOSYNOVIAL GIANT CELL TUMOR (TGCT): LONG-TERM EFFICACY, SAFETY, AND PATIENT-REPORTED OUTCOMES FROM THE PHASE 3 MANEUVER STUDY (CTOS 2025) - P3 | "Sustained treatment with pimi in pts with TGCT demonstrated continued improvements in efficacy, enhanced patient outcomes, and an acceptable safety profile, reinforcing its potential for long-term use in eligible pts.Clinical Outcome Assessments"

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

ABBISKO THERAPEUTICS PRESENTS LONGER-TERM EFFICACY AND SAFETY OUTCOMES FROM PHASE III MANEUVER STUDY OF PIMICOTINIB AT CTOS 2025 ANNUAL MEETING (HKEXnews) - "This longer-term analysis showed that sustained treatment with pimicotinib in patients with TGCT led to continued improvements in tumor response, patient-reported outcomes including pain and function, and maintained an acceptable safety profile, reinforcing its potential for long-term use in eligible patients."

P3 data • Patient reported outcomes • Tenosynovial Giant Cell Tumor

Extended efficacy and safety from the phase III MANEUVER trial of pimicotinib in patients with tenosynovial giant cell tumour (TGCT) (ESMO 2025) - P3 | "PROMIS-PF, Patient-Reported Outcomes Measurement Information System-Physical Function; SD, standard deviation. Conclusions This extended analysis of MANEUVER demonstrated that tumour responses to pimicotinib continued to improve and are durable, with ongoing improvements in COAs and a consistent safety profile, supporting the timely initiation and long-term treatment of eligible patients with pimicotinib."

Clinical • P3 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor

Pimicotinib Treatment Demonstrates Deep and Durable Tumor Responses and Continued Improvements in Pain and Function for Patients with TGCT (Businesswire) - "This latest analysis showed that, with a median follow-up of 14.3 months, the objective response rate (ORR) for people treated with pimicotinib from the beginning of the study increased considerably to 76.2% (95% CI: 63.8, 86.0) by blinded independent review committee (BICR) per RECIST v1.1, from 54% at Week 25....The results are being presented today in the Sarcoma mini-oral session at the European Society for Medical Oncology (ESMO) Congress 2025..."

P3 data • Tenosynovial Giant Cell Tumor

A Study to Evaluate the Relative Bioavailability of Pimicotinib Capsules Containing Free Base in Different Proportions in Healthy Subjects (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P1 trial

New molecules in the therapy of chronic graft-versus-host disease. (PubMed, Curr Opin Hematol) - "Expanding therapeutic options in cGvHD require decision-making based on organ involvement, prior therapy, and tolerability. Emerging compounds offer the potential to modulate chronic inflammation and fibrosis more precisely, supporting a move toward personalized and combinatorial approaches in advanced-line settings."

Journal • Chronic Graft versus Host Disease • Fibrosis • Graft versus Host Disease • Immunology • Inflammation • Transplantation

Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER) (clinicaltrials.gov) - P3 | N=90 | Active, not recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | Trial primary completion date: May 2026 ➔ Sep 2024

Trial primary completion date • Giant Cell Tumor of Bone • Tenosynovial Giant Cell Tumor

Pharmacologic inhibition of CSF-1R suppresses intrinsic tumor cell growth in osteosarcoma with CSF-1R overexpression. (PubMed, J Transl Med) - "These findings conclusively demonstrated that pharmacological inhibition of CSF-1R activity by ABSK021 resulted in significant anti-tumor effects in preclinical osteosarcoma models with CSF-1R overexpression. The high prevalence of CSF-1R expression observed in osteosarcoma patient samples highlights the potential clinical use of ABSK021, either as a monotherapy or in combination with chemotherapy, as a promising therapeutic strategy for osteosarcoma patients with CSF-1R as a potential predictive biomarker."

Journal • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • CASP3 • CSF1R • IL34

Merck KGaA, Darmstadt, Germany Completes Acquisition of SpringWorks Therapeutics to Accelerate Sustainable Growth of its Healthcare Business (Businesswire) - "Merck KGaA...announced that it has closed the acquisition of SpringWorks Therapeutics, Inc., for an enterprise value of $3.4 billion (approximately €3 billion), following regulatory clearances and the fulfillment of other customary closing conditions. The definitive agreement between Merck KGaA...and SpringWorks...was announced on 28 April, 2025. It represents one of the biggest M&A deals in the global biopharma sector so far in 2025. The business combination will immediately contribute to Merck KGaA, Darmstadt, Germany’s revenues and is expected to be accretive to the company’s earnings per share pre (EPS pre) by 2027....Acquisition marks the formation of a rare tumor business for Merck KGaA, Darmstadt, Germany including SpringWorks portfolio and pimicotinib. SpringWorks rare tumor portfolio includes Ogsiveo, a first-in-disease systemic therapy for adults with desmoid tumors and Gomekli a first and only approved therapy for adults and children with NF1-PN."

M&A • Desmoid Tumors • Neurofibromatosis • Tenosynovial Giant Cell Tumor

Pimicotinib Demonstrates Best-in-Class Potential with Significant Efficacy and Clinically Meaningful Improvements in Patients with Tenosynovial Giant Cell Tumor (EMD Serono Press Release) - P3 | N=90 | MANEUVER (NCT05804045) | Sponsor: Abbisko Therapeutics Co, Ltd | "Once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) compared with placebo at week 25 (54.0% vs. 3.2% for placebo (p<0.0001). The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT. These findings will be presented Sunday, June 1 in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting....In MANEUVER, which enrolled patients from China, Europe and North America, the effect of pimicotinib had an early onset, with 41.3 % (26 of 63) of patients experiencing objective response to therapy after 13 weeks."

P3 data • Tenosynovial Giant Cell Tumor

ABSK021-110: A Study to Investigate The Effect of Pimicotinib on The Pharmacokinetics of Metformin, Fexofenadine and Rosuvastatin In Healthy Subjects (clinicaltrials.gov) - P1 | N=39 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Active, not recruiting ➔ Completed

Trial completion

Abbisko Therapeutics Announces China NMPA Acceptance of New Drug Application for Pimicotinib for the Treatment of TGCT (The Manila Times) - "Abbisko Therapeutics Co., Ltd...announced that the China National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for pimicotinib, a highly selective, small-molecule CSF-1R inhibitor, as a Class 1 innovative drug for adult patients with Tenosynovial Giant Cell Tumor (TGCT)....The application is based on results from Part 1 of the global Phase 3 MANEUVER study, in which once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) compared with placebo at week 25 (54.0% vs. 3.2% for placebo; p<0.0001)."

China filing • Tenosynovial Giant Cell Tumor

Pimicotinib in tenosynovial giant cell tumor (TGCT): Efficacy, safety and patient-reported outcomes of phase 3 MANEUVER study. (ASCO 2025) - P3 | "MANEUVER is the first randomized pivotal study in TGCT to demonstrate > 50% ORR by RECIST v1.1 at Week 25 in a diverse, global patient population. Pimi produced statistically significant and clinically meaningful improvements in physical function and symptoms, representing an effective, well-tolerated and convenient daily treatment for patients with TGCT and addressing a critical unmet need."

Clinical • P3 data • Patient reported outcomes • Giant Cell Tumor of Bone • Hepatology • Liver Failure • Musculoskeletal Diseases • Musculoskeletal Pain • Oncology • Orthopedics • Pain • Tenosynovial Giant Cell Tumor

Merck KGaA To File for Global Approval of Pimicotinib After Positive Phase III TGCT Results (MSN News) - "Merck KGaA...having secured global rights from Abbisko in March 2025, has filed for approval in China and plans an FDA submission by year-end."

China filing • FDA filing • Tenosynovial Giant Cell Tumor

Abbisko Therapeutics Announces Receipt of US$85mn from Merck in Consideration for Pimicotinib (ABSK021) Global Commercialization Option (Abbisko Therap Press Release) - "Abbisko Therapeutics Co., Ltd...announced receipt of the global commercialization option exercise fee of US$85 million from Merck under the licensing agreement for pimicotinib (ABSK021). This option exercise fee will be recognized as revenue in 2025....In March 2025, Merck exercised its option to obtain the exclusive license to commercialize pimicotinib worldwide. Abbisko is eligible to receive up to US$605.5 million in milestone payments, including upfront, development, and commercial milestones, as well as double-digit percentage royalties on annual net sales of pimicotinib worldwide."

Financing • Tenosynovial Giant Cell Tumor

Medical Management of Tenosynovial Giant Cell Tumor. (PubMed, Curr Oncol Rep) - "For an alternative to surgery, the CSF1R inhibitors pexidartinib and vimseltinib are approved in the United States for TGCT, and other CSF1R inhibitors are in clinical development...The potential risks and benefits of available treatments should be carefully considered in collaboration with a bone tumor-experienced, multidisciplinary team to determine the best course of care. Increased D-TGCT awareness and support through patient advocacy groups have helped to reshape the patient journey."

Journal • Review • Giant Cell Tumor of Bone • Oncology • Osteosarcoma • Solid Tumor • Tenosynovial Giant Cell Tumor

Merck Showcases Innovation in Advanced Cancers and Rare Tumors at ASCO 2025 (Businesswire) - "Merck...announced the presentation of new oncology data across more than 12 tumor types at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, May 31 to June 4 in Chicago. The presentations include the Phase 3 MANEUVER data for potentially best-in-class pimicotinib in the treatment of the rare tumor TGCT, as well as data from both company- and investigator-sponsored studies highlighting the company’s focus on advancing differentiated molecules to tackle some of the most challenging cancers."

Clinical data • P3 data • Bladder Cancer • Colorectal Cancer • Merkel Cell Carcinoma • Tenosynovial Giant Cell Tumor • Urothelial Cancer

ABSK021-101: A Study to Assess the Safety, Tolerability, and Pharmacokinetics of ABSK-021 in Patients With Advanced Solid Tumor (clinicaltrials.gov) - P1 | N=276 | Recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | N=85 ➔ 276 | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Enrollment change • Trial completion date • Trial primary completion date • Giant Cell Tumor of Bone • Oncology • Solid Tumor • Tenosynovial Giant Cell Tumor

A Study to Evaluate the Effect of a High-fat Meal on the Exposure of Pimicotinib Capsule in Healthy Subjects (clinicaltrials.gov) - P1 | N=16 | Active, not recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | Not yet recruiting ➔ Active, not recruiting

Enrollment closed