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

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

Merck Exercised the Option for the Global Rights of Abbisko Therapeutics's Pimicotinib (PRNewswire) - "...Abbisko Therapeutics... announced that Merck has exercised the global commercialization option for pimicotinib (ABSK021), with an option exercise fee of USD85.0 million, under the licensing agreement signed in December 2023. The development represents further deepening of the collaboration, underscoring the unwavering commitment and confidence that both parties possess for the ongoing advancement of pimicotinib....Under the terms of the Agreement, Merck initially had an exclusive license to commercialize pimicotinib for all indications in the Chinese mainland, Hong Kong, Macau and Taiwan with an exclusive option for global commercial rights of pimicotinib. Merck now has exercised such option and has the exclusive license to commercialize pimicotinib worldwide. Abbisko Therapeutics has already received the one-time, non-refundable upfront payment of USD70.0 million in February 2024, and is now entitled to receive the additional option exercise fee of USD85.0 million..."

Licensing / partnership • 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 | Not yet recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P1 trial

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=38 | Active, not recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | Not yet recruiting ➔ Active, not recruiting

Enrollment closed

A Clinical Trial to Evaluate a High-Fat Meal and Omeprazole Enteric-coated Tablets on ASBK021 (clinicaltrials.gov) - P1 | N=36 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Not yet recruiting ➔ Completed | Trial completion date: Dec 2025 ➔ Nov 2024

Trial completion • Trial completion date

ABSK021-108: A Study to Evaluate the Relative Bioavailability of Two Different Pimicotinib Capsules in Healthy Subjects (clinicaltrials.gov) - P1 | N=26 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Active, not recruiting ➔ Completed | Trial completion date: May 2025 ➔ Jan 2025 | Trial primary completion date: May 2025 ➔ Jan 2025

Trial completion • Trial completion date • Trial primary completion date

ABSK021-106: The Study to Assess the Pharmacokinetics of Pimicotinib in Subjects With Mild and Moderate Hepatic Impairment Relative to Subjects With Normal Hepatic Function (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: Abbisko Therapeutics Co, Ltd | Enrolling by invitation ➔ Completed | Trial completion date: Jun 2025 ➔ Jan 2025

Trial completion • Trial completion date • Hepatology

A Study to Investigate the Effect of Pimicotinib on the Pharmacokinetics of Metformin, Fexofenadine and Rosuvastatin in Healthy Subjects (clinicaltrials.gov) - P1 | N=38 | Not yet recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P1 trial

ABSK021-108: A Study to Evaluate the Relative Bioavailability of Two Different Pimicotinib Capsules in Healthy Subjects (clinicaltrials.gov) - P1 | N=26 | Active, not recruiting | Sponsor: Abbisko Therapeutics Co, Ltd | Not yet recruiting ➔ Active, not recruiting

Enrollment closed

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

2024 ASH Oral Presentation----Abbisko presents promising preliminary phase 2 study results of pimicotinib in the treatment of Chronic Graft-versus-Host Disease (cGvHD) at the 66th ASH Annual Meeting (PRNewswire) - P2 | N=64 | NCT06186804 | Sponsor: Abbisko Therapeutics Co, Ltd | "Abbisko Therapeutics...announced the presentation of preliminary Phase 2 study results for pimicotinib (ABSK021) in patients with chronic Graft-versus-Host Disease (cGvHD) who have either progressed or not responded to one or more prior lines of therapy. Despite most enrolled patients having not yet completed the 6-month treatment cycle required for cGvHD response evaluation, preliminary data from the subset of patients receiving 20mg QD shows that pimicotinib achieved an ORR of 64%...the majority of enrolled patients have not yet completed the 6-month treatment cycle to determine the primary endpoint of the study, suggesting the possibility of improved outcomes with longer-term treatment with pimicotinib. The results show that pimicotinib demonstrated robust clinical efficacy and is well tolerated in heavily pre-treated patients with cGvHD."

P2 data • Chronic Graft versus Host Disease

LONG-TERM EFFICACY AND SAFETY PROFILE OF PIMICOTINIB ABSK021) IN TENOSYNOVIAL GIANT CELL TUMOR TGCT): PHASE 1B UPDATE. (CTOS 2024) - P1 | "Pimicotinib has exhibited robust and sustained antitumor efficacy with a favorable safety profile, demonstrating no evidence of cholestatic hepatotoxicity or drug-induced liver injury. Continuous tumor size reduction, along with improvements in symptoms of pain, stiffness, and joint function, has shown persistent benefits, and endorses the rationale for long-term dosing. Clinical trial information: NCT04192344."

Clinical • P1 data • Giant Cell Tumor of Bone • Oncology • Tenosynovial Giant Cell Tumor • CSF1R

ABSK021-108: A Study to Evaluate the Relative Bioavailability of Two Different Pimicotinib Capsules in Healthy Subjects (clinicaltrials.gov) - P1 | N=26 | Not yet recruiting | Sponsor: Abbisko Therapeutics Co, Ltd

New P1 trial

Updated long-term follow-up results from the phase 1b study of pimicotinib in TGCT (PRNewswire) - P1b | N=85 | NCT04192344 | Sponsor: Abbisko Therapeutics Co, Ltd | "Abbisko also announced the updated results from the Phase 1 open-label, multicenter study evaluating the safety and efficacy of pimicotinib in patients with TGCT. The poster...will be presented at the Connective Tissue Oncology Society 2024 Annual Meeting....As of June 30, 2024, data from 42 patients who received the 50 mg dose of pimicotinib showed: The best ORR was 85.0% by RECIST v1.1 per IRC. With a median treatment duration of 20.67 months (0.5, 30.1), 54.8% and 38.1% patients had an exposure of ≥ 18 months and ≥ 24 months, respectively. The median duration of response was not reached (NR) by Kaplan-Meier estimates, and 69.0% patients remained on treatment....The overall safety profile remains largely consistent, with no distinct adverse events emerging upon long-term follow-up."

P1 data • Tenosynovial Giant Cell Tumor

Pimicotinib Significantly Improved Outcomes for Patients with Tenosynovial Giant Cell Tumor in a Global Phase III Trial (PRNewswire) - P3 | N=90 | MANEUVER (NCT05804045) | Sponsor: Abbisko Therapeutics Co, Ltd | "Abbisko today announced that the Phase 3 MANEUVER study evaluating pimicotinib in the treatment of tenosynovial giant cell tumor (TGCT) achieved statistically significant improvements in objective response rate (ORR) at Week 25 of 54.0%, compared with 3.2% for placebo (p< 0.0001) based on RECIST v1.1 per Blinded Independent Review Committee (BIRC). Notably, the study also showed that treatment with pimicotinib provided statistically significant and clinically meaningful improvements in secondary endpoints associated with important patient outcomes in TGCT, including stiffness by Numeric Rating Scale (NRS; -3.00% mean change from baseline vs. -0.57 % for placebo, p<0.0001) and pain by Brief Pain Inventory (BPI; -2.32% vs. 0.23% mean change from baseline, p<0.0001). Further efficacy and safety data from Part 1 of the MANEUEVER study will be presented at an upcoming medical conference."

P3 data • Tenosynovial Giant Cell Tumor