Delving into the Latest Updates on Pimicotinib with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

CSF-1R

Mechanism

CSF-1R antagonists(Colony stimulating factor 1 receptor antagonists)

Therapeutic Areas

NeoplasmsNervous System DiseasesSkin and Musculoskeletal Diseases+ [5]

Active Indication

Pigmented Villonodular SynovitisPancreatic CancerChronic graft-versus-host disease+ [4]

Inactive Indication

Sarcoma

Originator Organization

Abbisko Therapeutics Co., Ltd.

Active Organization

Abbisko Therapeutics Co., Ltd.

Inactive Organization-

Drug Highest PhasePhase 3

First Approval Date-

RegulationPRIME (EU), Fast Track (US), Orphan Drug (EU), Breakthrough Therapy (EU)

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Structure

Molecular FormulaC22H24N6O3

InChIKeyNXFPMDWYDKHFMM-UHFFFAOYSA-N

CAS Registry2253123-16-7

主要目的：评价轻度和中度肝损伤成人受试者单次口服pimicotinib胶囊后的pimicotinib及其代谢物药代动力学（PK）。次要目的：在肝损伤受试者和健康受试者中评估pimicotinib单次口服给药的安全性和耐受性;评估肝损对pimicotinib及代谢产物血浆蛋白结合（PPB）的影响。

[Translation]

Primary objective: To evaluate the pharmacokinetics (PK) of pimicotinib and its metabolites after a single oral dose of pimicotinib capsules in adult subjects with mild and moderate hepatic impairment. Secondary objectives: To evaluate the safety and tolerability of a single oral dose of pimicotinib in subjects with hepatic impairment and healthy subjects; to evaluate the effect of hepatic impairment on plasma protein binding (PPB) of pimicotinib and its metabolites.

Start Date18 Sep 2024

Sponsor / Collaborator

Abbisko Therapeutics Co., Ltd.

NCT06322706 / Not yet recruitingPhase 1

A Single-Center, Open-Label, Multi-Cycle Study to Evaluate the Pharmacokinetic Profile of Different Doses of ABSK021 Capsules and the Effect of a High-Fat Meal and Omeprazole Enteric-coated Tablets on the Pharmacokinetic Profile of ABSK021 Capsules in Healthy Subjects

Part A: a single-center, randomized, open-label, three-cycle study to evaluate the pharmacokinetic (PK) profile of different doses of ABSK021 Capsules and the effect of a high-fat meal on the pharmacokinetic profile of ABSK021 Capsules in healthy subjects Part B: a single-center, open-label, fixed-sequence study to evaluate the effect of multiple oral doses of Omeprazole Enteric-coated Tablets on the PK profile of ABSK021 Capsules in healthy subjects

Start Date28 Mar 2024

Sponsor / Collaborator

Abbisko Therapeutics Co., Ltd.

100 Clinical Results associated with Pimicotinib

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100 Translational Medicine associated with Pimicotinib

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100 Patents (Medical) associated with Pimicotinib

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1

Literatures (Medical) associated with Pimicotinib

Future Oncology

MANEUVER: A Phase III study of pimicotinib to assess efficacy and safety in tenosynovial giant cell tumor patients

Article

Author: Shi, Haosong ; Gelderblom, Hans ; Zou, Qingping ; Niu, Xiaohui ; Guo, Qiuxiang ; Shan, Boyao ; Ravi, Vinod

Tenosynovial giant cell tumor (TGCT) is a rare, locally invasive soft tissue tumor arising from the synovium of joints, bursa and tendon sheaths and is associated with the overexpression of the colony-stimulating factor 1 (CSF-1) gene. Pimicotinib is an orally available, highly selective and potent small molecule CSF-1 receptor (CSF-1R) inhibitor with robust efficacy and safety profile in patients with TGCT and is under development in multiple diseases. In an open-label Phase I study in patients with TGCT not amenable to surgery, pimicotinib showed superior efficacy and safety. In this article, we elucidate the rationale and study design of the multi-region Phase III MANEUVER trial (NCT05804045), which is designed to assess the efficacy and safety of pimicotinib in patients with TGCT not amenable to surgical resection in Asia, North America and Europe.

25

News (Medical) associated with Pimicotinib

14 Nov 2024

·pmlive.com

Merck KGaA/Abbisko share positive phase 3 results for pimicotinib in rare tumour type

Merck KGaA and Abbisko Therapeutics have announced positive results from a late-stage study of pimicotinib in patients with tenosynovial giant cell tumours (TGCTs). The phase 3 MANEUVER trial has been evaluating the candidate in TGCT patients who are eligible for systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The trial met its primary endpoint, with an objective response rate at week 25 of 54% for pimicotinib versus 3.2% for placebo. Pimicotinib also provided statistically significant and clinically meaningful improvements in secondary endpoints associated with patient outcomes in TGCT, including stiffness and pain. The companies added that the drug was found to be well tolerated, and its safety profile was consistent with previously reported data. TGCT is a rare, benign and locally aggressive disease that originates in the synovial lining of joints, bursae and tendon sheaths. Patients can experience joint pain, stiffness, swelling and a reduced range of motion, and the disease can cause irreversible damage to bones, joints and surrounding tissues if left untreated or in recurrent cases. Pimicotinib, being developed by Abbisko, is an orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. The candidate has already been granted breakthrough therapy designation in the US and China, and priority medicine designation in the EU for TGCT. Merck and Abbisko entered into an agreement in December last year, giving Merck an exclusive licence to commercialise pimicotinib in mainland China, Hong Kong, Macau and Taiwan, with an option for the rest of the world. Danny Bar-Zohar, global head of research and development and chief medical officer for the healthcare business sector of Merck, said: “This phase 3 data from MANEUVER confirms results of Abbisko’s phase 1 study, indicating that targeting CSF-1R with pimicotinib has the potential to offer a new treatment option for patients. “As we work with Abbisko to review the data from this study and prepare to share it with regulators in China, we are focused on our shared goal of bringing pimicotinib to patients in need.”

Clinical ResultPhase 3Breakthrough TherapyLicense out/inPhase 1

12 Nov 2024

·endpts.com

Merck KGaA plans China submission after late-stage win for tumor drug

Merck KGaA’s experimental treatment for patients with tenosynovial giant cell tumors hit the primary endpoint in a Phase 3 study, and the company plans to submit the drug for approval in China with its Shanghai-based partner Abbisko. In the MANEUVER trial, patients on pimicotinib saw a 54% objective response rate at week 25 compared with 3.2% for placebo (p<0.0001), the company announced Tuesday . Merck KGaA also reported statistically significant and clinically meaningful improvements in secondary endpoints, including alleviation of stiffness and pain. It expects to share more data at an upcoming medical meeting. In a news release, Niu Xiaohui, one of the trial’s investigators and the director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, described tenosynovial giant cell tumors as rare and benign and a “disease of the young.” He also said that because they grow in and around joints, they cause swelling, pain, stiffness and mobility limitations. “This investigational medicine has the potential to establish a new treatment paradigm for patients with TGCT,” Xiaohui said. According to the company, the drug was well-tolerated, and only one patient discontinued the therapy, due to treatment-emergent adverse events. Five patients on the drug ended up taking lower doses of pimicotinib after adverse events. Danny Bar-Zohar, global head of R&D and CMO for the healthcare business sector of Merck KGaA, said in the release that the company is working with Abbisko to review the data and is preparing to share it with Chinese regulators. Abbisko and Merck KGaA inked a deal in December 2023 . The German drugmaker is now set to commercialize pimicotinib in the China mainland, Hong Kong, Macau and Taiwan, with an option for the rest of the world, including the US. Merck KGaA paid $70 million upfront, with the potential for up to $605.5 million in milestones, plus double-digit percentage royalties on any future sales. The drug is considered key to Merck KGaA’s growth strategy.

Phase 3License out/inClinical ResultADC

12 Nov 2024

·firstwordpharma.com

Abbisko, Merck KGaA drug hits main goal of pivotal study in rare tumour type

Abbisko Therapeutics' experimental drug pimicotinib met the primary endpoint of a Phase III study for the treatment of tenosynovial giant cell tumour (TGCT), as well as hitting all key secondary goals. Merck KGaA holds a marketing licence to the oral CSF-1R inhibitor in China and certain Asian countries under a deal signed late last year, with an option on global commercialisation rights.The MANEUVER trial's first double-blind part randomised 94 patients with TGCT who are eligible for systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy to receive pimicotinib or placebo once daily for 24 weeks. The study's main goal was objective response rate (ORR), which Abbisko said at week 25 was 54% for pimicotinib, compared with 3.2% for placebo. The Chinese pharma added that the drug also provided statistically significant and clinically meaningful improvements in secondary endpoints associated with patient outcomes in TGCT, including stiffness by Numeric Rating Scale and pain by Brief Pain Inventory.Abbisko noted that pimicotinib was well tolerated in the trial, with treatment-emergent adverse events (TEAEs) leading to treatment discontinuation occurring in 1.6% of those on the drug, and TEAEs leading to dose reduction at a rate of 7.9%.Advancing classYaochang Xu, chief executive of Abbisko, said "we look forward to collaborating with Merck as we pursue registration of pimicotinib as the first therapy option indicated for the systemic treatment of TGCT in China." Xu added that the drug represents "a key advancement within the emerging class of CSF-1R inhibitors."In 2019, Daiichi Sankyo's CSF1 inhibitor Turalio (pexidartinib) gained approval in the US for the treatment of adults with symptomatic TGCT. Clearance was based on Phase III data showing that 38% of those treated with the drug achieved an ORR, compared to none in the placebo group. Meanwhile, Ono Pharmaceutical's Deciphera Pharmaceuticals unit has filed for approval of its CSF1R inhibitor vimseltinib in TGCT, with a decision expected from US regulators by February 17 next year.

Phase 3Clinical ResultDrug Approval

100 Deals associated with Pimicotinib

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pigmented Villonodular Synovitis	Phase 3	CA	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	NL	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	US	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	CN	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	ES	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	PL	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pigmented Villonodular Synovitis	Phase 3	IT	Abbisko Therapeutics Co., Ltd.	27 Apr 2023
Pancreatic Cancer	Phase 2	CN	Abbisko Therapeutics Co., Ltd.	17 Oct 2023
Chronic graft-versus-host disease	Phase 2	CN	Abbisko Therapeutics Co., Ltd.	30 May 2023
Triple Negative Breast Cancer	Phase 1	US	Abbisko Therapeutics Co., Ltd.	02 Jan 2020

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05804045 (MANEUVER, Company_Website) Manual	Phase 3	Giant Cell Tumor of Tendon Sheath	94	匹米替尼	(jnkdbenpuz) = dpptlifzuv nrkxouxjqc (vsnszefoso ) Met View more	Positive	12 Nov 2024
				安慰剂	(jnkdbenpuz) = zsolvgrxky nrkxouxjqc (vsnszefoso ) Met View more
NCT04192344 (Corporate Publications) Manual	Phase 1	Giant Cell Tumor of Tendon Sheath	56	Pimicotinib 50mgQD	(wfutpcrdut) = ebbuxsfsot sdifgpwgsz (wrvmmajvyw ) View more	Positive	03 Nov 2023
				Pimicotinib 25mgQD	(wfutpcrdut) = wxyuhdufng sdifgpwgsz (wrvmmajvyw ) View more
NCT04192344 (ASCO2023) Manual	Phase 1	Giant Cell Tumor of Tendon Sheath	49	Pimicotinib 50 mg	(qsquamrwsu) = include LDH increase (75.5%), CPK increase (67.3%), α-HBDH increase (63.3%), AST increase (42.9%), amylase increase (26.5%), ALT increase (24.5%), pruritus (20.4%) and rash (20.4%) aoctrogtow (pagjkfyqsg ) View more	Positive	26 May 2023
				Pimicotinib 25 mg
NCT04192344 (NEWS) Manual	Phase 1	Giant Cell Tumor of Tendon Sheath	27	ABSK021	(enaauxscfd) = The majority of treatment-period adverse events (treatment-period adverse events) were grade 1 or grade 2 events. No cases of color change or severe liver injury were reported. byuqpqxzjv (czpesprnby )	Positive	15 Nov 2022