Pexidartinib

Generic Name: Pexidartinib

Brand Names: Turalio

Drug Type: Small Molecule

Chemical Formula: C_₂₀H_₁₅ClF_₃N_₅

CAS Number: 1029044-16-3

Unique Ingredient Identifier: 6783M2LV5X

Background: Pexidartinib is a selective tyrosine kinase inhibitor that works by inhibiting the colony-stimulating factor (CSF1)/CSF1 receptor pathway. Pexidartinib was originally developed by Daiichi Sankyo, Inc. and it was approved by the FDA in August 2019 as the first systemic therapy for adult patients with symptomatic tenosynovial giant cell tumor. Tenosynovial giant cell tumor is a rare form of non-malignant tumor that causes the synovium and tendon sheaths to thicken and overgrow, leading to damage in surrounding joint tissue. Debilitating symptoms often follow with tenosynovial giant cell tumors, along with a risk of significant functional limitations and a reduced quality of life in patients.

While surgical resection is a current standard of care for tenosynovial giant cell tumor, there are tumor types where surgeries are deemed clinically ineffective with a high risk of lifetime recurrence. Pexidartinib works by blocking the immune responses that are activated in tenosynovial giant cell tumors. In clinical trials, pexidartinib was shown to promote improvements in patient symptoms and functional outcomes in TGCT. Pexidartinib is available in oral formulations and it is commonly marketed as Turalio.

Indication: Pexidartinib is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.

Associated Conditions: Symptomatic Tenosynovial Giant Cell Tumor

Clinical Trials

PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors

Phase 1

Terminated

Conditions: Malignant Peripheral Nerve Sheath Tumors
Sarcoma

Interventions: Drug: PLX3397
Drug: sirolimus

First Posted Date: 2015-10-22

Last Posted Date: 2025-03-21

Lead Sponsor: Gulam Manji

Target Recruit Count: 39

Registration Number: NCT02584647

Locations: 🇺🇸
University of Iowa, Iowa City, Iowa, United States
🇺🇸
University of Michigan, Ann Arbor, Michigan, United States
🇺🇸
Early Drug Development Center, Boston, Massachusetts, United States

and more 2 locations

A Combination Clinical Study of PLX3397 and Pembrolizumab To Treat Advanced Melanoma and Other Solid Tumors

Phase 1

Terminated

Conditions: Gastrointestinal Stromal Tumor (GIST)
Ovarian Cancer
Squamous Cell Carcinoma of the Head and Neck
Melanoma
Non-small Cell Lung Cancer

Interventions: Drug: PLX3397
Biological: Pembrolizumab

First Posted Date: 2015-05-22

Last Posted Date: 2020-03-05

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 78

Registration Number: NCT02452424

Locations: 🇺🇸
HonorHealth Research Institute, Scottsdale, Arizona, United States
🇺🇸
Marin Cancer Care, Greenbrae, California, United States
🇺🇸
Ronald Reagan UCLA Medical Center, Los Angeles, California, United States

and more 9 locations

CGT9486 (Formerly Known as PLX9486) as a Single Agent and in Combination With PLX3397 (Pexidartinib) or Sunitinib in Participants With Advanced Solid Tumors

Phase 1

Completed

Conditions: Gastrointestinal Stromal Tumors

Interventions: Drug: CGT9486
Drug: Pexidartinib
Drug: Sunitinib

First Posted Date: 2015-03-30

Last Posted Date: 2025-02-14

Lead Sponsor: Cogent Biosciences, Inc.

Target Recruit Count: 51

Registration Number: NCT02401815

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States
🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States
🇺🇸
OSU Comprehensive Cancer Center, Columbus, Ohio, United States

and more 3 locations

Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

Phase 3

Completed

Conditions: Pigmented Villonodular Synovitis
Giant Cell Tumors of the Tendon Sheath
Tenosynovial Giant Cell Tumor

Interventions: Drug: Pexidartinib
Drug: Placebo

First Posted Date: 2015-02-25

Last Posted Date: 2022-05-11

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 120

Registration Number: NCT02371369

Locations: 🇺🇸
University of Southern California, Los Angeles, California, United States
🇺🇸
Mayo Clinic, Scottsdale, Arizona, United States
🇫🇷
Institut Gustave Roussy, Villejuif, France

and more 35 locations

PLX3397 KIT in Acral aNd mucOsal Melanoma

Phase 2

Completed

Conditions: Malignant Melanoma

Interventions: Drug: PLX3397

First Posted Date: 2014-02-26

Last Posted Date: 2021-08-12

Lead Sponsor: The Christie NHS Foundation Trust

Target Recruit Count: 7

Registration Number: NCT02071940

Locations: 🇬🇧
The Beatson West of Scotland Cancer Centre, Glasgow, Scotland, United Kingdom
🇬🇧
Abertawe Bro Morgannwg University Health Board, Singleton Hospital, Swansea, Wales, United Kingdom
🇬🇧
The Christie NHS Foundation Trust, Manchester, Greater Manchester, United Kingdom

and more 5 locations

A Phase 1b Open Label, Dose Escalation Study of PLX3397 in Combination With Vemurafenib in V600-mutated BRAF Melanoma

Phase 1

Terminated

Conditions: V600-mutated BRAF Unresectable Melanoma
V600-mutated BRAF Metastatic Melanoma
Stage III or Stage IV Metastatic Melanoma That Has Not Been Previously Treated With a Selective BRAF Inhibitor

Interventions: Drug: PLX3397
Drug: vemurafenib

First Posted Date: 2013-04-08

Last Posted Date: 2020-05-28

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 13

Registration Number: NCT01826448

Locations: 🇺🇸
University of Colorado, Denver, Aurora, Colorado, United States
🇺🇸
Vanderbilt University, Nashville, Tennessee, United States
🇺🇸
Seattle Cancer Care Alliance, Seattle, Washington, United States

and more 3 locations

A Phase 1b/2 Study of PLX3397 + Radiation Therapy + Temozolomide in Patients With Newly Diagnosed Glioblastoma

Phase 1

Completed

Conditions: Patients With Newly Diagnosed Glioblastoma

Interventions: Drug: PLX3397
Radiation: Radiation Therapy
Drug: Temozolomide

First Posted Date: 2013-02-13

Last Posted Date: 2020-06-30

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 65

Registration Number: NCT01790503

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸
James Cancer Hospital/Ohio State University, Columbia, Ohio, United States
🇺🇸
Seattle Cancer Care Alliance, Seattle, Washington, United States

and more 6 locations

Phase Ib/II Study of PLX 3397 and Eribulin in Patients With Metastatic Breast Cancer

Phase 1

Completed

Conditions: Metastatic Breast Cancer

Interventions: Drug: PLX3397
Drug: Eribulin

First Posted Date: 2012-05-11

Last Posted Date: 2020-07-17

Lead Sponsor: Hope Rugo, MD

Target Recruit Count: 67

Registration Number: NCT01596751

Locations: 🇺🇸
University of California, San Francisco, San Francisco, California, United States
🇺🇸
Duke University Cancer Center, Durham, North Carolina, United States
🇺🇸
Vanderbilt-Ingram Cancer Center, Nashville, Tennessee, United States

Safety Study of PLX3397 and Paclitaxel in Patients With Advanced Solid Tumors

Phase 1

Completed

Conditions: Solid Tumors

Interventions: Drug: PLX3397
Drug: Paclitaxel

First Posted Date: 2012-02-03

Last Posted Date: 2020-07-16

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 74

Registration Number: NCT01525602

Locations: 🇺🇸
Ohio State University, Columbus, Ohio, United States
🇺🇸
Univeristy of California, San Francisco, San Francisco, California, United States
🇺🇸
Sylvester Comprehensive Cancer Center/UMHC, Miami, Florida, United States

and more 5 locations

Pilot Study of PLX3397 in Patients With Advanced Castration-Resistant Prostate Cancer (CRPC)

Phase 2

Terminated

Conditions: Prostate Cancer

Interventions: Drug: PLX3397

First Posted Date: 2011-12-26

Last Posted Date: 2020-03-04

Lead Sponsor: Daiichi Sankyo

Target Recruit Count: 6

Registration Number: NCT01499043

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸
Memorial Sloan-Kettering Cancer Center, New York, New York, United States

Drug Development Updates

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