Efficacy data from the clinical trials

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ENLIVEN trial

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Pooled analysis

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Graph representing overall response rate (ORR) of TURALIO® (pexidartinib) for TGCT

Duration of response is defined as the time when the patient experiences first response to the time of progression, regardless of whether the patient has discontinued treatment.4

CI, confidence interval; ITT, intent-to-treat; ORR, overall response rate; RECIST, Response Evaluation Criteria In Solid Tumors.

aORR was determined by blinded independent central review (BICR).1

bA median follow-up of 31.2 (range: 2, 66) months (final database lock June 1, 2021).3

cFisher’s exact test.4

dPartial response was defined as 30% or more reduction in tumor length.2

eComplete response was defined as 100% reduction in tumor length.2

fData cutoff January 31, 2018.1

gAt completion of the ENLIVEN study.1

Graph representing overall response rate (ORR) of TURALIO® (pexidartinib) for TGCT

Duration of response is defined as the time when the patient experiences first response to the time of progression, regardless of whether the patient has discontinued treatment.4

CI, confidence interval; ITT, intent-to-treat; ORR, overall response rate; RECIST, Response Evaluation Criteria In Solid Tumors.

aORR was determined by blinded independent central review (BICR).1

bA median follow-up of 31.2 (range: 2, 66) months (final database lock June 1, 2021).3

cFisher’s exact test.4

dPartial response was defined as 30% or more reduction in tumor length.2

eComplete response was defined as 100% reduction in tumor length.2

fData cutoff January 31, 2018.1

gAt completion of the ENLIVEN study.1

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ENLIVEN trial

Secondary Endpoint:
ORR by Tumor Volume Score (TVS)1,3,4,6,a

The majority of TURALIO patients showed a reduction in tumor volume of 50% or more—at 25 weeks and at an ~5.5-year follow-up3,4,6

Graph representing overall response rate (ORR) by Tumor Volume Score (TVS) of TURALIO® (pexidartinib) Graph representing overall response rate (ORR) by Tumor Volume Score (TVS) of TURALIO® (pexidartinib)
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Over a follow-up period of ~5.5 years,
68% of patients achieved at least a
50% reduction in tumor volume3,7

CI, confidence interval; ITT, intent-to-treat; ORR, overall response rate; RECIST, Response Evaluation Criteria In Solid Tumors.

aTVS was defined in ENLIVEN as the estimated volume of the maximally distended synovial cavity or tendon sheath involved, measured in 10% increments.1

bORR was determined by blinded independent central review (BICR).1

cA median follow-up of 31.2 (range: 2, 66) months (final database lock June 1, 2021).3

dFisher’s exact test.4

ePartial response was defined as 50% or more reduction in tumor volume.7

fComplete response was defined as 100% reduction in tumor volume.7

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ENLIVEN trial

Secondary Endpoint:
Change from Baseline in Range of Motion4

2X greater relative difference in ROM at 25 weeks 2X greater relative difference in ROM at 25 weeks
How ROM was defined: Range of motion was defined as mean percentage change from baseline relative to a reference standard for the same joint

TURALIO delivered significant improvement in range of motion (ROM) vs placebo4,a

Graph representing improvement in range of motion (ROM) of TURALIO® (pexidartinib) compared to a placebo Graph representing improvement in range of motion (ROM) of TURALIO® (pexidartinib) compared to a placebo

Mean ROM in patients taking TURALIO at the start of the study was 62.5%; ROM had improved 15.1% from
baseline to about 77.6% of possible movement for the joint at 25 weeks4

Patients taking placebo had a 62.9% mean ROM at the start of the study and experienced an improvement
of 6.2% in ROM to a total of 69.1% at 25 weeks4

Joints represented in the analysis (TURALIO vs placebo, respectively) included the knee (n=25; n=28),
ankle (n=11; n=7), and other (n=9; n=8)8

A 6.7% improvement in ROM for the knee correlates with a 10-degree improvement in joint movement9

aThe total population studied was 120 patients. Results were excluded for 1 patient with missing baseline and 31 patients with a missing ROM assessment at week 25. Assessments were performed by a third-party clinical assessor using a goniometer.1

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Pooled analysis

Pooled long-term analysis of 130 patients on TURALIO from 3 cohorts7

TGCT patient cohort from the phase 1 study of pexidartinib in solid tumors (n=39)a

Phase 3 ENLIVEN patients randomized to TURALIO (n=61)

Phase 3 ENLIVEN crossover patients (n=30)

LIMITATION: This post hoc analysis lacked a control group for comparison; therefore, no conclusions about TURALIO can be drawn from this data. Additionally, this analysis did not evaluate time to disease progression in those patients who stopped TURALIO.

Graph representing overall response rate (ORR) by RECIST (response evaluation criteria in solid tumors) of TURALIO® (pexidartinib) Graph representing overall response rate (ORR) by RECIST (response evaluation criteria in solid tumors) of TURALIO® (pexidartinib)

ORR defined as CR+PR per RECIST v1.1 in the ITT population.

aThe pooled population had a median duration of treatment of 19 months, with treatment on-going in 54 patients (42%) as of the May 31, 2019 data cutoff.7

CR, complete response; ORR, overall response rate; PR, partial response; RECIST, Response Evaluation Criteria In Solid Tumors.

Additional long-term, pooled, follow-up analysis from 3 TURALIO-treated TGCT patient cohorts—May 31, 2019 data cutoff7

ORR=60% (n=78/130, 95% CI: 51.4%, 68.0%) defined by RECIST as CR or PR with a median follow-up of 39 months (range: 32-82 months)

The median time to an initial response was 3.4 months as assessed by RECIST v1.1

According to RECIST, of the 130 total patients, 16 (12%) progressed on treatment or after treatment discontinuation, 14 (11%) progressed on treatment, and 2 (2%) progressed after treatment

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Checkmark iconTURALIO is the first FDA-approved targeted therapy for adults with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery1,5

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References: 1. TURALIO [package insert]. Basking Ridge, NJ: Daiichi Sankyo, Inc; 2025. 2. Eisenhauer EA et al. Eur J Cancer. 2009;45(2):228-247. 3. Wagner AJ. Slides presented at: CTOS Connective Tissue Oncology Society; November 16-19, 2022; Vancouver, BC, Canada. 4. Tap WD et al; ENLIVEN investigators. Lancet. 2019;394(10197):478-487. 5. FDA approves pexidartinib for tenosynovial giant cell tumor. U.S. Food & Drug Administration; August 8, 2019. Accessed January 3, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pexidartinib-tenosynovial-giant-cell-tumor 6. Tap WD et al. N Engl J Med. 2015;373(5)(suppl):1-39. 7. Gelderblom H et al. Cancer. 2021;127(6):884-893. 8. Data on file. Daiichi Sankyo. 9. Data on File. Daiichi Sankyo.