Efficacy data from the clinical trials

Efficacy Efficacy

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ENLIVEN trial

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Pooled analysis

ENLIVEN trial

Primary Endpoint: ORR by RECIST v1.11-3

100%of patients experienced at least some reduction in the length of their tumor4

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

bFisher’s exact test.1

cAn exploratory analysis measured the best overall response achieved with TURALIO from the start of treatment until a median of 38 months (data cutoff of May 31, 2019). The analysis of this endpoint was not included in the prespecified statistical analysis plan to control for type 1 error; as such, it may be susceptible to bias.3

next: ORR TVS

ENLIVEN trial

Secondary Endpoint:
ORR by Tumor Volume Score (TVS)1,3,7

Most TURALIO patients showed reduction in tumor volume of 50% or more—at 25 weeks and over a 3-year follow-up

With a longer follow-up of 38 months,
66% of patients had at least
50% reduction in tumor volume3,7

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

bFisher’s exact test.5

cAn exploratory analysis measured the best overall response achieved with TURALIO from the start of treatment until a median of 38 months (data cutoff of May 31, 2019). The analysis of this endpoint was not included in the prespecified statistical analysis plan to control for type 1 error; as such, it may be susceptible to bias.3

next: DOR

ENLIVEN trial

Secondary Endpoint: DOR

Most responders experienced lasting tumor reduction with a median follow-up of 38 months

Durable response that lasts in ENLIVEN

Median duration of response was still not
reached at a median follow-up of 38 months
(response range: 0.0+ to 41.4+ months)3,a
No new safety signals were reported Signs of serious liver injury occurred in 5% of patients (3 of 61 patients)
receiving TURALIO. All cases occurred within the first 2 months of treatment1,5

Duration of response is defined as the time when the patient experiences first response to the time of first progression, regardless of whether the patient has discontinued treatment5

More than half of responders had an ongoing response. Median duration of response was not reached with a median follow-up of 22 months (response range: 6.9+ to 24.9+ months)1,5,b

aAs of May 31, 2019 assessment.3
bData cutoff January 31, 2018.1

next: ROM

ENLIVEN trial

Secondary Endpoint:
Change in Range of Motion5

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 placebo5,a

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 weeks5

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 weeks5

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)3

A 6.7% improvement in ROM for the knee correlates with a 10-degree improvement in joint movement8

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

next: LONG-TERM DATA

Pooled analysis

In the pooled analysis, 60% of patients achieved ORR; of these, most achieved results by 6 months on TURALIO

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

aThe phase 1 study was the first in-human study with a dose-escalation phase with an expansion cohort phase (39 patients with TGCT) conducted in patients with solid tumors. Pexidartinib at 1000 mg/d (split in twice-daily dosing) was taken until tumor progression or unacceptable toxicity.9

bThe pooled population had a median duration of treatment of 19 months, with treatment ongoing in 54 patients (42%) as of the May 31, 2019 data cutoff.9

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 cutoff9

Pooled long-term analysis of 130 patients on TURALIO from 3 cohorts9

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)

Post-hoc pooled analysis showed a durable long-term response with TURALIO9

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

TURALIO data from the pooled post-hoc analysis demonstrated that the safety profile is consistent
with data from the ENLIVEN study with no late-emerging toxicities9
See the full safety data from the pooled analysis

back: ROM

TURALIO is the only FDA-approved targeted therapy for TGCT1

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References: 1. TURALIO [package insert]. Basking Ridge, NJ: Daiichi Sankyo, Inc; 2021. 2. Eisenhauer EA et al. Eur J Cancer. 2009;45(2): 228-247. 3. Data on file. Daiichi Sankyo. 4. Healey JH et al. Slides presented at: Musculoskeletal Tumor Society Annual Meeting; October 2-4, 2019; Portland, Oregon. Presentation 46. 5. Tap WD et al; ENLIVEN investigators. Lancet. 2019;394(10197): 478-487. 6. West RB et al. Proc Natl Acad Sci U S A. 2006;103(3): 690-695. 7. Tap WD et al. N Engl J Med. 2015;373(5)(suppl):1-39. 8. Oncologic Drugs Advisory Committee. NDA 211810. Pexidartinib. U.S. Food & Drug Administration; May 14, 2019. Accessed October 19, 2021. https://www.fda.gov/media/124892/download. 9. Gelderblom H et al. Cancer. 2021;127(6):884-893.