a pilot study of plx3397, a selective colony-stimulating factor 1 receptor (csf1r) kinase inhibitor,...

<br /> A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS)

Presented By William Tap at 2014 ASCO Annual Meeting

Pigmented Villonodular Synovitis (PVNS)<br />Tenosynovial Giant Cell Tumor (TGCT)

Presented By William Tap at 2014 ASCO Annual Meeting

Pigmented Villonodular Synovitis (PVNS)

Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

Can we help patients (PVNS) <br />with a highly targeted therapy (PLX3397)<br />that blocks the CSF1R pathway <br />in this clonal neoplastic process frequently<br />initiated by a single genetic event?

Presented By William Tap at 2014 ASCO Annual Meeting

Study Goals

Presented By William Tap at 2014 ASCO Annual Meeting

Study Design

Presented By William Tap at 2014 ASCO Annual Meeting

Efficacy Assessments

Presented By William Tap at 2014 ASCO Annual Meeting

Tumor Volume Score (TVS)

Presented By William Tap at 2014 ASCO Annual Meeting

Patient Disposition

Presented By William Tap at 2014 ASCO Annual Meeting

Patient Characteristics: Safety Population (n=23)

Presented By William Tap at 2014 ASCO Annual Meeting

Safety: PVNS Expansion Cohort AEs<br />n = 23 Safety Population

Presented By William Tap at 2014 ASCO Annual Meeting

Treatment-Related AEs ≥ Grade 3

Presented By William Tap at 2014 ASCO Annual Meeting

Duration of Treatment (DOT), April 2014 data cutoff:<br />Median 256 days (range 21-585 days)

Presented By William Tap at 2014 ASCO Annual Meeting

14 patients evaluable with TVS

Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

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Presented By William Tap at 2014 ASCO Annual Meeting

Conclusions

Presented By William Tap at 2014 ASCO Annual Meeting

Thank You

Presented By William Tap at 2014 ASCO Annual Meeting