A Pilot Study of PLX 3397 a Selective

A Pilot Study of PLX 3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF 1 R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented By William Tap at 2014 ASCO Annual Meeting

Pigmented Villonodular Synovitis (PVNS) 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

Slide 4 Presented By William Tap at 2014 ASCO Annual Meeting

Can we help patients (PVNS) with a highly targeted therapy (PLX 3397) that blocks the CSF 1 R pathway in this clonal neoplastic process frequently 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 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: 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

Slide 16 Presented By William Tap at 2014 ASCO Annual Meeting

Slide 17 Presented By William Tap at 2014 ASCO Annual Meeting

Slide 18 Presented By William Tap at 2014 ASCO Annual Meeting

Slide 19 Presented By William Tap at 2014 ASCO Annual Meeting

Slide 20 Presented By William Tap at 2014 ASCO Annual Meeting

Slide 21 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