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

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<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

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Page 1: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

<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

Page 2: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

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

Presented By William Tap at 2014 ASCO Annual Meeting

Page 3: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Pigmented Villonodular Synovitis (PVNS)

Presented By William Tap at 2014 ASCO Annual Meeting

Page 4: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 4

Presented By William Tap at 2014 ASCO Annual Meeting

Page 5: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

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

Page 6: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Study Goals

Presented By William Tap at 2014 ASCO Annual Meeting

Page 7: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Study Design

Presented By William Tap at 2014 ASCO Annual Meeting

Page 8: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Efficacy Assessments

Presented By William Tap at 2014 ASCO Annual Meeting

Page 9: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Tumor Volume Score (TVS)

Presented By William Tap at 2014 ASCO Annual Meeting

Page 10: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Patient Disposition

Presented By William Tap at 2014 ASCO Annual Meeting

Page 11: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Patient Characteristics: Safety Population (n=23)

Presented By William Tap at 2014 ASCO Annual Meeting

Page 12: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

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

Presented By William Tap at 2014 ASCO Annual Meeting

Page 13: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Treatment-Related AEs ≥ Grade 3

Presented By William Tap at 2014 ASCO Annual Meeting

Page 14: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

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

Page 15: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

14 patients evaluable with TVS

Presented By William Tap at 2014 ASCO Annual Meeting

Page 16: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 16

Presented By William Tap at 2014 ASCO Annual Meeting

Page 17: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 17

Presented By William Tap at 2014 ASCO Annual Meeting

Page 18: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 18

Presented By William Tap at 2014 ASCO Annual Meeting

Page 19: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 19

Presented By William Tap at 2014 ASCO Annual Meeting

Page 20: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 20

Presented By William Tap at 2014 ASCO Annual Meeting

Page 21: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Slide 21

Presented By William Tap at 2014 ASCO Annual Meeting

Page 22: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Conclusions

Presented By William Tap at 2014 ASCO Annual Meeting

Page 23: A Pilot Study of PLX3397, a Selective Colony-Stimulating Factor 1 Receptor (CSF1R) Kinase Inhibitor, in Pigmented Villonodular Synovitis (PVNS) Presented

Thank You

Presented By William Tap at 2014 ASCO Annual Meeting