Leveraging Innovation To Deliver Precision Medicines

Pipeline

PRMT5

Strong scientific and clinical rationale for novel small molecule PRMT5 inhibitor

Rationale

  • PRMT5 is the predominant Type II PRMT that is responsible for symmetric dimethylation of arginine (SDMA) on histone and non-histone substrates
  • PRMT5 is highly overexpressed in many human cancers
  • Glioblastoma (GBM) with splicing dysregulation is selectively sensitive to inhibition of PRMT5
  • PRMT5 inhibition impairs the removal of detained introns leading to modulation of proliferation specific genes
  • GBM specific key tumor suppressor is silenced by PRMT5 leading to poor prognosis
  • Potential for patient stratification: targeting tumors with high CLNS1A/RIOK1 ratio

Opportunity

  • Brain-penetrant PRMT5 inhibitor to address the unmet medical need in treating GBM
  • Potential use in other cancers where PRMT5 is over expressed (lymphoma, pancreas, SCLC, etc) and in cancers with spliceosome dysregulation

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