Tumor-Agnostic TSC1 and TSC2 Inactivating Alterations

Inactivating alterations in TSC1 and TSC2 drive mTOR pathway activation
and tumor growth

  • TSC1 and TSC2 form a tumor suppressor complex that down regulates mTOR activity
  • TSC1 and TSC2 alterations occur across a broad range of cancers including bladder cancer, kidney cancer, breast cancer, melanoma, and liver cancer
  • No therapies are approved for TSC1 and TSC2 alterations but numerous case reports show durable responses to mTOR inhibition
  • Standard CLIA-certified NGS panels already capture TSC1 and TSC2 alterations

Proposed rationale for nab-sirolimus use in patients with tumor-agnostic TSC1 and TSC2 inactivating alterations

  • Exploratory analysis of data in AMPECT trial suggest responses to nab-sirolimus may occur in patients with TSC1 or TSC2 alterations
  • Data generated in non-PEComa patients with TSC1 or TSC2 alterations in ongoing Expanded Access Program (ASCO 2021 Abstract # 339405)
  • In preclinical animal models, nab technology demonstrates greater mTOR pathway inhibition vs. currently approved mTOR inhibitors

Aadi has initiated a registrational trial in Tumor-Agnostic TSC1 & TSC2 Alterations
Learn more