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

Rationale for FYARROTM use in patients with Tumor-agnostic 
TSC1 and TSC2 Inactivating Alterations

  • Evidence of responses in PEComa patients with TSC1 and TSC2 alterations in completed AMPECT registrational trial*
  • Data generated in non-PEComa patients with TSC1 or TSC2 alterations in ongoing Expanded Access Program (ASCO 2021 Abstract # 339405)*
  • FYARROTM demonstrates greater mTOR pathway inhibition vs. currently approved mTOR inhibitors in animal models
  • Strong precedent for tumor agnostic approvals in mutation driven indications established with FDA approvals of Viktravi, Rozlytrek, and Keytruda
* data not reviewed by the FDA


Aadi plans to initiate a registrational trial for FYARROTM in Tumor-Agnostic TSC1 & TSC2 Alterations