On March 31, 2022 Aadi Bioscience, Inc. (Nasdaq: AADI), a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, reported the commencement of dosing in PRECISION 1, a Phase 2 registrational trial of nab-sirolimus in patients 12 years and older with solid tumors with pathogenic inactivating alterations in TSC1 and TSC2 genes (Press release, Aadi Bioscience, MAR 31, 2022, View Source [SID1234611300]).
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The FDA granted Aadi Fast Track designation to evaluate nab-sirolimus for this patient population. Fast Track designation is an FDA process designed to facilitate the development, and expedite the review of, medicines to treat serious conditions and fill unmet medical need.
Neil Desai, Ph.D., Founder, Chief Executive Officer, and President of Aadi, stated "Following the approval and commercial launch of FYARRO (also known as nab-sirolimus) in advanced malignant PEComa, and initial demonstration of single agent activity outside of PEComa in patients with inactivating alterations in TSC1 or TSC2, we are now pursuing a tumor-agnostic indication for nab-sirolimus in patients with inactivating alterations in TSC1 or TSC2 following feedback on the study design from the U.S. Food and Drug Administration (FDA). These alterations are estimated to occur in over 10,000 advanced cancer patients per year, representing one of the larger targeted oncology opportunities."
Dr. Desai continued, "We believe the initiation of this registrational trial highlights our team’s ability to execute on drug development milestones and demonstrates meaningful progress towards expanding our nab-sirolimus program and establishing Aadi as a multi-indication, precision oncology company."
The PRECISION 1 trial is a multi-center, open-label, tumor-agnostic registrational clinical trial of nab-sirolimus. Designed as a "basket trial", the study will evaluate approximately 60 mTOR inhibitor naïve patients in each of two independent study arms (i.e., approximately 120 patients in total) comprised of patients with solid tumors harboring pathogenic inactivating alterations in either TSC1 or TSC2 genes. Preliminary data from PRECISION 1 are expected in the first half of 2023.
"We are excited to have initiated dosing in our PRECISION 1 clinical trial," stated Loretta Itri, M.D., Chief Medical Officer of Aadi. "There are currently no approved treatment options for patients with TSC1 or TSC2 alterations, and this trial is designed to evaluate the efficacy, safety and tolerability of nab-sirolimus to treat patients with tumors driven by those alterations. We believe that the early data we presented at ASCO (Free ASCO Whitepaper) 2021 from our Expanded Access Program for FYARROsuggested activity of nab-sirolimus in this patient population. In the PRECISION 1 trial, the two arms will be independently evaluable in order to separately assess therapeutic activity of nab-sirolimus in tumors harboring either TSC1 or TSC2 inactivating alterations."