Novel Treatment Given to First Patient in Trial Assessing the Drug’s Safety and Efficacy in Solid Tumors With Certain Genetic Alterations

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Researchers have treated the first patient in the PRECISION 1 trial with Fyarro to assess the drug’s efficacy across several endpoints including survival among more than 100 patients with solid tumors that exhibit certain genetic alterations.

Aadi Bioscience announced that researchers treated the first patient with Fyarro (nab-sirolimus) in the phase 2 PRECISION 1 trial designed to assess the mTOR inhibitor to treat patients with solid tumors with pathogenic inactivating alterations in TSC1 and TSC2 genes.

According to a company press release, the Food and Drug Administration (FDA) granted Aadi Bioscience fast track designation to assess Fyarro in patients aged 12 years and older with solid tumors exhibiting these genetic alterations. Fast track designation allows a company to simplify the development of and accelerate the review of a specific drug to treat serious conditions and potentially address an unmet need.

Researchers on the PRECISION 1 trial will assess the effects of Fyarro in approximately 120 patients with solid tumors that harbor alterations in either TSC1 or TSC2 genes. Of note, the trial will consist of two different groups of 60 patients each, allowing researchers to review the drug in each specific genetic alteration, according to the release.

“We are excited to have initiated dosing in our PRECISION 1 clinical trial,” said Dr. Loretta Itri, chief medical officer of Aadi Bioscience, in the release. “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 (Fyarro) to treat patients with tumors driven by those alterations. … In the PRECISION 1 trial, the two (patient groups) will be independently evaluable in order to separately assess therapeutic activity of (Fyarro) in tumors harboring either TSC1 or TSC2 inactivating alterations.”

There are several warnings and precautions related to treatment with Fyarro including myelosuppression (i.e., anemia, neutropenia and thrombocytopenia), stomatitis (i.e., mouth ulcers and tissue swelling in the mouth), hypokalemia (low potassium levels), infections, interstitial lung disease/noninfectious pneumonitis, high blood sugar, hypersensitivity reactions, bleeding, male infertility and embryo-fetal toxicity.

Fyarro, which was previously approved by the FDA for patients with perivascular epithelioid cell tumors, has been linked with side effects including stomatitis, infection, fatigue, rash, swelling of the legs and feet, nausea, decreased weight, diarrhea, musculoskeletal pain, cough, decreased appetite, altered taste and vomiting, according to the release.

“Following the approval and commercial launch of Fyarro (nab-sirolimus) in advanced malignant (perivascular epithelioid cell tumors), and initial demonstration of single-agent activity outside of (perivascular epithelioid cell tumors) in patients with inactivating alterations in TSC1 or TSC2, we are now pursuing a tumor-agnostic indication for (Fyarro) in patients with inactivating alterations in TSC1 or TSC2 following feedback on the study design from the U.S. Food and Drug Administration,” said Neil Desai, founder, chief executive officer and president of Aadi Bioscience, in the release. “These alterations are estimated to occur in over 10,000 advanced cancer patients per year, representing one of the larger targeted oncology opportunities.”

Preliminary data from this trial are expected in the first half of 2023, according to the release.

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