Aadi Bioscience Publishes Long-Term Data Supporting FYARRO® for Malignant PEComa

Aadi Bioscience, Inc has announced significant results from the AMPECT trial, detailing the long-term efficacy and safety of nab-sirolimus in treating malignant PEComa. Published in the Journal of Clinical Oncology, the study, titled "A Phase 2 Trial of nab-Sirolimus in Patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-term Efficacy and Safety Update," demonstrated promising outcomes with nab-sirolimus over a 3-year follow-up period.

The trial found that patients treated with nab-sirolimus showed a median response duration of 39.7 months and a median survival rate surpassing 53 months. Historically, median survival rates for metastatic/unresectable malignant PEComa range from 16 to 29 months, making these results noteworthy. The confirmed overall response rate (ORR) remained at 38.7%, including several complete responses, and the disease control rate (DCR) was 71%.

Dr. Andrew J. Wagner, who led the study, reported that the observed responses to nab-sirolimus were durable and maintained with acceptable safety profiles. Nab-sirolimus was generally well-tolerated, with common treatment-related adverse events (TRAEs) including stomatitis (82.4%), fatigue, and rash (each at 61.8%). No new or unexpected adverse events were reported, and no grade 4 or 5 TRAEs occurred.

Loretta Itri, Chief Medical Officer at Aadi, emphasized the potential of nab-sirolimus as a differentiated treatment for aggressive cancers, highlighting its ability to extend response duration and improve survival rates. She expressed gratitude to the trial's participants and investigators, noting the significance of their contributions to sarcoma research.

The AMPECT trial (NCT02494570) was the first prospective registrational study focusing on the efficacy and safety of nab-sirolimus in advanced malignant PEComa. This Phase 2, open-label, single-arm, multi-center study served as the basis for the FDA approval of FYARRO® as a treatment for advanced malignant PEComa, irrespective of mutational status.

Aadi Bioscience, Inc is a biopharmaceutical company that specializes in developing precision therapies targeting mTOR-driven cancers. The company has successfully commercialized FYARRO® for treating advanced malignant PEComa and is expanding its clinical trials to investigate nab-sirolimus alone or in combination with other treatments for different mTOR-driven cancers. Current trials include the PRECISION 1 study (NCT05103358) and studies involving neuroendocrine tumors and endometrioid endometrial cancer.

Overall, these findings from the AMPECT trial support the effectiveness of nab-sirolimus in treating malignant PEComa, offering hope for prolonged survival and improved therapeutic outcomes for patients with this rare and aggressive cancer.