On October 22, 2021 Aadi Bioscience, Inc. ("Aadi") (Nasdaq: AADI), a clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, reported the publication of "nab-Sirolimus for Patients with Malignant Perivascular Epithelioid Cell Tumors", detailing its AMPECT study of investigational ABI-009 in the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Journal of Clinical Oncology (Press release, Aadi Bioscience, OCT 22, 2021, View Source [SID1234592017]). The authors concluded that investigational nab-sirolimus (ABI-009, formerly known as nab-rapamycin), if approved, may represent an important new treatment option in malignant PEComa, a rare cancer and aggressive form of sarcoma, with no currently approved treatment.
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The content of the publication included the primary analysis, which occurred 6 months after the last patient on the AMPECT study initiated treatment, as well as a 1.5-year follow-up after the primary analysis with a data cutoff of November 23, 2020. In the trial, 34 patients were treated with ABI-009. In the 31 evaluable patients, the overall independently assessed response rate was 39% (12 of 31; with a 95% confidence interval, 22% to 58%) with 1 complete and 11 partial responses. In addition, 52% (16 of 31) of patients had stable disease. The responses were durable and median duration of response was not reached after a median follow-up for response of 2.5 years, and 7 of 12 responders with treatment ongoing (range 5.6-47.2+ months). Responses were of rapid onset (67% by week 6). The safety profile was found to be acceptable with most treatment-related adverse events characterized as low grade and manageable for long-term treatment.
Andrew Wagner, M.D., Ph.D., a senior oncologist at the Dana-Farber Cancer Institute and Principal Investigator of the study, said, "We are excited to publish the data obtained from this trial. nab-Sirolimus delivered highly durable responses in patients with advanced PEComa. Most of the responding patients rapidly achieved a response by their first assessment at 6 weeks following initiation of therapy and these patients have stayed on therapy for extended periods with an acceptable safety profile. We are encouraged by the outcomes in this first-ever prospective clinical trial in patients with this extremely rare disease".
Neil Desai, Ph.D., co-author of the article and Founder, Chief Executive Officer and President of Aadi Bioscience, said, "The Aadi Bioscience team is extremely grateful to the patients, families, and clinical trial teams who help expand the boundaries of available care through their participation in clinical trials. The publication of these results is an important milestone not only for malignant PEComa patients but also for the ongoing development of nab-sirolimus across the planned investigation in TSC1 and TSC2 indications and other combination strategies."
A New Drug Application for investigational ABI-009 is under review by the U.S. Food and Drug Administration for treatment of patients with PEComa and has a Prescription Drug User Fee Action target date of November 26, 2021. If approved, ABI-009 will be known as FYARRO.