Publication of long-term efficacy and safety data further support Fyarro (nab-sirolimus) for the treatment of malignant PEComa

The manuscript, "A Phase II Trial of nab-Sirolimus in Patients with Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-term Efficacy and Safety Update," is authored by Andrew J. Wagner, MD, PhD and colleagues.

"We are excited to report the final outcomes of this registrational trial after an additional 3 years of follow-up. Responses to nab-sirolimus in patients with advanced PEComa lasted a median of 39.7 months," commented Dr. Wagner, Senior Physician at the Dana-Farber Cancer Institute and Associate Professor of Medicine at Harvard Medical School. "The median survival outcomes were consistent with the primary analysis, and the confirmed overall response rate remained at 38.7% and included an additional patient with a complete response. The long-term, durable responses to nab-sirolimus with tolerable safety are great news for patients with this rare disease."

"The data published in JCO highlight the potential for nab-sirolimus to be an effective and highly differentiated treatment that may help patients suffering from this aggressive cancer achieve longer duration of responses and survival rates," said Loretta Itri, MD, Chief Medical Officer of Aadi. "We want to thank the principal investigators, study coordinators, and the patients who participated in AMPECT. We believe these results provide a significant contribution to sarcoma research and we look forward to continuing to advance nab-sirolimus."

The AMPECT trial (NCT02494570) evaluated the efficacy and safety of nab-sirolimus in patients with advanced malignant PEComa and was the first prospective registrational study in this setting . Data from this Phase II, open-label, single-arm, multi-center study served as the basis for the FDA approval of Fyarro in advanced malignant PEComa regardless of mutational status.

Key updates reported ::Efficacy: At study completion, the confirmed ORR on the basis of independent radiographic review using RECIST v1.1 remained at 38.7% (95% CI, 21.8%-57.8%), with an additional converted confirmed complete response (CR) since the previously published analysis. The disease control rate (DCR) remained at 71% (95% CI, 52.0%–85.8%). Durability: At the time of the primary analysis, the median duration of response (mDOR) had not been reached. At study completion, the mDOR was reached at 39.7 months (95% CI, 6.5 months to NR), median PFS remained the same at 10.6 months (95% CI, 5.5-41.2 months), and median OS was 53.1 months (95% CI, 22.2 months to NR). Safety: The most common TRAEs were stomatitis (82.4%) and fatigue and rash (each 61.8%). No new or unexpected adverse events occurred, and no grade 4 or 5 TRAEs were reported.

See- "Phase II Trial of nab-Sirolimus in Patients With Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT): Long-Term Efficacy and Safety Update."- Authors: Andrew J. Wagner, MD, PhD , Vinod Ravi, MD , Richard F. Riedel, MD , Kristen Ganjoo, MD, Brian A. Van Tine, MD, PhD , Rashmi Chugh, MD , Lee Cranmer, MD, PhDL, and Mark A. Dickson, MD .Journal of Clinical Oncology.