Multicenter, single-arm, open-label, phase 2 registrational study evaluating FYARRO in
34 patients (safety population)1

AMPECT design AMPECT design

Primary analysis at 6 months after the last patient initiated therapy1

Second analysis performed 2 years after last patient initiated therapy (1.5 years following primary analysis)1

Study-end analysis 3-year follow-up after the primary analysis (3.5 years after last patient initiated therapy)2

Primary endpoint1

  • Overall response rate (ORR by RECIST v1.1 criteria) assessed by independent radiology review

Secondary endpoints2

  • Duration of response (DOR)
  • Progression-free survival (PFS)
  • Overall survival (OS)

Exploratory endpoints1

  • Post hoc analysis of disease control rate (DCR; defined as the percentage of patients with a confirmed response or with SD of ≥12 weeks’ duration)
  • Tumor biomarker analyses

*A maximum of 2 dose reductions by 25% each (to 75 and then 56 mg/m2) were permitted for toxicity.1

The primary analysis was preplanned to occur when the last enrolled patient had been treated for 6 months.1

AMPECT=Advanced Malignant PEComa Trial; PEComa=perivascular epithelioid cell tumor; RECIST=Response Evaluation Criteria in Solid Tumors; SD=stable disease.

Baseline characteristics were consistent with published reports and retrospective studies of advanced malignant PEComa1,3

Baseline Patient Characteristics1

  • Metastatic or locally advanced (inoperable) malignant PEComa
  • mTOR inhibitor-naive
  • Most common primary tumor sites: uterus (24%), pelvis (18%), retroperitoneum (18%), kidney, and lung (12% each)
  • Most common metastatic sites: lung/thorax (72%), abdomen (28%), pelvis (24%), liver (21%), and colon (14%)
  • 34 patients received at least one dose of FYARRO and were included in the safety analysis
  • 31 patients were evaluable for efficacy (must have received ≥1 dose of FYARRO and must have centrally confirmed PEComa)

Included docetaxel, doxorubicin, gemcitabine, ifosfamide, and olaratumab.1

ECOG=Eastern Cooperative Oncology Group; mTOR=mechanistic target of rapamycin; PEComa=perivascular epithelioid cell tumor.

References: 1. Wagner AJ, Ravi V, Riedel RF, et al. nab-Sirolimus for patients with malignant perivascular epithelioid cell tumors. J Clin Oncol. 2021;39(33):3660-3670. doi:10.1200/JCO.21.01728 2. Wagner AJ, Ravi V, Riedel RF, et al. Study-end analysis from AMPECT, an open-label, phase 2 registration trial of patients with advanced malignant PEComa treated with nab-sirolimus, showing durability of response and long-term safety. Poster presented at: Connective Tissue Oncology Society Meeting; Vancouver, BC, Canada; November 16-19, 2022. 3. Sanfilippo R, Jones RL, Blay JY, et al. Role of chemotherapy, VEGFR inhibitors, and mTOR inhibitors in advanced perivascular epithelioid cell tumors (PEComas). Clin Cancer Res. 2019;25(17):5295-5300. doi:10.1158/1078-0432.CCR-19-0288