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

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

Final analysis performed at 2.5 years after the last patient initiated therapy2

Primary endpoint1

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

Secondary endpoints1,2

  • 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

  • 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 nab-sirolimus and must have centrally confirmed PEComa)

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

AMPECT=Advanced Malignant PEComa Trial; ECOG=Eastern Cooperative Oncology Group; mTOR=mechanistic target of rapamycin; nab=nanoparticle albumin-bound; 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. Final analysis from AMPECT, an open-label phase 2 registration trial of nab-SIROLIMUS for patients with advanced malignant perivascular epithelioid cell tumors (PEComa). Abstract presented at: CTOS Virtual Annual Meeting; November 12, 2021. 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