CLINICAL OUTCOMES AT STUDY-END ANALYSIS (N=31)1
PRIMARY ENDPOINT
OVERALL RESPONSE RATE (ORR)
39%
per independent review using
RECIST v1.1 (12/31 [efficacy
population]; 95% CI: 22%, 58%)1
Rapid Response
1.4months
median time to
response (95% CI: 1.3,
2.8 months)2
Durable Response
>3years
50% of patients had a DOR
of 39.7+ months (95% CI:
6.5 months to not reached)1
DISEASE CONTROL RATE
71% of patients with a confirmed response or with SD of ≥12 weeks’ duration (95% Cl: 52%, 85.8%)1*
HALF OF RESPONDERS WERE STILL RESPONDING AFTER 3 YEARS1
HALF OF RESPONDERS WERE STILL RESPONDING AFTER 3 YEARS1
* Disease control rate was a post hoc exploratory endpoint and was not prespecified. Therefore it should be interpreted with caution.1,3
AMPECT=Advanced Malignant PEComa Trial; CR=complete response; DOR=duration of response; PD=progressive disease; PEComa=perivascular epithelioid cell tumor; PR=partial response; RECIST=Response Evaluation Criteria in Solid Tumors; SD=stable disease.
In AMPECT, 2 patients experienced complete responses after 11 months and 34 months of treatment1,4
TARGET LESION CHANGES AT STUDY-END ANALYSIS (N=31)1,3
RECIST v1.1 criteria, per independent review
*Target tumor reduction may not match Best Overall Response Assessment, which also takes into consideration nontarget lesions and the observations of new lesions as per RECIST v1.1. SD and PD could be due to the natural course of the disease and not due to treatment.5
†One patient had an unconfirmed PR and thus best response is SD as per RECIST v1.1 (confirmation of response requirement).3,4
‡One patient with overall CR had a measurable lymph node as target tumor, but <10 mm.3
§Two patients with CRs of target tumor reduction had PRs as best response because of unresolved nontarget lesions.1
Image was adapted from Wagner et al. Target lesion changes per mutational status–final analysis figure. CTOS; 2022.
AMPECT=Advanced Malignant PEComa Trial; CR=complete response; PD=progressive disease; PR=partial response; RECIST=Response Evaluation Criteria in Solid Tumors; SD=stable disease.
FYARRO goes the distance for patients with PEComa
Secondary endpoints
(Kaplan-Meier estimates)1
MEDIAN PFS*
MEDIAN OS*
*
Survival data should be interpreted with caution given the single-arm study design.
nab=nanoparticle albumin-bound; OS=overall survival; PEComa=perivascular epithelioid cell tumor; PFS=progression-free survival.
References: 1. 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. 2. 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 3. Data on file. Aadi Bioscience, Inc.; 2021. 4. 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. 5. Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guidelines (version 1.1). Eur J Cancer. 2009;(45):228-247. doi:10.1016/j.ejca.2008.10.026
