Advanced PEComa

About Advanced PEComa

PEComas are a rare subset of soft tissue sarcomas recently recognized as a distinct entity by the World Health Organization in 2002 and are composed of histologically and immunohistochemically distinctive epithelioid cells.

PEComas appear to arise most commonly at visceral (especially gastrointestinal and uterine), retroperitoneal, and abdominopelvic sites.

Most PEComas are benign, but there is a subset of PEComas, i.e., advanced malignant PEComas [1], for which there are currently no approved therapies and for which there are only a few case reports in the literature.

The prognosis for this patient subset is poor, with a median survival estimated to be 12-17 months following diagnosis of advanced disease [2].

Overactivation of the mTOR pathway has been reported in malignant PEComa, and mTOR inhibitors have shown anecdotal efficacy in this indication in case reports or retrospective studies [1].

AADi’s phase 2 registrational trial for ABI-009 in patients with advanced PEComa (Perivascular Epithelioid Cell tumors) will be completed in 2019 followed by the NDA submission (NCT02494570).

ABI-009 has received Orphan Designation for PEComa and Fast Track Designation for advanced (metastatic or locally advanced) malignant PEComa.

AADi is also a recipient of FDA’s Orphan Product Development Grant for this indication.

You can check out more info about our PEComa study and eligibility in our dedicated website here: http://www.mypecomastudy.com/

1. Wagner AJ et al. (2010). J Clin Oncol 28, 835-840.
2. Bleeker JS et al(2012). Sarcoma 2012, 541626

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Advanced (unresectable or metastatic) PEComa
Ultra-orphan indication
Estimated prevalence 200-400 patients in US
mTOR activation, loss of TSC1 or TSC2 reported

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