Advanced PEComa

About Advanced PEComa

AADi has launched its phase 2 registrational trial in patients with advanced PEComa (Perivascular Epithelioid Cell tumors), a rare form of sarcoma, in which activation of the mTOR pathway is known to play an important role.

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].

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

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


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

Advanced PEComa

Aadi Bioscience, Inc. (Aadi), a clinical stage biopharmaceutical company focused on treating diseases driven by mTOR activation, today announced the completion of a $23 Million Series A round. The financing was led by Hermed Capital with participation from Celgene Corporation, Vivo Capital, Decheng Capital, the Helsinn Investment Fund and Star Summit Ventures. The company will use the capital to drive the clinical programs for ABI-009, its targeted albumin-bound mTOR inhibitor.

“We are extremely pleased with the participation and interest of our investors and now stand poised to develop the potential of what we believe is a best-in-class mTOR inhibitor,” said Neil Desai, Ph.D., Founder and CEO of Aadi.

Aadi is currently enrolling its registration trial for advanced Perivascular Epithelioid Cell tumors (PEComa), an extremely rare form of sarcoma. Aadi received agreement from the FDA on the design of its phase 2 registration study for the treatment of advanced PEComa with ABI-009. The study will enroll approximately 30-35 patients and the primary endpoint for the study is the overall response rate. Earlier this month, an independent data monitoring committee (DMC) reviewed the available data and concluded that the study should continue without modification.

Aadi’s other planned clinical programs include pulmonary hypertension (phase 1), early stage bladder cancer (phase 2) and pediatric cancers (phase 1). It continues to investigate applications of ABI-009 in cancer indications as well as cardiovascular and other diseases.

Dr. Desai is also pleased to welcome new directors to the board of Aadi including Carlo Montagner (Specialised Therapeutics), Richard Maroun (Frazier Healthcare Partners), Jerry Xiao (Hermed Capital) and Mahendra Shah (Vivo Capital).

About Aadi and ABI-009

Aadi is a clinical stage biopharmaceutical company led by Dr. Neil Desai, an inventor of ABRAXANE®, ABI-009 and the nab® technology platform. Aadi’s lead product is ABI-009, a nanoparticle albumin-bound mTOR inhibitor based on sirolimus or rapamycin, also known as nab-rapamycin. Aadi aims to develop the full potential of the albumin-bound mTOR inhibitor in diseases that are driven by mTOR activation and where the mTOR inhibitors have not or cannot be effectively exploited due to problems of effective drug delivery, safety or effective targeting to the disease site. In a phase 1 study ABI-009 was well tolerated at intravenous doses significantly higher than other mTOR inhibitors and its pharmacokinetic profile, with high Cmax and AUC, is very different from the approved mTOR inhibitors. In animal models, ABI-009 has greater antitumor efficacy than the oral mTOR inhibitors at the same dose. Similar to ABRAXANE®, albumin-bound rapamycin is expected to have high tumor penetration due to mechanisms of albumin uptake in tumors and other areas with tissue remodeling or inflammation. ABI-009 was licensed to Aadi in 2014 by Celgene Corporation (NASDAQ:CELG).

ABRAXANE® and nab® are registered trademarks of Celgene Corporation.