On June 25, 2020 Aadi Bioscience, Inc. (Aadi), a privately held biopharmaceutical company focused on development and commercialization of nab-sirolimus (sirolimus albumin-bound nanoparticles for injectable suspension) in mTOR driven diseases, reported that the company has initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the treatment of advanced (metastatic or locally advanced) malignant PEComa (perivascular epithelioid-cell tumor) – a rare form of sarcoma driven by mTOR activation for which there is currently no approved therapy (Press release, Aadi, JUN 25, 2020, View Source [SID1234561478]). The company expects to complete the NDA submission in the 4th quarter of 2020.
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Following receipt of Breakthrough Therapy, Fast Track, and Orphan Designations, the initiation of the NDA submission for nab-sirolimus is an important milestone for Aadi and a step towards the goal of treating high unmet need diseases with established mTOR pathway activation," said Neil Desai, Ph.D., Chief Executive Officer of Aadi. "We are proud to have completed the AMPECT Study, the first prospective clinical trial for advanced PEComa, and will continue to work expeditiously to complete the submission and bring this potential new therapy to patients.
About Aadi Bioscience and nab-sirolimus (ABI-009)
Aadi is a clinical stage biopharmaceutical company led by Dr. Neil Desai, an inventor of ABRAXANE and the albumin-based technology platform. Aadi’s lead product nab-sirolimus (sirolimus albumin-bound nanoparticles for injectable suspension, ABI-009) is an mTOR inhibitor complexed with human albumin that has significantly higher tumor accumulation, mTOR target suppression and improved efficacy over other mTOR inhibitors in preclinical models. mTOR as a therapeutic target is well recognized in oncology, however Aadi aims to develop the full potential of albumin-bound sirolimus in therapeutic areas and diseases that are driven by mTOR activation and where the mTOR inhibitors have not or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety or effective targeting to the disease site. These indications include oncology, cardiovascular, CNS, mitochondrial disease and diseases of ageing. Aadi’s ongoing and planned clinical trials include Oncology (first-line treatment of advanced colorectal cancer with respect to PTEN status, pediatric tumors, advanced sarcoma, newly diagnosed and recurrent glioblastoma, advanced neuroendocrine tumors), Cardiovascular indications (pulmonary arterial hypertension), CNS indications (surgically refractory epilepsy) and Mitochondrial disease (Leigh Syndrome).
About PEComa
Perivascular epithelioid-cell tumors, defined by the World Health Organization as "mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers," are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract) and can have an aggressive clinical course including distant metastases and ultimate death. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTORC1 pathway. Therefore, mTORC1 signaling is a promising therapeutic target for malignant PEComa.