On January 3, 2019 Aadi Bioscience, Inc. (Aadi), a clinical stage biopharmaceutical company focused on treating diseases driven by mTOR activation, reported that its drug TARZIFYX (sirolimus albumin-bound nanoparticles for injectable suspension, ABI-009) has received Breakthrough Therapy Designation status from the FDA for the indication of Advanced (metastatic or locally advanced) Malignant PEComa (perivascular epithelioid cell tumor) (Press release, Aadi, JAN 3, 2019, View Source [SID1234532410]). The designation was granted based on data provided to the FDA from Aadi’s ongoing phase 2 registration trial (NCT02494570) in Advanced Malignant PEComa. In October 2018, Aadi also received Fast Track Designation from the FDA for TARZIFYX for the same indication. Aadi received orphan drug designation for TARZIFYX for the treatment of PEComa in December 2017.

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Aadi’s Phase 2 registration trial for Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT), a rare form of sarcoma, has completed enrollment. Aadi previously received agreement from the FDA that this open label study in 30 efficacy evaluable patients with a primary endpoint of overall response rate could support the submission of an NDA for approval to treat this rare disease.

"We are extremely pleased with the progress of the AMPECT study and recognition of the high unmet need in this patient population via the Breakthrough Designation. Our small, but dedicated team is gearing up for NDA filing in 2019," said Neil Desai, Ph.D., Founder and Chief Executive Officer of Aadi. "There has been no prior clinical trial in this rare disease and standard chemotherapies used in sarcoma treatment have very limited activity in this patient population."

mTOR inhibition is relevant in a number of disease states and has not been fully exploited in oncology, cardiovascular disease, CNS diseases, mitochondrial disease, diseases of ageing, etc. Due to the unique properties of Aadi’s drug including effective CNS penetration, it is being tested in nine ongoing clinical studies across these indications with encouraging results even outside of oncology in early studies in pulmonary hypertension and surgically refractory epilepsy. Aadi’s ongoing and planned clinical trials include Oncology (pediatric tumors, advanced sarcoma, first-line treatment of advanced colorectal cancer, newly diagnosed and recurrent glioblastoma, advanced neuroendocrine tumors), Cardiovascular indications (pulmonary arterial hypertension), CNS indications (surgically refractory epilepsy) and Mitochondrial disease (Leigh Syndrome).