AADi Launches Its Phase 2 Registration Trial for ABI-009, a Targeted
`mTOR Inhibitor, for Patients with Advanced PEComa, a Rare Form of
`Sarcoma
`
`Study to be conducted at major cancer centers across the US
`
`November 02, 2015 09:00 AM Eastern Standard Time
`
`PACIFIC PALISADES, Calif.--(BUSINESS WIRE)--AADi, LLC, a clinical stage biopharmaceutical company focused on
`treating diseases driven by mTOR activation, today announced the initiation of its registration trial for advanced
`Perivascular Epithelioid Cell tumors (PEComa) with ABI-009, its targeted albumin-bound mTOR inhibitor. ABI-009 is the
`nanoparticle albumin-bound (nab®) version of the mTOR inhibitor sirolimus or rapamycin and leverages the same
`technology of the nab® platform that is behind the success of ABRAXANE® (paclitaxel protein-bound particles for
`injectable suspension) (albumin-bound). ABI-009 was licensed to AADi in 2014 by Celgene Corporation
`(NASDAQ:CELG). AADi plans to develop ABI-009 initially in oncology and cardiovascular indications.
`
`“We are extremely excited to launch our phase 2 registration trial in this very rare disease where activation of the mTOR
`pathway is known to play an important role. ABI-009 has a unique pharmacology amongst the mTOR inhibitors which we
`believe will translate into better outcomes for the patients,” said Neil Desai, Ph.D., Founder and Chief Executive Officer of
`AADi.
`
`In August 2015, AADi received agreement from the FDA on the design of its phase 2 registration study for the treatment
`of advanced (locally advanced or metastatic) PEComa with ABI-009. The study will enroll approximately 35 patients and
`the primary endpoint for the study is the overall response rate.
`
`“This is the first clinical trial focused on this rare form of sarcoma where the biology driving the disease matches the
`pharmacology and mechanism of action of the drug,” said Dr. Andrew Wagner, Principal Investigator of the study, Senior
`Physician, Center for Sarcoma and Bone Oncology and Assistant Professor of Medicine, Dana Farber Cancer Institute
`and Harvard Medical School. Dr. Wagner was the first to describe the relevance of mTOR inhibitors in the treatment of
`malignant PEComa due to the pathogenic activation of the mTOR pathway in these tumors. “It is important that we
`continue to search for new treatments for rare diseases and to scientifically test their safety and activity.”
`
`A phase 1 trial for ABI-009 was completed in patients with advanced non-hematologic malignancies in which the drug
`was well tolerated with evidence of activity in heavily pretreated patients. AADi also has an ongoing phase 1/2 trial of
`ABI-009 for the treatment of non-muscle invasive bladder cancer that is being conducted as part of a Fast-Track STTR
`grant awarded to AADi from the National Cancer Institute (NCI) of the National Institutes of Health (NIH). In addition to
`these studies, AADi plans to initiate a phase 2 trial in patients with various solid tumors that have been selected for
`mTOR pathway activations.
`
`As part of its focus in cardiovascular disease, AADi plans to initiate clinical studies with ABI-009 in the treatment of
`pulmonary arterial hypertension, a rare, progressive and debilitating disease that is highly dependent on mTOR
`activation, and which occurs due to abnormal constriction of the arteries in the lungs resulting in shortness of breath and
`increasing stress on the heart or heart failure.
`
`AADi is currently raising capital to fund its research programs through the end of phase 2 clinical studies.
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`Ex. 1101-0001
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`About PEComa
`
`Perivascular epithelioid cell tumors (PEComa) 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].
`
`About AADi and ABI-009
`
`AADi, LLC 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 available mTOR inhibitors. In animal models,
`ABI-009 has shown greater antitumor efficacy than the oral mTOR inhibitors at the same dose. Similar to Abraxane, the
`albumin-bound rapamycin is expected to have high tumor penetration by taking advantage of mechanisms of albumin
`uptake in tumors and other areas with tissue remodeling or inflammation.
`
`Abraxane® and nab® are registered trademarks of Celgene Corporation.
`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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`Contacts
`AADi
`Neil Desai, 310-309-9036
`info@aadibio.com
`www.aadibio.com
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`Ex. 1101-0002