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AAVP Biosystems, Inc. Maximizing Efficacy and Minimizing Toxicity Using Targeted Therapeutics DESCRIPTION: AAVP Biosystems (“AAVP”) is an early stage biotechnology company developing a targeted gene delivery vector that aims to direct therapy selectively to tumor cells while sparing normal cells and tissue. The core technology is fully and exclusively licensed from MD Anderson Cancer Center, and is undergoing IND-enabling studies in preparation for a late-2015 FDA filing. TECHNOLOGY: Adeno-associated virus phage or AAVP, is a hybrid vector platform developed by combining the targeting capability of bacteriophage and the sustained gene expression of adeno-associated virus. Bacteriophage is a virus that infects bacteria, but cannot infect human cells and has been used clinically for decades as an antimicrobial agent. AAVP has successfully developed a version of bacteriophage that can infect, but not replicate, in specific human cells. That specificity can be tailored to a cell of choice, such as a tumor cell or tumor associated cell. Normally, if a bacteriophage infects a human cell, no gene expression takes place. However, AAVP’s hybrid AAVP can express a gene product in a human cell based on the fact that it carries the gene expression machinery of an adeno-associated virus, but not the adeno-associated virus itself. This avoids the limitations well known to adeno-associated virus including the need for a helper virus and robust immunogenicity limiting clinical efficacy. The result is a platform that can be tailored to target any human cell and express exclusively in that cell, any gene of interest. The company has generated AAVP that infect a variety of cell types and express a variety of gene products, both for therapy and for imaging. AAVP’s lead therapeutic, RGD-AAVP-TNF, is designed to selectively infect tumor associated blood vessels and express TNF exclusively in the tumor vascular bed resulting in tumor necrosis. The company has validated this tumor targeting and selective gene expression, as well as therapeutic efficacy and absence of toxicity, in small and large animals. In fact, RGD-AAVP-TNF has been administered in the context of two veterinary clinical trials in dogs demonstrating safety and anti-tumor activity. AAVP has generated substantial pre-clinical data on stability, dosing and lack of toxicity and is readying to submit an FDA IND filing in preparation for a first in human clinical trial in patients with advanced stage malignancies. DEVELOPMENT PLAN: AAVP’s next steps are producing cGMP grade RGD-AAVP-TNF, completing an IND filing for the FDA, and initiating a Phase I clinical trial. AAVP has identified organizations capable of performing the cGMP production, assist with IND required toxicity studies, and build the IND package. IND-enabling projects will run through 2015, at which point the company anticipates requesting a pre-IND meeting. In late-2015, AAVP will focus on raising a round of capital, between $6 and $8 million, followed by the filing of the IND request to accrue the planned dose escalating Phase I clinical trial. In 2016, upon closing of the second capital raise, the company anticipates the start of enrollment of a ~30 patient first in human study, which should conclude in 2017. To accelerate IND-enabling activity, AAVP is in the process of leasing incubator research space at the Los Alamos National Laboratory. This fully-equipped research facility will house our key scientific advisors, as well as two research associates. This facility provides the Company with a greater level of control over its research endeavors at a greatly reduced cost than what would be required if this research was performed by contracted research organizations or in university laboratories. Furthermore, with two of the Company’s key scientific officers overseeing this research, the risks of erroneous or mistargeted data generation is greatly reduced. FINANCING: AAVP has raised over $600 thousand in initial seed funding, expects to close on the first part of a $3 million Series A round in early-2015, and the remainder of which it aims to close upon mid-year. Funding has been sourced from institutional investors and high-net worth family offices focused on early stage biotechnology companies. Cash needs through 2018 are approximately $11 million. These funds will support development through a first in human clinical trial. Over the next 18 months it is projected that $3 million of this total will be allocated to cGMP production, regulatory consultants, and support for the filing of an IND. The remaining funds are projected to carry through 2018 to support the completion of the Phase I clinical trial. KEY PERSONNEL: Jason Rifkin, J.D., M.S., President & CEO has managed all aspects of early stage biotechnology company operations. Prior to his role at AAVP, Jason was President of PreScience Labs, an early stage oncology company where he oversaw the build-up of a Phase I clinical trial program. Prior to joining PreScience, Jason held a number of executive clinical and commercial operations positions at Delcath Systems, Inc., a specialty pharmaceutical and medical device company focused on oncology. Timothy Rothwell, J.D., Chairman of the Board is also the Chairman of Emisphere Technologies Inc. and prior to that a Director of the Company. Mr. Rothwell is also Chairman of Speracura LLC, a healthcare consulting company and Chairman of the Pheo-Para Alliance. Mr. Rothwell was formerly Chairman of Sanofi in the U.S. from February 2007 to October 2009 and served as Chief Executive Officer and President of Sanofi-Aventis from September 2004 to February 2007. Steven Libutti, M.D., Founder and Director is the Director of the Montefiore-Einstein Center for Cancer Care, Professor and ViceChairman of the Department of Surgery and Professor in the Department of Genetics at the Albert Einstein College of Medicine and the Montefiore Medical Center. Dr. Libutti is Editor-in-Chief of the Nature Journal Cancer Gene Therapy. Renata Pasqualini, Ph.D., Founder is Associate Director for Translational Research at the University of New Mexico Cancer Center and co-leader of the UNM Cancer Center’s Program in Experimental Therapeutics and Drug Discovery. Dr. Pasqualini is also Professor in the Department of Internal Medicine at the University of New Mexico School of Medicine. Wadih Arap, M.D., Ph.D., Founder is the Deputy Director of the UNM Cancer Center and Chief of the Division of Hematology and Oncology in the Department of Internal Medicine in the University of New Mexico School of Medicine. CONTACT INFORMATION Jason Rifkin T: (917) 733-1542 Fax: (914) 723-1419 E-mail: [email protected] Website URL: www.aavpbiosystems.com