We are a biopharmaceutical company that was formed to explore new methods of use of AAT, also referred to as "plasma-derived AAT" (“p-AAT”). p-AAT is purified from human blood and is currently believed to be one of the body's most powerful anti-inflammatory proteins. p-AAT has a greater than 25-year safety record as an approved treatment for emphysema in AAT-deficient patients. Our initial strategy was based on licensing issued and pending "method- of-use" patents that cover new treatment indications for AAT and commercializing these through royalty agreements with existing AAT manufacturers. Our initial targeted markets were infectious diseases, including biohazards, but in 2009 we changed our focus to certain auto-immune and inflammatory diseases, such as Type 1 diabetes (also known as juvenile diabetes).
In 2012, we released initial findings of our human clinical trial using p-AAT for the treatment of Type 1 diabetes. The preliminary results demonstrated positive benefits that included stabilizing c‑peptide levels (a measure of the ability of pancreatic beta cells to produce insulin) and decreasing the insulin requirement in certain patients having recent onset of Type 1 diabetes. During the past three years, other organizations, such as the Immune Tolerance Network and Kamada, Ltd. (Israel), have also conducted human clinical trials in Type 1 diabetes using plasma-derived AAT and have reported positive results. We believe these studies support our premise that AAT can be used in the treatment of Type 1 diabetes and other immuno-modulated inflammatory diseases, such as bone marrow transplantation rejection, referred to as graft versus host disease (GvHD), treatment-refractory gout, acute myocardial infarction and inflammatory bowel disease.
We are currently monitoring the long-term follow up of patients from our Type 1 diabetes trial and are also supporting two new multicenter trials of the use of plasma-derived AAT in patients suffering from acute steroid-refractory GvHD. There are no satisfactory treatments for patients with this condition and most die within one year of diagnosis. Preclinical animal studies demonstrate a beneficial effect of AAT in models of this disease. Thus, a corresponding clinical solution to GvHD would represent a significant medical breakthrough and may even allow the broader use of bone marrow transplants to treat patients with otherwise lethal leukemias.
In January 2012, we announced our intention to pursue research and development on recombinant forms of AAT. These forms are created by fusing naturally occurring human AAT to the Fc portion of an immunoglobulin antibody in order to increase potency and provide for longer lasting blood levels (Fc-AAT). To date, a large number of in vitro and in vivo studies suggest that Fc-AAT may be 50-100x more potent than plasma-derived AAT and may also have a longer duration of effect. If borne out in clinical trials, this could lead to a product that can be made rapidly and in large quantities, is able to be self-administered subcutaneously and is able to be given less often than once per week. Each of these represents a significant competitive improvement over existing plasma-derived products which must be given intravenously, once per week in a doctor’s office or infusion clinic, and are very expensive due to the costs of the products themselves and the costs of the infusion procedures.
We have recently placed the first of these molecules on a formal development track. We are organizing preclinical activities to enable the initial clinical trials of Fc-AAT in 2014. Patent application filings are under review in the US and Europe and we are hopeful that we will have successful issuances later in 2013. If successful, it is expected that we would have market exclusivity for a minimum of 12 years from the time of introduction in the US and 10 years in Europe.
Bruce Schneider, Ph.D. - Chief Executive Officer
Dr. Schneider has served as our CEO and a member of our Board of Directors since January 1, 2013. Since 2010, he has provided R&D executive advisory and business development services to several large and small pharmaceutical companies, including an extensive role with Pfizer where he was engaged to facilitate strategic planning and decision-making for its early drug development pipeline. From 2009 to 2010, he was employed by Pfizer as Executive Vice President & R&D Integration Lead. From 1972-2009, he was employed by Wyeth Research, which was acquired by Pfizer in 2009, where he held numerous senior-level R&D positions, including Executive Vice President & Chief of Operations (2002 to 2009), Senior Vice President, Research Operations & Planning (1992-2002) and Vice President, Worldwide Clinical Operations (1987-1992). Dr. Schneider holds a Ph.D. in Applied Statistics from Temple University and a Sc.B in Applied Mathematics from Brown University.
Charles Dinarello, MD - Chief Scientific Officer
Dr. Dinarello has served as our Chief Scientific Officer and Director of Medical Affairs since February 2011. He also serves on our Scientific Advisory Board. From March 2009 to February 2011, he served as our Acting Chief Executive Officer and a member of our Board of Directors. Previously Professor of Medicine at Tufts University in Boston, since 1996, he is Professor of Medicine and Immunology at the University of Colorado Denver. Dr. Dinarello has published over 700 original research articles on cytokines, particularly interleukin-1 (IL-1) and tumor necrosis factor, and is considered a “founding father” of cytokine biology. Dr. Dinarello currently serves as a director of Techne Corporation (NASDAQ NNM: TECH), and is a Scientific Advisory Board member of Senesco Technologies, Inc., Source MDx, Inc., GlobeImmune, Inc., and Capstone Pharmaeuticals, Inc. He is also a member of the editorial board of the Proceedings of the National Academy of Sciences. Dr. Dinarello has received numerous international awards for his scientific contributions in cytokine biology.
Robert Ogden - Chief Financial Officer
Robert Ogden has served as our Chief Financial Officer, Treasurer and Secretary since May 2009. From 2007 to 2009, Mr. Ogden was a financial consultant, primarily providing services to public companies, including Fortune 500 and micro-cap entities. From May 2004 to June 2007, Mr. Ogden served as Chief Financial Officer, Treasurer and Secretary of SAN Holdings, Inc., a publicly-traded data storage solutions provider and software company, where he was responsible for all administrative and finance functions. From 2000 to May 2004, he was a multi-industry financial consultant providing financial controller and reporting services to both private and public companies. His prior experience includes financial executive positions with two micro-cap companies. Mr. Ogden began his career as a public accountant with PricewaterhouseCoopers LLP. Mr. Ogden received a BS in Commerce from the University of Virginia and is a CPA.
Bruce Schneider, Ph.D., Chairperson
Michael Kamarck, Ph.D.
Michael Kamarck has served as a member of our Board of Directors since January 2013. From 2010 to 2012, Dr. Kamarck was employed by Merck as President of Merck BioVentures and Senior Vice President of Vaccines and Biologics Manufacturing. From 2001 to 2009, he was employed by Wyeth, where he held various senior executive positions, including President, Technical Operations and Product Supply and was responsible for global technical operations for all of the Wyeth businesses. Dr. Kamarck also served as a member of the Wyeth Management Committee. Prior to Wyeth, he was employed by Bayer AG for 17 years in a variety of technical and leadership capacities. Dr. Kamarck received his B.A. from Oberlin College, his Ph.D. from MIT and was a Leukemia Society Fellow at Yale University.
Sandra Wrobel has served as a member of our Board of Directors since July 2013. Ms. Wrobel is currently CEO and Managing Director, Applied Strategies Consulting, LLC, a firm that provides strategy, business process, and operations expertise to life sciences and global healthcare organizations. She previously served as CFO and Vice President Business Operations at Intrabiotics Pharmaceuticals, Inc. and held a series of positions over twelve years at Strategic Decisions Group, culminating in Managing Director of their Life Sciences Consulting Practice. Ms. Wrobel received her B.S. degree in Chemical & Petroleum Refining Engineering from the Colorado School of Mines and her MBA from the Stanford University Graduate School of Business.
Michael Wort has served as a member of our Board of Directors since March 2009, and is a founder of Apro Bio Pharmaceutical Corporation. From October 2008 until March 2009, he was a director of Apro Bio and from October 2008 through December 2008, he served as its Interim Chief Executive Officer. Since 2007, Mr. Wort has served as the Managing Director of De Facto Communications, a subsidiary of Chime Communications, which is listed on the London Stock Exchange. In 2004, Mr. Wort founded MC BioCommunications, an investor and public relations firm specializing in emerging bioscience companies. He is also a founder and director of BeckPharma Limited, a technology transfer company, and PharmScape, a biotechnology consultancy company, both of which were founded in 2003. Mr. Wort’s other prior experience includes various sales, marketing and general management positions with GlaxoSmithKline plc. Additionally, he has served as an advisor and consultant on fund raising for more than 15 small-cap, biotechnology companies. Mr. Wort received his BSC in Applied Biology and is a Chartered Graduate Biologist in Industrial Microbiology.
Albert Kramer has served as a member of our Board of Directors since March 2009. From April 2008 until March 2009, he was a director of Apro Bio Pharmaceutical Corporation. Since 1993, Mr. Kramer has served as an attorney with the Kramer Law Firm. From 1993 to 2005, Mr. Kramer served as Special Counsel to Syratech Corporation. From 2004 to 2008, he served as Chief Counsel to Travelers Marketing, LLC. From 1975 to 1992, Mr. Kramer served as a District Court Judge for the Massachusetts Trial Court. He received his undergraduate and law degrees from Boston University.