We are a biopharmaceutical company that was originally formed to explore new methods of use of alpha-1 antitrypsin, a naturally occurring protein that can be purified from human blood and is often referred to as "plasma-derived AAT" (“p-AAT”). AAT has been shown in a variety of animal models to have profound anti-inflammatory and tissue protective properties. Plasma-derived AAT has a greater than 25-year safety record as an approved treatment for emphysema in AAT-deficient patients. The Company’s early strategy was based on licensing "method- of-use" patents that cover new treatment indications for AAT and commercializing these through royalty agreements with existing p-AAT manufacturers. More recently our focus has shifted to the pharmaceutical development of a novel recombinant version of AAT for use in a variety of inflammatory and immune-modulated diseases.
Clinical experience with new uses of p-AAT is growing. In 2012, we released initial findings of our own clinical study of p-AAT for the treatment of patients with recent onset Type 1 diabetes. The study has now been completed and shows stabilization of c‑peptide levels (a measure of the ability of pancreatic beta cells to produce insulin) in certain patients and suggests correlation of these effects with reductions in the pro-inflammatory mediator, IL-1β. During the past three years, other organizations, such as the Immune Tolerance Network and Kamada, Ltd. (Israel), have also conducted early clinical trials in Type 1 diabetes using plasma-derived AAT with similar results. Recently, Grifols and Kamada, two of the p-AAT manufacturers, have begun Phase 2/3 placebo-controlled trials in Type 1 diabetic patients in the US and Europe.
We are currently supporting two new multicenter trials of the use of plasma-derived AAT in patients suffering from acute steroid-refractory GvHD. A clinical solution for this life-threatening condition would represent a significant medical breakthrough and may even allow the broader use of bone marrow transplants to treat patients with otherwise lethal leukemias. We are also monitoring a pilot study of AAT in acute myocardial infarction being conducted at Virginia Commonwealth University.
While we continue to support potential new uses of p-AAT, our focus is now on the research and development of recombinant “Fc fusion” 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 roughly 50x more potent than p-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 both product and infusion procedure costs.
We have recently placed the first of these Fc-AAT molecules on a formal development track and are organizing preclinical activities to enable the initial clinical trials in 2015/16. A patent covering our lead molecule was issued in the US in late 2013 and corresponding applications are under review in Europe and Canada. We now expect to have market exclusivity for a minimum of 12 years from the time of introduction in the US and, if successful, for at least 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 Pharmaceuticals, 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.
Bruce Forrest, MD - Chief Development Officer
Dr. Forrest was appointed to serve as our CDO in May, 2014. Concurrently he is President of B D Forrest & Company, Inc. a biopharmaceutical consulting firm that provides scientific strategy and business development support to emerging and established pharmaceutical companies. The Company retained Dr. Forrest to provide such services from late 2012 through April 2014. In his expanded role of Chief Development Officer he will now also oversee implementation of the development plan for Fc-AAT. Dr. Forrest has over 25 years of industry experience in pharmaceutical development, including vaccines and biopharmaceuticals. He was previously Senior Vice President in Vaccines R&D at Wyeth Pharmaceuticals where he was responsible for clinical and development activities for all late phase programs. From 2004-2008 he was head of Wyeth’s R&D organization in Japan where he oversaw the successful regulatory approvals of several new drug entities. He previously worked in various clinical research roles at Chiron Corporation and United Biomedical, Inc. Dr. Forrest is a medical graduate (MD) from the University of Adelaide (Australia) where he specialized in immunology and has an MBA from the Warwick Business School (UK).
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.
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.
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.
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.