FDA grants orphan drug designation for PNH to Amyndas Pharmaceuticals’ novel complement inhibitor

Amyndas Pharmaceuticals SA, the complement therapeutics company, announced that the U.S. Food and Drug Administration (FDA) has granted AMY-101, a potent complement C3 inhibitor, an Orphan Drug Designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). The U.S. FDA orphan designation comes after the European Medicines Agency (EMA) orphan status approval of AMY-101 for the same indication, less than 2 months ago. “We are pleased to receive orphan drug designation for AMY-101 and are encouraged by the FDA’s recognition for the need of innovative new treatments for PNH” said Dr. Lambris, Founder of Amyndas and Professor of Research Medicine in the Department of Pathology and Laboratory Medicine at the University of Pennsylvania. In PNH, it is clear that about 30 percent of the patients are not optimally responding to therapy; when current treatment fails, clinicians have limited options to offer these patients. AMY-101 acts through a novel mechanism of action, inhibiting complement centrally, at the level of C3 instead of C5; this approach could increase the number of PNH therapy responders and benefit many more patients suffering from PNH”. “We believe that AMY-101 represents an opportunity to make a significant impact in the lives of patients with PNH, bringing a therapeutic advantage over current treatments” said Dr. Lambris. “Additionally, since AMY-101 binds tightly to C3 with a long half-life, the drug could be self-administered subcutaneously every 12 hours, giving patients better control and dramatically reducing the need for clinic visits.” “The Orphan Drug Designation is an important regulatory advancement, and brings Amyndas one step closer to making this novel compound available to PNH patients,” said Dr. Lambris. Under the U.S. Orphan Drug Act (ODA) orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. Orphan drug designation entitles Amyndas to a seven-year period of marketing exclusivity from product launch in the United States, if AMY-101 is approved by the FDA for the treatment of PNH, and enables the company to apply for research funding, tax credits for certain research expenses, assistance in clinical research study designs and a waiver from the FDA's Prescription Drug User Fee. Amyndas is planning to move AMY-101 into the clinic for first-in-human trials in 2015, and the Orphan Drug Designation should prove valuable in the clinical development of this drug candidate. About AMY-101 AMY-101 is a novel therapeutic based on the second-generation compstatin analogue Cp40, discovered by Dr. John Lambris of the University of Pennsylvania, a leading researcher in the field of complement and the founder of Amyndas Pharmaceuticals. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade, which plays a pivotal role in PNH. Compared to previous compstatin analogues, AMY-101 shows 6,000-fold improvements in binding affinity to human C3, enhanced inhibitory potency and an extended circulating half-life in vivo. Amyndas has an exclusive, worldwide licensing agreement with the University of Pennsylvania that provides broad rights to develop and commercialize University of Pennsylvania’s second-generation compstatin analogues, which are potent complement-inhibiting peptides for the treatment of a range of complement-mediated disorders. This agreement allows Amyndas to move forward with the development of AMY-101 aimed at providing a new treatment option for PNH. About PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare but life-threatening hematological disorder. In PNH, important regulatory proteins are missing from the surface of blood cells, leaving them vulnerable to complement attack. Activation of complement can cause red blood cells to break apart, a process called hemolysis, which clinically results in severe anemia and contributes to a high risk of clotting. By targeting complement component C5, the monoclonal antibody eculizumab (Soliris®, an Alexion Pharmaceuticals drug) - to date the only approved therapeutic for PNH – reduces hemolysis and the need for blood transfusions for many PNH patients. However, one third of PNH patients continue to require blood transfusions to manage their anemia; this insufficient response is due to fragments of complement C3 proteins on the surface of their red blood cells, which are eventually attacked by immune cells. Inhibition of the “complement cascade” at the level of C3 might be a superior strategy as it prevents both hemolysis and immune cell recognition. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade. About Amyndas Pharmaceuticals Amyndas Pharmaceuticals S.A. and Amyndas Pharmaceuticals LLC, two transatlantic sister companies (based in Glyfada, Greece and Philadelphia, Pa., respectively), are developing novel therapeutics to treat complement-mediated disorders that could be more potent than other complement inhibitors, potentially opening up prospects for treating new indications. The companies work closely together to move their novel drug candidates to the clinic, starting with applications in PNH and ABO-incompatible kidney transplantation and potentially including other complement-mediated disorders, such as periodontal diseases, hemodialysis and ischemia-reperfusion injury. For more information please contact Dr. Despina Yancopoulou Business Development Amyndas Pharmaceuticals SA N. Zerva 28, Glyfada 16675, Greece T: +30 (210) 894-1213 e-mail: [email protected]

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