Amyndas Pharmaceuticals SA, which is committed to developing innovative therapeutics to treat complement-mediated disorders, announced today that its lead candidate, the C3 complement inhibitor AMY-101, has been granted orphan designation by the European Medicines Agency (EMA) and the U.S. FDA for the treatment of C3 glomerulopathy (C3G). C3 glomerulopathy refers to a group of rare renal disorders characterized by undesirable activation of complement and the accumulation of complement component C3 in renal glomeruli, leading to proteinuria, hematuria and kidney failure (Pickering et al. Kidney Int. 2013; 84:1079-89). Half of all patients with C3G progress to end-stage renal failure within 10 years of diagnosis; and allograft loss occurs following kidney transplantation secondary to disease relapse in more than 50% of transplant recipients. Currently, there is no treatment for C3G. Management is symptom-based and includes drugs for blood pressure control, such as angiotensin-converting enzyme inhibitors, and immunosuppressive therapy. As the pathology of C3G is due to malfunction of the alternative pathway (AP) of complement, inhibition of complement component C3 is a promising strategy for the development of an effective treatment. Indeed, in recent studies AMY-101 was shown to inhibit complement dysregulation in C3G in vitro, presenting a promising therapeutic opportunity and potentially a major advancement for patients with C3G (Zhang et al. Immunobiology 2015; 220:993-8). AMY-101 is the first drug to be granted an orphan drug status by the EMA and the U.S. FDA for the indication of C3G. “Receiving the orphan drug designation for C3G speaks to the urgent unmet need for a treatment for patients with this serious and life-threatening condition” said Prof. Lambris, founder of Amyndas and Professor of Research Medicine in the Department of Pathology and Laboratory Medicine at the University of Pennsylvania. “There is currently no treatment for C3G and clinicians have no options to offer these patients; we are committed to bringing AMY-101 to C3G patients for this very purpose. This designation, which is based on the potential clinical benefit of AMY-101 in C3G patients, is an important achievement, as Amyndas continues the clinical development program for AMY-101. Moreover, it signifies a landmark event, as it marks the first C3 glomerulopathy drug to achieve this regulatory milestone” added Prof. Lambris. Amyndas’ collaborator Prof. Richard Smith, a leading researcher in C3G and Director of the Institute of Human Genetics and the Molecular Otolaryngology and Renal Research Laboratories (MORL) at the University of Iowa, commented: “The decision of the two leading regulatory agencies, the EMA and the FDA, to grant AMY-101 orphan designation underscores the urgent need for therapy for C3 glomerulopathy and validates the scientific rationale of Amyndas’ approach”. The FDA and EMA Orphan Drug programs provide orphan designation to novel drugs that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States or 5 in 10,000 in the EU). The designation provides sponsors with development and commercial incentives including seven years of market exclusivity in the US, 10 years of market exclusivity in the EU, consultation by FDA and EMA on clinical study design, potential for expedited drug development, and certain fee exemptions and reductions. 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 C3G. 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 new treatment options for complement-mediated diseases, such as C3G. About Amyndas Pharmaceuticals Amyndas Pharmaceuticals is 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 company plans to move its novel drug candidates to the clinic, starting with applications in C3G, paroxysmal nocturnal hemoglobinuria (PNH), ABO-incompatible kidney transplantation and periodontal disease, and potentially including other complement-mediated disorders, such as age-related macular degeneration (AMD) and ischemia-reperfusion injury.