Prothena Achieves Orphan Drug Status In EU For Lead Program NEOD001

DUBLIN, Ireland, Feb. 27, 2013 (GLOBE NEWSWIRE) -- Prothena Corporation plc (Nasdaq:PRTA), a biotechnology company focused on the discovery and development of novel antibodies for the potential treatment of a broad range of diseases, today announced that the European Medicines Agency (EMA) granted orphan designation for its lead program, NEOD001, for the potential treatment of amyloid light-chain (AL) amyloidosis. In 2012, the US Food and Drug Administration (FDA) granted orphan drug status to this program. NEOD001 is a monoclonal antibody that specifically targets the amyloid that accumulates in the organs of patients with the disease.

"AL amyloidosis is the most common and pathogenic form of systemic amyloidoses, and securing orphan designation for NEOD001 in the European Union is an important milestone for Prothena and for patients," said Dale Schenk, PhD, Prothena's President and Chief Executive Officer. "NEOD001 targets systemic amyloidoses, diseases in which misfolded proteins accumulate in the body's organs, causing progressive damage and death. We expect to start Phase I clinical trials of NEOD001 in primary amyloidosis patients early this year. If proven effective in clinical trials, our approach has the potential to provide a novel therapy for this orphan disease with significant unmet medical need." 

"Orphan drug designations support our global development strategy for NEOD001 and our goal of providing improved therapies for patients with amyloidoses," said Gene Kinney, PhD, Prothena's Chief Scientific Officer and Head of Research and Development. "Our preclinical experience to date with NEOD001 has indicated its potential in this disease area, where limited treatment options exist. The orphan drug designations in the EU and the US provide recognition of the need for therapeutics in this area and help advance the development of potential treatments for patients."

About NEOD001

NEOD001 is a monoclonal antibody that specifically targets the amyloid that accumulates in both AL and secondary systemic amyloidosis. If proven safe and effective in clinical trials, this approach has the potential to be a first-in-class agent for this orphan disease with a significant unmet medical need. In 2012, NEOD001 was granted orphan drug designation by the FDA, and an Investigational New Drug application for NEOD001 in systemic amyloidosis was filed. Prothena plans to initiate a Phase 1 clinical trial for NEOD001 in this indication in early 2013. NEOD001 is being developed by Onclave Therapeutics, Limited, a wholly-owned subsidiary of Prothena. More information on the planned clinical trial for NEOD001 is available on clinicaltrials.gov.

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