Clinical stage biotechnology company Prothena has dosed the first AL amyloidosis patient in its Phase I study of NEOD001, an antibody therapeutic candidate.
The potent NEOD001 monoclonal antibody will be evaluated for safety and tolerability in patients with AL amyloidosis.
Prothena president and chief executive officer Dr Dale Schenk said preclinical data indicated NEOD001’s potential in the amyloidosis disease area, where limited treatment options exist for patients.
"If proven to be safe and effective in clinical trials, our approach has the potential to provide a novel therapy for this orphan disease with significant unmet medical need," Schenk said.
The multi-centre study will determine a recommended dose of NEOD001 for testing in Phase II trials.
NEOD001, which specifically targets the amyloid that accumulates in both AL and AA forms of amyloidosis, obtained orphan drug designation from FDA in 2012, and from the European Medicines Agency in 2013.
Prothena research and development head and chief scientific officer Gene Kinney said; "The orphan drug designations we have received in the US and EU recognise the rarity of this disease and will help us advance the development of our potential treatment for these patients."