Pharmaceutical Business review

Prothena doses first patient in NEOD001 Phase I AL amyloidosis study

The multi-centre study will determine a recommended NEOD001 dose for testing in Phase 2 trials.

Prothena president and chief executive officer 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 added.

NEOD001 specifically targets the amyloid that accumulates in both AL and AA forms of amyloidosis.

The FDA granted orphan drug designation to the investigational agent in 2012 and the European Medicines Agency in 2013.