PRX004 is an investigational antibody designed to deplete the pathogenic, non-native forms of the TTR protein (misTTR found in ATTR amyloidosis), without affecting the normal tetrameric form (TTR) of the protein. Positive 9 month results from the Phase 1 long-term extension study were reported in December 2020. PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months, including improvement in neuropathy in 3 of the 7 patients, and improved cardiac systolic function for all 7 patients. In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels.
More information on the Phase 1 clinical study of PRX004 in patients with ATTR amyloidosis can be found on www.clinicaltrials.gov, by searching NCT03336580.