28 Apr, 2014 Positive Initial Data from Phase 2 Open-Label Extension (OLE) Study with Patisiran
We have reported positive initial data from our Phase 2 open-label extension (OLE) study with patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of TTR-mediated amyloidosis (ATTR). Data were presented at the International Symposium on Amyloidosis, held April 27 – May 1, 2014. Preliminary results showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the approximately 80% target level through 168 days. Moreover, OLE results showed a favorable tolerability profile with up to eight doses administered. These data provide the first clinical evidence of sustained RNAi-mediated TTR knockdown in FAP patients beyond two doses of patisiran.
In addition, we have presented results of a 283-patient natural history study of patients with FAP. This cross-sectional analysis demonstrated a rapid progression in Neuropathy Impairment Score (NIS) and a high correlation of this measurement with disease severity. Moreover, these results give us confidence that our APOLLO Phase 3 trial of patisiran in FAP patients is robustly powered to show the potential impact of TTR lowering on the mNIS+7 endpoint used in that study.