Alnylam Announces Data From Patisiran Phase 2 OLE Study In Patients With FAP

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) announced six-month clinical data from its ongoing Phase 2 open-label extension or OLE study with patisiran or ALN-TTR02, an investigational RNAi therapeutic in development for the treatment of transthyretin or TTR-mediated amyloidosis or ATTR in patients with familial amyloidotic polyneuropathy or FAP.

Results showed a mean 0.95 point decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients with mNIS+7 data available for the current analysis. This decrease in neuropathy progression compares favorably with the 7 to 10 point increase in mNIS+7 at six months that can be estimated from historical data sets in untreated FAP patients with similar baseline characteristics.

In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for over nine months, with an up to 89.6% knockdown achieved between doses.

Patisiran was found to be generally well tolerated in this study out to one year of therapy, with no drug-related serious adverse events to date, and all 27 patients enrolled in the study continue to receive drug treatment.