Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today initial 12-month clinical data from its ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP).
These new clinical data are being presented at the 67th Annual Meeting of the American Academy of Neurology (AAN) being held April 18 – 25 in Washington, D.C. Study results showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff.
This decrease in neuropathy progression compares favorably to the 13 to 18 point increase in mNIS+7 at 12 months that can be estimated from the literature 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 approximately 16 months, with an up to 88% mean knockdown achieved between doses.
