Alnylam: Patisiran Has Potential To Halt Neuropathy Progression In FAP Patients

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), an RNAi therapeutics company, announced new results from its ongoing Phase 2 open-label extension studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin or TTR for the treatment of TTR-mediated amyloidosis called ATTR amyloidosis.

Data from the patisiran Phase 2 OLE study provided continued evidence following 18 months of dosing that patisiran has the potential to halt neuropathy progression in patients with Familial Amyloidotic Polyneuropathy or FAP.

The familial amyloid neuropathies are a rare group of autosomal dominant diseases wherein the autonomic nervous system and/or other nerves are compromised by protein aggregation and/or amyloid fibril formation.

The company expects to complete enrollment in the APOLLO Phase 3 trial of patisiran over the next three to four months, supporting a potential New Drug Application filing in 2017 if the study is positive.

In addition, initial data from the revusiran Phase 2 OLE study showed robust and sustained knockdown of serum TTR. It was found to be generally well tolerated in the majority of patients with TTR cardiac amyloidosis, including patients with Familial Amyloidotic Cardiomyopathy and Senile Systemic Amyloidosis, out to 10 months of treatment.

These new clinical data were presented at the 1st European Congress on Hereditary ATTR amyloidosis held November 2 - 3, 2015 in Paris.