Positive Phase II Data with Patisiran (ALN-TTR02)

Positive Phase II Data with Patisiran (ALN-TTR02)

We presented positive data from our Phase II clinical trial of patisiran (ALN-TTR02) for the treatment of transthyretin-mediated amyloidosis (ATTR) at the International Symposium on Familial Amyloidotic Polyneuropathy, November 10 – 13, 2013. Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%.  Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein.   In addition, patisiran was found to be generally safe and well tolerated in this study.

These new data are consistent with our earlier experience from pre-clinical and Phase I clinical studies showing excellent translation of RNAi therapeutics. In total, we are very encouraged by these clinical activity and safety data with patisiran.

We have also initiated the APOLLO Phase III trial of patisiran, with the study now open for enrollment.  APOLLO is designed to evaluate the efficacy and safety of patisiran in ATTR patients with familial amyloidotic polyneuropathy (FAP), and, if successful, is intended to support marketing authorization.


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