Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today the achievement of positive clinical results from its Phase II trial of patisiran (ALN-TTR02), an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The data are being presented at the IXth International Symposium on Familial Amyloidotic Polyneuropathy (ISFAP) being held in Rio de Janeiro, Brazil, November 10 – 13. 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. The company also announced today the initiation of the APOLLO Phase III trial of patisiran in ATTR patients with FAP, with the study now open for enrollment.
“These new data confirm what we have seen consistently with our patisiran program, namely that we can achieve robust knockdown of circulating wild-type and mutant TTR. Specifically, in this Phase II study, we demonstrated up to 96% knockdown of TTR, the disease-causing protein in ATTR, and established a dose and dose regimen for evaluation in our Phase III trial. Knockdown of circulating TTR is expected to result in improved clinical outcomes for patients with ATTR based on data from FAP patients receiving liver transplants. Further, evidence from other systemic amyloidotic diseases shows that as little as a 50% reduction of the disease-causing protein can result in disease improvement or stabilization,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “In addition, we continue to be very encouraged with the safety profile of patisiran which has now been extended with this experience in ATTR patients and with multi-dose regimens. By all accounts, these new data are consistent with our earlier experience from pre-clinical and Phase I clinical studies showing excellent translation of RNAi therapeutics, and support our belief that patisiran has the potential to be best-in-class for the treatment of ATTR patients with polyneuropathy.”