30 Jun, 2013 Positive Phase II Clinical Data with ALN-TTR02
We presented positive data from our Phase II clinical trial of ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR) at the Biennial Meeting of the Peripheral Nerve Society being held June 29 – July 3. Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant knockdown of serum TTR protein levels of up to 93%. 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, ALN-TTR02 was found to be generally safe and well tolerated in this study.