Positive Phase II Clinical Data with ALN-TTR02

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.


These results represent a major milestone in our TTR program. For the first time in patients with ATTR, we have demonstrated robust knockdown of circulating wild-type and mutant TTR in a multi-dose study. The safety profile of ALN-TTR02, which has now been further supported with this experience in ATTR patients and with multi-dose regimens, continues to be very encouraging. TTR protein reduction in ATTR patients has the potential to delay or even reverse disease progression with associated clinical benefits.

We plan to review these data, as well as progress from other Alnylam 5×15 programs, at our upcoming R&D Day on July 11, 2013 in New York City.


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