Alnylam: Positive Clinical Results for Gene Knockdown Therapy Validates RNAi Mechanism, Candidate Advances to Phase II

Alnylam (NASDAQ: ALNY) reported positive Phase I proof-of-concept and safety results for ALN-TTR02, the company’s most advanced wholly-owned compound. ALN-TTR02 is being developed to treat Transthyretin-Mediated Amyloidosis (or ATTR), which is a hereditary disease caused by mutations in the TTR gene. In its severest form, ATTR represents a major unmet medical need with significant morbidity and mortality as an orphan disease. At least 50,000 people are affected worldwide. Results from this study show that administration of ALN-TTR02 leads to robust knockdown of serum TTR protein levels of up to 94%. The results were highly statistically significant (p<0.00001). The drug was found to be generally safe and well tolerated, and importantly, results from the study validate the ability of RNAi technology to safely and effectively target and “silence” genes implicated in disease. Given the favorable results, ALNY is advancing the drug in a Phase II study, and plans to initiate pivotal trials next year. The company is hosting a conference call on the news for investors at 8:30am. Shares up strong in pre-market, expect continued momentum through the day.