Alnylam Reports Positive Top-line Results From Ongoing ALN-TTRsc Phase I Trial

(RTTNews) - Alnylam Pharmaceuticals, Inc.(ALNY), a RNAi therapeutics company, Thursday announced positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin or TTR gene. The targeted compound is intended for the treatment of TTR-mediated amyloidosis or ATTR, an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene.

The company said ALN-TTRsc achieved statistically significant knockdown of serum TTR protein levels of greater than 80% in healthy volunteer subjects, in line with results for ALN-TTRsc previously reported in non-human primates. In addition, to date ALN-TTRsc was found to be generally safe and well tolerated. The company plans presentation of data from the Phase I trial at the HFSA meeting in September, and to start Phase II study in familial amyloidotic cardiomyopathy patients by the end of this year.