Alnylam Pharma Announces Positive Initial Phase 2 Data With Revusiran

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), a RNAi therapeutics company, announced positive initial Phase 2 data with revusiran, an investigational RNAi therapeutic targeting transthyretin or TTR for the treatment of TTR cardiac amyloidosis.

In the pilot Phase 2 study, revusiran was found to be generally well tolerated in TTR cardiac amyloidosis patients. Revusiran showed clinical activity with an up to 98.2% knockdown of serum TTR, the disease causing protein. This included similar knockdown effects toward wild type and mutant TTR protein within V122I patients, who represent the most common genotype associated with inherited forms of TTR cardiac amyloidosis. In the five-week course of treatment, there were no significant changes observed in several exploratory clinical measurements. Revusiran utilizes Alnylam's proprietary GalNAc-conjugate delivery platform that enables subcutaneous delivery of RNAi therapeutics with a wide therapeutic index.

"Our pilot Phase 2 study was designed to evaluate the tolerability and initial clinical activity of revusiran in patients with TTR cardiac amyloidosis. These initial results demonstrate that revusiran is generally well tolerated in patients with significant disease burden. In addition, we continue to be impressed with the level of knockdown - up to 98.2% - achieved with revusiran toward both mutant and wild-type TTR. In fact, this level of knockdown is the greatest ever reported for an RNAi therapeutic in clinical studies. As would be expected with the short treatment duration of five weeks, there were no significant changes in the exploratory clinical measurements performed," said Akshay Vaishnaw, M.D., Executive Vice President and Chief Medical Officer of Alnylam.

TTR-mediated amyloidosis or ATTR is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene.