Alnylam Pharmaceuticals, Inc. ALNY announced that pre-clinical results on ALN-AT3, the company’s investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), were published in Nature Medicine.
Results from the study revealed that subcutaneous administration of ALN-AT3 helped in potent, dose-dependent and durable knockdown of AT in wild-type mice, hemophilia A mice and non-human primates. Moreover, ALN-AT3 treatment improved hemostasis in hemophilia mice and normalized thrombin generation in a non-human primate “inhibitor” model of hemophilia A. ALN-AT3 was well tolerated in hemophilia mice in exaggerated doses.
ALN-AT3 is being developed for the treatment of hemophilia in a two-part phase I study. The company has already completed the Part A of the study and Part B is ongoing. The primary objective of the second part of the study is to evaluate the safety and tolerability of ALN-AT3 in hemophilia subjects.
Alnylam currently has no approved product in its portfolio. Although the company has a number of pipeline candidates, most of the candidates are in early or mid stages of development. The company has only a couple of late-stage candidates in its pipeline – patisiran (TTR-mediated amyloidosis in familial amyloidotic polyneuropathy patients – phase III) and revusiran (familial amyloidotic cardiomyopathy – phase III). The successful development and commercialization of these candidates are crucial for the company.
We expect investor focus to remain on the company’s pipeline.