Alnylam Pharma Says Positive Interim Results From Ongoing Study With Fitusiran

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) announced positive interim results from Part D of its ongoing Phase 1 study with fitusiran, an investigational RNAi therapeutic, in patients with hemophilia with inhibitors. The company plannned to initiate Phase 3 program in early 2017.

The results were presented at 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 - 6, 2016 in San Diego, California.

New clinical data showed that once-monthly subcutaneous administration of fitusiran achieved lowering of AT and increases in thrombin generation, resulting in a median estimated annualized bleeding rate (ABR) of zero in patients with hemophilia A or B with inhibitors (N=16).

In addition, fitusiran was generally well tolerated through the data cut-off date, October 6, 2016, with no thromboembolic events, were presented from Part D of the ongoing fitusiran Phase 1 study, which included patients with hemophilia A or B with inhibitors who were enrolled in two separate dose cohorts of 50 mg, once-monthly (N=6) or 80 mg, once-monthly (N=10). Treatment with fitusiran resulted in potent and dose-dependent lowering of AT and increases in thrombin generation.

Additional data on longer-term administration of fitusiran in patients without inhibitors will be presented in a separate poster presentation at ASH on December 4.

In an exploratory analysis of bleeding events, a median ABR of zero was achieved for patients in combined dose cohorts in the observation period, compared to the pre-study median ABR of 31. The majority of patients treated in both cohorts (9 of 16; 56 percent) were bleed-free and most patients (11 of 16; 69 percent) experienced zero spontaneous bleeds. In the 80 mg cohort, 70 percent (7 out of 10) of patients were bleed-free and 90 percent (9 out of 10) of patients experienced zero spontaneous bleeds.