We are hosting a series of online “RNAi Roundtables” in which Alnylam scientists and key clinical collaborators review recent progress in many of the company’s development-stage pipeline programs and discuss the related disease areas. Click here to access our current and previous presentations.
Interim Clinical Results from Fitusiran, an investigational RNAi therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders
We reported positive interim results from our Phase 1 study of fitusiran in patients with hemophilia with inhibitors as well as from our Phase 2 open-label extension (OLE) study in hemophilia patients without inhibitors at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.
Initial Evidence of Clinical Activity Shown with Givosiran (ALN-AS1) in Acute Intermittent Porphyria Patients with Recurrent Attacks
We reported positive initial results from Cohorts 1 and 2 in Part C of our Phase 1 study with givosiran (gi-VOH-si-ran), the International Nonproprietary Name for ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias. These results were presented at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.
We reported interim results from our ongoing EXPLORE study, a multinational, prospective, observational study aimed at characterizing the natural history and clinical management of patients with acute hepatic porphyrias (AHP) who experience recurrent attacks or receive prophylactic treatment to prevent attacks. Results from 112 patients, of which 104 have acute intermittent porphyria (AIP), showed a mean of 9.5 acute attacks reported in the prior year with severe neuropathic pain as the cardinal feature in 100% of attacks, along with other symptoms including nausea or vomiting (>80%), fatigue (77%), and weakness (79%). Approximately 64% of patients reported experiencing porphyria symptoms between attacks, with about 46% experiencing symptoms daily. Patients also reported a diminished quality of life and significant healthcare utilization, with a mean of 4.6 overnight hospitalizations per year (range 0-70) and a mean hospital stay duration of 6.6 days (range 1-60). Of the attacks reported on study, approximately 76% of them required treatment with heme or at a healthcare facility.