wo fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophiliannBoth Phase 3 studies achieved their primary and secondary endpoints; fitusiran prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all hemophilia populations, presented at ASH 2021nnParis – April 4, 2023 – Two studies, published in The Lancet and The Lancet Haematology, evaluating the efficacy and safety of fitusiran, an investigational siRNA therapy for the prophylactic treatment of adults and adolescents with hemophilia A or B, reinforce the potential of this investigational therapy to transform the current standard of care and address unmet needs for all types of hemophilia, regardless of inhibitor status.nnHemophilia A and B are rare congenital lifelong bleeding disorders in which the ability of a person’s blood to clot is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and significantly impact quality of life. Fitusiran has the potential to provide prophylaxis for all types of hemophilia, regardless of inhibitor status, with as few as six subcutaneous injections per year.nnDietmar Berger, M.D., Ph.D.nHead of Global R&D ad interim and Chief Medical Officer at Sanofin“Sanofi is committed to advancing the standard of care for all people with hemophilia through innovative science, providing consistent bleed protection while reducing treatment burden. We are entering a new era in hemophilia where, for the first time, people can choose therapies that meet their personal needs. These published data validate our science and add to a growing body of evidence supporting fitusiran’s potential to transform the treatment landscape. We look forward to sharing additional data on fitusiran later this year.”nnBoth Phase 3 studies compared once-monthly subcutaneous fitusiran prophylaxis (80mg) with on-demand/episodic use of clotting factor concentrates in the ATLAS-A/B study, and on-demand/episodic use of bypassing agents in the ATLAS-INH study. Across both clinical studies, prophylactic treatment with fitusiran reduced annualized bleeding rates by 90% (95% CI [84.1%; 93.6%], P <0.0001) compared to the control arms, showing a statistically significant and clinically meaningful improvement in bleeding episodes when compared to on-demand treatments; and showed improvement in quality of life.nnIn the study ATLAS-INH study published by The Lancet, 66% of participants with inhibitors (25 out of 38) receiving fitusiran 80mg monthly experienced zero bleeding episodes compared to 5% (1 out of 19) receiving an on-demand bypassing agent after nine months of treatment.nnThe ATLAS A/B study published in The Lancet Haematology showed 51% of participants without inhibitors (40 out of 79) who received fitusiran 80mg monthly prophylaxis experienced zero bleeds compared to 5% (2 out of 40) in the comparator group, receiving on-demand clotting factor concentrates.nnSanofi is currently investigating the efficacy and safety of fitusiran under a revised regimen which includes lower doses and less frequent dosing (as few as six subcutaneous injections per year), maintaining an antithrombin target range of 15-35% in all ongoing studies.nnATLAS-AB Phase 3 StudynATLAS-A/B is a Phase 3 randomized, open-label study investigating the efficacy and safety of fitusiran in males ?12 years with severe hemophilia A or B without inhibitors who had previously been treated with on-demand clotting factor concentrates. Study participants (n=120) were randomized 2:1 to receive either once-monthly 80mg subcutaneous fitusiran prophylaxis or on-demand clotting factor concentrates. The primary endpoint is annualized bleeding rate.nnATLAS-INH Phase 3 StudynThe ATLAS-INH study is a randomized, open-label Phase 3 study designed to evaluate the safety and efficacy of fitusiran in males ?12 years with severe hemophilia A or B with inhibitors to factor VIII or IX. Study participants (n=57) receiving on-demand treatment with bypassing agents (BPA) were randomized in a 2:1 ratio to receive once-monthly 80mg subcutaneous fitusiran prophylaxis or continue with on-demand BPA. The primary endpoint is annualized bleeding rate.nnAbout fitusirannFitusiran is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors. Fitusiran is designed to lower antithrombin, a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Fitusiran utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability. Fitusiran is currently under clinical investigation and has not been evaluated by any regulatory authority.
Sanofi R&D in its core haemophilia category is advancing on three fronts – engineered proteins, RNA interference drugs and gene therapies – and highlighted results in two of those at the International Society on Thrombosis and Haemostasis (ISTH) congress over the weekend.
The data come from a trial of fitusiran, an RNA-modulating drug, in both hemophilia A and hemophilia B patients who have not developed inhibitors — a type of immune system response that reduces the effectiveness of standard treatments. Over a period of more than six months, 40 of the 79 patients given fitusiran didn't have bleeds that required treatment with so-called factor replacement therapies, compared with an annual bleed rate of 21.8 for those receiving typical drugs.
Paris – December 14, 2021 - Positivendata from two Phase 3 studies evaluating the efficacy and safety of nfitusiran, an investigational small interference RNA (siRNA) therapy fornthe prophylactic treatment of adults and adolescents with hemophilia A nor B, with or without inhibitors, are presented at the 63rd nAmerican Society of Hematology (ASH) Annual Meeting. Results from the nATLAS-A/B study, investigating fitusiran in people without inhibitors, nare being presented today in the Late-Breaking Abstract Session and nfindings from the ATLAS-INH study, which evaluated fitusiran in people nwith inhibitors to factor VIII or IX, were shared in the Plenary nScientific Session on December 12, 2021.
Pfizer/BioNTech, Moderna expect data on shot`s protection against new variant
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2021 and reviewed recent business highlights.
Sanofi’s response to nonfatal thrombotic events in a phase 3 hemophilia program has delayed its plans to seek approval for RNAi drug fitusiran by 18 months. The delay will “allow for the appropriate collection and assessment of safety and efficacy data under the amended protocols.”
Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors. Sanofi’s first priority is patient safety. The company voluntarily paused dosing in all ongoing fitusiran clinical studies on October 30, 2020 to assess reports of non-fatal thrombotic events in patients participating in the Phase 3 program.
Sanofi is getting a big part of its late-stage clinical program for fitusiran back on track after working out an agreement with the FDA on a new set of protocols. They’re getting back into the clinic with adult and adolescent patients.
When Alnylam scored an FDA approval for its second RNAi therapy, Givlaari, late last year, some market watchers cast a wary eye on the drug's short stay in the clinic and questionable safety profile. But after eight months on the market, Alnylam is touting longer-term, open-label data for Givlaari that could put some of those worries to rest.