Genzyme Corporation, a biotechnology company, engages in the discovery and development of products and services primarily in the areas of rare genetic diseases, multiple sclerosis, cardiovascular diseases, and endocrinology. It offers Cerezyme to treat gaucher disease type I; Fabrazyme to treat fabry disease; Aldurazyme for mucopolysaccharidosis I; Myozyme and Lumizyme for pompe disease; Renagel that reduces phosphorous levels in chronic kidney disease (CKD) patients who are on dialysis; Renvela for use in dialysis patients and patients with earlier stage CKD; Hectorol, a vitamin D2 treatment for secondary hyperparathyroidism in CKD patients; Thyrogen to use in thyroid cancer follow-up; Leuk...
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Alnylam Pharmaceuticals, Inc., Genzyme Corporation - Special Call
Jul 10 17
To discuss new positive results from the ongoing phase 2 open-label extension (OLE) study with fitusiran in patients with hemophilia A and B, with or without inhibitors (N=33)
Sanofi Genzyme and Alnylam Pharmaceuticals, Inc Announces New Positive Results from the Ongoing Phase 2 Open-Label Extension (OLE) Study with Fitusiran in Patients with Hemophilia A and B, with or Without Inhibitors (N=33)
Jul 10 17
Sanofi Genzyme and Alnylam Pharmaceuticals, Inc. announced new positive results from the ongoing phase 2 open-label extension (OLE) study with fitusiran in patients with hemophilia A and B, with or without inhibitors (N=33). These results were presented July 10, 2017 in an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, being held from July 8 - 13, 2017 in Berlin, Germany. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT with the goal of promoting sufficient thrombin generation upon activation of the clotting cascade to restore hemostasis and prevent bleeding. The companies also announced that phase 1 clinical trial results demonstrating an encouraging preliminary safety and tolerability profile and initial evidence that monthly subcutaneously administered fitusiran lowered AT levels and increased thrombin generation in patients with hemophilia A and B without inhibitors were published online July 10, 2017 and will appear in the September 7, 2017, print issue of The New England Journal of Medicine (NEJM). The updated clinical results in the fitusiran phase 2 OLE study showed that the safety and tolerability profile of fitusiran remains encouraging, with no thromboembolic events, including during co-administration of replacement factor or bypassing agents. The majority of adverse events (AEs) were mild or moderate in severity, with the most common AEs consisting of transient, mild injection site reactions (ISRs). In addition, once-monthly subcutaneous (SC) administration of fitusiran achieved lowering of AT, increases in thrombin generation, and, in a post-hoc exploratory analysis, reductions in the median estimated annualized bleeding rate (ABR) in patients with and without inhibitors. Based on these results, the companies announced last week the initiation of the ATLAS phase 3 program for fitusiran in patients with hemophilia A and B with or without inhibitors. The ongoing fitusiran phase 2 OLE study includes patients (N=33) with hemophilia A (N=27) and hemophilia B (N=6). The study includes 14 patients with inhibitors, including one with hemophilia B. Fitusiran was administered as a low volume (less than 1 mL), monthly, subcutaneous, fixed dose of 50 mg (N=13) or 80 mg (N=20). All results are as of a June 15, 2017 data transfer date. Patients were treated for up to 20 months in the phase 2 OLE, with a median of 11 months on study. The majority of AEs were mild or moderate in severity, with the most common non-laboratory AEs consisting of transient, mild ISRs (18% of patients). There was one discontinuation due to an AE, an asymptomatic alanine aminotransferase (ALT) elevation in a patient with chronic hepatitis C virus (HCV) infection. Serious adverse events (SAEs) considered possibly related to drug were reported in two patients: asymptomatic ALT elevation in one patient with chronic HCV infection, as noted above, and seizure with confusion in one patient with a prior history of seizure disorder. Asymptomatic ALT increases greater than 3x the upper limit of normal (ULN), without concurrent elevations in bilirubin greater than 2x ULN, were observed in 11 patients, all of whom were hepatitis C antibody positive; at current follow-up, all ALT elevations are resolved (N=10) or resolving (N=1). No thromboembolic events, laboratory evidence for pathological clot formation, or instances of anti-drug antibody (ADA) formation were reported. Regarding clinical activity results, treatment with fitusiran resulted in approximately 80% lowering of AT with corresponding increases in thrombin generation. Increases in thrombin generation remained within the lower end of the range of values observed in normal healthy volunteers. In an exploratory post-hoc analysis of bleeding events, a median ABR of one (interquartile range [IQR]: 0-3) was achieved for all patients (N=33), and a median ABR of zero (IQR: 0-3) was achieved for the subset of patients with inhibitors (N=14), corresponding favorably to pre-study median ABR values of 20 (IQR: 4-36) in all patients and 38 (IQR: 20-48) in inhibitor patients. There was a high proportion of patients (16 of 33; 48%) who remained bleed-free in the observation period, and most patients (22 of 33; 67%) experienced zero spontaneous bleeds. All breakthrough bleed events were successfully managed with replacement factor (recombinant factor VIII or recombinant factor IX) or bypassing agents (recombinant factor VIIa or activated prothrombin complex concentrate).
Sanofi Genzyme Presents at BIO International Conference 2017, Jun-19-2017 through Jun-22-2017
May 24 17
Sanofi Genzyme Presents at BIO International Conference 2017, Jun-19-2017 through Jun-22-2017. Venue: San Diego Convention Center, San Diego, CA 2865, United States. Presentation Date & Speakers: Jun-19-2017, Franqui Jimenez, Sr. Director, MSAT. Jun-20-2017, Carlo Incerti, Head Global Medical Affairs, David Meeker, Executive Vice President and Head, Stacy Coen, Head of Global Rare Disease Business Development and Licensing.