July 21, 2017 1:58 AM ET

Biotechnology

Company Overview of Alnylam Pharmaceuticals, Inc.

Company Overview

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, discovers, develops, and commercializes novel therapeutics based on RNA interference. Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases. The company’s clinical development programs include Patisiran, which is in Phase III clinical trial for the treatment of transthyretin-mediated amyloidosis (ATTR); Fitusiran, an investigational RNAi therapeutic that is in Phase I/II clinical trial for the treatment of hemophilia and rare bleeding disorders; and Inclisiran (ALN-PCSsc), which is in Phase II clinical trial for hypercholesterolemia. Its early st...

300 Third Street

3rd Floor

Cambridge, MA 02142

United States

Founded in 2002

514 Employees

Phone:

617-551-8200

Fax:

617-551-8101

Key Executives for Alnylam Pharmaceuticals, Inc.

CEO & Executive Director
Age: 54
Total Annual Compensation: $731.6K
President
Age: 54
Total Annual Compensation: $546.4K
COO & Executive VP
Age: 54
Total Annual Compensation: $244.2K
Executive Vice President of Research & Development
Age: 54
Total Annual Compensation: $526.4K
Principal Accounting Officer, Vice President of Finance and Treasurer
Age: 42
Total Annual Compensation: $309.1K
Compensation as of Fiscal Year 2016.

Alnylam Pharmaceuticals, Inc. Key Developments

Alnylam Pharmaceuticals, Inc., Genzyme Corporation - Special Call

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)

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).

Alnylam Pharmaceuticals, Inc. Announces Initiation of the ATLAS Phase 3 Clinical Program for Fitusiran

Alnylam Pharmaceuticals, Inc. announced the initiation of the ATLAS Phase 3 clinical program for fitusiran. The global, multicenter program is designed to evaluate the safety and efficacy of fitusiran in three separate trials, including patients with hemophilia A and B with or without inhibitors and patients receiving prophylactic therapy. 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 to restore hemostasis and prevent bleeding. The three separate trials are: ATLAS-INH, a nine-month, open-label, randomized, active-controlled trial designed to enroll approximately 50 patients with hemophilia A or B with inhibitors receiving prior on-demand therapy. The study’s primary endpoint is the annualized bleeding rate (ABR), and key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score. ATLAS-A/B, a nine-month, open-label, randomized, active-controlled trial designed to enroll approximately 100 patients with hemophilia A or B without inhibitors receiving prior on-demand therapy. The study’s primary endpoint is the ABR, and key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score. ATLAS-PPX, an open-label, one-way crossover study designed to enroll approximately 100 patients with hemophilia A or B with or without inhibitors receiving prophylaxis therapy as prior standard of care. In this study, patients will receive standard of care prophylaxis for six months and then transition to fitusiran treatment for seven months. The ABR will be prospectively measured in both periods. The study’s primary endpoint is the ABR in the fitusiran period and in the factor/bypassing agent prophylaxis period. Key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score.

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