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April 27, 2015 12:45 PM 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. The company’s clinical development programs include Patisiran and Revusiran, which are in Phase III clinical trials for the treatment of transthyretin-mediated amyloidosis; ALN-AT3 that is in Phase I clinical trial for hemophilia and rare bleeding disorders; ALN-CC5, which is in Phase I/II clinical trial for the treatment of complement-mediated diseases; and ALN-PCSsc that is in Phase I clinical trial for hypercholesterolemia. Its product development programs also include ALN-AS1 for the treatment of hepatic porphyrias; ALN-AAT for the treatment of...

300 Third Street

3rd Floor

Cambridge, MA 02142

United States

Founded in 2002

256 Employees

Phone:

617-551-8200

Fax:

617-551-8101

Key Executives for Alnylam Pharmaceuticals, Inc.

Chief Executive Officer and Executive Director
Age: 51
Total Annual Compensation: $689.6K
President and Chief Operating Officer
Age: 51
Total Annual Compensation: $515.0K
Vice President of Finance and Treasurer
Age: 39
Total Annual Compensation: $257.2K
Chief Medical Officer and Executive Vice President of Research and Development
Age: 51
Total Annual Compensation: $463.5K
Senior Vice President, General Counsel and Secretary
Age: 60
Total Annual Compensation: $210.8K
Compensation as of Fiscal Year 2014.

Alnylam Pharmaceuticals, Inc. Key Developments

Alnylam Pharmaceuticals, Inc. - Special Call

To discuss these new phase 2 open-label extension study results with patisiran for the treatment of familial amyloidotic polyneuropathy

Alnylam Reports 12-Month Clinical Data from Phase 2 Open Label Extension (OLE) Study of Patisiran

Alnylam Pharmaceuticals, Inc. announced initial 12-month clinical data from its ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP). These new clinical data are being presented at the 67th Annual Meeting of the American Academy of Neurology (AAN) being held April 18 to 25 in Washington, D.C. Study results showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. This decrease in neuropathy progression compares favorably to the 13 to 18 point increase in mNIS+7 at 12 months that can be estimated from the literature in untreated FAP patients with similar baseline characteristics. In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for approximately 16 months, with an up to 88% mean knockdown achieved between doses. In aggregate, these results are consistent with the therapeutic hypothesis that TTR knockdown has the potential to halt neuropathy progression in patients with FAP. Patisiran was also found to be generally well tolerated out to 17 months of drug administration, with no drug-related serious adverse events to date; all 27 patients enrolled in the study continue to receive patisiran. Alnylam has also announced that it plans to report 18-month OLE data in late 2015. Patisiran is being administered once every 3 weeks at a dose of 0.3 mg/kg by intravenous infusion. The study is measuring a number of clinical endpoints every six months, including mNIS+7 which is an evaluation of muscle weakness, sensory and autonomic function, and nerve conductance, where neuropathy progression leads to an increased score over time. The change in the mNIS+7 measurement from baseline to 18 months is the primary endpoint in the company's Phase 3 APOLLO trial of patisiran in FAP patients. New results presented at the AAN meeting for patients (N=20) who reached the 12-month endpoint as of a data cut off of March 13, 2015 showed that neuropathy impairment scores were essentially unchanged from baseline values after 12 months of treatment. A number of additional exploratory clinical measures are also being assessed in the OLE study, including: quality of life (QOL); timed 10-meter walk test (10MWT) to evaluate mobility; hand grip strength test; modified body mass index (mBMI) as a measure of nutritional status; level of disability by R-ODS; autonomic neuropathy symptoms by COMPASS-31; and nerve fiber density in skin biopsies. New results presented at the AAN meeting showed that these clinical measures were largely unchanged over the 12-month evaluation period. Patients with cardiac abnormalities at baseline comprise a cardiac subgroup (N=11) in the study, where cardiac biomarkers (NT-proBNP and troponin I) and echocardiographic parameters are measured at baseline and every three or six months, respectively. Results in the cardiac subgroup showed no clinically significant changes in cardiac biomarkers (N=7-8) or in echocardiographic parameters (N=6-7) after 12 months of dosing. Finally, serum TTR levels are being measured throughout the OLE study. New results showed that repeat dosing with patisiran achieved sustained mean TTR knockdown at the 80% target level for approximately 16 months, and an up to 88% mean level of TTR knockdown was achieved in between doses. A similar degree of TTR knockdown was observed in patients with or without concurrent use of TTR tetramer stabilizers. Patisiran administration was also found to be generally well tolerated in FAP patients (N=27), with minimal adverse events reported for a period of up to 17 months. As of the time of the current data cutoff on March 13, 2015, 511 doses had been administered with a median of 19 doses per patient. Mean treatment duration was 13 months and the longest treatment duration was out to 17 months. There were no drug-related serious adverse events. The most common drug-related or possibly drug-related adverse events were flushing (22.2%) and infusion-related reactions (18.5%), which were both mild in severity and did not result in any discontinuations. Additional reported drug-related adverse events seen in >5% of patients were mild to moderate in severity and included diarrhea (7.4%) and peripheral edema (7.4%). There were no clinically significant changes in liver function tests, renal function tests, or other laboratory or hematological parameters.

Alnylam Pharmaceuticals, Inc. Announces the Publication in Nature Medicine of Pre-Clinical Results with ALN-AT3

Alnylam Pharmaceuticals, Inc. announced the publication in Nature Medicine of pre-clinical results with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD). The paper, titled 'An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia' (Sehgal et al., Nat Med, doi:10.1038/nm.3847),documents a broad set of pre-clinical data supporting the clinical advancement of ALN-AT3. Among the many findings reported, subcutaneous administration of ALN-AT3 led to potent, dose-dependent, and durable knockdown of AT in wild-type mice, hemophilia A mice, and non-human primates (NHPs). In addition, ALN-AT3 treatment improved hemostasis in hemophilia mice and normalized thrombin generation in a non-human primate 'inhibitor' model of hemophilia A (HA). Furthermore, long-term ALN-AT3 administration, even at highly exaggerated doses, was shown to be well tolerated in hemophilia mice, supporting a wide therapeutic index in the disease setting. As documented in the new publication, single and multiple subcutaneous doses of ALN-AT3 led to dose-dependent and durable knockdown of serum AT in wild-type and HA mice and in NHPs. In microvessel laser injury and saphenous vein bleeding models in HA mice, subcutaneous administration of ALN-AT3 provided hemostatic protection that was comparable to or better than that achieved with intravenously administered factor VIII replacement therapy. Furthermore, in wild type NHPs, repeat dosing with ALN-AT3 resulted in potent, titratable, and reversible knockdown of plasma AT. Studies were also performed in an NHP hemophilia 'inhibitor' model, in which a hemophilia phenotype was induced via administration of a polyclonal anti-factor VIII antibody. ALN-AT3 treated animals showed robust AT knockdown as well as dose-dependent increases in thrombin generation, restoring this hemostatic parameter back to normal levels. These results demonstrate that ALN-AT3 can normalize thrombin generation in the absence of functional levels of factor VIII and/or in the presence of anti-factor VIII antibodies in a large animal model, providing key pre-clinical proof of concept for the program. Alnylam is currently evaluating ALN-AT3 in a Phase 1 clinical trial in subjects with hemophilia. The ongoing study is being conducted in Bulgaria, Switzerland and the U.K. as a single- and multi-dose, dose-escalation study comprised of two parts. Part A, which is complete, was a randomized, single-blind, placebo-controlled, single-dose, dose-escalation study (n=4 per cohort; 3:1 randomization of ALN-AT3:placebo) in healthy volunteer subjects. This part of the study was completed after the first dose cohort that received a single subcutaneous dose of ALN-AT3 at 30 micrograms/kilogram (mcg/kg). Part B of the study, which is ongoing, is an open-label, multi-dose, dose-escalation study enrolling up to 18 subjects with moderate or severe hemophilia A or B. The primary objective of this part of the study is to evaluate the safety and tolerability of multiple doses of subcutaneously administered ALN-AT3 in hemophilia subjects. Secondary objectives include assessment of clinical activity as determined by knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3. In addition, the study is recording potential effects of ALN-AT3 on the incidence of bleeding.

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