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Alnylam Pharmaceuticals Reports Second Quarter 2016 Financial Results and Highlights Recent Period Progress
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8-4-16 4:00 PM EDT | Email Article
Alnylam Pharmaceuticals Reports Second Quarter 2016 Financial Results and Highlights Recent Period Progress

– Advanced Two Programs into Phase 1 Clinical Studies: ALN-TTRsc02 for Transthyretin-Mediated Amyloidosis and ALN-HBV for Chronic Hepatitis B Virus Infection –

– Presented Clinical Data from Patisiran, Revusiran, Fitusiran, and ALN-CC5 Programs –

– Maintained Strong Balance Sheet with $1.28 Billion in Cash and Remains On Track to End 2016 with Greater than $1.0 Billion in Cash –

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter 2016, and highlighted recent progress in advancing its pipeline.

“We continue to advance our pipeline of now ten investigational RNAi therapeutics, an entirely new and innovative class of medicines, across a broad range of diseases. We believe our two latest stage programs, patisiran and revusiran, have demonstrated encouraging progress for patients with hATTR amyloidosis,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We also recently presented new results in our hemophilia program showing that a once-monthly, subcutaneous regimen of fitusiran can achieve a median estimated annualized bleeding rate of zero in hemophilia A and B patients. In addition, we presented encouraging initial data in hemophilia patients with inhibitors. Through the end of the year, we are anticipating a very data rich period marked with over six planned clinical readouts – we look forward to sharing our continued progress.”

Second Quarter 2016 and Recent Significant Corporate Highlights

Advanced investigational pipeline programs in Genetic Medicine Strategic Therapeutic Area (STAr).
Advanced investigational RNAi therapeutic programs for the treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Reported positive initial 24-month data from ongoing Phase 2 open-label extension (OLE) study with patisiran for the treatment of hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Results showed that patisiran can potentially halt or improve neuropathy progression. Patisiran administration was also found to be generally well tolerated in hATTR-PN patients out to 25 months, with no drug-related serious adverse events (SAEs) reported through the data transfer date.
Presented baseline demographic data from APOLLO Phase 3 study of patisiran, revealing enrollment of a globally representative patient population with a wide range of disease severity and TTR mutations.
Reported initial 12-month results from ongoing Phase 2 OLE study with revusiran for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM).
Initiated Phase 1 clinical trial for ALN-TTRsc02, an Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a low volume, once quarterly, subcutaneous dosing regimen.
Reported positive interim clinical results from Phase 1 study of fitusiran for the treatment of hemophilia and rare bleeding disorders (RBD).
Fitusiran achieved a median estimated annualized bleeding rate (ABR) of zero in hemophilia patients without inhibitors. In the initial low-dose cohort of patients with inhibitors, fitusiran achieved antithrombin lowering, increased thrombin generation, and preliminary evidence for reduced bleeding. Fitusiran administration was generally well tolerated in patients with and without inhibitors, with no SAEs related to study drug, and no thromboembolic events or laboratory evidence (based on D-dimer, platelet count, fibrinogen, and/or PT/INR) of pathologic clot formation through the data transfer date.
Continued dosing hemophilia patients in ongoing Phase 1/2 OLE study, with patients currently having received up to 13 months of dosing.
The Company also updated its guidance for Phase 3 initiation, and now plans to start studies in early 2017.
Reported initial clinical results in patients with paroxysmal nocturnal hemoglobinuria (PNH) from ongoing Phase 1/2 study of ALN-CC5 for the treatment of complement-mediated diseases.
Initial data support the potential for ALN-CC5 to reduce the dose and frequency of eculizumab, as well as to improve disease control in eculizumab inadequate responders.
ALN-CC5 was generally well tolerated in patients with PNH after multiple doses, with the majority of adverse events (AEs) being mild or moderate in severity. There were no drug related SAEs or discontinuations due to AEs in the study through the data transfer date.
The Company announces today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to ALN-AS1 for the treatment of acute hepatic porphyrias.
The Company also announces today the publication of pre-clinical data with ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1) in the Journal of the American Society of Nephrology (Liebow et al., J Am Soc Nephrol, 2016; doi:10.1681/ASN.2016030338).
Advanced investigational pipeline programs in Cardio-Metabolic Disease STAr.
The Medicines Company announced completion of enrollment in its Phase 2 ORION-1 study for ALN-PCSsc (also known as PCSK9si). The trial enrolled 501 patients with atherosclerotic cardiovascular disease (ASCVD), exceeding the original enrollment target of 480 patients.
Advanced investigational pipeline programs in Hepatic Infectious Disease STAr.
Initiated Phase 1/2 clinical trial with ALN-HBV for the treatment of hepatitis B virus (HBV) infection.
Broke ground on new manufacturing facility in Norton, Massachusetts. The 200,000 square foot, state-of-the-art facility is being built to support the Company’s expanding development pipeline and transition toward commercial stage.
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