Full Press Release Details
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
Presented Clinical Data from Patisiran, Revusiran, Fitusiran, and
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 -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--August 4, 2016--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
"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
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
Reported positive interim clinical results from Phase 1 study of
fitusiran for the treatment of hemophilia and rare bleeding
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
Continued dosing hemophilia patients in ongoing Phase 1/2 OLE
study, with patients currently having received up to 13 months
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
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
Advanced investigational pipeline programs in Hepatic Infectious
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.
Expanded Management Team with appointments of Adrian Dana, M.D., Vice
President of Drug Safety and Pharmacovigilance; Brendan Martin,
General Manager, UK and Ireland; Jeffrey Miller, Vice President,
General Manager, CC5 Program; and Andrew Slugg, Vice President of
Upcoming Events in 2H 2016
Alnylam announces today that it plans to present clinical data at