Full Press Release Details
Pharmaceuticals Reports Third Quarter 2015 Financial Results and
Highlights from Recent Period
Advanced Late-Stage Clinical Pipeline: Patisiran APOLLO Phase 3 Trial on
Track to Complete Enrollment in Next 3-4 Months; if Positive, Expect NDA
Presented Positive Clinical Data for ALN-PCSsc for Hypercholesterolemia
and ALN-AS1 for Acute Hepatic Porphyrias -
Maintained Strong Balance Sheet with $1.34 Billion in Cash and Expect to
End 2015 with Greater than $1.2 Billion in Cash -
Hosting R&D Day on December 10, 2015 -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 9, 2015--Alnylam
Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics
company, today announced consolidated financial results for the third
quarter 2015 and highlighted recent progress in its advancing pipeline.
"The past quarter and recent period were marked by tremendous progress
in our efforts to advance innovative medicines to patients. Notably, we
made significant progress with our late-stage clinical programs,
including new data on patisiran and revusiran. Importantly, we're on
track to complete enrollment in our APOLLO Phase 3 trial of patisiran
within the next three to four months; if the study is positive, APOLLO
data are expected to support an NDA submission in 2017," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "Overall, we
believe our new clinical data confirm the reproducible and modular
characteristics of our platform with all six programs for which we've
reported human data to date showing positive target gene knockdown
results and associated clinical pharmacology. As we enter the final
months of 2015, we look forward to additional important data read outs
at AHA and ASH and an in depth review of our pipeline at our R&D Day."
Third Quarter 2015 and Recent Significant Corporate Highlights
Advanced pipeline programs in Genetic Medicine Strategic Therapeutic
Advanced investigational RNAi therapeutic programs for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
Continued enrollment in APOLLO Phase 3 study of patisiran in
ATTR amyloidosis patients with Familial Amyloidotic
Polyneuropathy (FAP).
The company announced last week that it is on track to
complete enrollment in APOLLO within the next three to
four months. If positive, the company expects that the
APOLLO study results will support an NDA submission for
Reported positive 12- and 18-month clinical data from
patisiran Phase 2 open-label extension (OLE) study, showing
sustained mean maximal TTR knockdown of 93% and continued
evidence for potential halting of neuropathy progression.
Importantly, we also documented first-ever objective evidence
for an effect of patisiran toward improvement of nerve fiber
density. Patisiran was also found to be generally well
tolerated out to nearly two years of drug administration.
Initiated Phase 3 OLE study ("APOLLO-OLE") with patisiran.
Continued enrollment in ENDEAVOUR Phase 3 study of revusiran
in ATTR amyloidosis patients with Familial Amyloidotic
Cardiomyopathy (FAC).
Reported initial 6-month clinical data from revusiran Phase 2
OLE study, showing sustained TTR knockdown representing the
longest dosing experience reported to date for target gene
knockdown with a GalNAc-siRNA conjugate. In majority of
patients, 6 minute walk distance (6MWD) results at 6 months
were stable compared with baseline, and revusiran was
generally well tolerated out to 10 months of administration.
Advanced Development Candidate (DC) for ALN-TTRsc02, an
ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of
ATTR amyloidosis, with goal of filing a Clinical Trial
Application (CTA) in early 2016.
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
Continued dosing hemophilia patients with once-monthly
subcutaneous dose regimen in ongoing Phase 1 study.
Initiated Phase 1 OLE study with ALN-AT3.
Announced Genzyme's election to opt into ALN-AT3 program for
development and potential future commercialization in
territories outside of North America and Western Europe.
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
Continued dosing healthy volunteers in multiple ascending dose
part of ongoing Phase 1 trial.
Advanced ALN-AS1 for the treatment of acute hepatic porphyrias
Presented initial positive results from ongoing Phase 1 trial,
showing that single subcutaneous doses of ALN-AS1 resulted in
lowering of aminolevulinic acid (ALA) and porphobilinogen
(PBG), the toxic heme synthesis intermediates that mediate
porphyria attacks. In addition, ALN-AS1 treatment resulted in
potent, dose-dependent, and durable silencing of
aminolevulinic acid synthase-1 (ALAS1) mRNA in liver. Further,
ALN-AS1 was found to be generally well tolerated with no
clinically significant drug-related adverse events through the
Presented initial data from EXPLORE trial, a multinational,
prospective observational study of patients with hepatic
porphyrias suffering from recurrent attacks.
Initiated Phase 1/2 trial with ALN-AAT for the treatment of
alpha-1 antitrypsin (AAT) deficiency-associated liver disease
(alpha-1 liver disease).
Advanced ALN-GO1 for the treatment of primary hyperoxaluria type 1
Selected ALN-GO1 DC, with plans to file CTA in late 2015 and
initiate Phase 1 study in early 2016.
Advanced investigational pipeline programs in Cardio-Metabolic Disease
and Hepatic Infectious Disease STArs.
Alnylam and The Medicines Company presented positive initial data
from the ongoing Phase 1 trial of ALN-PCSsc for the treatment of
hypercholesterolemia. The effects of ALN-PCSsc toward LDL-C were
found to be comparable to reported results with anti-PCSK9
monoclonal antibodies, but showed a durability profile supportive
of a once-quarterly and possibly bi-annual subcutaneous dose
regimen. Further, ALN-PSCsc was found to be generally well
Continued pre-clinical studies to support a CTA filing for ALN-HBV