Full Press Release Details
Pharmaceuticals Reports Second Quarter 2015 Financial Results and
Highlights Recent Period Activities
Advanced Pipeline of Seven Investigational Clinical Programs and
Presented Positive Clinical Results for Three: Patisiran for Familial
Amyloidotic Polyneuropathy (FAP), ALN-AT3 for Hemophilia, and ALN-CC5
for Complement-Mediated Diseases -
Addition to Upcoming Presentation of ALN-PCSsc Clinical Results,
Announces New Plan to Present Initial Clinical Data for ALN-AS1
Porphyria Program at Medical Meeting in September and Provides Update on
Revusiran Phase 2 Open-Label Extension Study -
Maintained Strong Balance Sheet with $1.40 Billion in Cash and Remains
On Track to End 2015 with Greater than $1.2 Billion in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--August 6, 2015--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, today reported its consolidated financial results for the
second quarter 2015, and highlighted recent progress in advancing its
"The second quarter of 2015 and recent period were marked by tremendous
progress in our efforts to bring innovative medicines to patients, as we
execute on our Alnylam 2020' strategy. In our patisiran Phase 2
open-label extension - or OLE' - study, we believe to have generated
increased evidence for a possible halting of neuropathy progression
after the first 12 months of treatment, with study drug administration
generally well tolerated out to 17 months. In our hemophilia program, we
believe that recent results from our ongoing Phase 1 trial provide
confirmatory evidence that ALN-AT3 has the potential to re-balance
hemostasis in people with severe hemophilia through normalization of
thrombin generation. We also reported initial positive data from our
ongoing Phase 1/2 trial with ALN-CC5 - an investigational RNAi
therapeutic for complement-mediated diseases - where we demonstrated
what we believe to be promising preliminary clinical activity for single
doses of study drug with potent and highly durable knockdown of serum C5
and inhibition of serum complement activity," said John Maraganore,
Ph.D., Chief Executive Officer of Alnylam. "We also completed enrollment
in our ALN-PCSsc Phase 1 study with results to be presented later this
month, and we started dosing in our patisiran APOLLO-OLE study and Phase
1 studies with ALN-AS1 and ALN-AAT. We now enter a very data-rich back
half of the year, with important results being presented from six
distinct clinical programs. We very much look forward to sharing these
results in the weeks and months to come, as they highlight what we
believe to be the significant potential for RNAi therapeutics as a new
class of innovative medicines."
Second Quarter 2015 and Recent Significant Corporate Highlights
Advanced pipeline of investigational programs in Genetic Medicine
Strategic Therapeutic Area (STAr).
Advanced RNAi therapeutic programs for the treatment of
transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Continued enrollment in APOLLO Phase 3 study of patisiran in
ATTR patients with Familial Amyloidotic Polyneuropathy (FAP).
Initiated Phase 3 open-label extension study ("APOLLO-OLE")
Reported positive 12-month clinical data from patisiran Phase
2 OLE study, showing sustained TTR knockdown of up to a mean
88% and continued evidence for potential halting of neuropathy
progression with a mean 2.5 point decrease in neuropathy
impairment score (mNIS+7) at 12 months, comparing favorably to
a 13-18 point increase estimated from the literature in
untreated FAP patients with similar baseline characteristics;
patisiran was also found to be generally well tolerated out to
17 months of study drug administration.
Continued enrollment in ENDEAVOUR Phase 3 study of revusiran
in ATTR patients with Familial Amyloidotic Cardiomyopathy
Continued dosing FAC and senile systemic amyloidosis (SSA)
patients in revusiran Phase 2 OLE study, which was initiated
in November 2014 and is designed to evaluate the tolerability
and clinical activity of revusiran with long-term dosing for
The company reports today that three patients have
discontinued from the revusiran Phase 2 OLE study as of
August 5 due to injection site reactions (ISRs), including
some with associated diffuse rash; the study drug remains
otherwise generally well tolerated in the broader
revusiran Phase 2 OLE study population. The company plans
to present initial clinical results from the revusiran
Phase 2 OLE study in late 2015.
Received Orphan Drug Designation (ODD) from the United States
Food & Drug Administration (FDA) for revusiran.
The company announces today that it is advancing a Development
Candidate (DC) for an ESC-GalNAc-siRNA targeting TTR that is
expected to support a once-monthly and possibly once-quarterly
subcutaneous dose regimen. Further details and guidance on
this program will be presented at the Oligonucleotide
Therapeutics Society meeting to be held October 11 - 14, 2015
in Leiden, The Netherlands.
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
Reported positive interim results from the ongoing Phase 1
study, including an up to 86% knockdown of antithrombin (AT),
and evidence for a potential re-balancing of hemostasis
resulting in normalization of thrombin generation up to a mean
increase of 350% and marked improvements in whole blood
clotting. In addition, in a post hoc exploratory analysis, AT
knockdown was found to be associated with reduced bleeding
frequency, with the maximum bleed-free interval of 114 days in
a patient with severe hemophilia A. ALN-AT3 was also shown to
be generally well tolerated, without any clinically
significant increases in D-dimer, a marker of pathologic clot
Presented new pre-clinical results with ALN-AT3 and published
pre-clinical study results in Nature Medicine.
These published pre-clinical data were the centerpiece of
a recent "Clinical Implications" article in the New
England Journal of Medicine.
Presented initial positive Phase 1/2 study results with ALN-CC5
for the treatment of complement-mediated diseases.