Full Press Release Details
Pharmaceuticals Reports First Quarter 2015 Financial Results and
Highlights Recent Period Progress
Launched "Alnylam 2020" Guidance for Advancement and Commercialization
of RNAi Therapeutics -
Advanced Multiple Clinical Programs and Presented Evidence for Potential
Disease-Modifying Activity for Patisiran in Familial Amyloidotic
Polyneuropathy (FAP) and ALN-AT3 in Hemophilia -
Plans to Present New Clinical Data for ALN-AT3 and Initial Clinical Data
for ALN-CC5 in Oral Presentations at Medical Meetings in June -
Maintained Strong Balance Sheet with $1.45 Billion in Cash and Expects
to End 2015 with Greater than $1.2 Billion in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 7, 2015--Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported
its consolidated financial results for the first quarter 2015, and
highlighted recent progress in advancing its pipeline.
"These are exciting times for RNAi therapeutics and Alnylam's efforts to
bring innovative medicines to patients. With our Alnylam 2020' strategy
we aim to transition from a late-stage clinical development company to a
multi-product commercial-stage company with a sustainable development
pipeline - a profile that we believe has rarely been achieved in the
biopharmaceutical industry. Amongst many achievements in the first
quarter and recent period, we reported promising data on potential
disease modifying effects for patisiran in Familial Amyloidotic
Polyneuropathy (FAP) and ALN-AT3 in hemophilia. Specifically, in our
patisiran Phase 2 open-label extension - or OLE' - study, we generated
what we believe to be continued evidence for a possible halting of
neuropathy progression after the first 12 months of treatment, with drug
administration generally well tolerated out to 17 months. Furthermore,
recent results from our Phase 1 trial with ALN-AT3 provide initial
evidence for potential correction of the hemophilia phenotype with an
encouraging tolerability profile. In aggregate, we believe these results
strengthen the bridge that we are building between RNAi-mediated
knockdown and potential clinical benefit for patients," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We have also
continued enrolling subjects in our ALN-PCSsc Phase 1 study, and we
initiated dosing in our Phase 1/2 trial with ALN-CC5. With these
advancements we are now poised for a very data-rich mid-year period,
with confirmed oral presentations of clinical results for ALN-AT3 and
ALN-CC5 in June and initial Phase 1 data for ALN-PCSsc expected in
mid-2015. We look forward to sharing these data in the weeks and months
First Quarter 2015 and Recent Significant Corporate Highlights
Launched "Alnylam 2020" Guidance for advancement and commercialization
of RNAi Therapeutics. Specifically, by the end of 2020, Alnylam
expects to achieve a company profile with 3 marketed products, as well
as 10 RNAi therapeutic clinical programs - including 4 in late stages
of development - across its 3 Strategic Therapeutic Areas (STArs).
Advanced pipeline programs in Genetic Medicine STAr.
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).
Reported positive 12-month clinical data from patisiran Phase
2 open-label extension (OLE) study, showing sustained TTR
knockdown of up to a mean 88% and continued evidence for
potential halting of neuropathy progression. Specifically, a
mean 2.5 point decrease in neuropathy impairment score
(mNIS+7) was observed after 12 months of patisiran
administration, comparing favorably to a 13-18 point increase
in untreated patients with similar baseline characteristics,
as estimated from published historical data sets. Patisiran
was also found to be generally well tolerated out to 17 months
of drug administration.
Continued enrollment in ENDEAVOUR Phase 3 study of revusiran
in ATTR amyloidosis patients with Familial Amyloidotic
Cardiomyopathy (FAC).
Presented complete Phase 2 data with revusiran in patients
with TTR cardiomyopathy, showing tolerability and an up to
98.2% knockdown of serum TTR.
Continued dosing FAC patients in revusiran Phase 2 OLE study
designed to evaluate the tolerability and clinical activity of
revusiran with long-term dosing for up to two years.
Presented results from a retrospective natural history study
evaluating disease progression in ATTR amyloidosis patients
with FAC, showing a 140 meter decline in 6 minute walk
distance (6MWD) at 18 months; the change in 6MWD at 18 months
is a co-primary endpoint measure in ENDEAVOUR.
Advanced ALN-AT3 for the treatment of hemophilia and rare bleeding
Reported positive initial results from a small number of
subjects in a Phase 1 trial of ALN-AT3, including an up to 70%
knockdown of antithrombin (AT) and initial evidence for the
potential correction of the hemophilia phenotype with an up to
334% increase in thrombin generation and marked improvement in
whole blood clotting.
The company announces today that it is transitioning to
once-monthly subcutaneous dose cohorts in its ongoing Phase 1
The company also announces today that it has completed its
chronic GLP toxicology studies of ALN-AT3, including a 9-month
study in non-human primates and 6-month studies in rat and
hemophilia A mice, with No Adverse Effect Level (NOAEL) doses
that support further advancement of the program.
Published pre-clinical study results in Nature Medicine
documenting safety, efficacy, and durability of ALN-AT3 in
rodent and non-human primate (NHP) models of hemophilia.
Initiated Phase 1/2 trial with ALN-CC5. The trial is being
conducted initially in normal human volunteers, and then is
expected to move to patients with paroxysmal nocturnal
hemoglobinuria (PNH).
Advanced pipeline programs in Cardio-Metabolic Disease STAr
Continued dosing in Phase 1 trial with ALN-PCSsc in normal human