Full Press Release Details
Pharmaceuticals Reports Fourth Quarter and Full Year 2014 Financial
Results and Highlights Recent Period Progress
Introduced Pipeline Growth Strategy for RNAi Therapeutics in Three
Strategic Therapeutic Areas, or "STArs," and Launched "Alnylam 2020"
Guidance for Advancement and Commercialization of RNAi Therapeutics -
Presented Positive Data from Multiple Clinical Programs, Including
Initial Evidence of Potential Disease Modifying Effects with Patisiran
Advanced Revusiran into ENDEAVOUR Phase 3 Trial and Added Three New
Programs into Clinical Stages -
Maintained Strong Balance Sheet with $882 Million in Cash and Expects to
End 2015 with Greater than $1.2 Billion in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 12, 2015--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, today reported its consolidated financial results for the
fourth quarter and full year 2014, and highlighted recent progress in
advancing its pipeline.
"Alnylam made excellent progress in the fourth quarter of 2014 and the
recent period, as we continued advancing RNAi therapeutics in clinical
studies. Amongst other highlights, we reported clinical data from our
patisiran and ALN-AT3 programs showing what we believe to be early
evidence for the translation of RNAi-mediated knockdown into potential
clinical benefit for patients. We also executed on our pipeline goals,
with strong enrollment in our patisiran APOLLO Phase 3 study and
advancement of revusiran into our ENDEAVOUR Phase 3 trial. In addition,
we filed three Clinical Trial Applications for ALN-PCSsc, ALN-CC5, and
ALN-AS1, and have initiated Phase 1 studies for two of those programs,"
said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
"Alnylam also introduced its pipeline growth strategy and new guidance.
Specifically, Alnylam intends to advance its pipeline in three Strategic
Therapeutic Areas, or STArs' - Genetic Medicines, Cardio-Metabolic
Disease, and Hepatic Infectious Disease - where we believe there are
significant opportunities for RNAi therapeutics as high impact
medicines. Further, with Alnylam 2020' we have launched new guidance
that marks our expected 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."
"Alnylam continues to maintain a very strong balance sheet, ending 2014
with approximately $882 million in cash," said Michael Mason, Vice
President, Finance & Treasurer. "We also further strengthened our
balance sheet earlier this year with a public offering and concurrent
private placements from Genzyme that resulted in net proceeds of
approximately $567 million. This financing results in a balance sheet
that allows us to invest in a broad pipeline of RNAi therapeutics across
all three STArs, which we believe should enable us to achieve our
Alnylam 2020' guidance. As for financial guidance this year, we expect
to end 2015 with greater than $1.2 billion in cash."
Fourth Quarter 2014 and Recent Significant Corporate Highlights
Introduced pipeline growth strategy for RNAi therapeutics in three
Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad
pipeline of RNAi therapeutics for the treatment of rare diseases;
Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward
genetically validated, liver-expressed disease targets for unmet needs
in dyslipidemia, hypertension, non-alcoholic steatohepatitis (NASH),
and type 2 diabetes; and Hepatic Infectious Disease, with a pipeline
of RNAi therapeutics that address the major global health challenges
of hepatic infectious diseases.
Launched "Alnylam 2020" Guidance for advancement and intended
commercialization of RNAi Therapeutics. Specifically, by the end of
2020, Alnylam expects to achieve a company profile with 3 marketed
products, 10 RNAi therapeutic clinical programs - including 4 in late
stages of development - across its 3 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 patients with Familial Amyloidotic Polyneuropathy (FAP)
Reported positive six-month clinical data from patisiran Phase
2 open-label extension (OLE) study, showing tolerability,
sustained TTR knockdown, and promising initial evidence for
potential stabilization of neuropathy progression.
Initiated ENDEAVOUR Phase 3 study with revusiran. ENDEAVOUR is
a randomized, double-blind, placebo-controlled, global study
designed to evaluate the efficacy and safety of revusiran in
ATTR patients with Familial Amyloidotic Cardiomyopathy (FAC).
Presented positive initial Phase 2 data with revusiran, with
up to 98.2% knockdown of serum TTR; revusiran administration
was found to be generally well tolerated in patients with
advanced cardiac disease.
Initiated Phase 2 OLE study with revusiran to evaluate
tolerability and clinical activity with long-term dosing for
Reported positive initial results from Phase 1 trial of ALN-AT3,
including 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.
Initiated Phase 1/2 trial with ALN-CC5. The trial is being
conducted initially in normal human volunteers, and then in
patients with paroxysmal nocturnal hemoglobinuria (PNH).
Filed Clinical Trial Application (CTA) to initiate a Phase 1 trial
with ALN-AS1 in acute intermittent porphyria (AIP) patients who
are asymptomatic "high excreters" (ASHE), and then in AIP patients
who experience recurrent porphyria attacks.
Expanded Genetic Medicine pipeline with ALN-GO1, an
investigational RNAi therapeutic targeting glycolate oxidase (GO)
in development for the treatment of Primary Hyperoxaluria Type 1
Advanced pipeline programs in Cardio-Metabolic Disease STAr
Initiated Phase 1 trial with ALN-PCSsc in normal human volunteers
with elevated LDL-C at baseline.
Advanced pipeline programs in Hepatic Infectious Disease STAr
Selected Development Candidate (DC) for ALN-HBV, showing an up to