Full Press Release Details
Pharmaceuticals Reports Fourth Quarter and Full Year 2015 Financial
Results and Highlights Recent Period Progress
Advanced Pipeline of Eight Clinical Stage Programs in 2015, with Ten or
More Major Clinical Data Readouts, Start of Fitusiran Phase 3 Trials,
and Three New IND Filings Planned in 2016 -
Completed Enrollment in Patisiran Phase 3 APOLLO Trial, Positioning the
Company for First Filing for Regulatory Approval in 2017 -
Maintained Strong Balance Sheet with $1.28 Billion in Cash and Expects
to End 2016 with Greater than $850 Million in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 11, 2016--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, today reported its consolidated financial results for the
fourth quarter and full year 2015, and highlighted recent progress on
"In 2015, including the fourth quarter, we made strong progress
advancing our pipeline and executing on our broader business objectives.
Through our focused efforts, we grew our pipeline to eight clinical
stage programs, achieved human proof of concept in multiple programs,
and accrued patients in our two pivotal Phase 3 trials, including the
APOLLO study, where we have now completed enrollment," said John
Maraganore, Ph.D., Chief Executive Officer at Alnylam. "We believe 2016
will be a key year for Alnylam as we expect ten or more clinical data
readouts, the start of two pivotal Phase 3 trials in our fitusiran
hemophilia program, and the filing of three new INDs. In addition, with
our progress in APOLLO, we're entering the dawn of our commercial
transition with planned regulatory submissions in 2017 for patisiran
approval, if the trial is positive. We believe our 2016 plans place us
firmly on track to achieve our Alnylam 2020' goals of building a
multi-product, commercial-stage company with a robust and sustainable
pipeline across our three Strategic Therapeutic Areas, or STArs.'"
Fourth Quarter 2015 and Recent Significant Corporate Highlights
Advanced investigational pipeline programs in Genetic Medicine STAr.
Advanced RNAi therapeutics programs for the treatment of
transthyrethin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Completed APOLLO Phase 3 enrollment with 225 patients for
patisiran, in development for patients with Transthyretin
(TTR)-Mediated Amyloidosis (ATTR Amyloidosis).
If the study is positive, the Company expects to submit a
New Drug Application (NDA) and Marketing Authorisation
Application (MAA) for patisiran, based on an analysis of
the full APOLLO data set, in late 2017.
Reported positive initial 18-month clinical data from
patisiran Phase 2 open-label extension (OLE) study, showing
continued evidence for potential halting of neuropathy
progression. Patisiran was also found to be generally well
tolerated out to nearly two years of drug administration
through the data cutoff date.
Complete 18-month clinical data from the patisiran Phase 2
OLE is expected to be reported in an oral session at the
upcoming American Academy of Neurology (AAN) annual
meeting on April 20, 2016, in Vancouver, Canada.
Continued enrollment in ENDEAVOUR Phase 3 study of revusiran
in ATTR amyloidosis patients with Familial Amyloidotic
Cardiomyopathy (FAC), with data expected in 2018.
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, revusiran was generally well tolerated out to 10
months of administration through the data cutoff date.
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, starting a Phase 1 study in
mid-2016 with initial data in late 2016, and initiating a
Phase 3 trial in 2017.
Advanced fitusiran (ALN-AT3) for the treatment of hemophilia and
rare bleeding disorders (RBD).
Presented positive interim data from ongoing Phase 1 trial of
Interim results showed that monthly subcutaneous doses
achieved antithrombin (AT) lowering associated with
statistically significant and clinically meaningful
increases in thrombin generation and decreases in bleeding
frequency in patients with hemophilia A and B.
Fitusiran was also found to be generally well tolerated
through the data cutoff date, including no clinically
significant increases in D-dimer, a biomarker of excessive
Announced that Sanofi Genzyme elected to opt into the
fitusiran program for development and commercialization
outside of North America and Western Europe, while retaining
its future opt-in right to co-develop and co-promote fitusiran
with Alnylam in North America and Western Europe, subject to
certain restrictions.
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
Presented positive initial data from an ongoing Phase 1/2
Results showed that ALN-CC5 achieved clinically meaningful
reductions in serum C5 and inhibition of complement
ALN-CC5 was also shown to be generally well tolerated,
with no clinically significant, drug-related adverse
events through the data cutoff date.
Initiated Part C of ongoing Phase 1/2 trial in patients with
paroxysmal nocturnal hemoglobinuria (PNH).
Advanced ALN-AS1 for the treatment of acute hepatic porphyrias.
Alnylam announces today that it has enrolled its first patient
in Part C of the ongoing Phase 1 clinical trial. Part C is
being conducted in Acute Intermittent Porphyria (AIP) patients
experiencing multiple recurrent porphyria attacks, and will
evaluate the safety and tolerability of multiple doses of
ALN-AS1 as well as measures of clinical activity, including
reduction in frequency and severity of attack symptoms,
hospitalizations, quality of life, and reduction in the use of