Full Press Release Details
Pharmaceuticals Reports First Quarter 2016 Financial Results and
Highlights Recent Period Progress
Pipeline of Ten Clinical Stage Programs, with Ten Additional Clinical
Data Readouts Anticipated This Year -
Expects to Complete Enrollment of Revusiran Phase 3 ENDEAVOUR Trial in
Late 2016 with Data Readout in Mid-2018 -
Track to Start Fitusiran Phase 3 Program in Mid-2016; Plans to Report
Updated Hemophilia Patient Data in July -
Provides Update on ALN-CC5 Program and Development Path Forward; Plans
to Present Initial Data in Paroxysmal Nocturnal Hemoglobinuria (PNH)
Quarter with $1.2 Billion in Cash and Increases Year-End Cash Guidance
to Over $1 Billion, Including $150 Million of Restricted Marketable
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 2, 2016--Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today
reported its consolidated financial results for the first quarter 2016,
and highlighted recent progress in advancing its pipeline.
"At Alnylam, we continue to advance a broad pipeline of investigational
RNAi therapeutics - including 10 programs in clinical development and 2
programs in Phase 3 - across a broad range of disease indications. A
major milestone in the first quarter was completion of enrollment in our
APOLLO Phase 3 trial for patisiran, and we're on track to view results
in mid-2017. If positive, we expect to file our first regulatory
applications for approval later that same year. We're also making strong
progress in our ENDEAVOUR Phase 3 trial of revusiran, where we now
expect completion of enrollment in late 2016 and data readout in
mid-2018. In our fitusiran program in hemophilia, we look forward to
presenting important new results in July, including initial results in
patients with inhibitors, and are on track to start our two Phase 3
studies shortly thereafter," said John Maraganore, Ph.D., Chief
Executive Officer at Alnylam. "We're also making progress with our
earlier stage clinical programs. In our ALN-CC5 program, initial results
in PNH patients, which will be presented at EHA next month, point to an
optimal development path forward in PNH for eculizumab poor responders
and for eculizumab sparing, with parallel efforts in other
complement-mediated diseases. Finally, we also filed Clinical Trial
Applications for ALN-HBV and ALN-TTRsc02, and initiated our Phase 1
study for our ALN-GO1 program in primary hyperoxaluria. We look forward
to sharing our continued progress throughout the course of a very data
First Quarter 2016 and Recent Significant Corporate Highlights
Advanced investigational pipeline programs in Genetic Medicine
Strategic Therapeutic Area (STAr).
Advanced investigational RNAi therapeutics programs for the
treatment of transthyrethin (TTR)-mediated amyloidosis (ATTR
Completed enrollment in the APOLLO Phase 3 trial with
patisiran for the treatment of hereditary TTR-mediated
amyloidosis with polyneuropathy (hATTR-PN), also known as
familial amyloidotic polyneuropathy (FAP).
Based on strong investigator and patient interest, the
study was substantially over enrolled with 225 patients.
Data from APOLLO are expected in mid-2017, and assuming
positive results, the Company expects to submit an NDA and
MAA for patisiran by the end of 2017 and launch in 2018.
Announced complete 18-month data from ongoing Phase 2
open-label extension (OLE) study with patisiran.
Data presented at the American Academy of Neurology (AAN)
Meeting provided continued evidence that patisiran has the
potential to halt neuropathy progression in patients with
hATTR-PN. In the first reported exploratory analysis of
its kind, the degree of TTR knockdown observed in patients
was shown to correlate with improvement in neuropathy
impairment scores. Further, patisiran was found to be
generally well tolerated with no drug related serious
adverse events up to 25 months of treatment. The majority
of adverse events were mild to moderate.
Continued enrollment in ENDEAVOUR Phase 3 study with revusiran
for the treatment of hereditary TTR-mediated amyloidosis with
cardiomyopathy (hATTR-CM), also known as familial amyloidotic
cardiomyopathy (FAC).
The Company announced today that it expects to complete
ENDEAVOUR enrollment in late 2016 and report results in
Filed Clinical Trial Application (CTA) for ALN-TTRsc02, an
ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of
ATTR amyloidosis, which is expected to enable a once-
quarterly subcutaneous dosing regimen.
Assuming a positive Phase 1 study, the Company plans to
initiate a Phase 3 trial in 2017.
Advanced fitusiran (ALN-AT3) for the treatment of hemophilia and
rare bleeding disorders (RBD).
Initiated dosing of hemophilia patients with inhibitors in
Part D of an ongoing Phase 1 clinical trial evaluating a
once-monthly subcutaneous dose regimen of fitusiran. Both
patients with hemophilia A with inhibitors and hemophilia B
with inhibitors have now been dosed with fitusiran.
Continued dosing patients in an ongoing Phase 1 OLE, where
once-monthly doses of fitusiran are administered to patients
with moderate or severe hemophilia A or B with or without
Alnylam is on track to initiate two Phase 3 trials: the first
in mid-2016 in hemophilia A and B patients with inhibitors;
and, the second in late 2016 in moderate or severe hemophilia
A and B patients without inhibitors.
The Company has initiated discussions with global
regulatory authorities to confirm specific trial designs.
Advanced ALN-CC5 for the treatment of complement-mediated diseases.
The Company announced today that it has achieved preliminary
evidence for clinical activity in a small number of paroxysmal
nocturnal hemoglobinuria (PNH) patients enrolled in Part C of
its ongoing Phase 1/2 trial, and it believes that based on LDH
data, the optimal development path for ALN-CC5 in PNH is for
eculizumab poor responders and for eculizumab sparing.
The Company now plans to transition toward a new Phase 2