Full Press Release Details
Pharmaceuticals Reports Second Quarter 2018 Financial Results and
Highlights Recent Period Activity
Received Positive CHMP Opinion for ONPATTRO in Europe for the Treatment
of hATTR Amyloidosis in Adult Patients with Stage 1 or Stage 2
Polyneuropathy and On Track for August 11 PDUFA Date in U.S. -
Achieved Robust Enrollment in ENVISION Phase 3 Study of Givosiran and On
Track to Report Topline Results from Interim Analysis for Potential
Accelerated Approval -
Achieved Regulatory Alignment for Phase 3 Studies of Lumasiran and
Maintained Strong Balance Sheet with $1.48 Billion in Cash and Expects
to End 2018 with Approximately $1.0 Billion in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--August 2, 2018--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, today reported its consolidated financial results for the
second quarter 2018 and highlighted recent progress in advancing its
"The second quarter and recent period mark a milepost in the history of
Alnylam that has been 16 years in the making - the recommendation from
the EU Committee for Medicinal Products for Human Use (CHMP) that the
European Medicines Agency approve the first-ever RNAi therapeutic,
ONPATTRO. We believe the positive CHMP opinion in the EU signals the
potential for a new therapeutic paradigm in medicine, raising hope for
patients and caregivers impacted by hATTR amyloidosis. With this
achievement, the expected FDA action by our August 11 PDUFA date, and
plans to file our JNDA in Japan later this year, we believe we are
poised to deliver on the promise of ONPATTRO on a truly global scale,"
said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
"In parallel, we have advanced our three other wholly owned late-stage
programs. We achieved robust enrollment in our ENVISION Phase 3 study of
givosiran and are on track for an interim analysis by the end of
September in support of a potential accelerated approval. In addition,
with our recent alignment with the FDA on a Phase 3 trial design for
lumasiran, we are gearing up to initiate a pivotal study for this
program in the coming weeks. Finally, we're pleased to announce today
that we have reached alignment with the FDA on a Phase 3 trial design
for ALN-TTRsc02 in hATTR amyloidosis patients, where we're on track to
start the study by year's end. All together, we believe our efforts
position us to achieve our Alnylam 2020 strategy of building a
multi-product, global, commercial-stage company with a deep and
sustainable clinical pipeline by the end of 2020."
Second Quarter 2018 and Recent Significant Corporate Highlights
Received a positive opinion from CHMP recommending marketing
authorization of ONPATTRO (patisiran) - a first-of-its-kind RNAi
therapeutic - for the treatment of hATTR amyloidosis in adult patients
with stage 1 or stage 2 polyneuropathy.
The European Commission (EC) decision on approval of ONPATTRO is
now expected in September, and the recommended Summary of Product
Characteristics (SmPC) includes data from secondary and
exploratory study endpoints in the APOLLO Phase 3 trial, including
The Company is on track in the U.S. with an August 11 PDUFA date
for ONPATTRO with the FDA.
Published APOLLO study results for patisiran in the July 5, 2018
issue of The New England Journal of Medicine.
Presented additional data from the APOLLO Phase 3 study at the 4th
Congress of the European Academy of Neurology (EAN) and the
Peripheral Nerve Society (PNS) 2018 Annual Meeting.
To date the Company has fulfilled over 200 requests by physicians
for eligible patients to begin treatment in the early access or
compassionate use programs for patisiran in the U.S. and EU.
Advanced givosiran, an investigational RNAi therapeutic in development
for the treatment of acute hepatic porphyrias (AHPs).
Completed enrollment of the cohort of patients in the ENVISION
Phase 3 study that will comprise the planned interim analysis in
support of a potential accelerated approval.
The Company remains on track to report topline results of the
interim analysis by the end of September and, pending Company
and FDA review of the program at the time of interim analysis
and assuming positive results and acceptable safety, the
Company expects to submit an NDA at or around year-end 2018
seeking an accelerated approval.
The interim analysis is based on lowering of urinary
aminolevulinic acid (ALA) levels at three months of treatment
as a surrogate biomarker that is reasonably likely to predict
Alnylam announced today that it has achieved robust enrollment in
ENVISION and expects to complete full patient accrual by the end
of September, ahead of schedule.
As a result, the Company now expects to report topline results
on the primary endpoint of annualized attack rate in early
Advanced lumasiran, an investigational RNAi therapeutic in development
for the treatment of primary hyperoxaluria type 1 (PH1), with new
positive data from the Phase 1/2 study presented at the OxalEurope
European Hyperoxaluria Consortium.
The Company is on track to initiate a Phase 3 pivotal trial in
mid-2018, with results expected in late 2019 supporting a
potential NDA filing in early 2020.
Advanced ALN-TTRsc02, a subcutaneously administered investigational
RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Alnylam announced today that it has reached alignment with the FDA
on the design of a pivotal Phase 3 study for ALN-TTRsc02 in
patients with hATTR amyloidosis.
The Phase 3 pivotal trial will be an open-label study with
co-primary endpoints of mNIS+7 and Norfolk-QOL at nine months
comparing the effects of ALN-TTRsc02 in approximately 120
patients with hATTR amyloidosis to results from the placebo
arm from the APOLLO Phase 3 study of patisiran. In addition,
certain cardiac parameters will be included as endpoints.
An additional reference arm of approximately 30 patients
receiving patisiran will be included.
The Company is on track to start the Phase 3 study in late 2018
and plans to start additional Phase 3 studies of ALN-TTRsc02,
including in wild-type ATTR amyloidosis, in 2019.