− Expects to Exceed Alnylam 2020 Goals with
Four Marketed Products, 14 Organic Clinical Stage Programs,
Including 6 in Late-Stage Development, Across 4 Strategic
Therapeutic Areas (STArs), by End of 2020 –
– Initiates HELIOS-B Phase 3 Study of
Investigational Vutrisiran in ATTR Amyloidosis Cardiomyopathy –
– Announces Initial Positive Clinical Results
with ALN-AAT02 and ALN-HBV02 (VIR-2218), Providing Human Proof of
Concept for “Enhanced Stabilization Chemistry Plus” (ESC+) GalNAc
Conjugate Delivery Technology –
– Presents Initial Positive Clinical Data for
ALN-AGT, an Investigational RNAi Therapeutic for Hypertension,
Expanding Opportunities for RNAi Therapeutics in Highly Prevalent
Chronic Diseases –
– Reports Strong Progress in CNS and Ocular
Delivery of RNAi Therapeutics with Seven Initial Programs Selected
as Part of Regeneron Collaboration, Including ALN-HTT, an
Investigational RNAi therapeutic for Huntington’s Disease –
– Alnylam to Webcast its R&D Day Today at
8:00 a.m. ET –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, is hosting an R&D Day in New York City
today. During the event, the Company plans to discuss its
commercial and R&D progress, including its product and pipeline
goals for 2020, focusing on the potential for global approval
and/or commercialization of four RNAi therapeutic products and the
advancement of six late-stage investigational programs.
Alnylam also announces today that it has initiated HELIOS-B, a
global Phase 3 placebo-controlled mortality and cardiovascular
hospitalization trial with investigational vutrisiran in patients
with ATTR amyloidosis with cardiomyopathy. In addition, the Company
is reporting initial positive clinical data with ALN-AAT02,
ALN-HBV02 (VIR-2218), and ALN-AGT, representing the first human
proof of concept for Alnylam’s ESC+ GalNAc conjugate delivery
technology – having the potential to enable greater target
selectivity and a wider therapeutic index – and expansion of the
Alnylam pipeline in highly prevalent chronic disease opportunities.
The Company is also reporting progress in its efforts with CNS and
ocular delivery of RNAi therapeutics, including an update on the
status of its collaborative work with Regeneron, and announces a
new investigational CNS program, ALN-HTT, in development for the
treatment of Huntington’s disease. Finally, Alnylam is presenting
its general framework for progressing toward a self-sustainable
financial profile for future growth and value creation.
“At Alnylam, we believe that we’re at an exciting stage of
growth, with a large number of commercial and development-stage
opportunities with potentially transformative impact for patients
across both rare and common diseases. Over the course of 2020 we
expect to deliver on a Company profile with four medicines on the
market, assuming additional regulatory approvals, while also
advancing a large number of late- and earlier-stage programs, all
while focusing on our pathway toward a self-sustainable financial
profile. Our plans include continued global commercialization of
ONPATTRO as a market-leading program, with ongoing clinical studies
aimed at broadening our ATTR amyloidosis franchise to potentially
address the full spectrum of disease, including the now initiated
HELIOS-B study with investigational vutrisiran that we’re
announcing today ,” said John Maraganore, Ph.D., Chief Executive
Officer of Alnylam. “With the FDA approval just this week
for GIVLAARI and through our efforts and those of our partners, we
now believe that we’re poised to exceed our Alnylam 2020 goals of
building a multi-product, global, commercial-stage biopharma
company with a deep and sustainable clinical pipeline for future
growth and a robust, organic research engine for sustainable
innovation and great potential for patient impact.”
2020 Product and Pipeline Goals
ONPATTRO® (patisiran), a commercial-stage RNAi
therapeutic targeting transthyretin (TTR) for the treatment of
polyneuropathy in patients with hATTR amyloidosis. Alnylam plans
to:
- Continue global commercial execution.
- Receive approval of ONPATTRO in Brazil in mid-2020, with
planned launches in additional countries throughout 2020.
- Complete enrollment in the APOLLO-B study in late 2020.
GIVLAARI™ (givosiran), a commercial-stage RNAi
therapeutic for the treatment of adults with acute hepatic
porphyria (AHP). Alnylam plans to:
- Achieve regulatory approval from the European Medicines Agency
(EMA) in early 2020.
- Execute on U.S. and EU commercial launches throughout
2020.
- Report additional results from the ENVISION Phase 3 study in
mid-2020.
- File for regulatory approvals and launch in additional
countries in 2020.
Vutrisiran, an investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis. Alnylam plans
to:
- Complete enrollment in the HELIOS-A Phase 3 study in early
2020.
- Continue enrollment in the HELIOS-B Phase 3 study throughout
2020.
Lumasiran, an investigational RNAi therapeutic in
development for the treatment of primary hyperoxaluria type 1
(PH1). Alnylam plans to:
- File a New Drug Application (NDA) and a Marketing Authorisation
Application (MAA) with the FDA and EMA, respectively, in early
2020, assuming positive results from the ILLUMINATE-A Phase 3
study.
- Report topline results from the ILLUMINATE-B pediatric Phase 3
study in mid-2020.
- Achieve FDA and EMA regulatory approvals in late 2020.
Alnylam also plans to support The Medicines Company's continued
efforts with respect to the ORION Phase 3 studies of
inclisiran, an investigational RNAi therapeutic targeting
PCSK9 in development for the treatment of hypercholesterolemia.
Specifically, The Medicines Company has guided that it plans
to:
- File for regulatory approval with the FDA in late 2019.
- File for regulatory approval with the EMA in early 2020.
- Continue enrollment in the ORION-4 Phase 3 CVOT study in
2020.
Alnylam also plans to support Sanofi’s continued efforts in
advancing fitusiran, an investigational RNAi therapeutic in
development for the treatment of hemophilia.
In addition, the Company plans to continue advancement of its
mid/earlier-stage clinical pipeline, independently or with
partners, including:
- Cemdisiran, in development for the treatment of
complement-mediated diseases, which is currently in a Phase 2 study
in patients with IgA nephropathy, in addition to planned
collaborative studies with Regeneron on combinations of cemdisiran
with pozelimab, an anti-C5 monoclonal antibody;
- ALN-AAT02, in development for the treatment of alpha-1
antitrypsin deficiency-associated liver disease, which is currently
in a Phase 1/2 study in healthy volunteers and patients with
alpha-1 liver disease;
- ALN-HBV02 (also known as VIR-2218), partnered with Vir
and in development for the treatment of chronic hepatitis B virus
(HBV) infection, which is currently in a Phase 1/2 study; and
- ALN-AGT, in development for the treatment of
hypertension, which is currently in a Phase 1 study.
- The Company also expects to submit 3 new Investigational New
Drug (IND) or equivalent applications in 2020, including for
ALN-HSD – an investigational RNAi therapeutic targeting
HSD17B13 for non-alcoholic steatohepatitis (NASH) in collaboration
with Regeneron – and ALN-LEC – an investigational RNAi
therapeutic targeting LECT2 for ALECT2 renal amyloidosis – amongst
other potential programs.
HELIOS-B Phase 3 Study of Vutrisiran
Alnylam is announcing today that it has initiated the HELIOS-B
Phase 3 study of vutrisiran in patients with hereditary and
wild-type ATTR amyloidosis patients with cardiomyopathy. HELIOS-B
will evaluate the efficacy of vutrisiran versus placebo toward the
composite outcome of all-cause mortality and recurrent
cardiovascular hospitalizations at 30 months, the primary study
endpoint. The study protocol includes an optional interim analysis
to be conducted at the Company’s discretion. HELIOS-B complements
the ongoing HELIOS-A Phase 3 study in patients with hereditary ATTR
amyloidosis with polyneuropathy, creating a comprehensive clinical
development program to evaluate the safety and efficacy of
vutrisiran across the entire disease spectrum of ATTR
amyloidosis.
Initial Positive Clinical Results with ESC+ GalNAc Conjugate
and Highly Prevalent Chronic Disease Programs
- ALN-AAT02 Phase 1 Results
- Initial results demonstrate an improved hepatic safety profile
for ALN-AAT02 – an ESC+ GalNAc conjugate – compared with ALN-AAT01,
with robust knockdown of alpha-1 antitrypsin and favorable
tolerability with no subjects showing clinically significant
elevations in liver alanine transaminase levels.
- ALN-HBV02 Phase 1/2 Results
- Initial results demonstrate improved hepatic safety profile
for ALN-HBV02 – an ESC+ GalNAc conjugate – compared with ALN-HBV01,
with substantial multi-log decreases in HBsAg and favorable
tolerability with no subjects or patients showing clinically
significant elevations in liver alanine transaminase levels.
- In the initial single-ascending dose cohorts of a Phase 1
study, ALN-AGT achieved dose-dependent lowering of angiotensinogen
– a genetically defined mediator of hypertension – with robust and
durable knockdown of angiotensinogen and favorable
tolerability.
Progress in CNS and Ocular RNAi Therapeutic Programs and
Status of Regeneron Collaboration
Alnylam is announcing today the selection of the initial seven
programs in the Regeneron collaboration, including ALN-APP – an
investigational RNAi therapeutic targeting amyloid precursor
protein (APP) for hereditary cerebral amyloid angiopathy (hCAA) and
early onset familial Alzheimer’s disease (EOFAD) – and ALN-HTT – an
investigational RNAi therapeutic for the treatment of Huntington’s
disease. In addition, the Regeneron collaboration includes an
undisclosed additional CNS target program, two undisclosed ocular
disease target programs, and two undisclosed liver target programs.
The Regeneron collaboration is expected to achieve 1-2 new INDs per
year starting in 2021, adding to the Company’s expected proprietary
flow of 1-2 new INDs per year.
The Company’s R&D Day will be held today, November 22, 2019
from 8:00 am to 12:30 pm ET at the Westin New York at Times Square
in New York City and will include a live video stream on the
Investors section of the Company’s website, www.alnylam.com. A
replay will be available on the Alnylam website within 48 hours
after the event. Presentations showcased during the event will be
featured on Capella (www.alnylam.com/capella).
About RNAi
RNAi (RNA interference) is a natural cellular process of gene
silencing that represents one of the most promising and rapidly
advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough
that happens once every decade or so,” and was recognized with the
award of the 2006 Nobel Prize for Physiology or Medicine. By
harnessing the natural biological process of RNAi occurring in our
cells, a new class of medicines, known as RNAi therapeutics, is now
a reality. Small interfering RNA (siRNA), the molecules that
mediate RNAi and comprise Alnylam's RNAi therapeutic platform,
function upstream of today’s medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors – that encode for
disease-causing proteins, thus preventing them from being made.
This is a revolutionary approach with the potential to transform
the care of patients with genetic and other diseases.
About Alnylam
Alnylam (Nasdaq: ALNY) is leading the translation of RNA
interference (RNAi) into a whole new class of innovative medicines
with the potential to transform the lives of people afflicted with
rare genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning
science, RNAi therapeutics represent a powerful, clinically
validated approach for the treatment of a wide range of severe and
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust RNAi therapeutics platform. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), approved in the
U.S., EU, Canada, Japan, and Switzerland, and GIVLAARI™
(givosiran), approved in the U.S. Alnylam has a deep pipeline of
investigational medicines, including five product candidates that
are in late-stage development. Alnylam is executing on its "Alnylam
2020" strategy of building a multi-product, commercial-stage
biopharmaceutical company with a sustainable pipeline of RNAi-based
medicines to address the needs of patients who have limited or
inadequate treatment options. Alnylam employs over 1,200 people
worldwide and is headquartered in Cambridge, MA. For more
information about our people, science and pipeline, please visit
www.alnylam.com and engage with us on Twitter at @Alnylam or on
LinkedIn.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's views and plans with respect to the potential for RNAi
therapeutics, including lumasiran, vutrisiran, inclisiran,
fitusiran, cemdisiran, ALN-AAT02, ALN-HBV02, ALN-AGT, ALN-HSD,
ALN-LEC, ALN-APP, its program for Huntington’s disease and other
pre-clinical programs, including in the eye and CNS, its plans for
additional regulatory filings and the continuing product launch of
ONPATTRO, its plans for additional regulatory filings and launch of
GIVLAARI, its 2020 product and pipeline goals, including the
expected timing of data readouts, regulatory filings and potential
regulatory approvals, its expectations and plans with respect to a
general framework for progressing toward a self-sustainable
financial profile, expectations with respect to future INDs, and
expectations regarding the potential to exceed its “Alnylam 2020”
guidance for the advancement and commercialization of RNAi
therapeutics, constitute forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. Actual results and future plans may
differ materially from those indicated by these forward-looking
statements as a result of various important risks, uncertainties
and other factors, including, without limitation, Alnylam's ability
to discover and develop novel drug candidates and delivery
approaches, successfully demonstrate the efficacy and safety of its
product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to
occur in other subjects or in additional studies or otherwise
support further development of product candidates for a specified
indication or at all, actions or advice of regulatory agencies,
which may affect the design, initiation, timing, continuation
and/or progress of clinical trials or result in the need for
additional pre-clinical and/or clinical testing, delays,
interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting
intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, including ONPATTRO and GIVLAARI, progress in continuing
to establish a commercial and ex-United States infrastructure,
successfully launching, marketing and selling its approved products
globally, including ONPATTRO and GIVLAARI, Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future,
competition from others using technology similar to Alnylam's and
others developing products for similar uses, Alnylam's ability to
manage its growth and operating expenses and achieve a
self-sustainable financial profile in the future, obtain additional
funding to support its business activities, and establish and
maintain strategic business alliances and new business initiatives,
Alnylam's dependence on third parties, including Regeneron, for
development, manufacture and distribution of products, and
Ironwood, for assistance with the education about and promotion of
GIVLAARI, the outcome of litigation, the risk of government
investigations, and unexpected expenditures, as well as those risks
more fully discussed in the "Risk Factors" filed with Alnylam's
most recent Quarterly Report on Form 10-Q filed with the Securities
and Exchange Commission (SEC) and in other filings that Alnylam
makes with the SEC. In addition, any forward-looking statements
represent Alnylam's views only as of today and should not be relied
upon as representing its views as of any subsequent date. Alnylam
explicitly disclaims any obligation, except to the extent required
by law, to update any forward-looking statements.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191122005105/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340 Joshua Brodsky (Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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