– Company to Host Conference Call January 21st
at 8:30 am ET to Discuss Results –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the Company will present
full 18-month results from the HELIOS-A Phase 3 study of
vutrisiran, an investigational RNAi therapeutic in development for
the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis, at the Société
Francophone du Nerf Périphérique (SFNP) Annual Meeting. The meeting
will be held January 21-22 in Paris, France, and convenes
healthcare professionals focused on the peripheral nervous system.
The Company previously announced positive topline 18-month results
from the HELIOS-A study in October 2021. David Adams M.D., Ph.D.,
Department of Neurology, Coordinator of the National Reference
Center for Familial Amyloid Polyneuropathy (FAP) and Rare
Neuropathies, Bicêtre Hospital, Greater Paris University Hospitals,
AP-HP, will present “HELIOS-A: Study of Vutrisiran in Patients with
hATTR Amyloidosis” on January 21, 2022.
Vutrisiran is under review by the U.S. Food and Drug
Administration (FDA), the European Medicines Agency (EMA), the
Brazilian Health Regulatory Agency (ANVISA), and the Japanese
Pharmaceuticals and Medical Devices Agency (PMDA). Vutrisiran has
been granted Orphan Drug Designation in the U.S. and the European
Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has
also been granted a Fast Track designation in the U.S. for the
treatment of the polyneuropathy of hATTR amyloidosis in adults. In
the U.S., vutrisiran has received an action date under the
Prescription Drug User Fee Act (PDUFA) of April 14, 2022. The
Company received Orphan Drug Designation in Japan for transthyretin
type familial amyloidosis with polyneuropathy.
Conference Call
Alnylam Management will discuss the HELIOS-A 18-month results
via a conference call on Friday, January 21, 2022, at 8:30 am ET.
To access the call, please dial 877-312-7507 (domestic) or
+1-631-813-4828 (international) five minutes prior to the start
time and refer to conference ID 2239918. A replay of the call will
be available beginning at 11:30 am ET on the day of the call. To
access the replay, please dial 855-859-2056 (domestic) or
+1-404-537-3406 (international) and refer to conference ID
2239918.
About hATTR Amyloidosis
Hereditary transthyretin-mediated (hATTR) amyloidosis is an
inherited, progressively debilitating, and fatal disease caused by
variants (i.e., mutations) in the TTR gene. TTR protein is
primarily produced in the liver and is normally a carrier of
vitamin A. Variants in the TTR gene cause abnormal amyloid proteins
to accumulate and damage body organs and tissue, such as the
peripheral nerves and heart, resulting in intractable peripheral
sensory-motor neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis, represents a major unmet medical need with significant
morbidity and mortality affecting approximately 50,000 people
worldwide. The median survival is 4.7 years following diagnosis,
with a reduced survival (3.4 years) for patients presenting with
cardiomyopathy.
About Vutrisiran
Vutrisiran is an investigational, subcutaneously administered
RNAi therapeutic in development for the treatment of ATTR
amyloidosis, which encompasses both hATTR and wild-type ATTR
(wtATTR) amyloidosis. It is designed to target and silence specific
messenger RNA, potentially blocking the production of wild-type and
variant transthyretin (TTR) protein before it is made. Quarterly,
and potentially biannual, administration of vutrisiran may help to
reduce deposition and facilitate the clearance of TTR amyloid
deposits in tissues and potentially restore function to these
tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization
Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for
increased potency and high metabolic stability that may allow for
infrequent subcutaneous injections. The safety and efficacy of
vutrisiran have not been evaluated by the U.S. Food and Drug
Administration, European Medicines Agency, or any other health
authority.
About HELIOS-A Phase 3 Study
HELIOS-A (NCT03759379) is a Phase 3 global, randomized,
open-label study to evaluate the efficacy and safety of vutrisiran.
The study enrolled 164 patients with hATTR amyloidosis with
polyneuropathy at 57 sites in 22 countries. Patients were
randomized 3:1 to receive either 25mg of vutrisiran (N=122) via
subcutaneous injection once every three months or 0.3 mg/kg of
patisiran (N=42) via intravenous infusion once every three weeks
(as a reference comparator) for 18 months. The efficacy results of
vutrisiran in HELIOS-A are compared to the external placebo group
from the landmark APOLLO Phase 3 study, which evaluated the
efficacy and safety of patisiran in a patient population similar to
that studied in HELIOS-A. The primary endpoint was the change from
baseline in mNIS+7 at nine months. Secondary endpoints at nine
months were the change from baseline in the Norfolk QoL-DN score
and the timed 10-MWT. Additional secondary endpoints at 18 months
evaluated in the HELIOS-A study included change from baseline in
mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI),
Rasch-built Overall Disability Scale (R-ODS), and serum
transthyretin (TTR) levels. Additional exploratory endpoint data at
18-months, included NT-proBNP, echocardiographic measures and
cardiac amyloid burden as measured by technetium scintigraphy
imaging. Following the 18-month treatment period, all patients are
eligible to receive vutrisiran for an additional 18 months as part
of the randomized treatment extension where they will receive
either 25mg vutrisiran once quarterly or 50mg vutrisiran once every
six months.
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 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 Pharmaceuticals
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), GIVLAARI®
(givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran) being
developed and commercialized by Alnylam’s partner Novartis. Alnylam
has a deep pipeline of investigational medicines, including six
product candidates that are in late-stage development. Alnylam is
executing on its “Alnylam P5x25” strategy to deliver transformative
medicines in both rare and common diseases benefiting patients
around the world through sustainable innovation and exceptional
financial performance, resulting in a leading biotech profile.
Alnylam 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, on
LinkedIn, or on Instagram.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's
expectations, plans, aspirations, and goals, including, without
limitation, those related to the ongoing regulatory review of
vutrisiran, including the PDUFA date in the U.S., and the planned
achievement of its “Alnylam P5x25” strategy, 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: the direct or indirect impact of the
COVID-19 global pandemic or any future pandemic on Alnylam’s
business, results of operations and financial condition and the
effectiveness or timeliness of Alnylam’s efforts to mitigate the
impact of the pandemic; the potential impact of the recent
leadership transition on Alnylam’s ability to attract and retain
talent and to successfully execute on its “Alnylam P5x25” strategy;
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches and successfully demonstrate the efficacy and
safety of its product candidates; the pre-clinical and clinical
results for its product candidates; actions or advice of regulatory
agencies and Alnylam’s ability to obtain and maintain regulatory
approval for its product candidates, including vutrisiran, as well
as favorable pricing and reimbursement; successfully launching,
marketing and selling its approved products globally; delays,
interruptions or failures in the manufacture and supply of its
product candidates or its marketed products; obtaining, maintaining
and protecting intellectual property; Alnylam’s ability to
successfully expand the indication for ONPATTRO (and potentially
vutrisiran, if approved) in the future; Alnylam's ability to manage
its growth and operating expenses through disciplined investment in
operations and its ability to achieve a self-sustainable financial
profile in the future without the need for future equity financing;
Alnylam’s ability to maintain strategic business collaborations;
Alnylam's dependence on third parties for the development and
commercialization of certain products, including Novartis,
Regeneron and Vir; the outcome of litigation; the potential impact
of a current government investigation and the risk of future
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 its other SEC
filings. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20220114005025/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Josh Brodsky (Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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