− APOLLO-B is the Industry’s Furthest
Progressed Randomized, Double-Blind, Placebo-Controlled Pivotal
Study of an Investigational TTR Silencer in Hereditary and
Wild-type ATTR Amyloidosis with Cardiomyopathy −
− Topline Results Expected in Mid-2022 −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that it has achieved full
patient enrollment in its APOLLO-B Phase 3 study of patisiran, an
investigational RNAi therapeutic in development for the treatment
of cardiomyopathy in patients with transthyretin-mediated (ATTR)
amyloidosis. Enrollment was completed with over 300 ATTR
amyloidosis patients across 90 sites in more than 20 countries.
ATTR amyloidosis is a rare, progressively debilitating, and fatal
disease that is caused by misfolded transthyretin (TTR) proteins
that accumulate as amyloid deposits in multiple tissues, including
the nerves, heart and gastrointestinal (GI) tract and encompasses
hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR)
amyloidosis. hATTR amyloidosis is estimated to impact approximately
50,000 people worldwide and is a multisystem disease that can
include sensory and motor, autonomic, and cardiac symptoms. wtATTR
amyloidosis is estimated to impact between 200,000 and 300,000
people worldwide and primarily manifests as cardiomyopathy, which
can lead to heart failure and mortality. APOLLO-B is the industry’s
furthest progressed randomized, double-blind, placebo-controlled
study of a TTR silencer investigational medicine for the treatment
of cardiomyopathy in hATTR and wtATTR amyloidosis patients.
Patisiran is the established name for ONPATTRO®, which is
approved in the United States, Canada and Japan for the treatment
of the polyneuropathy of hATTR amyloidosis in adults and in the
European Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.
“The APOLLO-B trial was initiated based on encouraging data from
previous studies that support further investigation of patisiran in
ATTR amyloidosis patients with cardiomyopathy. Specifically,
encouraging results were obtained from the landmark Phase 3 APOLLO
study in hATTR amyloidosis patients with polyneuropathy on a number
of exploratory cardiac endpoints. Today’s milestone marks an
important step forward as we continue to study the safety and
efficacy of patisiran in the treatment of cardiovascular-related
manifestations of ATTR amyloidosis, which can often be devastating
for these patients,” said Rena Denoncourt, Vice President, TTR
Franchise Lead. “We look forward to announcing topline APOLLO-B
results in mid-2022 and are excited about the potential of
patisiran to treat multiple manifestations of this rapidly
progressive disease and further advance our aspiration to build the
industry leading franchise of RNAi therapeutics for the treatment
of ATTR amyloidosis.”
About the APOLLO-B Phase 3 Study Design
The APOLLO-B study is a Phase 3, randomized, double-blind,
placebo-controlled multicenter global study designed to evaluate
the efficacy and safety of patisiran in patients with
transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy,
which enrolled over 300 adult patients with ATTR amyloidosis
(hereditary or wild-type) with cardiomyopathy. Patients were
randomized on a 1:1 basis to receive 0.3 mg/kg of patisiran or
placebo intravenously administered every three weeks over a
12-month treatment period. After 12 months, all patients will
receive patisiran in an open-label extension period. The primary
outcome measure of APOLLO-B is the change from baseline in the
6-minute walk test at 12 months compared to placebo. Key secondary
and exploratory endpoints will evaluate the efficacy of patisiran
on health-related quality of life, mortality and cardiovascular
events, cardiac biomarkers and additional manifestations of cardiac
amyloid involvement. For more information on APOLLO-B (NCT03997383)
please visit www.clinicaltrials.gov.
About ONPATTRO® (Patisiran)
ONPATTRO is an RNAi therapeutic that is approved in the United
States and Canada for the treatment of the polyneuropathy of hATTR
amyloidosis in adults. ONPATTRO is also approved in the European
Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with
polyneuropathy. ONPATTRO is an intravenously administered RNAi
therapeutic targeting transthyretin (TTR). It is designed to target
and silence TTR messenger RNA, thereby blocking the production of
TTR protein before it is made. ONPATTRO blocks the production of
TTR in the liver, reducing its accumulation in the body’s tissues
in order to halt or slow down the progression of the polyneuropathy
associated with the disease. For more information about ONPATTRO,
including full Prescribing Information and Important Safety
Information, visit ONPATTRO.com.
ONPATTRO Indication and Important Safety Information
Indication ONPATTRO is indicated for the treatment of the
polyneuropathy of hereditary transthyretin-mediated amyloidosis in
adults.
Infusion-Related Reactions Infusion-related reactions (IRRs)
have been observed in patients treated with ONPATTRO® (patisiran).
In a controlled clinical study, 19% of ONPATTRO-treated patients
experienced IRRs, compared to 9% of placebo-treated patients. The
most common symptoms of IRRs with ONPATTRO were flushing, back
pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, acetaminophen, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
ONPATTRO infusion. Monitor patients during the infusion for signs
and symptoms of IRRs. If an IRR occurs, consider slowing or
interrupting the infusion and instituting medical management as
clinically indicated. If the infusion is interrupted, consider
resuming at a slower infusion rate only if symptoms have resolved.
In the case of a serious or life-threatening IRR, the infusion
should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels.
Supplementation at the recommended daily allowance (RDA) of vitamin
A is advised for patients taking ONPATTRO. Higher doses than the
RDA should not be given to try to achieve normal serum vitamin A
levels during treatment with ONPATTRO, as serum levels do not
reflect the total vitamin A in the body. Patients should be
referred to an ophthalmologist if they develop ocular symptoms
suggestive of vitamin A deficiency (e.g., night blindness).
Adverse Reactions The most common adverse reactions that
occurred in patients treated with ONPATTRO were upper respiratory
tract infections (29%) and infusion-related reactions (19%).
About ATTR Amyloidosis
Transthyretin-mediated (ATTR) amyloidosis is a rare, rapidly
progressive, debilitating disease caused by misfolded transthyretin
(TTR) proteins which accumulate as amyloid fibrils in multiple
tissues including the nerves, heart, and gastrointestinal (GI)
tract. There are two different types of ATTR amyloidosis –
Hereditary ATTR (hATTR) amyloidosis, caused by a TTR gene variant,
and Wild-type ATTR amyloidosis (wtATTR), which occurs without a TTR
gene variant. hATTR amyloidosis affects approximately 50,000 people
worldwide, while wtATTR amyloidosis is estimated to impact 200,000
– 300,000 people worldwide.
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 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 those
related to patisiran and its potential to treat
cardiovascular-related manifestations of ATTR amyloidosis in both
in hATTR and wtATTR amyloidosis patients, the expected timing for
topline results from the APOLLO-B Phase 3 study, Alnylam’s
aspiration to build the industry leading franchise of RNAi
therapeutics for the treatment of ATTR amyloidosis, and the
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; 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,
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 (or 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 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 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.
This release is not intended to convey conclusions about
efficacy or safety as to any investigational uses or dosing
regimens of any investigational RNAi therapeutics. There is no
guarantee that any investigational therapeutics or dosing regimens
for such therapeutics will successfully complete clinical
development or gain health authority approval.
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version on businesswire.com: https://www.businesswire.com/news/home/20210601005231/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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