– APOLLO-B Will Enroll Patients with Both
Hereditary and Wild-Type Amyloidosis –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the Company has
initiated APOLLO-B, a global Phase 3 placebo-controlled clinical
trial of patisiran, an intravenously administered RNAi therapeutic,
for the treatment of transthyretin amyloidosis (ATTR amyloidosis)
with cardiomyopathy. The primary endpoint is the change from
baseline in the 6-minute walk test (6-MWT) at 12 months. Secondary
endpoints will evaluate the efficacy of patisiran on quality of
life using the Kansas City Cardiomyopathy Questionnaire Overall
Summary, and composite endpoints of mortality and
hospitalization.
“ATTR amyloidosis is a rare, debilitating, and life-threatening
disease encompassing hereditary ATTR (hATTR) amyloidosis and
wild-type ATTR (wtATTR) amyloidosis. Based on the encouraging
exploratory results on cardiac endpoints in the Phase 3 APOLLO
study, we are investigating the potential for patisiran to treat
cardiovascular-related manifestations of ATTR amyloidosis,” said
Eric Green, Senior Vice President and General Manager, TTR Program.
“The initiation of APOLLO-B represents a significant milestone in
our commitment to explore the full potential of patisiran for
patients living with all types of ATTR amyloidosis.”
Patisiran is the non-branded drug name for ONPATTRO®. It is
approved by the U.S. Food and Drug Administration (FDA) for the
treatment of the polyneuropathy of hATTR amyloidosis in adults.
ONPATTRO is also approved in the European Union, Canada, and
Japan.
APOLLO-B Phase 3 Study Design The APOLLO-B Phase 3 trial
is a randomized, double-blind, placebo-controlled multicenter
global study designed to evaluate the efficacy and safety of
patisiran in approximately 300 adult patients with ATTR amyloidosis
(hereditary or wild type) with cardiomyopathy. Patients will be
randomized on a 1:1 basis to receive 0.3 mg/kg of patisiran or
placebo intravenously administered every three weeks over a
24-month treatment period. After 12 months, all patients will
receive patisiran in an open-label treatment period. For more
information on APOLLO-B (NCT03997383), including the full list of
eligibility criteria, please visit www.clinicaltrials.gov, email
clinicaltrials@alnylam.com or call 877-256-9526 in North America
and +31 20 369 7861 in Europe.
About ONPATTRO■ (Patisiran) ONPATTRO is an RNAi
therapeutic that is approved by the U.S. Food and Drug
Administration (FDA) for the treatment of the polyneuropathy of
hATTR amyloidosis in adults. ONPATTRO is also approved in the
European Union for the treatment of hATTR amyloidosis in adults
with Stage 1 or Stage 2 polyneuropathy, in Canada for the treatment
of hATTR amyloidosis with polyneuropathy by Health Canada, and in
Japan for the treatment of hATTR amyloidosis with polyneuropathy by
the Japanese Ministry of Health, Labour and Welfare (MHLW).
Patisiran is also being investigated in patients with ATTR
amyloidosis (hereditary [hATTR] or wild type [wtATTR]) with
cardiomyopathy in the APOLLO-B study. Based on Nobel Prize-winning
science, ONPATTRO is an intravenously administered RNAi therapeutic
targeting transthyretin (TTR) for the treatment of hereditary ATTR
amyloidosis. 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 disease.
ONPATTRO Indication and Important Safety Information
Infusion-Related Reactions Infusion-related reactions (IRRs)
have been observed in patients treated with ONPATTRO. In a
controlled clinical study, 19 percent of ONPATTRO-treated patients
experienced IRRs, compared to 9 percent 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, paracetamol, 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 respiratory-tract
infection (29 percent) and infusion-related reactions (19
percent).
About Transthyretin (ATTR) Amyloidosis Transthyretin
amyloidosis (ATTR) amyloidosis is a rare, serious,
life-threatening, multisystemic disease encompassing hereditary
ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis,
which result from either hereditary (genetic mutation) or
nonhereditary (ageing) causes, respectively. In ATTR amyloidosis,
misfolded TTR proteins accumulate as amyloid fibrils in multiple
organs and tissue types. hATTR amyloidosis can include sensory and
motor, autonomic and cardiac symptoms and is estimated to impact
50,000 people worldwide. wtATTR amyloidosis predominantly manifests
as cardiomyopathy and heart failure symptoms, although patients may
experience other manifestations due to extra-cardiac amyloid
deposition. The disease 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 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 discovery platform. Alnylam’s first
commercial RNAi therapeutic is ONPATTRO® (patisiran), approved in
the U.S., EU, Canada, and Japan. Alnylam has a deep pipeline of
investigational medicines, including five product candidates that
are in late-stage development. Looking forward, Alnylam will
continue to execute 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 plans
concerning the APOLLO-B study of patisiran and the potential for
patisiran to treat cardiovascular-related manifestations of ATTR
amyloidosis in patients qualifying for the study, and expectations
regarding 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, progress in establishing a commercial and ex-United
States infrastructure, successfully launching, marketing and
selling its approved products globally, 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 or marketing products for similar uses, Alnylam's
ability to manage its growth and operating expenses, 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 for
development, manufacture and distribution of products, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190916005255/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340 Josh Brodsky (Investors)
617-551-8276
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