Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation to
vutrisiran, an investigational therapeutic for the treatment of the
polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults. According to the FDA, Fast Track designation
is designed to facilitate the development and expedite the review
of drugs that treat serious conditions and fill an unmet medical
need. With this designation, Alnylam will be eligible to submit a
rolling New Drug Application for vutrisiran.
“Vutrisiran has demonstrated an encouraging safety profile in
the Phase 1 study, with infrequent quarterly dosing with
low-volume, subcutaneous administration which potentially reduces
the burden of care for this progressive, life-threatening and
multisystem disease. We are therefore pleased that the FDA has
granted vutrisiran Fast Track designation,” said Rena Denoncourt,
Vutrisiran Program Leader at Alnylam. “After completing enrollment
earlier this year, we look forward to sharing topline results of
the HELIOS-A Phase 3 study of vutrisiran in early 2021. More
broadly, we remain committed to developing additional therapeutic
options for the treatment of ATTR amyloidosis to augment the
market-leading position of ONPATTRO® (patisiran), approved for the
treatment of the polyneuropathy of hATTR amyloidosis in
adults.”
In addition to Fast Track designation, vutrisiran has been
granted Orphan Drug designation in the United States and the
European Union for the treatment of ATTR amyloidosis. The safety
and efficacy of vutrisiran are being evaluated in the ongoing
HELIOS-A and HELIOS-B Phase 3 clinical trials. Together, these
studies comprise a comprehensive clinical development program
intended to demonstrate the broad impact of vutrisiran across the
multisystem manifestations of disease and the full spectrum of
patients with ATTR amyloidosis.
About Vutrisiran Vutrisiran is an investigational,
subcutaneously-administered RNAi therapeutic in development for the
treatment of ATTR amyloidosis, which encompasses both hereditary
(hATTR) and wild-type (wtATTR) amyloidosis. It is designed to
target and silence specific messenger RNA, blocking the production
of wild-type and mutant transthyretin (TTR) protein before it is
made. Quarterly 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 next-generation delivery platform
known as the Enhanced Stabilization Chemistry
(ESC)-GalNAc-conjugate delivery platform. 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 is a Phase 3
global, randomized, open-label study to evaluate the efficacy and
safety of vutrisiran in patients with hATTR amyloidosis with
polyneuropathy. The trial randomized patients 3:1 to receive either
25mg of vutrisiran subcutaneously once every 12 weeks or 0.3 mg/kg
of patisiran intravenously once every three weeks. For most
endpoints, results from the vutrisiran arm will be compared to
results from the placebo arm of the landmark APOLLO Phase 3 study,
which evaluated the efficacy and safety of patisiran in people with
hATTR amyloidosis with polyneuropathy. The co-primary endpoints of
HELIOS-A are the change from baseline in the modified Neurologic
Impairment Score +7 (mNIS+7) and in the Norfolk Quality of
Life-Diabetic Neuropathy (Norfolk QoL-DN) score, at 9 months.
Secondary endpoints include the change from baseline in key
clinical evaluations including the timed 10-meter walk test
(10-MWT), modified body mass index (mBMI), and Rasch-built Overall
Disability Scale (R-ODS). The percent reduction in serum
transthyretin (TTR) levels in the vutrisiran arm will be compared
to the within-study patisiran arm. Additional exploratory endpoints
will be assessed to determine the effect of vutrisiran on other
aspects of the multisystem nature of this disease, including
manifestations of cardiac amyloid involvement.
About HELIOS-B Phase 3 Study 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.
ONPATTRO Important Safety Information 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%).
Indication ONPATTRO is indicated
for the treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults.
For additional information about ONPATTRO, please see the full
Prescribing Information.
About Transthyretin (ATTR) Amyloidosis Transthyretin
(ATTR) amyloidosis is a rare, progressively debilitating, and fatal
disease caused by misfolded TTR proteins that accumulate as amyloid
deposits in multiple tissues including the nerves, heart and
gastrointestinal (GI) tract. There are two types of ATTR
amyloidosis: hereditary ATTR (hATTR) amyloidosis and wild-type
(wtATTR) amyloidosis. hATTR amyloidosis is an inherited disease
resulting in intractable peripheral sensory-motor neuropathy,
autonomic neuropathy, and/or cardiomyopathy. It is estimated to
affect 50,000 people worldwide. The condition can have a
debilitating impact on a patient’s life and may lead to premature
death within 4.7 years of diagnosis. wtATTR amyloidosis is a
nonhereditary, progressive type of the disease with undefined
etiology. It affects an estimated 200,000-300,000 people worldwide.
It primarily manifests as cardiomyopathy, which leads to heart
failure and mortality within 2 to 6 years.
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, Switzerland and Brazil,
and GIVLAARI® (givosiran), approved in the U.S. and EU. 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 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.
Forward Looking Statements Various statements in this
release, including, without limitation, Alnylam's views and plans
with respect to the potential for RNAi therapeutics, including
vutrisiran, its expectations with respect to the encouraging safety
profile of vutrisiran in the Phase 1 study, timing for reporting
topline results from its HELIOS-A Phase 3 study, its commitment to
developing multiple therapeutic options for the treatment of ATTR
amyloidosis, the intended goals of the HELIOS-A and -B studies to
demonstrate the broad impact of vutrisiran across the multisystem
manifestations of disease and the full spectrum of patients with
ATTR amyloidosis, and expectations regarding the continued
execution on 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: potential risks to Alnylam’s business, activities and
prospects as a result of the COVID-19 pandemic, or delays or
interruptions resulting therefrom; Alnylam's ability to discover
and develop novel drug candidates and delivery approaches and
successfully demonstrate the efficacy and safety of its product
candidates, including vutrisiran; 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 or its marketed products; obtaining, maintaining
and protecting intellectual property; intellectual property matters
including potential patent litigation relating to its platform,
products or product candidates; obtaining regulatory approval for
its product candidates, including inclisiran and lumasiran, and
maintaining regulatory approval and obtaining pricing and
reimbursement for its 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, and achieve net product revenues for ONPATTRO within our
expected range during 2020; 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 within the ranges of our expected guidance
and achieve a self-sustainable financial profile in the future
without the need for future equity financing; Alnylam’s ability to
establish and maintain strategic business alliances and new
business initiatives; Alnylam's dependence on third parties,
including Regeneron, for development, manufacture and distribution
of certain products, including eye and CNS 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
Annual Report on Form 10-K 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/20200414005125/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Joshua Brodsky
(Investors) 617-551-8276
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