– HELIOS-A Topline Results Expected in Early
2021 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that it has achieved full
enrollment in its HELIOS-A Phase 3 study of vutrisiran, an
investigational RNAi therapeutic in development for the treatment
of ATTR amyloidosis. The study was designed to enroll 160 patients
with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across
68 sites in 22 countries. Alnylam is on track to report topline
results from HELIOS-A in early 2021.
“We are pleased to have reached another important milestone for
our TTR program by completing enrollment of the HELIOS-A study,”
said Rena Denoncourt, Program Leader, Vutrisiran Program at
Alnylam. “We are committed to developing multiple therapeutic
options for this progressive, life-threatening and multisystem
disease, and believe that vutrisiran, as a low-dose,
once-quarterly, subcutaneously administered investigational
therapeutic, can be an attractive option for patients. We look
forward to sharing topline results in early 2021.”
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.
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 also being
evaluated in the ongoing HELIOS-B Phase 3 clinical trial in
patients with ATTR amyloidosis with cardiomyopathy. The HELIOS-B
study was initiated in late 2019 and is currently enrolling at
sites around the world. Together, the HELIOS-A and -B 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 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, 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 1,300 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, including, without limitation, Alnylam's views and
plans with respect to the potential for RNAi therapeutics,
including vutrisiran, its expectations with respect to the 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: 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 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; 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 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/20200218005307/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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