− Achieved 28% and 33% Reduction in Composite
of All-Cause Mortality and Recurrent Cardiovascular Events in the
Overall and Monotherapy Populations, Respectively –
− Reduced All-Cause Mortality by 36% and 35% in
the Overall and Monotherapy Populations, Respectively, in a
Pre-Specified Secondary Endpoint –
− Demonstrated Clinically Significant Benefits
on 6-Minute Walk Test, Kansas City Cardiomyopathy Questionnaire and
NYHA Class – Key Measures of Disease Progression –
− Observed Consistent Effects in All Key
Subgroups, Including Baseline Tafamidis –
− Demonstrated Encouraging Safety, Consistent
with Established Profile –
− Alnylam to File a U.S. Supplemental New Drug
Application Using a Priority Review Voucher –
− Alnylam to Host Conference Call Today at 8:00
am ET –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced positive topline results from
its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi
therapeutic in development for the treatment of ATTR amyloidosis
with cardiomyopathy (ATTR-CM).
The study met the primary endpoint, demonstrating a
statistically significant reduction in the composite of all-cause
mortality and recurrent cardiovascular (CV) events during the
double-blind period in both the overall population (HR 0.718,
p-value 0.0118; n=654) and in the monotherapy population (patients
not receiving tafamidis at baseline; HR 0.672, p-value 0.0162;
n=395).
The study also demonstrated statistically significant
improvements across all secondary endpoints in both the overall and
monotherapy populations. This includes key measures of disease
progression: 6-minute walk test (6-MWT), Kansas City Cardiomyopathy
Questionnaire (KCCQ) and New York Heart Association (NYHA) Class at
Month 30 (p<0.025 for all). Importantly, treatment with
vutrisiran also reduced all-cause mortality in the overall
population (HR 0.645, p<0.025) and in the monotherapy population
(HR 0.655, p<0.05) up to Month 42. This was a pre-specified,
intent-to-treat analysis that included up to six months of data
from the open-label extension.
“I’m thrilled by these overwhelmingly positive data from the
HELIOS-B study, which suggest that vutrisiran has the potential to
address the needs of patients with ATTR amyloidosis with
cardiomyopathy, a steadily progressive, debilitating, and
ultimately fatal disease,” said Pushkal Garg, M.D., Chief Medical
Officer of Alnylam. “The results showed that vutrisiran improved
cardiovascular outcomes, including survival, function and quality
of life in all patient groups with ATTR cardiomyopathy. We are
moving with urgency to file these compelling data with regulators
to bring this medicine to patients around the world.”
In addition, vutrisiran demonstrated consistent effects on the
primary composite endpoint and all secondary endpoints across all
key subgroups, including baseline tafamidis use, ATTR disease type
and measures of disease severity.
In the HELIOS-B study, vutrisiran demonstrated encouraging
safety and tolerability, consistent with its established profile.
Rates of adverse events (AEs), serious AEs and AEs leading to study
drug discontinuation were similar between the vutrisiran and
placebo arms. No AEs were seen ≥3% more frequently in the
vutrisiran arm compared to the placebo arm.
“I am overjoyed by the results of the HELIOS-B study, which
suggest the potential for vutrisiran to be a transformative
medicine for patients with ATTR amyloidosis with cardiomyopathy,”
said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam.
“Assuming favorable regulatory review, vutrisiran has the potential
to become the new standard of care for the treatment of this
disease, driving Alnylam’s next era of substantial growth.”
HELIOS-B (NCT: NCT04153149) is a Phase 3, randomized,
double-blind, placebo-controlled multicenter global study designed
and powered to evaluate the efficacy and safety of vutrisiran on
the reduction of all-cause mortality and recurrent cardiovascular
events as a primary composite endpoint in patients with ATTR
amyloidosis with cardiomyopathy in the overall and monotherapy
populations. The study randomized 655 adult patients with ATTR
amyloidosis (hereditary or wild-type) with cardiomyopathy. Patients
were randomized 1:1 to receive vutrisiran 25mg or placebo
subcutaneously once every three months during a double-blind
treatment period of up to 36 months. After the double-blind period,
all eligible patients remaining on the study may receive vutrisiran
in an open-label extension period.
Detailed results from the HELIOS-B study have been submitted as
a late-breaking abstract to the European Society of Cardiology for
presentation. The Company plans to proceed with global regulatory
submissions starting later this year, including filing a
supplemental New Drug Application with the U.S. Food and Drug
Administration using a Priority Review Voucher.
Investor Webcast Information
Management will discuss the HELIOS-B topline results via
conference call on Monday, June 24, 2024, at 8:00 am ET. To access
the call, please register online at
https://register.vevent.com/register/BId4cad1b9b91f4f41a7907f0222ed55c7.
Participants are requested to register at a minimum of 15 minutes
before the start of the call. A replay of the call will be
available two hours after the call and archived on the same web
page for six months.
A live audio webcast of the call will be available on the
Investors section of the Company’s website at
www.alnylam.com/events. An archived webcast will be available on
the Company’s website approximately two hours after the event.
IMPORTANT SAFETY INFORMATION
Reduced Serum Vitamin A Levels and Recommended
Supplementation
AMVUTTRA® (vutrisiran) 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
AMVUTTRA. Higher doses than the RDA should not be given to try to
achieve normal serum vitamin A levels during treatment with
AMVUTTRA, as serum vitamin A 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 AMVUTTRA for polyneuropathy of hereditary
transthyretin-mediated amyloidosis (hATTR-PN) were arthralgia
(11%), dyspnea (7%), and vitamin A decreased (7%).
For additional information about AMVUTTRA, please see the
full Prescribing Information.
About ATTR
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly
progressive, debilitating and fatal disease caused by misfolded
transthyretin (TTR) proteins, which accumulate as amyloid deposits
in various parts of the body, including the nerves, heart and
gastrointestinal tract. Patients may present with polyneuropathy,
cardiomyopathy, or both manifestations of disease. There are two
different forms of ATTR – hereditary ATTR (hATTR), which is caused
by a TTR gene variant and affects approximately 50,000 people
worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR
gene variant and impacts an estimated 200,000 – 300,000 people
worldwide.
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers
rapid knockdown of mutant and wild‑type transthyretin (TTR),
addressing the underlying cause of transthyretin (ATTR)
amyloidosis. Administered quarterly via subcutaneous injection,
AMVUTTRA is approved and marketed in more than 15 countries for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran
is also in development for the treatment of ATTR amyloidosis with
cardiomyopathy (ATTR-CM), which encompasses both wild-type and
hereditary forms of the disease. For more information about
AMVUTTRA, including the full U.S. Prescribing Information, visit
AMVUTTRA.com.
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 or
disease pathway 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) has led 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 and prevalent diseases with unmet need. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach yielding transformative medicines.
Since its founding in 2002, Alnylam has led the RNAi Revolution and
continues to deliver on a bold vision to turn scientific
possibility into reality. Alnylam’s commercial RNAi therapeutic
products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran),
GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio®
(inclisiran), which is being developed and commercialized by
Alnylam’s partner, Novartis. Alnylam has a deep pipeline of
investigational medicines, including multiple 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 X (formerly Twitter) at @Alnylam, or on LinkedIn,
Facebook, or Instagram.
Alnylam Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. All statements
other than historical statements of fact regarding Alnylam’s
expectations, beliefs, goals, plans or prospects including, without
limitation, Alnylam’s expectations regarding the safety and
efficacy of vutrisiran for the treatment of ATTR amyloidosis with
cardiomyopathy, including its potential to be a transformative
medicine for patients with ATTR amyloidosis with cardiomyopathy;
the potential for vutrisiran to become the new standard of care for
the treatment of ATTR amyloidosis with cardiomyopathy; the
potential for vutrisiran to obtain regulatory approval for the
treatment of ATTR amyloidosis with cardiomyopathy; the potential
for vutrisiran to drive Alnylam’s next era of substantial growth;
the expected timing of the presentation of full data from the
HELIOS-B clinical trial and the filing of a U.S. Supplemental New
Drug Application for vutrisiran; Alnylam’s plans to use a Priority
Review Voucher in connection with the Supplemental New Drug
Application for vutrisiran; the potential for vutrisiran’s clinical
profile to support first-line positioning in newly diagnosed
patients and in those patients who continue to experience disease
progression with stabilizers; and the potential for Alnylam to
achieve its Alnylam P5x25 vision of becoming a leading biopharma
company should be considered forward-looking statements. 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, risks and uncertainties relating to: Alnylam’s
ability to successfully execute on its “Alnylam P5x25” strategy;
Alnylam’s ability to successfully demonstrate the efficacy and
safety of its product candidates; the pre-clinical and clinical
results for Alnylam’s product candidates, including vutrisiran;
actions or advice of regulatory agencies and Alnylam’s ability to
obtain regulatory approval for its product candidates, including
vutrisiran, as well as favorable pricing and reimbursement;
successfully launching, marketing and selling Alnylam’s approved
products globally; and any delays, interruptions or failures in the
manufacture and supply of Alnylam’s product candidates or its
marketed products; as well as those risks more fully discussed in
the “Risk Factors” filed with Alnylam’s 2023 Annual Report on Form
10-K filed with the Securities and Exchange Commission (SEC), as
may be updated from time to time in Alnylam’s subsequent Quarterly
Reports on Form 10-Q 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/20240624263080/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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