Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the Company will present
new results for patisiran, an investigational RNAi therapeutic in
development for the treatment of the cardiomyopathy of
transthyretin-mediated (ATTR) amyloidosis, at the Heart Failure
Society of America (HFSA) Annual Scientific Meeting (ASM) 2023,
October 6-9, 2023. Data from an interim analysis of the ongoing
open-label extension (OLE) period of the APOLLO-B Phase 3 study of
patisiran in patients with the cardiomyopathy of ATTR amyloidosis
through 24 months will be presented. In addition, findings from a
post-hoc analysis of the APOLLO-B Phase 3 study that further
characterize the impact of patisiran on health status and quality
of life in patients with the cardiomyopathy of ATTR amyloidosis
will be presented, as well as demographics, baseline
characteristics and safety data for patients who enrolled in the
expanded access program (EAP) for patisiran.
HFSA Presentation Details
- Baseline Characteristics of Patients with Transthyretin
Cardiac Amyloidosis Enrolled in the Patisiran Expanded Access
Program Poster 087: General ePoster Viewing Session 1 Friday,
October 6, 2023, 7:00 – 7:30 p.m. ET Presenting Author: Naveen
Saha
- Primary Results from APOLLO-B Open-label Extension Study of
Patisiran in Patients with Transthyretin Cardiac Amyloidosis
Poster 021: Oral Abstract Session 4 Saturday, October 7, 2023, 5:21
– 5:31 p.m. ET Presenting Author: Mathew Maurer
- Impact of Patisiran on Health Status and Quality of Life in
Patients with Transthyretin Cardiac Amyloidosis Poster 030:
Oral Abstract Session 6 Sunday, October 8, 2023, 8:24 – 8:34 a.m.
ET Presenting Author: Parag Kale
As previously announced, a supplemental New Drug Application
(sNDA) for patisiran for the treatment of the cardiomyopathy of
ATTR amyloidosis is under review by the U.S. Food and Drug
Administration (FDA), with an action date of October 8, 2023 under
the Prescription Drug User Fee Act (PDUFA). Patisiran is the
established name for ONPATTRO®, which is approved by the U.S. FDA
for the treatment of the polyneuropathy of hereditary ATTR
amyloidosis in adults.
ONPATTRO Indication and Important Safety Information
Indication
ONPATTRO is indicated for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with ONPATTRO. 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%).
For additional information about ONPATTRO, please see the full
U.S. Prescribing Information.
About ONPATTRO® (patisiran)
ONPATTRO (patisiran) is an RNAi therapeutic that is approved in
the United States and Canada for the treatment of the
polyneuropathy of hereditary ATTR (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 reducing
the production of TTR protein before it is made. Reducing the
pathogenic protein leads to a reduction in amyloid deposits in
tissues. Patisiran is also being evaluated for the treatment of the
cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis; the
safety and efficacy of patisiran in this indication have not been
established or evaluated by the FDA, EMA or any other health
authority.
About ATTR Amyloidosis
Transthyretin-mediated (ATTR) amyloidosis 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 amyloidosis – hereditary ATTR
(hATTR) amyloidosis, which is caused by a TTR gene variant and
affects approximately 50,000 people worldwide, and wild-type ATTR
(wtATTR) amyloidosis, which occurs without a TTR gene variant and
impacts an estimated 200,000 – 300,000 people worldwide.
About APOLLO-B Phase 3 Study
APOLLO-B is a Phase 3, randomized, double-blind,
placebo-controlled multicenter global study designed and powered to
evaluate the effects of patisiran on functional capacity and
quality of life in patients with ATTR amyloidosis with
cardiomyopathy. The study enrolled 360 adult patients with ATTR
amyloidosis (hereditary or wild-type) with cardiomyopathy at 69
sites in 21 countries. Patients were randomized 1:1 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 received patisiran in a 36-month open-label extension
period.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual
property for use in RNAi therapeutic products using LNP
technology.
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 Pharmaceuticals (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, statements, express or implied, relating to Alnylam’s
aspiration to become a leading biotech company and the planned
achievement of its “Alnylam P5x25” strategy, 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: the FDA may not
approve the sNDA for patisiran by the application PDUFA date or at
all; Alnylam’s ability to successfully execute on its “Alnylam
P5x25” strategy; 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 Alnylam’s product candidates,
including patisiran and vutrisiran; actions or advice of regulatory
agencies and Alnylam’s ability to obtain and maintain regulatory
approval for its product candidates, including patisiran and
vutrisiran, as well as favorable pricing and reimbursement;
successfully launching, marketing and selling Alnylam’s approved
products globally; delays, interruptions or failures in the
manufacture and supply of Alnylam’s product candidates or its
marketed products; delays or interruptions in the supply of
resources needed to advance Alnylam’s research and development
programs, including as may arise from recent disruptions in the
supply of non-human primates; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
indication for ONPATTRO or AMVUTTRA 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 Roche, Novartis, Sanofi, Regeneron and Vir; the
outcome of litigation; the potential risks of future government
investigations; and unexpected expenditures; as well as those risks
more fully discussed in the “Risk Factors” filed with Alnylam's
2022 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.
This release discusses investigational RNAi therapeutics and
uses of previously approved RNAi therapeutics in development and is
not intended to convey conclusions about efficacy or safety as to
those investigational therapeutics or uses. Patisiran has not been
approved by any regulatory agency for the treatment of ATTR
amyloidosis with cardiomyopathy. No conclusions can or should be
drawn regarding its safety or effectiveness in treating
cardiomyopathy in this population. There is no guarantee that any
investigational therapeutics or expanded uses of commercial
products will successfully complete clinical development or gain
health authority approval.
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Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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