– Data from Ongoing Open-Label Extension Study
Highlight Durable Halting or Reversal of Neuropathy Disease
Progression Relative to Baseline and Consistent Safety Profile of
ONPATTRO in Patients with hATTR Amyloidosis –
– Alnylam to Share Data Evaluating Treatment
with ONPATTRO in Patients Previously Treated with Tafamidis –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the Company will present
new results from the Global Open-Label Extension (OLE) study of
ONPATTRO® (patisiran) in patients with hereditary ATTR (hATTR)
amyloidosis with polyneuropathy and data from a post-hoc analysis
of the APOLLO study, evaluating efficacy of ONPATTRO in patients
with previous tafamidis treatment at the Peripheral Nerve Society
(PNS) Annual Meeting, June 22-26, in Genoa, Italy. Additional data
examining ONPATTRO and hATTR amyloidosis disease burden will be
presented at PNS, and at the 5th Congress of the European Academy
of Neurology (EAN), June 29-July 2, in Oslo, Norway.
“The results being presented from the ongoing Open-Label
Extension study continue to reinforce the long-term ability of
ONPATTRO to halt or reverse neuropathy progression and improve
quality of life relative to baseline in patients with hATTR
amyloidosis with polyneuropathy, while maintaining a safety profile
consistent with previous studies,” said Eric Green, Senior Vice
President and General Manager, TTR Program at Alnylam. “In
addition, the data to be presented at the PNS and EAN meetings
illustrate the critical need to recognize, properly diagnose, and
treat the disease as early as possible.”
PNS Data Presentations:
- Long-Term Safety and Efficacy of
Patisiran in Patients with hATTR Amyloidosis: Global OLE
StudyLead Author: Michael PolydefkisPlatform Presentation
Session – Monday, June 24, 15:30 – 15:45 p.m. (CET)
- Efficacy of Patisiran in Patients
with hATTR Amyloidosis and Prior Tafamidis Use: Analysis of
APOLLOLead Author: Hollis LinPoster Session II – Monday, June
24, 12:00 – 14:00 p.m. (CET)
- Hereditary Transthyretin-Mediated
(hATTR) Amyloidosis: French Perspective on the Patient
JourneyLead Author: David AdamsPoster Session I – Sunday, June
23, 12:00 – 14:00 p.m. (CET)
- Impact of Patisiran, an RNAi
Therapeutic, on Diarrhea Symptoms in Patients with Hereditary
Transthyretin-Mediated AmyloidosisLead Author: Laura
ObiciPoster Session II – Monday, June 24, 12:00 – 14:00 p.m.
(CET)
- Variable Presentation of Hereditary
Transthyretin-Mediated (hATTR) Amyloidosis: A Single Center
Experience with the Patisiran PAAPLead Author: Yessar
HussainPoster Session II – Monday, June 24, 12:00 – 14:00 p.m.
(CET)
- Indirect Treatment Comparison of the
Efficacy of Patisiran and Inotersen for hATTR Amyloidosis with
PolyneuropathyLead Author: Laura ObiciPoster Session III –
Tuesday, June 25, 12:00 – 14:00 p.m. (CET)
EAN Data Presentations:
- Impact of Baseline Neuropathy State
in Patients with Hereditary Transthyretin-Mediated (hATTR)
Amyloidosis With or Without Patisiran TreatmentLead Author:
Theodoros KyriakidesEAN e-Presentation Session – Monday, July 1,
13:30 – 14:15 p.m. (CET)
IMPORTANT SAFETY INFORMATION
ONPATTRO is a medicine that treats the polyneuropathy caused by
an illness called hereditary transthyretin-mediated amyloidosis
(hATTR amyloidosis). ONPATTRO is used in adults only.
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 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. 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.
About hATTR amyloidosis
Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is
an inherited, progressively debilitating, and often fatal disease
caused by mutations in the TTR gene. TTR protein is primarily
produced in the liver and is normally a carrier of vitamin A.
Mutations in the TTR gene cause abnormal amyloid proteins to
accumulate and damage body organs and tissue, such as the
peripheral nerves and heart, resulting in intractable peripheral
sensory-motor neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis represents a major unmet medical need with significant
morbidity and mortality, affecting approximately 50,000 people
worldwide. The median survival is 4.7 years following diagnosis,
with a reduced survival (3.4 years) for patients presenting with
cardiomyopathy.
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 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/ocular diseases. Based on Nobel Prizewinning science, RNAi
therapeutics represent a powerful, clinically validated approach
for the treatment of diseases with high unmet need. ONPATTRO®
(patisiran) is the first-ever RNAi therapeutic approved by the U.S.
FDA for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults and by the EMA
for the treatment of hATTR amyloidosis in adults with stage 1 or
stage 2 polyneuropathy. Alnylam has a deep pipeline of
investigational medicines, including six product candidates in
Phase 3 studies. 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. Headquartered in
Cambridge, MA, Alnylam employs over 1,200 people worldwide. For
more information, 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 to present additional data concerning ONPATTRO
(patisiran), 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/20190613005348/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
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