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Alnylam to Report New Clinical Results for Patisiran at the 4th Congress of the European Academy of Neurology

Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, today announced that additional results from the APOLLO Phase 3
study of patisiran, an investigational RNAi therapeutic for the
treatment of hereditary ATTR (hATTR) amyloidosis, will be presented at
the 4th Congress of the European Academy of Neurology (EAN),
being held June 16-19, 2018 in Lisbon, Portugal.

“The results being presented at EAN add to the wealth of data from the
APOLLO Phase 3 study of patisiran that provide evidence for the
potential of this investigational RNAi therapeutic for the treatment of
hATTR amyloidosis,” said Eric Green, Vice President and General Manager,
TTR Program at Alnylam.

Presentations include:

In addition, Alnylam and collaborators will give encore presentations of
results from the Phase 1 and Phase 1/2 OLE studies of givosiran, an
investigational RNAi therapeutic for the treatment of patients with
Acute Hepatic Porphyrias (AHPs) with recurrent attacks.

Presentations include:

Alnylam is also sponsoring a Scientific Theatre presentation titled, “Inherited
Metabolic Diseases with Porphyric Neuropathy” on Saturday,
June 16, 4:30 to 4:45 pm WET and Sunday, June 17, 9:30 to 9:45 am WET,
with Prof. Jean-Charles Deybach, M.D., Ph.D., Director of the European
Porphyria Network and EXPLORE Investigator, as the speaker.

About PatisiranPatisiran is an investigational,
intravenously administered RNAi therapeutic targeting transthyretin
(TTR) in development for the treatment of hereditary ATTR amyloidosis.
It is designed to target and silence specific messenger RNA, potentially
blocking the production of TTR protein before it is made. This may help
to reduce the deposition and facilitate the clearance of TTR amyloid in
peripheral tissues and potentially restore function to these tissues.
Patisiran is currently under Priority Review as a Breakthrough Therapy
with the U.S. Food and Drug Administration (FDA) and under accelerated
assessment by the European Medicines Agency (EMA) for the treatment of
patients with hATTR amyloidosis. The FDA has set a PDUFA date of August
11, 2018. The safety and efficacy of patisiran have not been evaluated
by the FDA, the EMA or any other health authority.

About APOLLO Phase 3 StudyThe APOLLO Phase 3 study (N=225)
was a randomized, double-blind, placebo-controlled, global study
designed to evaluate the efficacy and safety of patisiran in hATTR
amyloidosis patients with polyneuropathy. The study was completed
in August 2017 and detailed study results were presented at the 1st European
ATTR Amyloidosis Meeting for Patients and Doctors on November 2, 2017.
All patients who completed the APOLLO Phase 3 study were eligible to
screen for the Global OLE study, in which they had the opportunity to
receive patisiran on an ongoing basis.

About hATTR amyloidosisHereditary transthyretin
(TTR)-mediated (hATTR) amyloidosis is an inherited, progressively
debilitating, and often fatal disease caused by mutations in the TTR
gene. TTR protein is produced primarily in the liver and is normally a
carrier of vitamin A. Mutations in TTR cause abnormal amyloid proteins
to accumulate and damage body organs and tissue, such as the peripheral
nerves and heart, resulting in intractable peripheral sensory
neuropathy, autonomic neuropathy, and/or cardiomyopathy. 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. The only approved treatment options are liver
transplantation for early stage disease and tafamidis (approved
in Europe, Japan and certain countries in Latin America, specific
indication varies by region). There is a significant need for novel
therapeutics to help treat patients with hATTR amyloidosis.

About GivosiranGivosiran is an investigational,
subcutaneously administered RNAi therapeutic targeting aminolevulinic
acid synthase 1 (ALAS1) in development for the treatment of acute
hepatic porphyrias (AHPs). Monthly administration of givosiran has the
potential to significantly lower induced liver ALAS1 levels in a
sustained manner and thereby decrease neurotoxic heme intermediates,
aminolevulinic acid (ALA) and porphobilinogen (PBG) to near normal
levels. By reducing accumulation of these intermediates, givosiran has
the potential to prevent or significantly reduce the occurrence of
severe and life-threatening attacks, control chronic symptoms, and
decrease the burden of the disease. Givosiran utilizes Alnylam’s
Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology, which
enables subcutaneous dosing with increased potency and durability and a
wide therapeutic index. Givosiran has been granted Breakthrough Therapy
designation by the U.S. Food and Drug Administration (FDA) and PRIME
designation by the European Medicines Agency (EMA). Givosiran has also
been granted orphan drug designations in both the U.S. and the EU for
the treatment of AHPs. The safety and efficacy of givosiran are
currently being investigated in the ENVISION Phase 3 clinical trial and
ongoing Phase 1/2 OLE study and have not been evaluated by the FDA, the
EMA or any other health authority.

About RNAiRNAi (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 major new
class of medicines, known as RNAi therapeutics, is on the horizon. 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 PharmaceuticalsAlnylam (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, and hepatic
infectious 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 discovery platform and deep
pipeline of investigational medicines, including four product candidates
that are in late-stage development. 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. Alnylam
employs over 800 people in the U.S. and Europe 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 StatementsVarious statements in
this release concerning Alnylam’s future expectations, plans and
prospects, including, without limitation, Alnylam’s views with respect
to data to be presented for patisiran and givosiran, and the potential
implications of such data for patients, 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, 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, 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.

Neither patisiran nor givosiran have been approved by the U.S. Food and
Drug Administration, European Medicines Agency, or any other regulatory
authority and no conclusions can or should be drawn regarding the safety
or effectiveness of these investigational therapeutics.

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