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Alnylam Doses First Patient in Phase 1 Study of ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension

− Initial Results Expected in Late 2019 –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam
Pharmaceuticals, Inc.
 (Nasdaq: ALNY), the leading RNAi therapeutics
company, announced today that the first patient has been dosed in the
Company’s Phase 1 study of ALN-AGT, an investigational RNAi therapeutic
targeting angiotensinogen (AGT) for the treatment of hypertension in
high unmet need populations, including patients with resistant or
refractory hypertension, chronic kidney disease or heart failure. The
Company expects to report initial results in late 2019.

“We are pleased to have initiated dosing in this Phase 1 study with the
goal of evaluating the safety and preliminary pharmacokinetic and
pharmacodynamic activity of ALN-AGT in patients with hypertension,” said
Lauren Melton, Senior Director, Program Leader, ALN-AGT program at
Alnylam. “We believe RNAi-mediated angiotensinogen silencing represents
a novel and targeted approach with the potential to offer robust and
highly durable control of blood pressure for patients with resistant or
refractory hypertension.”

The Phase 1 study is a multi-center, randomized, double-blind,
placebo-controlled trial designed to evaluate the safety, tolerability,
pharmacokinetic, and pharmacodynamic effects of subcutaneously
administered ALN-AGT in patients with essential hypertension. The study
will be conducted in four parts: Part A: single ascending dose phase in
hypertensive patients; Part B: single dose in hypertensive patients with
controlled salt intake; Part C: multi-dose phase in hypertensive
patients; and Part D: multi-dose phase in hypertensive patients who are
obese. In Parts C and D, once daily oral doses of irbesartan
(angiotensin II receptor blocker) will be used as the active comparator.
Patients will be randomized 2:1 ALN-AGT to placebo or ALN-AGT to
irbesartan. The planned enrollment for this study, including optional
cohorts, is up to 168 patients.

About ALN-AGT
ALN-AGT is an investigational, subcutaneously
administered RNAi therapeutic targeting angiotensinogen (AGT) in
development for the treatment of hypertension in high unmet need
populations. ALN-AGT utilizes Alnylam’s Enhanced Stabilization Chemistry
Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous
dosing with increased selectivity and a wide therapeutic index. The
safety and efficacy of ALN-AGT have not been evaluated by the FDA, EMA
or any other health authority.

About Hypertension
Hypertension is a complex multifactorial
disease clinically defined as a systolic blood pressure of above 130 or
a diastolic blood pressure of greater than 80 mmHg. Approximately 47
percent of U.S. adults live with hypertension with more than half of
patients on medication remaining above the blood pressure target level.
Despite the availability of antihypertensive medications, there remains
an unmet medical need, particularly given the poor rates of adherence to
existing therapies and peak and trough effects. In particular, there are
a number of high unmet need settings where novel approaches to
hypertension are warranted, including resistant and refractory
hypertension, chronic kidney disease, and heart failure.

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 Pharmaceuticals
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 Prize-winning
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 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 concerning Alnylam’s future expectations, plans and
prospects, including, without limitation, the potential of RNAi
therapeutics, in particular ALN-AGT, and its expectations regarding the
anticipated timing for initial results from the Phase 1 study of
ALN-AGT, 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 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 10Q 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.

ALN-AGT has not been evaluated by the FDA, EMA, or any other regulatory
authority and no conclusions can or should be drawn regarding the safety
or effectiveness of this investigational therapeutic.

Contacts

Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors
and Media)
617-682-4340

Josh Brodsky
(Investors)
617-551-8276

leverton

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