− Initial Results Expected in Late 2019 –
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-AGTALN-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 HypertensionHypertension 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 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 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 PharmaceuticalsAlnylam (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 StatementsVarious 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190531005042/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
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