Alnylam Submits CTA Application for ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension in High Un...
March 11 2019 - 7:00AM
Business Wire
− Company Expects to Initiate a Phase 1 Study
in Mid-2019 –
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading
RNAi therapeutics company, announced today that the Company has
submitted a clinical trial authorization (CTA) application to The
Medicines and Healthcare products Regulatory Agency (MHRA) in the
United Kingdom to initiate a 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 plans to
initiate a Phase 1 study in mid-2019, upon obtaining MHRA
approval.
“We are excited to expand our efforts in the cardiovascular
disease setting in pursuit of a novel approach to treat
hypertension, particularly given that hypertensive disorders are
the leading preventable cause of death in modern society. With
RNAi, we believe there’s an opportunity to reimagine the treatment
of hypertension in high unmet need settings,” said Lauren Melton,
Senior Director, Program Leader, ALN-AGT program at Alnylam. “We
believe RNAi-mediated angiotensinogen silencing represents a novel
approach with the potential to provide effective and highly durable
control of blood pressure for patients with resistant or refractory
hypertension, chronic kidney disease or heart failure. Pending
feedback from the MHRA, we look forward to evaluating the safety
and preliminary pharmacodynamic activity of this molecule in
patients with hypertension.”
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 whole 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 (CNS)/ocular 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. Alnylam’s first U.S. FDA-approved RNAi
therapeutic is ONPATTRO® (patisiran) lipid complex injection
available in the U.S. for the treatment of the polyneuropathy of
hereditary transthyretin-mediated (hATTR) amyloidosis in adults. In
the EU, ONPATTRO is approved for the treatment of hATTR amyloidosis
in adults with stage 1 or stage 2 polyneuropathy. Alnylam has a
deep pipeline of investigational medicines, including five 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 1,000 people worldwide 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 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,
Alnylam's filing of a CTA for ALN-AGT and its expectations
regarding the anticipated timing for initiation of a Phase 1 study,
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 Annual Report on Form 10K 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/20190311005243/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340Josh
Brodsky(Investors)617-551-8276
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