-- Company to Host Conference Call on Saturday,
April 13, 2019, at 8:00 am ET (2:00 pm CET) to Discuss Results
--
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi
therapeutics company, announced today that the Company and its
collaborators will present at the European Association for the
Study of the Liver (EASL) 54th Annual International Liver
Congress™, being held April 10-14, 2019 in Vienna, Austria.
Presentations include full clinical results from the ENVISION Phase
3 study of givosiran, an investigational RNAi therapeutic targeting
aminolevulinic acid synthase 1 (ALAS1) in development for the
treatment of acute hepatic porphyria (AHP). The Company announced
positive topline results from the ENVISION Phase 3 study earlier
this month.
Presentations include:
- ENVISION, a Phase 3 Study to
Evaluate Efficacy and Safety of Givosiran, an Investigational RNAi
Therapeutic Targeting Aminolevulinic Acid Synthase 1, in Acute
Hepatic Porphyria Patients
Oral presentation, Saturday, April 13, 2019
at 10:15 am CET
Lead authors: Balwani M, Gouya L, Rees DC
- Acute Hepatic Porphyria Disease
Manifestations and Daily Life Impacts in EXPLORE International,
Prospective, Natural History Study
Poster presentation, Friday, April 12, 2019
starting at 9:00 am CET
Lead authors: Ventura P, Gouya L, Balwani
M
- Management of Acute Hepatic
Porphyria (AHP) Attacks in Europe and United States: EXPLORE
International, Prospective, Natural History Study
Poster presentation, Friday, April 12, 2019
starting at 9:00 am CET
Lead authors: Sardh E, Balwani M, Gouya L
The ENVISION study of givosiran has been selected for inclusion
in the ‘Best of ILC’, a resource prepared by EASL to highlight the
most noteworthy contributions to the scientific program each
year.
Conference Call
Alnylam management will discuss these results via a conference
call on Saturday, April 13, 2019 at 8:00 am ET (2:00 pm CET). A
slide presentation will also be available on the Investors page of
the Company’s website, www.alnylam.com, to accompany the conference
call. To access the call, please dial 866-548-4713 (domestic) or
323-794-2093 (international) five minutes prior to the start time
and refer to conference ID 3368636. A replay of the call will be
available beginning at 11:00 am ET on the day of the call. To
access the replay, please dial 888-203-1112 (domestic) or
719-457-0820 (international) and refer to conference ID
3368636.
About Acute Hepatic Porphyria
AHP refers to a family of rare, genetic diseases characterized
by potentially life-threatening attacks and for some patients
chronic debilitating symptoms that negatively impact daily
functioning and quality of life. AHP is comprised of four subtypes,
each resulting from a genetic defect leading to deficiency in one
of the enzymes of the heme biosynthesis pathway in the liver: AIP,
hereditary coproporphyria (HCP), variegate porphyria (VP), and
ALAD-deficiency porphyria (ADP). These defects cause the
accumulation of neurotoxic heme intermediates aminolevulinic acid
(ALA) and porphobilinogen (PBG), with ALA believed to be the
primary neurotoxic intermediate responsible for causing both
attacks and ongoing symptoms between attacks. Common symptoms of
AHP include severe, diffuse abdominal pain, weakness, nausea, and
fatigue. The nonspecific nature of AHP signs and symptoms can often
lead to misdiagnoses of other more common conditions such as
irritable bowel syndrome, appendicitis, fibromyalgia, and
endometriosis, and consequently, patients afflicted by AHP often
remain without a proper diagnosis for up to 15 years. In addition,
long-term complications of AHP and its treatment can include
chronic neuropathic pain, hypertension, chronic kidney disease and
liver disease, including iron overload, fibrosis, cirrhosis and
hepatocellular carcinoma. Currently, there are no treatments
approved to prevent debilitating attacks or to treat the chronic
manifestations of the disease.
About Givosiran
Givosiran is an investigational, subcutaneously administered
RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)
in development for the treatment of AHP. 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 PBG, to near normal
levels. By reducing accumulation of these intermediates, givosiran
has the potential to prevent or 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. The safety and efficacy of givosiran
were evaluated in the ENVISION Phase 3 trial with positive results;
these results have not been evaluated by the FDA, the EMA or any
other health authority and no conclusions should be drawn regarding
the safety and effectiveness of this investigational
therapeutic.
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,
Alnylam's views with respect to the potential benefits of
givosiran, 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 10-K 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.
Author Disclosures
Dr. Manisha Balwani (Principal Investigator in the ENVISION
study) receives financial compensation as an advisory board member
for Alnylam (the study sponsor and manufacturer of the study drug
givosiran).
The Icahn School of Medicine at Mount Sinai (“ISMMS”) holds
issued and pending patents related to the study drug givosiran and
has licensed these patents to Alnylam. As part of the license to
Alnylam, ISMMS will receive payments from Alnylam, including a
payment when givosiran entered Phase 3 clinical studies, as well as
future payments if givosiran becomes a marketed treatment for acute
hepatic porphyria. ISMMS, as well as the ISMMS faculty that are
named inventors on the licensed patents, will benefit
financially.
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version on businesswire.com: https://www.businesswire.com/news/home/20190327005223/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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