Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the Company and its
collaborators will present at the 2019 International Congress on
Porphyrins and Porphyria (ICPP), being held September 8-11, 2019 in
Milan, Italy. Presentations will include additional results from
the ENVISION Phase 3 study and Phase 1/2 open-label extension study
of givosiran, an investigational RNAi therapeutic for the treatment
of acute hepatic porphyria (AHP).
Oral presentations:
- ENVISION, a Phase 3 Study of Safety and Efficacy of
Givosiran, an Investigational RNAi Therapeutic, in Acute Hepatic
Porphyria Patients Tuesday, September 10, 2019 at 11:30 am CET
Leading Authors: Gouya L, Sardh E, Rees DC
- Acute Hepatic Porphyria (AHP) Disease Manifestations and
Daily Life Impacts in EXPLORE International, Prospective, Natural
History Study Tuesday, September 10, 2019 at 9:15 am CET
Leading Authors: Anderson K, Ventura P, Balwani M
- A Drug-Drug Interaction Study to Investigate the Effect of
Givosiran on the Activity of Five Major Drug Metabolizing CYP450
Enzymes in Subjects with Acute Intermittent Porphyria (AIP) who are
Chronic High Excreters (CHE) Tuesday, September 10, 2019 at
12:00 pm CET Leading Authors: Vassiliou D, Sardh E, Harper P
Poster presentations:
- A Phase 1/2 Open-Label Extension Study of Givosiran, an
Investigational RNAi Therapeutic, in Patients with Acute
Intermittent Porphyria Monday, September 9, 2019, 10:30 to
11:00 am and 12:45 to 2:00 pm CET Leading authors: Bonkovsky HL,
Bissell DM, Sardh E
- Overall Health, Daily Functioning, and Quality of Life in
Acute Hepatic Porphyria Patients: ENVISION, a Phase 3 Global,
Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial
Monday, September 9, 2019, 10:30 to 11:00 am and 12:45 to 2:00 pm
CET Leading authors: Sardh E, Gouya L, Rees DC
- Burden of Illness in Acute Hepatic Porphyria (AHP): Insights
from Patient and Caregiver Members of the British Porphyria
Association Monday, September 9, 2019, 10:30 to 11:00 am and
12:45 to 2:00 pm CET Leading authors: Gill L, Burrell S,
Chamberlayne J
- Disease Characteristics of Acute Hepatic Porphyria Patients:
ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind,
Placebo-Controlled Trial Monday, September 9, 2019, 10:30 to
11:00 am and 12:45 to 2:00 pm CET Leading authors: Balwani M, Sardh
E, Gouya L
- The Evolving Diagnosis and Care of Patients with Acute
Hepatic Porphyria (AHP) in the UK: from 2006 to 2018 Monday,
September 9, 2019, 10:30 to 11:00 am and 12:45 to 2:00 pm CET
Leading authors: Gill L, Burrell S, Chamberlayne J
- A Review of the Predominant Symptoms of Acute Hepatic
Porphyria: Pain, Fatigue, and Nausea Monday, September 9, 2019,
10:30 to 11:00 am and 12:45 to 2:00 pm CET Leading authors: Ko J,
McHorney C, Safikhani S
About Acute Hepatic Porphyria 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: acute intermittent
porphyria (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 acute hepatic porphyria (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 porphobilinogen
(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.
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 AHP. 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.
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. Alnylam’s first commercial RNAi
therapeutic is ONPATTRO® (patisiran), approved in the U.S., EU,
Canada, and Japan. Alnylam has a deep pipeline of investigational
medicines, including five product candidates in Phase 3 studies and
one in registration. 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, Alnylam's 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 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.
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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Christine Regan
Lindenboom (Investors and
Media) 617-682-4340
Josh Brodsky
(Investors) 617-551-8276
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