− Updated Results to be Presented from Clinical
Studies of Givosiran, an Investigational RNAi Therapeutic for the
Treatment of AHPs −
− In Addition, Data to be Presented from
EXPLORE Natural History Study Characterizing the Burden of Disease
in AHPs −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that Alnylam scientists and
collaborators will present new results from the Phase 1 and Phase
1/2 open-label extension (OLE) studies of givosiran, its
investigational RNA interference (RNAi) therapeutic, in patients
with acute hepatic porphyrias (AHPs) at the European Association
for the Study of the Liver (EASL) 53rd Annual International Liver
Congress™, being held April 11-15, 2018 in Paris, France. Data will
also be presented from the ongoing EXPLORE study, which aims to
characterize the natural history and clinical management of
patients with AHPs related to medical history, healthcare
utilization, and quality of life.
Presentations include:
- A Phase 1/2, Randomized, Placebo
Controlled and Open Label Extension Studies of Givosiran an
Investigational RNA Interference (RNAi) Therapeutic, in Patients
with Acute Intermittent PorphyriaOral presentation, Saturday,
April 14, 2018 at 10:00 am CETLead authors: E Sardh, P Harper, M
Balwani
- EXPLORE: A Prospective,
Multinational Natural History Study of Patients with Acute Hepatic
Porphyria with Recurrent AttacksOral presentation, Saturday,
April 14, 2018 at 8:15 am CETLead authors: L Gouya, J Bloomer, M
Balwani
- Impact of Acute Hepatic Porphyrias
on Quality of Life and Work Loss: An Analysis of the EXPLORE
Natural History StudyPoster presentation, Saturday, April 14,
2018 starting at 9:00 am CETLead authors: L Gouya, M Balwani, DM
Bissell
- Trends in Healthcare Utilization in
the United States and Europe associated with Patients with Acute
Hepatic Porphyria with Recurrent Attacks in EXPLORE: A Prospective,
Multinational Natural History Study of Patients with Acute Hepatic
PorphyriasPoster presentation, Saturday, April 14, 2018
starting at 9:00 am CETLead authors: L Gouya, M Balwani, DM
Bissell
Alnylam is also sponsoring a mini-workshop titled “Developing
Innovative Treatments for Patients with Rare Diseases:
Liver-Targeted RNA Interference Therapeutics” on Friday, April 13
from 6:30-7:30 pm CET.
Conference CallAlnylam management will discuss these
results via conference call on Saturday, April 14, 2018 at 2:00 pm
CET (8:00 am ET). A slide presentation will also be available on
the Investors page of the Company’s website, www.alnylam.com, to
accompany the conference call.
About GivosiranGivosiran (ALN-AS1) is an investigational
RNAi therapeutic specifically targeting liver ALAS1 mRNA and given
by subcutaneous injection for the treatment of acute hepatic
porphyrias (AHPs). 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
significantly 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 AHPs. The safety and
efficacy of givosiran are currently being investigated in the
ENVISION Phase 3 clinical trial and have not been evaluated by the
FDA, the EMA or any other health authority.
About Acute Hepatic PorphyriasAcute hepatic porphyrias
(AHPs) are a family of rare, genetic diseases characterized by
potentially life-threatening attacks and for many patients chronic
debilitating symptoms that negatively impact daily functioning and
quality of life. AHPs are 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)
– that may cause attacks and ongoing symptoms between attacks.
Common symptoms of AHPs include severe, diffuse abdominal pain,
weakness, nausea, and fatigue. The symptoms of AHPs can often
resemble that of other more common conditions such as irritable
bowel syndrome, appendicitis, fibromyalgia, and endometriosis and
consequently, patients afflicted with AHPs are often misdiagnosed
or remain undiagnosed for up to 15 years. Currently, there are
no treatments approved to prevent debilitating attacks and treat
the chronic symptoms of the disease.
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 major new class of medicines, known as
RNAi therapeutics, is on the horizon. 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 whole new
class of innovative medicines with the potential to transform the
lives of people afflicted with rare genetic, cardio-metabolic, and
hepatic infectious 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 and deep pipeline of investigational
medicines, including four 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 700 people in the U.S. and Europe 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 StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's planned
development of investigational RNAi therapeutic givosiran, its
views with respect to the potential for givosiran, and its
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, 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.
Givosiran has not been approved by the FDA, EMA, or any other
regulatory authority and no conclusions can or should be drawn
regarding its safety or effectiveness.
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version on businesswire.com: https://www.businesswire.com/news/home/20180328005740/en/
Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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