Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi
therapeutics company, announced today that the European Medicines
Agency (EMA) has granted access to its Priority Medicines (PRIME)
scheme for givosiran (ALN-AS1), an investigational RNAi therapeutic
targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment
of acute hepatic porphyrias. The purpose of the PRIME initiative is
to bring treatments to patients faster by enhancing the EMA’s
support for the development of medicines for diseases where there
is an unmet medical need and where early clinical data show
potential to benefit patients.
Promising results from the Phase 1 study of givosiran formed the
basis of the application for PRIME. The ongoing Phase 1 trial is
being conducted as a randomized, double-blind, placebo-controlled
study. Specifically, data were recently reported in patients with
acute intermittent porphyria (AIP) experiencing recurrent attacks.
As presented at the 2016 American Society of Hematology (ASH)
meeting, givosiran demonstrated initial evidence for clinical
activity in AIP patients with meaningful reductions in the number
and frequency of porphyria attacks. In the first two dose cohorts,
givosiran was found to be generally well tolerated with no
drug-related serious adverse events. In the third dose cohort,
which remains blinded, one death due to acute pancreatitis,
considered unlikely related to givosiran or placebo, was reported
after the data transfer date.
“We are pleased to have givosiran accepted into the PRIME
program. We believe givosiran could be a potentially transformative
treatment option for patients with acute hepatic porphyrias, a
family of debilitating and life threatening diseases with enormous
unmet medical need,” said Jeff Miller, Vice President, General
Manager, Givosiran Program at Alnylam. “We look forward to
collaborating with the EMA on the accelerated assessment of
givosiran, with the goal of advancing this investigational medicine
into a Phase 3 trial in late 2017.”
Givosiran has previously been granted Orphan Drug Designations
in both the EU and the U.S. for the treatment of acute hepatic
porphyrias. Through the PRIME program Alnylam will have enhanced
scientific and regulatory support from the EMA, including its
advice on optimization of the development pathway and the potential
for accelerated assessment of the Marketing Authorisation
Application (MAA).
About GivosiranAlnylam is developing givosiran (formerly
known as ALN-AS1), a subcutaneously administered, investigational
RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)
for the treatment of acute hepatic porphyrias, including acute
intermittent porphyria (AIP). AIP is an ultra-rare autosomal
dominant disease caused by loss of function mutations in
porphobilinogen deaminase (PBGD), an enzyme in the heme
biosynthesis pathway that can result in accumulation of toxic heme
intermediates, including aminolevulinic acid (ALA) and
porphobilinogen (PBG). Patients with AIP can suffer from acute
and/or recurrent life-threatening attacks characterized by severe
abdominal pain, neuropathy (affecting the central, peripheral or
autonomic nervous system), and neuropsychiatric manifestations.
Givosiran is an ESC-GalNAc-siRNA conjugate targeting ALAS1, a
liver-expressed, rate-limiting enzyme upstream of PBGD in the heme
biosynthesis pathway. Inhibition of ALAS1 is known to reduce the
accumulation of heme intermediates that cause the clinical
manifestations of AIP. Givosiran has the potential to be a novel
treatment approach for the prevention of recurrent attacks.
Givosiran is an investigational compound, currently in early stage
clinical development. The safety and efficacy of givosiran have not
been evaluated by the U.S. Food and Drug Administration or any
other health authority.
About Acute Hepatic PorphyriasThe porphyrias are a family
of rare metabolic disorders with mostly autosomal dominant
inheritance predominantly caused by a genetic mutation in one of
the eight enzymes responsible for heme biosynthesis. Acute hepatic
porphyrias (AHP) constitute a subset where the enzyme deficiency
occurs within the liver, and includes acute intermittent porphyria
(AIP), hereditary coproporphyria (HCP), and variegate porphyria
(VP). Exposure of AHP patients to certain drugs, dieting, or
hormonal changes can trigger strong induction of aminolevulinic
acid synthase 1 (ALAS1), another enzyme in the heme biosynthesis
pathway, which can lead to accumulation of neurotoxic heme
intermediates that precipitate disease symptoms. Patients with AHP
can suffer from a range of symptoms that, depending on the specific
type, can include acute and/or recurrent life-threatening attacks
with severe abdominal pain, peripheral and autonomic neuropathy,
neuropsychiatric manifestations, cutaneous lesions and possibly
paralysis and death if untreated or if there are delays in
treatment. There are no approved treatments for the prevention of
attacks; the only approved treatment for acute attacks is hemin for
injection (Panhematin® or Normosang®), a preparation of heme
derived from human blood. Hemin requires administration through a
large vein or a central intravenous line and is associated with a
number of complications including thrombophlebitis or coagulation
abnormalities. Chronic administration of hemin may result in renal
insufficiency, iron overload, systemic infections (due to the
requirement for central venous access) and, in some instances,
tachyphylaxis.
About GalNAc Conjugates and Enhanced Stabilization Chemistry
(ESC)-GalNAc ConjugatesGalNAc-siRNA conjugates are a
proprietary Alnylam delivery platform and are designed to achieve
targeted delivery of RNAi therapeutics to hepatocytes through
uptake by the asialoglycoprotein receptor. Alnylam's Enhanced
Stabilization Chemistry (ESC)-GalNAc-conjugate technology enables
subcutaneous dosing with increased potency and durability, and a
wide therapeutic index. This delivery platform is being employed in
nearly all of Alnylam's pipeline programs, including programs in
clinical development.
About RNAiRNAi (RNA interference) is a revolution in
biology, representing a breakthrough in understanding how genes are
turned on and off in cells, and a completely new approach to drug
discovery and development. Its discovery has been heralded as "a
major scientific breakthrough that happens once every decade or
so," and represents one of the most promising and rapidly advancing
frontiers in biology and drug discovery today which was awarded the
2006 Nobel Prize for Physiology or Medicine. RNAi is a natural
process of gene silencing that occurs in organisms ranging from
plants to mammals. By harnessing the natural biological process of
RNAi occurring in our cells, the creation of 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, target the cause of
diseases by potently silencing specific mRNAs, thereby preventing
disease-causing proteins from being made. RNAi therapeutics have
the potential to treat disease and help patients in a fundamentally
new way.
About Alnylam PharmaceuticalsAlnylam is a
biopharmaceutical company developing novel therapeutics based on
RNA interference, or RNAi. The company is leading the translation
of RNAi as a new class of innovative medicines. Alnylam's pipeline
of investigational RNAi therapeutics is focused in 3 Strategic
Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline
of RNAi therapeutics for the treatment of rare diseases;
Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics
toward genetically validated, liver-expressed disease targets for
unmet needs in cardiovascular and metabolic diseases; and Hepatic
Infectious Disease, with a pipeline of RNAi therapeutics that
address the major global health challenges of hepatic infectious
diseases. In early 2015, Alnylam launched its "Alnylam 2020"
guidance for the advancement and commercialization of RNAi
therapeutics as a whole new class of innovative medicines.
Specifically, by the end of 2020, Alnylam expects to achieve a
company profile with 3 marketed products, 10 RNAi therapeutic
clinical programs – including 4 in late stages of development –
across its 3 STArs. The company's demonstrated commitment to RNAi
therapeutics has enabled it to form major alliances with leading
companies including Ionis, Novartis, Roche, Takeda, Merck,
Monsanto, The Medicines Company, and Sanofi Genzyme. In addition,
Alnylam holds an equity position in Regulus Therapeutics Inc., a
company focused on discovery, development, and commercialization of
microRNA therapeutics. Alnylam scientists and collaborators have
published their research on RNAi therapeutics in over 200
peer-reviewed papers, including many in the world's top scientific
journals such as Nature, Nature Medicine, Nature
Biotechnology, Cell, New England Journal of
Medicine, and The Lancet. Founded in 2002, Alnylam
maintains headquarters in Cambridge, Massachusetts. For more
information about Alnylam's pipeline of investigational RNAi
therapeutics, please visit www.alnylam.com.
Alnylam Forward Looking StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including without limitation, Alnylam's views with
respect to the potential for RNAi therapeutics, including
givosiran, its expectations regarding the timing of clinical
studies, including the initiation of a Phase 3 trial for givosiran
following interactions with regulatory authorities, its
expectations regarding scientific and regulatory support for
givosiran from the EMA and collaborating with the EMA on the
accelerated assessment of givosiran, its expectations regarding its
STAr pipeline growth strategy, and 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 our 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.
The scientific information referenced in this news release
relating to givosiran is preliminary and investigative. Givosiran
has not been approved by the U.S. Food and Drug Administration,
European Medicines Agency, 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: http://www.businesswire.com/news/home/20170301005711/en/
Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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