CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that Alnylam scientists and collaborators will present new results from clinical studies of three of its investigational RNAi therapeutics – fitusiran, ALN-AS1 and ALN-CC5 – at the 58th Annual Meeting of the American Society of Hematology (ASH) being held December 3-6, 2016 in San Diego, California.
“The data being presented at ASH demonstrate our commitment to the development of novel treatment options for patients living with rare hematologic diseases, an area of high unmet need,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We look forward to presenting updated safety and clinical activity data from studies of fitusiran and ALN-CC5 and interim data from the ALN-AS1 phase 1 study in acute hepatic porphyria patients with recurrent porphyria attacks, for which there are limited treatment options.”
Presentations include:
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Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin
for the Treatment of Hemophilia: Updated Results from a Phase 1 and
Phase 1/2 Extension Study in Patients with Inhibitors
Author: K. John Pasi, Royal London Haemophilia Centre
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster I
Date/Time: Saturday, December 3, 5:30 – 7:30 p.m. PT
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Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin
for the Treatment of Hemophilia: Updated Results from a Phase 1 and
Phase 1/2 Extension Study in Patients without Inhibitors
Author: Margaret V. Ragni, University of Pittsburgh and Hemophilia Center of Western Pennsylvania
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Date/Time: Sunday, December 4, 6:00 – 8:00 p.m. PT
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Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of
ALN-AS1, an Investigational RNAi Therapeutic for the Treatment of
Acute Hepatic Porphyria
Author: Eliane Sardh, Karolinska University Hospital
Session: 801. Gene Therapy and Transfer: Poster I
Date/Time: Saturday, December 3, 5:30 – 7:30 p.m. PT
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A Subcutaneously Administered Investigational RNAi Therapeutic
(ALN-CC5) Targeting Complement C5 for Treatment of PNH and
Complement-Mediated Diseases: Preliminary Phase 1/2 Study Results in
Patients with PNH
Author: Anita Hill, University of Leeds
Session: 508. Bone Marrow Failure: Poster III
Date/Time: Monday, December 5, 6:00 – 8:00 p.m. PT
About Fitusiran
Fitusiran is a subcutaneously administered,
investigational RNAi therapeutic targeting antithrombin (AT) for the
treatment of hemophilia A and B and rare bleeding disorders (RBD)
currently in early stage clinical development. Fitusiran is designed to
lower levels of AT with the goal of promoting sufficient thrombin
generation to restore hemostasis and prevent bleeding in patients with
hemophilia and RBD. AT, also known as "antithrombin III" and "SERPINC1"
is a liver-expressed plasma protein and member of the "serpin" family of
proteins that acts by inactivating thrombin and other coagulation
factors. AT plays a key role in normal hemostasis by helping to limit
the process of fibrin clot formation. However, in hemophilia,
insufficient thrombin generation results in impaired fibrin clot
formation. Lowering AT in the hemophilia setting may promote the
generation of sufficient levels of thrombin needed to form an effective
fibrin clot and prevent bleeding. This rationale is supported by human
genetic data suggesting that co-inheritance of thrombophilic mutations,
including AT deficiency, may ameliorate bleeding in hemophilia. Lowering
of AT is a unique and innovative strategy for restoring hemostasis in
people with hemophilia. Fitusiran utilizes Alnylam's ESC-GalNAc
conjugate technology, which enables subcutaneous dosing with increased
potency and durability and a wide therapeutic index.
About ALN-AS1
ALN-AS1 is a subcutaneously administered,
investigational RNAi therapeutic targeting aminolevulinic acid synthase
1 (ALAS1) for the treatment of acute hepatic porphyrias, including acute
intermittent porphyria (AIP), currently in early stage clinical
development. 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. ALN-AS1 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. ALN-AS1 has
the potential to be a prophylactic approach for the prevention of
recurrent attacks, as well as for the treatment of acute porphyria
attacks.
About ALN-CC5
ALN-CC5 is a subcutaneously administered,
investigational RNAi therapeutic targeting the C5 component of the
complement pathway, currently in early stage clinical development for
the treatment of complement-mediated diseases. The complement system
plays a central role in immunity as a protective mechanism for host
defense, but its dysregulation results in life-threatening complications
in a broad range of human diseases including paroxysmal nocturnal
hemoglobinuria (PNH), atypical hemolytic-uremic syndrome (aHUS),
myasthenia gravis, neuromyelitis optica, and membranous nephropathy,
amongst others. Complement component C5, which is predominantly
expressed in liver cells, is a genetically and clinically validated
target; loss-of-function human mutations are associated with an
attenuated immune response against certain infections and intravenous
anti-C5 monoclonal antibody (mAb) therapy has demonstrated clinical
activity and tolerability in a number of complement-mediated diseases. A
subcutaneously administered RNAi therapeutic that silences C5 represents
a novel approach to the treatment of complement-mediated diseases.
ALN-CC5 utilizes Alnylam's ESC-GalNAc conjugate technology, which
enables subcutaneous dosing with increased potency and durability and a
wide therapeutic index.
The safety and efficacy of fitusiran, ALN-AS1, and ALN-CC5 have not been evaluated by the U.S. Food and Drug Administration or any other health authority.
Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi
Genzyme, the specialty care global business unit of Sanofi, formed an
alliance to accelerate and expand the development and commercialization
of RNAi therapeutics across the world. The alliance is structured as a
multi-product geographic alliance in the field of rare diseases. Alnylam
retains product rights in North America and Western Europe, while Sanofi
Genzyme obtained the right to access certain programs in Alnylam's
current and future Genetic Medicines pipeline in the rest of the world
(ROW) through the end of 2019, together with certain broader
co-development/co-commercialization rights and global rights for certain
products. In the case of fitusiran, Sanofi Genzyme has elected to opt
into the program for its ROW rights, while retaining its further opt-in
right to co-develop and co-promote fitusiran with Alnylam in North
America and Western Europe, subject to certain restrictions.
About RNAi
RNAi (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 Pharmaceuticals
Alnylam 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 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 for RNAi therapeutics, including fitusiran, ALN-AS1, and
ALN-CC5, its plans regarding the reporting of clinical data from its
clinical trials of fitusiran, ALN-AS1 and ALN-CC5, and 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 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 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.
None of Alnylam's investigational therapeutics have 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 the safety or effectiveness of these therapeutics.