Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that Alnylam scientists and collaborators will present new results from ongoing clinical studies with two of its subcutaneously administered investigational RNAi therapeutics – ALN-AT3 and ALN-CC5 – at the 57th Annual Meeting of the American Society of Hematology (ASH) being held December 5-8, 2015 in Orlando, Florida.
“We very much look forward to sharing results from these ongoing clinical studies with the global hematology community,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Significant need remains for safe and effective treatment options for patients with rare hematologic diseases, and we remain focused on delivering innovative new therapies to address these serious conditions."
Presentations by Alnylam scientists and collaborators at the meeting include:
- A Subcutaneously Administered Investigational RNAi Therapeutic
(ALN-CC5) Targeting Complement C5 for Treatment of PNH and
Complement-Mediated Diseases: Interim Phase 1 Study Results
Author: Anita Hill, University of Leeds
Session: Bone Marrow Failure: Poster II
Date/Time: Sunday, December 6, 6:00 – 8:00 p.m. ET
- A Subcutaneously Administered Investigational RNAi Therapeutic
(ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim
Weekly and Monthly Dosing Results in Patients with Hemophilia
Author: John K. Pasi
Oral Session: Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia
Date/Time: Monday, December 7, 11:30 a.m. ET
Location: Room W311ABCD
- Antithrombin Reduction Corrected Thrombin Generation in Samples
from Hemophilia A and B Patients with Inhibitors
Author: Gili Kenet
Oral Session: Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia
Date/Time: Monday, December 7, 11:45 a.m. ET
Location: Room W311ABCD
Genzyme Alliance
In January 2014, Alnylam and Genzyme, a
Sanofi company, 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 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 ALN-AT3, Genzyme
has elected to opt into the program for its rest-of-world rights, while
retaining their further opt-in right to co-develop and co-promote
ALN-AT3 with Alnylam in North America and Western Europe, subject to
certain restrictions.
About Hemophilia and Rare Bleeding Disorders
Hemophilias are
hereditary disorders caused by genetic deficiencies of various blood
clotting factors, resulting in recurrent bleeds into joints, muscles,
and other major internal organs. Hemophilia A is defined by
loss-of-function mutations in Factor VIII, and there are greater than
40,000 registered persons in the U.S. and E.U. with Hemophilia A.
Hemophilia B, defined by loss-of-function mutations in Factor IX,
affects greater than 9,500 registered persons in the U.S. and E.U. Other
Rare Bleeding Disorders (RBD) are defined by congenital deficiencies of
other blood coagulation factors, including Factors II, V, VII, X, and
XI, and there are about 1,000 persons worldwide with a severe bleeding
phenotype. Standard treatment for persons living with hemophilia
involves replacement of the missing clotting factor either as
prophylaxis or on-demand therapy. However, as many as one third of
people with severe hemophilia A will develop an antibody to their
replacement factor - a very serious complication; persons in this
‘inhibitor' subset become refractory to standard replacement therapy.
There exists a small subset of persons living with hemophilia who have
co-inherited a prothrombotic mutation, such as Factor V Leiden,
antithrombin deficiency, protein C deficiency, and prothrombin G20210A.
People who have co-inherited these prothrombotic mutations are
characterized as having a later onset of disease, lower risk of
bleeding, and reduced requirements for Factor VIII or Factor IX
treatment as part of their disease management. There exists a
significant need for novel therapeutics to treat people living with
hemophilia.
About Antithrombin (AT)
Antithrombin (AT, also known as
"antithrombin III" and "SERPINC1") is a liver expressed plasma protein
and member of the "serpin" family of proteins that acts as an important
endogenous anticoagulant by inactivating Factor Xa and thrombin. AT
plays a key role in normal hemostasis, which has evolved to balance the
need to control blood loss through clotting with the need to prevent
pathologic thrombosis through anticoagulation. In hemophilia, the loss
of certain procoagulant factors (Factor VIII and Factor IX, in the case
of hemophilia A and B, respectively) results in an imbalance of the
hemostatic system toward a bleeding phenotype. In contrast, in
thrombophilia (e.g., Factor V Leiden, protein C deficiency, antithrombin
deficiency, amongst others), certain mutations result in an imbalance in
the hemostatic system toward a thrombotic phenotype. Since
co-inheritance of prothrombotic mutations may ameliorate the clinical
phenotype in hemophilia, inhibition of AT defines a novel strategy for
improving hemostasis.
About ALN-CC5
ALN-CC5 is an investigational RNAi therapeutic
targeting the C5 component of the complement pathway 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, 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.
About GalNAc Conjugates and Enhanced Stabilization Chemistry
(ESC)-GalNAc Conjugates
GalNAc-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 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 Merck, Medtronic, Novartis, Biogen, Roche, Takeda,
Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The
Medicines Company, and Genzyme, a Sanofi company. 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 ALN-AT3 and ALN-CC5, and
its plans regarding reporting data from its clinical trials of ALN-AT3
and ALN-CC5 and its plans for the commercialization of RNAi
therapeutics, including ALN-AT3, constitute forward-looking statements
for the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important 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 drug 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, actions of regulatory agencies, which
may affect the initiation, timing and progress of clinical trials,
obtaining, maintaining and protecting intellectual property, Alnylam's
ability to enforce its patents against infringers and defend its patent
portfolio against challenges from third parties, obtaining regulatory
approval for products, competition from others using technology similar
to Alnylam's and others developing products for similar uses, Alnylam's
ability to manage operating expenses, Alnylam's ability to 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,
marketing, sales and distribution of products, the outcome of
litigation, 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
to update any forward-looking statements.
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