Mar 18, 2013 Press Release for Alnylam


Alnylam Initiates Phase I Clinical Study for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR)
Mar 18, 2013
— ALN-TTRsc is Company's First Subcutaneously Delivered RNAi Therapeutic to Enter Clinical Trials —
— Data from Trial Expected Mid-2013 —
"RNAi therapeutics hold great promise for the treatment of ATTR since
they have demonstrated rapid, potent, and durable knockdown of TTR, the
disease-causing protein. We are advancing what we believe to be the
industry leading effort in ATTR; this includes ALN-TTRsc for the
treatment of FAC and ALN-TTR02 for the treatment of FAP which is
currently enrolling patients in a Phase II trial," said
The Phase I trial of ALN-TTRsc is being conducted in the
"ATTR is a genetic disease with significant unmet medical need and
limited treatment options for patients. RNAi therapeutics are a novel
and compelling approach for the treatment of ATTR, as this novel
modality has been shown to achieve robust knockdown of serum levels of
both wild-type and mutant TTR," said
ATTR is an autosomal dominant inherited disease caused by mutations in the TTR gene, which is expressed predominantly in the liver. Pre-clinical studies have shown that subcutaneous administration of ALN-TTRsc resulted in potent and sustained suppression of TTR. In non-human primates, ALN-TTRsc administration resulted in an approximately 80% reduction of TTR at doses as low as 2.5 mg/kg. In single- and multi-dose pre-clinical safety studies in rodents and non-human primates, ALN-TTRsc was found to be generally safe and well tolerated. Specifically, at doses as high as 300 mg/kg in non-human primates, ALN-TTRsc was well tolerated with no clinical signs, no adverse laboratory or histopathologic findings, no elevations in cytokines or complement, and no significant injection site reactions.
Alnylam entered
into an exclusive alliance with Genzyme, a Sanofi company, to
develop and commercialize RNAi therapeutics, including ALN-TTR02 and
ALN-TTRsc, for the treatment of ATTR in
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited,
progressively debilitating, and fatal disease caused by mutations in the
TTR gene. TTR protein is produced primarily in the liver and is normally
a carrier for retinol binding protein. Mutations in TTR cause abnormal
amyloid proteins to accumulate and damage body organs and tissue, such
as the peripheral nerves and heart, resulting in intractable peripheral
sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. ATTR
represents a major unmet medical need with significant morbidity and
mortality; familial amyloidotic polyneuropathy (FAP) affects
approximately 10,000 people worldwide and familial amyloidotic
cardiomyopathy (FAC) affects at least 40,000 people worldwide. FAP
patients have a life expectancy of five to 15 years from symptom onset,
and the only treatment options for early stage disease are liver
transplantation and tafamidis (approved in
About 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. Research findings demonstrate potent and durable target gene silencing, as well as a wide therapeutic index, with subcutaneously administered GalNAc-siRNAs from multiple "Alnylam 5x15" programs. GalNAc-siRNAs are being employed in Alnylam's ALN-TTRsc, ALN-AT3, and ALN-AS1 RNAi therapeutic programs for the treatment of transthyretin-mediated amyloidosis (ATTR), hemophilia and rare bleeding disorders, and acute intermittent porphyria, respectively, among other ‘Alnylam 5x15' programs.
About RNA Interference (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 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 with a core
focus on RNAi therapeutics for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia and rare
bleeding disorders (RBD), ALN-AS1 for the treatment of acute
intermittent porphyria (AIP), ALN-PCS for the treatment of
hypercholesterolemia, and ALN-TMP for the treatment of
hemoglobinopathies. As part of its "Alnylam 5x15TM" strategy,
the company expects to have five RNAi therapeutic products for
genetically defined diseases in clinical development, including programs
in advanced stages, on its own or with a partner by the end of 2015.
Alnylam has additional partnered programs in clinical or development
stages, including ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection and ALN-VSP for the treatment of liver cancers.
The company's leadership position on RNAi therapeutics and intellectual
property have enabled it to form major alliances with leading companies
including Merck, Medtronic, Novartis, Biogen Idec, Roche,
About "Alnylam 5x15™"
The "Alnylam 5x15" strategy, launched in
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including without limitation,
statements regarding Alnylam's views with respect to the potential for
RNAi therapeutics and its proprietary GalNAc-siRNA delivery platform,
its expectations regarding the development of ALN-TTR02 and ALN-TTRsc,
as well as other "Alnylam 5x15" programs, its expectations with respect
to the timing and success of its clinical trials, including for
ALN-TTR02 and ALN-TTRsc, its expectations regarding the reporting of
data from its ALN-TTRsc clinical trial, and its expectations regarding
its "Alnylam 5x15" product strategy, 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, successfully demonstrate the safety and efficacy of its drug
candidates, including drug candidates utilizing GalNAc-siRNA delivery,
the pre-clinical and clinical results for these product candidates,
which may not support further development of such product candidates,
actions of regulatory agencies, which may affect the initiation, timing
and progress of clinical trials for such product candidates, obtaining,
maintaining and protecting intellectual property, obtaining regulatory
approval for products, competition from others using technology similar
to Alnylam's and others developing products for similar uses, and
Alnylam's ability to establish and maintain strategic business alliances
and new business initiatives, as well as those risks more fully
discussed in the "Risk Factors" section of its Annual Report on Form
10-K for the year ended
Vice
President, Investor Relations and
or
Spectrum
Source:
News Provided by Acquire Media
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Christine Akinc
Chief Corporate Communications Officer media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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