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| Alnylam Initiates Phase I Clinical Study of ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) |
– Data Demonstrating TTR Knockdown Expected in Third Quarter of 2012 – “Advancement of ALN-TTR02 into the clinic is an important milestone in our ‘Alnylam 5x15’ product strategy. Indeed, ALN-TTR02 is our lead program in this effort and we expect to initiate a Phase II trial later this year, followed by a pivotal trial starting in 2013,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “ALN-TTR02 utilizes our proprietary second-generation LNP technology which has shown significant potency improvements in both pre-clinical and clinical studies. We very much look forward to our expected reporting of TTR knockdown data from the Phase I study in the third quarter of this year.”
“RNAi therapeutics represent a novel and exciting approach for the
treatment of ATTR, as this new drug modality has the potential to make a
meaningful impact in the treatment of this devastating disease. I am
very encouraged by the recent results from Alnylam’s Phase I study with
ALN-TTR01 which showed knockdown in serum levels of TTR, the pathogenic
protein in this disease. These results are important because we believe
TTR suppression has the potential of halting or even reversing disease
pathology in ATTR patients,” said
The Phase I trial of ALN-TTR02 is being conducted in the About Transthyretin-Mediated Amyloidosis Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary, systemic disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormones and retinol binding proteins. 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. In its severest form, ATTR represents a major unmet medical need with significant morbidity and mortality as an orphan disease; FAP (familial amyloidotic polyneuropathy) affects approximately 10,000 people worldwide and FAC (familial amyloidotic cardiomyopathy) affects at least 40,000 people worldwide. ATTR patients with FAP have a mean life expectancy of five to 15 years from symptom onset and the only treatment option is liver transplantation; as a result there is a significant need for novel therapeutics to treat patients who have inherited mutations in the TTR gene. About ALN-TTR Program
ALN-TTR is a systemically delivered RNAi therapeutic being developed for
the treatment of ATTR. Pre-clinical
studies have shown that treatment with Alnylam’s first-generation
product candidate, ALN-TTR01, resulted in both prevention and regression
of pathogenic TTR deposits in peripheral tissues including dorsal root
ganglia, sciatic nerve, stomach, and intestines in animal models. In 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 RNAs (siRNAs), 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 5x15™”
The “Alnylam 5x15” strategy, launched in
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-PCS for the treatment of severe
hypercholesterolemia, ALN-HPN for the treatment of refractory anemia,
ALN-APC for the treatment of hemophilia, 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 partner-based programs in clinical or development
stages, including ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and
ALN-HTT for the treatment of Huntington’s disease. The company’s
leadership position on RNAi therapeutics and intellectual property have
enabled it to form major alliances with leading companies including
Merck, 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, including ALN-TTR02 and ALN-TTRsc, its expectations
with respect to the timing and success of its clinical and pre-clinical
trials, the expected timing of regulatory filings, including its plan to
file an IND or IND equivalent application for ALN-TTRsc and to initiate
clinical trials for ALN-TTR02 and ALN-TTRsc, its expectations regarding
the reporting of data from its ALN-TTR01, ALN-TTR02 and ALN-TTRsc
clinical trials, and Alnylam’s 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
efficacy and safety of its drug candidates, including ALN-TTR02 and
ALN-TTRsc, the pre-clinical and clinical results for its product
candidates, which may not 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, 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 most recent annual report
on Form 10-K on file with the
Source:
Alnylam Pharmaceuticals, Inc.
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