– Company Demonstrates Continued Commitment to
ATTR Amyloidosis Patients with Potential for Low Volume, Once
Quarterly, Subcutaneous Dose Regimen of ALN-TTRsc02 –
– Company Expects to Present Initial Clinical
Results in Late 2016 and to Initiate a Phase 3 Study in 2017 –
Alnylam Pharmaceuticals, Inc.
(Nasdaq: ALNY), the leading RNAi therapeutics company, today
announced that it has initiated a Phase 1 clinical trial with
ALN-TTRsc02, a subcutaneously administered investigational RNAi
therapeutic for the treatment of transthyretin (TTR)-mediated
amyloidosis (ATTR amyloidosis). The Phase 1 trial will be conducted
in normal healthy volunteers. Initiation of this trial is based on
encouraging pre-clinical results, including data presented last
year at the Oligonucleotide Therapeutics Society (OTS) meeting held
October 11 – 14, 2015. The Company has guided that it expects to
report initial clinical data from this study in late 2016, and if
positive, plans to initiate a Phase 3 study in 2017.
"We believe ALN-TTRsc02 has the potential to become a
best-in-class, once-quarterly, subcutaneous treatment regimen for
the treatment of ATTR amyloidosis," said Eric Green, Vice
President, General Manager, TTR Program. "With just four doses
anticipated per year, ALN-TTRsc02 could offer patients with ATTR
amyloidosis an important new therapeutic option to manage their
disease. In parallel, we continue to make great progress with
patisiran and revusiran, with data from both programs expected to
be presented at the International Symposium on Amyloidosis meeting
next month.”
The Phase 1 trial of ALN-TTRsc02 is a randomized,
placebo-controlled, single ascending-dose study designed to enroll
up to a total of 100 normal healthy volunteers (NHVs). The primary
objective of the study is to evaluate safety and tolerability of a
single subcutaneous dose of ALN-TTRsc02. Secondary objectives
include evaluation of pharmacokinetics and clinical activity for
ALN-TTRsc02, as measured by knockdown of serum TTR levels in NHVs,
and identification of the appropriate dose and regimen for a
pivotal study.
ALN-TTRsc02 utilizes the company's Enhanced Stabilization
Chemistry (ESC)-GalNAc-siRNA conjugate delivery platform, which
enables high potency and durability with a very wide therapeutic
index. Clinical results from other RNAi therapeutic programs in
Alnylam's pipeline that utilize ESC-GalNAc technology have
demonstrated robust target gene knockdown and durability supportive
of the potential for a quarterly and, possibly, bi-annual dosing
regimen.
ALN-TTRsc02 Pre-clinical DataIn pre-clinical studies,
including those in non-human primates (NHPs), ALN-TTRsc02 achieved
potent and highly durable knockdown of serum TTR of up to 99% with
multi-month durability achieved after just a single dose at 1
mg/kg, supportive of a potentially once quarterly dose regimen. In
13-week toxicology studies, ALN-TTRsc02, when given monthly for
four doses, was generally well-tolerated with no significant
adverse events at doses as high as 120 mg/kg in rats or 300 mg/kg
in NHPs. There were no effects on platelet counts observed in
either study.
About ATTR AmyloidosisTransthyretin (TTR)-mediated
amyloidosis (ATTR amyloidosis) is a progressively debilitating and
often fatal disease caused by deposition of TTR in peripheral
tissues. TTR protein is produced primarily in the liver and is
normally a carrier of vitamin A. In hereditary ATTR amyloidosis
(hATTR), 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.
hATTR represents a major unmet medical need with significant
morbidity and mortality; hATTR with polyneuropathy (hATTR-PN) –
also known as familial amyloidotic polyneuropathy (FAP) – affects
approximately 10,000 people worldwide and hATTR with cardiomyopathy
(hATTR-CM) – also known as familial amyloidotic cardiomyopathy
(FAC) – is estimated to affect at least 40,000 people worldwide.
hATTR-PN patients have a life expectancy of 5 to 15 years from
symptom onset, and the only approved treatment options for early
stage disease are liver transplantation and tafamidis (approved
in Europe, certain countries in Latin America
and Japan, where it is approved for all stages of disease).
hATTR-CM is fatal within 2.5 to 5 years of diagnosis and treatment
is currently limited to supportive care. Wild-type amyloidosis
(wtATTR) – also called senile systemic amyloidosis (SSA) – is a
non-hereditary form of TTR cardiac amyloidosis caused by idiopathic
deposition of wild-type TTR; its prevalence is generally unknown,
but is associated with advanced age. There is a significant need
for novel therapeutics to treat patients with ATTR amyloidosis.
Sanofi Genzyme AllianceIn 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/or global rights for
certain programs. In the case of patisiran, Alnylam will advance
the product in North America and Western Europe,
while Sanofi Genzyme will advance the product in the ROW. In the
case of revusiran, Alnylam and Sanofi Genzyme will
co-develop/co-commercialize the product in North America and
Western Europe, while Sanofi Genzyme will advance the product in
the ROW. In the case of ALN-TTRsc02, Sanofi Genzyme will have the
right to opt into the program with
co-development/co-commercialization rights.
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
ALN-TTRsc02, patisiran, and revusiran, its expectations regarding
the timing of the start of clinical studies for ALN-TTRsc02 and
presentation of clinical data for ALN-TTRsc02, patisiran, and
revusiran, 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
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 for a specified indication or at all, 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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version on businesswire.com: http://www.businesswire.com/news/home/20160609005113/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276
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