– Provides Opportunity for Patients who
Participate in APOLLO Phase 3 Trial to Receive Patisiran on Ongoing
Basis –
– Consistent With Previous Guidance, Company
Expects APOLLO, if Positive, to Enable New Drug Application (NDA)
Filing in 2017 Timeframe –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, announced today that it has initiated its
Phase 3 open-label extension study (abbreviated “APOLLO-OLE”) with
patisiran, an investigational RNAi therapeutic targeting
transthyretin (TTR) for the treatment of TTR-mediated amyloidosis
(ATTR amyloidosis) in patients with Familial Amyloidotic
Polyneuropthy (FAP). All patients who complete the APOLLO Phase 3
trial with patisiran are eligible to enroll in APOLLO-OLE. The
study will evaluate the long-term safety and efficacy of patisiran
and will also measure effects of treatment toward a number of
clinical endpoints, including the modified Neuropathy Impairment
Score, or “mNIS+7,” which is an evaluation of muscle weakness,
sensory and autonomic function, and nerve conductance. The company
is also reiterating its previous guidance that – assuming positive
study results in the APOLLO Phase 3 study – it expects to be in a
position to file a New Drug Application (NDA) for patisiran in the
2017 timeframe.
“Patisiran is the leading investigational candidate in our
pipeline of RNAi therapeutics. We are very encouraged with the
clinical activity and tolerability seen to date with patisiran,
including in our Phase 2 OLE study where we have recently reported
what we believe to be continued evidence for a possible halting of
neuropathy progression after the first 12 months of treatment,”
said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of
R&D and Chief Medical Officer of Alnylam. “The initiation of
APOLLO-OLE marks our continued progress with patisiran, and we are
pleased to provide patients previously treated in APOLLO with the
opportunity to receive patisiran on an ongoing basis. The APOLLO
trial continues enrolling patients with FAP, and, assuming positive
results, we expect to be in a position to file an NDA in the 2017
timeframe. Accordingly, we believe successful execution of the
APOLLO Phase 3 trial is a critical step toward achieving our
‘Alnylam 2020’ goals where, by the end of 2020, we expect to have 3
marketed products, as well as 10 programs in clinical development,
including 4 in late stages of development, across our 3 Strategic
Therapeutic Areas, or ‘STArs.’”
“Therapeutic options for ATTR amyloidosis patients with FAP are
limited, and there is a significant need for novel therapies to
treat this debilitating disease. I am pleased with Alnylam’s
progress to date with patisiran, and find the recent Phase 2 OLE
data to be very encouraging. In particular, the possibility of
halting neuropathic progression over 12 months of treatment is
promising in light of the rapid increase in neuropathy impairment
scores observed in analysis of other historical data sets,” said
Juan Buades, M.D., Ph.D., Internal Medicine Specialist, and Head of
the TTR-FAP Clinic, Hospital Son Llatzer, Palma de Mallorca, Spain.
“I very much look forward to continuing to participate in the
clinical advancement of this investigational medicine.”
APOLLO-OLE is an open-label, multi-center study designed to
evaluate the long-term safety and tolerability of patisiran in ATTR
amyloidosis patients with FAP who were previously enrolled in the
APOLLO Phase 3 study. Eligible patients treated in APOLLO can
enroll in the study, where they will receive patisiran at a dose of
0.3 mg/kg every three weeks for up to the earlier of two years or
until the drug is commercially available in their market. The
primary objective of APOLLO-OLE is to evaluate the long-term safety
and efficacy of patisiran administration. The study will measure a
number of clinical endpoints, including Neuropathy Impairment Score
at baseline and every 12 months thereafter. A number of additional
clinical endpoints will be assessed, including: quality of life;
timed 10-meter walk test to evaluate mobility; modified body mass
index as a measure of nutritional status; level of disability; and
nerve fiber density in skin biopsies.
In addition to the APOLLO and APOLLO-OLE trials, Alnylam is also
currently conducting a Phase 2 OLE study, where patients previously
treated in a Phase 2 study are receiving open-label patisiran at a
dose of 0.3 mg/kg every 3 weeks. Initial 12-month clinical data
from this study were presented recently at the 67th Annual Meeting
of the American Academy of Neurology (AAN) held April 18 – 25,
2015. Patisiran was found to be generally well tolerated out to 17
months of drug administration, with no drug-related serious adverse
events to date. In addition, patisiran treatment achieved a
sustained mean serum TTR knockdown at the 80% target level for
approximately 16 months, with an up to 88% mean knockdown achieved
between doses. Results of neuropathy assessments showed a mean 2.5
point decrease in mNIS+7 at 12 months in patients (N=20) who had
reached the 12-month endpoint at the time of data cutoff. This
decrease in neuropathy progression compares favorably to the 13 to
18 point increase in mNIS+7 at 12 months that can be estimated from
the literature in untreated FAP patients with similar baseline
characteristics. In aggregate, these results are consistent with
the therapeutic hypothesis that TTR knockdown has the potential to
halt neuropathy progression in patients with FAP. Alnylam plans to
report complete 12-month data (N=27) and preliminary 18-month data
from the patisiran Phase 2 OLE study in late 2015.
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), including co-development/co-commercialization and/or global
product rights in certain defined instances. In the case of
patisiran, Alnylam will advance the product in North America and
Western Europe, while Genzyme will advance the product in the
ROW.
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited,
progressively debilitating, and often fatal disease caused by
mutations in the TTR gene. TTR protein is produced primarily in the
liver and is normally a carrier of vitamin A. 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) is
estimated to affect at least 40,000 people worldwide. FAP 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). FAC is fatal
within 2.5 to 5 years of diagnosis and treatment is currently
limited to supportive care. 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 TTR amyloid
polyneuropathy and/or cardiomyopathy.
About LNP Technology
Alnylam has licenses to Tekmira LNP intellectual property for
use in RNAi therapeutic products using LNP technology.
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 patisiran for the treatment of ATTR
amyloidosis in patients with FAP, expectations regarding the
reporting of data from clinical studies, in particular the ongoing
Phase 2 OLE study with patisiran, expectations regarding the filing
of a New Drug Application and/or foreign equivalent for patisiran,
expectations regarding its STAr pipeline growth strategy, and its
plans regarding commercialization of RNAi therapeutics, including
patisiran, 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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version on businesswire.com: http://www.businesswire.com/news/home/20150720005361/en/
Alnylam Pharmaceuticals, Inc.Michael Mason,
617-551-8327Vice President, Finance and
TreasurerorMedia:SpectrumLiz Bryan, 202-955-6222 x2526
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