CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that Alnylam scientists and collaborators will present results from ongoing Phase 2 open-label extension (OLE) studies with its investigational RNAi therapeutics in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) at the First European Congress on Hereditary ATTR Amyloidosis being held November 2-3, 2015 in Paris, France. The presentations will include 18-month patisiran data for the treatment of patients with Familial Amyloidotic Polyneuropathy (FAP) and 6-month revusiran data for the treatment of patients with TTR cardiac amyloidosis, including Familial Amyloidotic Cardiomyopathy (FAC) and Senile Systemic Amyloidosis (SSA), a non-hereditary form of TTR cardiac amyloidosis. The Company will host a conference call on Tuesday, November 3 at 7:00 a.m. ET to discuss these results.
“We are excited to share these data with world leading experts and clinicians in the ATTR community,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We continue to collect safety and patient experience data in our OLE studies with the goal of providing much needed therapeutic options for patients with this difficult-to-treat condition."
Presentations by Alnylam scientists and collaborators at the meeting include:
- DISCOVERY: A Study Examining the Prevalence of Transthyretin Mutations in Subjects Suspected of Having Cardiac Amyloidosis
| Author: | Olakunle Akinboboye, Queens Heart Institute | |||
| Session: | Epidemiology and Phenotypes in ATTR Amyloidosis | |||
| Date/Time: |
Monday, November 2, 9:30 – 10:30 a.m. CET |
- Analysis of Disease Progression in Patients with Transthyretin Cardiac Amyloidosis
| Author: | Julian D. Gillmore, University College London Medical School | |||
| Session: | New Tools for Diagnosis | |||
| Date/Time: | Monday, November 2, 4:10 – 5:10 p.m. CET |
- Patients with Hereditary ATTR Amyloidosis Experience an Increasing Burden of Illness as the Disease Progresses
| Author: | David Adams, French Reference Centre for FAP | |||
| Session: | Poster session II | |||
| Date/Time: | Tuesday, November 3, 7:45 – 8:30 a.m. CET |
- Phase 2 Open-Label Extension (OLE) Study of Patisiran, an Investigational siRNA Agent for Familial Amyloidotic Polyneuropathy (FAP)
| Author: | David Adams, French Reference Centre for FAP | |||
| Session: | Disease Modifying Therapy | |||
| Date/Time: | Tuesday, November 3, 10:00 – 11:00 a.m. CET |
- Phase 2 Open-Label Extension (OLE) Study of Revusiran, an Investigational RNAi Therapeutic for the Treatment of Patients with Transthyretin Cardiac Amyloidosis
| Author: | Julian D. Gillmore, University College London Medical School | |||
| Session: | Disease Modifying Therapy | |||
| Date/Time: | Tuesday, November 3, 10:00 – 11:00 a.m. CET |
Conference Call Information
Alnylam management will discuss
these data in a webcast conference call on Tuesday, November 3 at 7:00
a.m. ET. A slide presentation will also be available on the Investors
page of the company's website, www.alnylam.com,
to accompany the conference call. To access the call, please dial
877-312-7507 (domestic) or 631-813-4828 (international) five minutes
prior to the start time and refer to conference ID 70778772. A replay of
the call will be available beginning at 9:00 a.m. ET. To access the
replay, please dial 855-859-2056 (domestic) or 404-537-3406
(international), and refer to conference ID 70778772.
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 for
certain programs. 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. In the case of revusiran, Alnylam and Genzyme
will co-develop/co-commercialize 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 amyloidosis) 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 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 and revusiran
for the treatment of TTR-mediated amyloidosis, and its plans regarding
commercialization of RNAi therapeutics, including patisiran and
revusiran, 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.
