CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today at a presentation in New York City will discuss pipeline progress across its three strategic therapeutic areas (STArs) including: Genetic Medicines, Cardio-Metabolic Disease, and Hepatic Infectious Disease. The Company’s management will profile their mid-to-late stage RNAi therapeutic programs, specifically discussing the unmet medical need, clinical development plans and market potential for several innovative investigational medicines. In January 2016, the Company expects to provide additional specific 2016 guidance on pipeline programs in its three STArs.
Alnylam CEO John Maraganore, along with other members of the company’s leadership team, will provide a detailed review of key clinical development programs including the ATTR portfolio, fitusiran (ALN-AT3), ALN-CC5, ALN-AS1, and ALN-PCSsc. The Company will further underscore how it intends to deliver on its 2020 goals by:
- Leveraging a strong cash position and strategic alliances;
- Focusing on a reproducible and modular platform for drug discovery and development;
- Generating competitive and differentiated therapeutic profiles; and
- Tapping the large number of product opportunities focused on liver-expressed disease genes.
“With human proof-of-concept currently demonstrated in six programs and eight programs in total expected to be in clinical development by year-end 2015, we believe we are well on our way to achieve our Alnylam 2020 goals, where – by the end of 2020 – we expect to achieve a company profile with three marketed products, as well as ten clinical programs, including four in late stages of development, across our three STArs,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “With the financial resources to drive our pipeline forward, a reproducible and modular platform, demonstrated potent and durable knockdown of target proteins, and competitive and differentiated target profiles, we believe we have the key components in place to make a meaningful difference in the lives of patients and to maximize value for shareholders.”
At the event, the Company will also provide additional guidance on notable advances it anticipates over the next two years, including:
- Ten major clinical data readouts expected in 2016; and,
- Five or more Phase 3 trials ongoing and the first Phase 3 readout expected by the end of 2017.
Genetic Medicines STAr
Alnylam is advancing a broad pipeline
of genetically validated liver disease targets for rare orphan diseases,
with high unmet patient needs. This includes patisiran and revusiran,
investigational RNAi therapeutics for the treatment of
transthyretin-mediated amyloidosis (ATTR amyloidosis), now in Phase 3
clinical trials with the Company's APOLLO and ENDEAVOUR studies,
respectively. APOLLO remains on track to complete enrollment in the next
2-3 months, which is expected to enable a New Drug Application (NDA)
submission in 2017, while ENDEAVOUR continues enrollment. The Company
will also discuss ALN-TTRsc02, which is expected to be filed as a
Clinical Trial Application (CTA) in early 2016 and to enter Phase 3 in
2017.
In addition, the Company is advancing fitusiran (ALN-AT3), an investigational RNAi therapeutic in development for the treatment of hemophilia and rare bleeding disorders, where positive Phase 1 data including monthly dosing results were recently reported. The Company will discuss the potential commercial opportunity for fitusiran and will detail plans to advance to pivotal studies in mid-2016.
The clinical plan for ALN-CC5, being investigated for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) and complement-mediated diseases, will also be covered. Recent positive Phase 1 data were reported, including robust and dose-dependent knockdown of serum C5 with very low levels of residual C5, in addition to effects on multiple measures of complement activity and a durability profile supportive of a monthly, or possibly quarterly, subcutaneous dose regimen. Enrollment in Part C in PNH patients is expected to start before year-end, with initial data planned to read out in mid-2016, ahead of an anticipated 2017 Phase 3 start.
Amongst other programs, the Company will provide an update on ALN-AS1, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyrias in which initial recently reported Phase 1 clinical data demonstrated rapid and durable single-dose activity. ALN-AS1 could address a major unmet need in an ultra-rare orphan disease, with potential to make a difference in patients afflicted with this condition.
Across its Genetic Medicines STAr, Alnylam plans to commercialize its products through direct marketing and sales in the U.S. and EU, while leveraging its landmark partnership with Genzyme, a Sanofi company, for commercialization in those countries of North America, Europe, and the rest-of-world where Genzyme has the right to access Alnylam’s current and future Genetic Medicines pipeline.
Cardio-Metabolic Disease STAr
In addition, Alnylam is
advancing its pipeline of RNAi therapeutics toward genetically
validated, liver-expressed disease targets for unmet needs in
dyslipidemias, diabetes, NASH, and hypertension, amongst other diseases.
The validated profile of ESC-GalNAc conjugates with a once-quarterly and
possibly bi-annual, low-volume, subcutaneous dose regimen and a wide
therapeutic index supports advancement of new investigational medicines
in this disease area.
Alnylam is advancing ALN-PCSsc, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia, in collaboration with The Medicines Company. Phase 1 clinical data with ALN-PCSsc was recently reported demonstrating PCSK9 knockdown and LDL-cholesterol lowering supportive of a highly differentiated target product profile with a potential for a bi-annual subcutaneous dose regimen. ORION-1, a Phase 2 clinical study in 480 patients with elevated LDL-C, is being conducted by The Medicines Company and expects to start by the end of this year.
Alnylam intends to seek strategic partnership opportunities for programs in its Cardio-Metabolic Disease STAr, while retaining significant product commercialization rights in the U.S. and EU.
Hepatic Infectious Disease
Finally, Alnylam is advancing a
pipeline of RNAi therapeutics that address major global health
challenges, including hepatitis B virus (HBV) and hepatitis D virus
(HDV) infections, amongst other hepatic infectious disease opportunities.
ALN-HBV is an ESC-GalNAc-siRNA targeting the HBV genome and the company now plans to file a CTA for this program in early 2016, representing potentially a 3-4 month shift from earlier guidance. Alnylam is also advancing additional investigational programs including for the treatment of HDV infection and chronic liver infections.
The Company intends to seek strategic partnership opportunities for programs in its Hepatic Infectious Disease STAr, while retaining significant product commercialization rights in the U.S. and EU.
Alnylam will webcast its R&D Day live on the Investors section of the company's website, www.alnylam.com. The event will be held today from 8:00 a.m. to 11:30 a.m. ET at the Sofitel New York in New York City. An audio replay of the event will be available on the Alnylam website approximately 90 minutes after the event.
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) through the end of 2019,
together with certain broader co-development/co-commercialization rights
and global rights for certain products. 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. In the case of fitusiran, Genzyme has elected to
opt into the program for its rest-of-world rights, while retaining their
further opt-in right to co-develop and co-promote fitusiran with Alnylam
in North America and Western Europe, subject to certain restrictions.
About GalNAc Conjugates and Enhanced Stabilization Chemistry
(ESC)-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. Alnylam's Enhanced Stabilization
Chemistry (ESC)-GalNAc-conjugate technology enables subcutaneous dosing
with increased potency and durability, and a wide therapeutic index.
This delivery platform is being employed in nearly all of Alnylam's
pipeline programs, including programs in clinical development.
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 its ATTR portfolio,
fitusiran (ALN-AT3), ALN-CC5, ALN-AS1, ALN-PCSsc, and ALN-HBV, its plans
and expected timing regarding regulatory filings, including for
patisiran, ALN-TTRsc02 and ALN-HBV, the expected timing for the
initiation of additional clinical trials for its candidates, including
the expected timing for the initiation of Phase 3 trials for ALN-TTRsc02
and fitusiran, plans for reporting data from its clinical trials,
including ten major clinical data readouts expected in 2016, and its
plans for the 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, 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.
