Patisiran Expected to be First Commercially
Available RNAi Therapeutic in Mid-2018
Restructured Sanofi Alliance Provides Alnylam
with Global Commercial Footprint for ATTR Amyloidosis
Givosiran Interim Phase 3 Data Expected in
Mid-2018 and U.S. New Drug Application Filing Anticipated in Late
2018
Company Plans to Advance Three Additional Phase
3 Programs: Fitusiran, Inclisiran, and ALN-TTRsc02
Company Updates Guidance to End 2017 with
Greater Than $1.7 Billion in Cash
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi
therapeutics company, today announced its corporate goals for 2018.
The goals focus on clinical and regulatory progress against its
five mid- to late-stage programs, including patisiran, which is
advancing toward potential global regulatory approvals and
commercialization, paving the way for the Company to achieve its
Alnylam 2020 goals.
"In the year ahead, we look forward to the potential global
commercial launch of patisiran, the first-ever RNAi therapeutic to
enter the market, marking the birth of a whole new class of
medicines with the potential to transform the treatment of diseases
with high unmet need. Together with the advancements made in our
other late-stage programs, we now look forward to our transition
from a development-stage company toward a multi-product, global
commercial-stage biopharmaceutical company with a deep and
sustainable pipeline and research engine," said John Maraganore,
Ph.D., Chief Executive Officer of Alnylam. “With continued
diligence and investment in our pipeline and commercial execution
we believe we are well positioned to realize our Alnylam 2020
goals, making RNAi therapeutics broadly available to patients in
need. Further, with our recently completed strategic restructuring
of our Sanofi alliance, we are now poised to optimize our ATTR
amyloidosis programs, patisiran and ALN-TTRsc02, for patients
around the world.”
“The recently restructured alliance with Sanofi provides
strategic and operational clarity and a global launching pad for
patisiran and our entire ATTR amyloidosis business. Given the
transformational potential of patisiran based on our recently
completed APOLLO Phase 3 study, we’re excited to accelerate our
build of capabilities to bring this investigational RNAi
therapeutic to patients around the world,” said Barry Greene,
President of Alnylam. “As part of a staged global effort, we are
preparing for the patisiran launch in mid-2018 in the U.S. and late
2018 in Europe, and we will then focus on Japan and other
rest-of-world markets.”
2018 Corporate Goals
Patisiran, an investigational RNAi therapeutic currently
under regulatory review for the treatment of hATTR amyloidosis.
Alnylam plans to:
- Present additional data from the APOLLO
Phase 3 study in early, mid-, and late 2018;
- File for regulatory approval in Japan
and other global markets, including a Japanese NDA filling with the
Pharmaceuticals and Medical Device Agency (PMDA) in mid-2018;
- File for regulatory approvals in
additional rest-of-world countries in late 2018;
- Gain regulatory approvals from the U.S.
Food and Drug Administration and the European Medicines Agency in
mid- and late 2018, respectively; and
- Assuming regulatory approval, launch
patisiran in the U.S. and EU in mid- and late 2018,
respectively.
Givosiran, an investigational RNAi therapeutic in
development for the treatment of acute hepatic porphyrias (AHPs).
Alnylam plans to:
- Present Phase 1/2 open-label extension
(OLE) data in early 2018;
- Report topline interim results from the
ENVISION Phase 3 trial in mid-2018;
- Assuming a positive interim readout,
file for accelerated regulatory approval with submission of a New
Drug Application (NDA) in the U.S. in late 2018; and
- Complete enrollment in the ENVISION
Phase 3 study in late 2018.
ALN-TTRsc02, an investigational, subcutaneously
administered RNAi therapeutic in development for the treatment of
ATTR amyloidosis. Alnylam plans to:
- Initiate a Phase 3 pivotal study
program in late 2018.
Alnylam also intends to support Sanofi’s efforts in advancing
fitusiran, an investigational RNAi therapeutic in development for
the treatment of hemophilia and rare bleeding disorders. Throughout
2018, Sanofi expects to enroll patients in the ATLAS Phase 3
program of fitusiran in patients with hemophilia A or B with and
without inhibitors. If successful, fitusiran will be commercialized
globally by Sanofi Genzyme, the specialty care global business unit
of Sanofi. Alnylam is eligible to receive 15 to 30 percent
royalties on net sales.
Alnylam also plans to support The Medicines Company's continued
efforts with respect to the ORION Phase 3 studies of inclisiran –
an investigational RNAi therapeutic targeting PCSK9 in development
for the treatment of hypercholesterolemia – throughout 2018.
Specifically, The Medicines Company has guided to complete
enrollment in the ORION 9, 10, and 11 LDL-C pivotal studies in
early 2018 and to initiate enrollment in the ORION 4 cardiovascular
outcomes (CVOT) study in mid-2018. Alnylam is eligible to receive
milestones and royalties of up to 20 percent from The Medicines
Company based on the successful development and commercialization
of inclisiran.
In addition, the Company plans to continue advancement of its
earlier-stage clinical pipeline programs with multiple data
read-outs expected throughout 2018. Alnylam also plans to file one
or more new clinical trial applications (CTA) in 2018, and advance
its infectious disease collaboration with Vir Biotechnology.
The Company now expects to end 2017 with greater than $1.7
billion in cash, cash equivalents, marketable securities, and
restricted cash and intends to provide financial guidance for 2018
in connection with its year-end 2017 financial results in
February.
Alnylam management will present a company overview detailing
these goals and guidance at the 36th Annual J.P. Morgan
Healthcare Conference tomorrow, Monday, January 8,
2018 at 8:00 a.m. PT (11:00 a.m. ET) at
the Westin St. Francis Hotel in San Francisco,
California. In addition, the Company plans to webcast its Q&A
breakout session immediately following the presentation
at 8:30 a.m. PT (11:30 a.m. ET). A live audio webcast of
the presentation and breakout session will be available on the
Investors section of the Company's website, www.alnylam.com. A
replay will be available on the Alnylam website within 48 hours
after the event.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene
silencing that represents one of the most promising and rapidly
advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough
that happens once every decade or so,” and was recognized with the
award of the 2006 Nobel Prize for Physiology or Medicine. By
harnessing the natural biological process of RNAi occurring in our
cells, 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,
function upstream of today’s medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors – that encode for
disease-causing proteins, thus preventing them from being made.
This is a revolutionary approach with the potential to transform
the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals, Inc.
Alnylam (Nasdaq:ALNY) is leading the translation of RNA
interference (RNAi) into a whole new class of innovative medicines
with the potential to transform the lives of people afflicted with
rare genetic, cardio-metabolic, and hepatic infectious diseases.
Based on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of severe and debilitating diseases. Founded in 2002, Alnylam
is delivering on a bold vision to turn scientific possibility into
reality, with a robust discovery platform and deep pipeline of
investigational medicines, including four product candidates that
are in late-stage development. Looking forward, Alnylam will
continue to execute on its "Alnylam 2020" strategy of building a
multi-product, commercial-stage biopharmaceutical company with a
sustainable pipeline of RNAi-based medicines to address the needs
of patients who have limited or inadequate treatment options.
Alnylam employs over 700 people in the U.S. and Europe and is
headquartered in Cambridge, MA. For more information about our
people, science and pipeline, please visit www.alnylam.com and
engage with us on Twitter at @Alnylam or on LinkedIn.
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, ALN-TTRsc02, givosiran,
fitusiran, and inclisran, its expectations regarding the potential
regulatory approval and commercial launch of patisiran in the
United States and Europe and the expected timing of regulatory
filings seeking approval for patisiran in Japan and other global
markets, its expectations regarding the timing of clinical studies
for givosiran, ALN-TTRsc02, fitusiran, and inclisiran and expected
regulatory filings for givosiran, its expectations regarding the
further development of fitusiran by its collaborator, Sanofi
Genzyme, its plans for the development and commercialization of
RNAi therapeutics for infectious diseases with Vir Biotechnology,
its expected cash, cash equivalents, fixed income, marketable
securities, and restricted investments position as of December 31,
2017, and expectations regarding 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 and future plans may differ materially
from those indicated by these forward-looking statements as a
result of various important risks, uncertainties and other 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 product 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 or
advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional pre-clinical and/or clinical
testing, delays, interruptions or failures in the manufacture and
supply of its product candidates, obtaining, maintaining and
protecting intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, competition from others using technology
similar to Alnylam's and others developing products for similar
uses, Alnylam's ability to manage its growth and operating
expenses, 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 and distribution of products, the
outcome of litigation, the risk of government investigations, 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, except to the extent required
by law, to update any forward-looking statements.
None of Alnylam’s investigational therapeutics have been
approved by the U.S. Food and Drug Administration, European
Medicines Agency, or any other regulatory authority and no
conclusions can or should be drawn regarding the safety or
effectiveness of such investigational therapeutics.
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version on businesswire.com: http://www.businesswire.com/news/home/20180107005098/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276
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