− Product and Pipeline Goals Detail Execution
across Four Commercial Brands, Including One Potential Label
Expansion, and Ten Clinical Data Readouts From Proprietary and
Partner Programs –
− Pipeline Programs and Scientific Innovation
Highlighted at R&D Day Exemplify Strength of RNAi Platform and
Organic Product Engine –
− Alnylam to Webcast its R&D Day Event
Today at 8:30 a.m. ET –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, is hosting a virtual R&D Day today.
During the event, the Company plans to showcase its R&D
progress and platform innovation, as well as its product and
pipeline goals for 2023 focused on continued commercial execution
of four RNAi therapeutic products, and advancement of early-, mid-,
and late-stage investigational programs.
“Throughout 2022, Alnylam has continued to demonstrate the
strength of RNAi therapeutics, including the commercial launch of
the fifth Alnylam-discovered product within four years, while
advancing an organic pipeline of potentially transformative
investigational medicines in both rare and common diseases,” said
Akshay Vaishnaw, M.D., Ph.D., President of Alnylam. “We expect our
track record of strong execution will continue in 2023, with plans
to deliver strong commercial performance across four commercial
brands, achieve one label expansion, and report ten clinical
datasets across Alnylam- and partner-led programs. We believe these
milestones will help advance us toward achieving our ‘Alnylam
P5x25’ goals of becoming a top tier biotech company over the coming
years.”
2023 Product and Pipeline Goals
Alnylam plans to provide guidance on 2023 combined net product
revenue for ONPATTRO®, AMVUTTRA®, GIVLAARI®,
and OXLUMO® with its fourth quarter and full year 2022
results.
Patisiran, a commercial-stage RNAi therapeutic for the
treatment of polyneuropathy in patients with hATTR amyloidosis, and
in development for the treatment of the cardiomyopathy of ATTR
amyloidosis. Alnylam plans to:
- Achieve approval from the U.S. Food and Drug Administration
(FDA) in late 2023, for the treatment of the cardiomyopathy of ATTR
amyloidosis
Vutrisiran, a commercial-stage RNAi therapeutic for the
treatment of polyneuropathy in patients with hATTR amyloidosis, and
in development for the treatment of the cardiomyopathy of ATTR
amyloidosis. Alnylam plans to:
- Report data from the evaluation of a biannual dosing regimen in
early 2023
- Submit a Supplemental New Drug Application (sNDA) for a
biannual dosing regimen in early 2023
ALN-TTRsc04, an investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis. Alnylam plans
to:
- Report topline Phase 1 results in late 2023
Zilebesiran, an investigational RNAi therapeutic in
development for the treatment of hypertension. Alnylam plans
to:
- Complete enrollment in the KARDIA-2 Phase 2 study in early
2023
- Report topline results from the KARDIA-1 Phase 2 study in
mid-2023
- Report topline results from the KARDIA-2 Phase 2 study at or
around year-end 2023
ALN-APP, an investigational RNAi therapeutic in
development for the treatment of Alzheimer’s Disease and Cerebral
Amyloid Angiopathy. Alnylam plans to:
- Report topline Phase 1 results in early 2023
ALN-KHK, an investigational RNAi therapeutic in
development for the treatment of Type 2 Diabetes. Alnylam plans
to:
- Initiate a Phase 1 study in early 2023
- Report topline Phase 1 results in late 2023
The Company also plans to continue advancement of its
preclinical portfolio of investigational RNAi therapeutics
targeting genes expressed in multiple tissue types, and plans to
advance between 2-4 new Investigational New Drug (IND) applications
from its organic product engine in 2023.
Partnered Program Milestones
Fitusiran, an investigational RNAi therapeutic in
development for the treatment of hemophilia A and B, with or
without inhibitors. Alnylam’s partner Sanofi plans to:
- Report Phase 3 results from the ATLAS-OLE-005 Phase 3 study
evaluating a revised dose and dose regimen in late 2023
ALN-HBV02, an investigational RNAi therapeutic in
development for the treatment of chronic hepatitis B and D virus
infection. Alnylam’s partner Vir Biotechnology plans to:
- Report additional results from Part A of the MARCH trial,
evaluating the combination of ALN-HBV02 (VIR-2218) and VIR-3434, an
anti-HBV monoclonal antibody, for the treatment of patients with
chronic HBV infection, in early 2023
- Report initial results from Part B of the MARCH trial in late
2023
- Report additional results from the Phase 2 study evaluating the
combination of ALN-HBV02 (VIR-2218) and PEG-IFN alpha in early
2023
- Report initial results from the Phase 2 study evaluating the
combination of ALN-HBV02 (VIR-2218) and VIR-3434, an anti-HBV
monoclonal antibody, in viremic patients, in late 2023
- Report initial results from the Phase 2 study evaluating the
combination of ALN-HBV02 (VIR-2218) and VIR-3434 in patients with
Hepatitis Delta Virus (HDV) infection, in late 2023
ALN-PNP, an investigational RNAi therapeutic in
development for the treatment of non-alcoholic steatohepatitis
(NASH). Alnylam’s Partner Regeneron plans to:
- Initiate a Phase 1 study in early 2023
R&D Day Webcast Information
The Company’s R&D Day event will be held today, Thursday,
December 15th, from 8:30 a.m. to 12:30 p.m. ET and will include a
video stream on the Investors section of the Company’s website,
investors.alnylam.com/events. A replay will be available on the
Alnylam website within 48 hours after the event. Presentations
showcased during the event will be featured on Capella
(https://capella.alnylam.com/2022/12/15/alnylam-rd-day-2022).
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 new class of medicines known as RNAi therapeutics is now a
reality. 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
or disease pathway 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
Alnylam (Nasdaq: ALNY) has led 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 and prevalent diseases with unmet need. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach yielding transformative medicines.
Since its founding 20 years ago, Alnylam has led the RNAi
Revolution and continues to deliver on a bold vision to turn
scientific possibility into reality. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), GIVLAARI®
(givosiran), OXLUMO® (lumasiran), AMVUTTRA® (vutrisiran) and
Leqvio® (inclisiran), which is being developed and commercialized
by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of
investigational medicines, including multiple product candidates
that are in late-stage development. Alnylam is executing on its
“Alnylam P5x25” strategy to deliver transformative medicines in
both rare and common diseases benefiting patients around the world
through sustainable innovation and exceptional financial
performance, resulting in a leading biotech profile. Alnylam 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, on LinkedIn, or on
Instagram.
Alnylam Forward Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. All statements
other than historical statements of fact regarding Alnylam’s
expectations beliefs, goals, plans or prospects, including without
limitation expectations regarding Alnylam’s aspiration to become a
top tier biotech company, the potential for Alnylam to identify new
potential drug development candidates and advance its research and
development programs, Alnylam’s ability to obtain approval for new
commercial products or additional indications for its existing
products, and Alnylam’s projected commercial and financial
performance, should be considered forward-looking statements.
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: the direct or indirect impact of the
COVID-19 global pandemic or any future pandemic on Alnylam’s
business, results of operations and financial condition and the
effectiveness or timeliness of Alnylam’s efforts to mitigate the
impact of the pandemic; the potential impact of the January 2022
leadership transition on Alnylam’s ability to attract and retain
talent and to successfully execute on its “Alnylam P5x25” strategy;
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches, including using Alnylam’s IKARIA and GEMINI
platforms, and successfully demonstrate the efficacy and safety of
its product candidates; the pre-clinical and clinical results for
its product candidates, including ALN-APP, patisiran and
vutrisiran; actions or advice of regulatory agencies and Alnylam’s
ability to obtain and maintain regulatory approval for its product
candidates, including patisiran and vutrisiran, as well as
favorable pricing and reimbursement; successfully launching,
marketing and selling its approved products globally; delays,
interruptions or failures in the manufacture and supply of its
product candidates or its marketed products; obtaining, maintaining
and protecting intellectual property; Alnylam’s ability to
successfully expand the indication for ONPATTRO or AMVUTTRA in the
future; Alnylam's ability to manage its growth and operating
expenses through disciplined investment in operations and its
ability to achieve a self-sustainable financial profile in the
future without the need for future equity financing; Alnylam’s
ability to maintain strategic business collaborations; Alnylam's
dependence on third parties for the development and
commercialization of certain products, including Novartis, Sanofi,
Regeneron and Vir; the outcome of litigation; the potential impact
of a current government investigation and the risk of future
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 its other SEC
filings. 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.
This release discusses investigational RNAi therapeutics and
uses of previously approved RNAi therapeutics in development and is
not intended to convey conclusions about efficacy or safety as to
those investigational therapeutics or uses. There is no guarantee
that any investigational therapeutics or expanded uses of
commercial products will successfully complete clinical development
or gain health authority approval.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221215005187/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
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