– Company Anticipates Investigational New Drug
(IND) Applications for Nine or More Programs with Targets Expressed
in the Liver, Central Nervous System, Adipose Tissue, and Muscle by
End of 2025 –
– Positive Initial Phase 1 Results with
ALN-TTRsc04 Demonstrate Rapid Knockdown with Mean Serum TTR
Reduction up to 97% with Durability Supporting Potential for Annual
Dosing and an Encouraging Safety Profile –
– Positive Initial Phase 1 Results with ALN-KHK
Demonstrate Robust Target Engagement and an Encouraging Safety
Profile, Supporting Continued Development as a Novel Treatment for
Type 2 Diabetes Mellitus –
– 2024 Product and Pipeline Goals Detail
Expected Execution Across Four Commercial Brands and Robust
Clinical Pipeline –
– Alnylam to Webcast its R&D Day Event
Today at 8:30 am E.T. –
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 2024, including continued commercial execution
of four RNAi therapeutic products and the advancement of early-,
mid-, and late-stage investigational programs.
“Alnylam continues to drive the next wave of RNAi therapeutics
that is changing medicine, with a sustainable innovation engine
generating a robust pipeline and a multi-product commercial
portfolio setting the stage for the future. We expect our track
record of strong pipeline and commercial execution to continue
through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D.,
Ph.D., Chief Innovation Officer of Alnylam. “As we look ahead to
the next frontier of RNAi therapeutic innovation, we are excited to
be presenting promising results that speak to our ambition to reach
many new tissues, with new data on delivery to the central nervous
system, adipose tissue, muscle, and tumors. We believe this
progress—with nine or more Alnylam proprietary INDs planned by the
end of 2025—will help build our pipeline to enable sustainable
future growth and advance us toward achieving our ‘Alnylam P5x25’
goals of becoming a top-tier biotech company.”
Novel Extrahepatic Delivery Systems Extend Alnylam’s
Leadership in RNAi Platform Innovation
Alnylam scientists will present new preclinical findings
demonstrating RNAi-mediated knockdown of targets expressed in
adipose and muscle. The data provide evidence for best-in-class
delivery to these tissues using subcutaneous administration, with
potent and sustained knockdown of specific genes. The Company is
progressing candidates that employ the new delivery technology
toward Development Candidate selection in 2024.
The Company will also share preclinical data demonstrating
systemic delivery of an RNAi therapeutic across the blood-brain
barrier to the central nervous system (CNS). The results, while
early, have the potential to broaden applications of RNAi
therapeutics in the CNS by eliminating the need for direct
administration (i.e., intrathecally).
Next Wave of RNAi Therapeutics Fuel Robust Clinical
Pipeline
Alnylam scientists will present new genetically validated
targets for rare, specialty, and prevalent diseases, including for
cholestatic liver diseases, a range of bleeding disorders, obesity,
and type 2 diabetes mellitus (T2DM). By the end of 2025, the
Company plans to file Investigational New Drug (IND) applications
for at least nine new Alnylam-led RNAi programs, including five for
targets expressed in the liver, two for the CNS, and one each for
adipose tissue and muscle. The Company also anticipates that INDs
will be filed for at least six additional programs led by partners
during this timeframe.
New Positive Phase 1 Data Reinforce Strength of Organic
Product Engine
Alnylam scientists will present positive initial results from
Phase I clinical trials for ALN-TTRsc04, in development for the
treatment of ATTR amyloidosis, and ALN-KHK, in development for the
treatment of T2DM. In addition, Alnylam will share an update on
delivery advances driving ALN-BCAT, which will soon begin clinical
testing in patients with hepatocellular carcinoma (HCC).
ALN-TTRsc04 Phase 1 Study
ALN-TTRsc04 is an investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis that utilizes
Alnylam’s IKARIA technology and provides the potential for greater
than 90% target gene silencing with once annual dosing. In the
Phase 1 study in healthy volunteers, single 300 mg doses of
ALN-TTRsc04 achieved rapid knockdown with a mean reduction of
greater than 90% at Day 15. A peak mean TTR reduction of 97% was
achieved at Day 29 and a mean TTR reduction of 93% was sustained at
Day 180. At all dose levels evaluated to date, single doses of
ALN-TTRsc04 have been well tolerated, with no adverse events deemed
to be related to study drug by the investigator.
These initial results suggest the potential for ALN-TTRsc04 to
offer over 90% TTR reduction with once annual dosing. The Phase 1
study is ongoing. Additional data will help inform selection of
dose level and regimen for a Phase 3 study in ATTR amyloidosis with
cardiomyopathy, which the Company expects to start at or around
year-end 2024.
ALN-KHK Phase 1 Study
In the Phase 1 study in overweight to obese healthy volunteers,
single doses of investigational ALN-KHK at the highest dose cohort
available resulted in a mean increase in fructose area under the
curve to 3-fold relative to baseline levels measured at Day 85,
providing clear evidence of target engagement. Decreases in
circulating FGF21 levels were also observed, consistent with the
known biology of KHK.
ALN-KHK was shown to be safe and well tolerated, with an
encouraging safety profile supporting continued clinical
development, and target engagement that suggests the potential for
quarterly or less frequent dosing. The study is ongoing, and
additional data from the two highest dose levels will enable dose
selection for Part B, which will evaluate multiple doses of ALN-KHK
in obese patients with T2DM.
ALN-BCAT Phase 1 Study
Alnylam scientists will also present promising preclinical data
with ALN-BCAT, an investigational RNAi therapeutic targeting
β-catenin for the treatment of HCC. The
Wnt/β-catenin pathway is implicated in many human
cancers, including HCC, a leading cause of cancer death worldwide.1
Alnylam announces today that it recently filed an IND application
with ALN-BCAT and plans to initiate a Phase 1 study in early 2024.
The study will evaluate the investigational drug’s potential both
as a monotherapy and in combination with immunotherapy in patients
with advanced disease. ALN-BCAT employs Alnylam’s advanced lipid
nanoparticle (LNP) delivery technology termed “reLNP,” which
provides improved tolerability due to enhanced
biodegradability.
2024 Product and Pipeline Goals
Alnylam plans to provide guidance on 2024 combined net product
revenue for ONPATTRO®, AMVUTTRA®, GIVLAARI®,
and OXLUMO® with its fourth quarter and full year 2023
results.
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 (including both hATTR and wtATTR amyloidosis). Alnylam
plans to:
- Report topline results from the HELIOS-B Phase 3 study in early
2024.
- Submit a Supplemental New Drug Application (sNDA) for the
cardiomyopathy of ATTR amyloidosis in mid-2024, assuming positive
HELIOS-B results.
ALN-TTRsc04, an investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis. Alnylam plans
to:
- Initiate a Phase 3 study in patients with ATTR amyloidosis with
cardiomyopathy at or around year-end 2024.
Zilebesiran,* an investigational RNAi therapeutic in
development for the treatment of hypertension. Alnylam plans
to:
- Report topline results from the KARDIA-2 Phase 2 study in early
2024.
- Initiate the KARDIA-3 Phase 2 study in early 2024.
ALN-APP,* an investigational RNAi therapeutic in
development for the treatment of Alzheimer’s disease and cerebral
amyloid angiopathy (CAA). Alnylam plans to:
- Initiate a Phase 2 study in CAA in early 2024.
- Report initial Part B multi-dose data from the Phase 1 study in
Alzheimer’s disease in late 2024.
- Initiate a Phase 2 study in Alzheimer’s disease at or around
year-end 2024.
ALN-KHK, an investigational RNAi therapeutic in
development for the treatment of type 2 diabetes mellitus. Alnylam
plans to:
- Initiate Part B of the Phase 1 study in early 2024.
ALN-BCAT, an investigational RNAi therapeutic in
development for the treatment of hepatocellular carcinoma. Alnylam
plans to:
- Initiate a Phase 1 study in early 2024.
In addition, the Company plans to file INDs for three new
Alnylam-led programs by the end of 2024.
Partner-Led Program Highlights
Alnylam also plans to support its partners in advancing multiple
mid- and late-stage programs, including:
- Fitusiran, an investigational RNAi therapeutic partnered
with Sanofi in late-stage development for the treatment of
hemophilia A and B, with or without inhibitors. Sanofi plans to
file a New Drug Application for fitusiran in 2024.
- ALN-HBV02 (VIR-2218), an investigational RNAi
therapeutic partnered with Vir Biotechnology in development for the
treatment of chronic hepatitis B and D virus infection. Vir expects
to report clinical data from multiple Phase 2 studies throughout
2024.
R&D Day Webcast Information
The Company’s R&D Day event will be held today, Wednesday,
Dec. 13, from 8:30 a.m. to 12:45 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.
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 Pharmaceuticals (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 in 2002, 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), AMVUTTRA®
(vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), 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 X (formerly Twitter) at @Alnylam, or on LinkedIn,
Facebook, or 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, Alnylam’s projected commercial and financial performance
and the planned achievement of its “Alnylam P5x25” strategy, 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:
Alnylam’s ability to successfully execute on its “Alnylam P5x25”
strategy; Alnylam’s ability to discover and develop novel drug
candidates and delivery approaches and successfully demonstrate the
efficacy and safety of its product candidates; the pre-clinical and
clinical results for Alnylam’s product candidates, including
vutrisiran, zilebesiran, ALN-APP, ALN-TTRsc04 and ALN-KHK; actions
or advice of regulatory agencies and Alnylam’s ability to obtain
and maintain regulatory approval for its product candidates,
including vutrisiran, as well as favorable pricing and
reimbursement; successfully launching, marketing and selling
Alnylam’s approved products globally; delays, interruptions or
failures in the manufacture and supply of Alnylam’s product
candidates or its marketed products; delays or interruptions in the
supply of resources needed to advance Alnylam’s research and
development programs; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
approved indications for 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; 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; Alnylam’s
ability to maintain strategic business collaborations; Alnylam’s
dependence on third parties for the development and
commercialization of certain products, including Roche, Novartis,
Sanofi, Regeneron and Vir; the outcome of litigation; the risks of
future government investigations; and unexpected expenditures; as
well as those risks more fully discussed in the “Risk Factors”
filed with Alnylam’s 2022 Annual Report on Form 10-K filed with the
Securities and Exchange Commission (SEC), as may be updated from
time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q
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.
*ALN-APP is an Alnylam-led program partnered with Regeneron.
Zilebesiran is an Alnylam-led program partnered with Roche.
Reference
1) Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I,
Jemal A, Bray F. Global cancer statistics 2020: GLOBOCAN estimates
of incidence and mortality worldwide for 36 cancers in 185
countries. CA Cancer J Clin. 2021;71(3):209–249. doi:
10.3322/caac.21660.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231213336564/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340 Josh Brodsky (Investors)
617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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