– Full Year 2024 Preliminary Net Product
Revenues of $1,646 Million for ONPATTRO®, AMVUTTRA®, GIVLAARI®, and
OXLUMO®, Representing 33% Annual Growth –
– 2025 Combined Net Product Revenue Guidance**
of $2,050 Million to $2,250 Million Positions Company to Achieve
Alnylam P5x25 Goal of Non-GAAP Profitability –
– Robust Clinical Pipeline with
Multi-Billion-Dollar Opportunities for Sustainable Growth –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced its preliminary* fourth
quarter and full year 2024 global net product revenues for
ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO. In addition, the Company
provided 2025 net product revenue, non-GAAP operating income
profitability, and pipeline goals guidance.
“Alnylam’s commercial and clinical achievements in 2024 position
us very well for another transformative year in 2025, as we
continue to evolve into a global, top-tier biotech company,” said
Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. “We
generated net product revenues for the year of over $1.6 billion,
representing growth of 33% compared to 2023, at the high end of our
revised guidance range, and demonstrating the strength of our
underlying hATTR-PN and Rare businesses. We expect 2025 will
represent an important inflection point for our TTR franchise, with
the potential launch of vutrisiran in ATTR-CM delivering
significant topline growth as reflected in our net product sales
guidance announced today. If we are successful in meeting this
product revenue guidance, we anticipate achieving non-GAAP
profitability in 2025.”
Dr. Greenstreet continued, “We’re also looking forward to a year
of major advancements in our pipeline and RNAi platform, with key
goals outlined today. This remarkable pace of progress positions us
well to finish the year achieving key Alnylam P5x25 goals and to
continue delivering sustainable innovation to patients through our
global commercial infrastructure, broad pipeline, and organic
platform.”
Preliminary Fourth Quarter and Full Year 2024 Commercial and
Financial Performance*
Total TTR: ONPATTRO® (patisiran) & AMVUTTRA®
(vutrisiran)
- Preliminary* global net product revenues for ONPATTRO and
AMVUTTRA for the fourth quarter were approximately $56 million and
$287 million, respectively, representing together 35% total TTR
annual growth compared to Q4 2023, and for the full year 2024 were
approximately $253 million and $970 million, respectively,
representing together 34% total TTR annual growth compared to full
year 2023.
Total Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Preliminary* global net product revenues for GIVLAARI and
OXLUMO for the fourth quarter were approximately $65 million and
$44 million, respectively, representing together 18% total Rare
annual growth compared to Q4 2023, and for the full year 2024 were
approximately $256 million and $167 million, respectively,
representing together 29% total Rare annual growth compared to full
year 2023.
2025 Combined Net Product Revenue & Non-GAAP Operating
Income Guidance
Alnylam announced today full year 2025 combined net product
revenue guidance for ONPATTRO, AMVUTTRA (PN & CM**), GIVLAARI
and OXLUMO of $2,050 million to $2,250 million, representing
combined full year growth compared to 2024 of 31% at the mid-point
of the guidance range. On a franchise level, the guidance is broken
down as follows:
- Total TTR (ONPATTRO, AMVUTTRA (PN & CM**)): $1,600 million
to $1,725 million, representing full year growth compared to 2024
of 36% at the mid-point of the guidance range.
- Total Rare (GIVLAARI, OXLUMO): $450 million to $525 million,
representing full year growth compared to 2024 of 15% at the
mid-point of the guidance range.
In addition, the Company anticipates delivering non-GAAP
operating income profitability in 2025.
The Company plans to provide additional guidance for
collaboration and royalty revenue and operating expenses at the
time fourth quarter and full year 2024 earnings are released.
2025 Product and Pipeline Goals
Vutrisiran – an RNAi therapeutic marketed in various
countries globally as a treatment of adults with hATTR amyloidosis
with polyneuropathy, and in development for the treatment of adults
with ATTR amyloidosis with cardiomyopathy. Alnylam expects to:
- Achieve U.S. Food and Drug Administration (FDA) approval of the
supplemental New Drug Application for the treatment of adults with
ATTR amyloidosis with cardiomyopathy by the PDUFA target action
date of March 23, 2025.
- Secure additional global approvals and reimbursement in Japan
and the EU for the treatment of adults with ATTR amyloidosis with
cardiomyopathy in the second half of 2025.
Nucresiran (ALN-TTRsc04) – an investigational RNAi
therapeutic in development for the treatment of ATTR amyloidosis.
Alnylam expects to:
- Initiate a Phase 3 study in patients with ATTR amyloidosis with
cardiomyopathy in the first half of 2025.
Zilebesiran – an investigational RNAi therapeutic in
development for the treatment of hypertension, in collaboration
with Roche. Alnylam expects to:
- Report results from the KARDIA-3 Phase 2 study in the second
half of 2025.
- Initiate a Phase 3 cardiovascular outcomes trial in the second
half of 2025.
Mivelsiran – an investigational RNAi therapeutic in
development for the treatment of Alzheimer’s disease and cerebral
amyloid angiopathy (CAA). Alnylam expects to:
- Report interim results from Part B of the Phase 1 study in
Alzheimer’s disease in the second half of 2025.
- Initiate a Phase 2 study in Alzheimer’s disease in the second
half of 2025.
ALN-6400 – an investigational RNAi therapeutic in
development for the treatment of bleeding disorders. Alnylam
expects to:
- Initiate a Phase 2 study in a bleeding disorder in the second
half of 2025.
In addition, the Company plans to file Investigational New Drug
(IND) applications for four new Alnylam-led programs by the
end of 2025.
Partner-Led Program Highlights
Alnylam partnered programs continue to progress, including:
- Fitusiran – an investigational RNAi therapeutic
partnered with Sanofi in development for the treatment of
hemophilia A and B, with or without inhibitors. Sanofi expects to
secure FDA approval by the PDUFA target action date of March 28,
2025.
- Elebsiran – an investigational RNAi therapeutic
partnered with Vir Biotechnology in development for the treatment
of chronic hepatitis B and chronic hepatitis delta. In 2025, Vir
expects to initiate a Phase 3 chronic hepatitis delta
registrational study and to report functional cure results from a
Phase 2 chronic hepatitis B study.
Alnylam management will discuss its preliminary 2024 net product
revenues, as well as 2025 goals and guidance during a webcast
presentation at the 43rd Annual J.P. Morgan Healthcare Conference
in San Francisco, California tomorrow, Monday, January 13, 2025 at
9:45 am PT (12:45 pm ET).
About RNAi Therapeutics
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 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, statements regarding Alnylam’s evolution into a
leading, global, top-tier biotech company; Alnylam’s expectations
regarding the potential approval and launch of AMVUTTRA for the
treatment of ATTR amyloidosis with cardiomyopathy in the U.S. in
early 2025 and in other territories in the second half of 2025; the
potential that the launch of AMVUTTRA for ATTR-CM will deliver
significant topline growth for Alnylam; the potential for 2025 to
be a transformative year for Alnylam and that 2025 will represent
an important inflection point for Alnylam’s TTR franchise;
Alnylam’s ability to deliver non-GAAP operating income
profitability in 2025; the potential for 2025 to be a year of major
advancements in Alnylam’s pipeline and RNAi platform; Alnylam’s
potential achievement of the goals in its “Alnylam P5x25” strategy;
Alnylam’s ability to continue to deliver sustainable innovation to
patients through its global commercial infrastructure, broad
pipeline and organic platform; the potential for Alnylam to advance
its research and development programs, including its goals and
expectations regarding the clinical development of vutrisiran,
nucresiran, zilebesiran, mivelsiran, ALN-6400, and its earlier
stage programs, and its partners’ expectations for Alnylam’s
partnered programs; and Alnylam’s projected commercial and
financial performance, including the expected range of net product
revenues and non-GAAP operating income for 2025, 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,
risks and uncertainties relating to: 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, nucresiran,
zilebesiran, mivelsiran and ALN-6400; 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; 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; 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 risk of future government
investigations; and unexpected expenditures; as well as those risks
and uncertainties more fully discussed in the “Risk Factors” filed
with Alnylam’s 2023 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 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.
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. Vutrisiran has not been
approved by any regulatory agency for the treatment of ATTR
amyloidosis with cardiomyopathy. No conclusions can or should be
drawn regarding its safety or effectiveness in treating
cardiomyopathy in this population. There is no guarantee that any
investigational therapeutics or expanded uses of commercial
products will successfully complete clinical development or gain
health authority approval.
Use of Non-GAAP Financial Measures
This press release contains a non-GAAP financial measure of
non-GAAP operating income. This measure is not in accordance with,
or an alternative to, GAAP, and may be different from non-GAAP
financial measures used by other companies. Stock-based
compensation expense is included in GAAP operating income but
excluded for purposes of determining non-GAAP operating income. The
Company has excluded the impact of stock-based compensation expense
as it may fluctuate from period to period based on factors
including the variability associated with performance-based grants
for stock options and restricted stock units and changes in the
Company’s stock price, which impacts the fair value of these
awards.
* The preliminary selected financial results are unaudited,
subject to adjustment, and provided as an approximation in advance
of the Company’s announcement of complete financial results in
February 2025. ** Guidance assumes FDA approval of the sNDA for
vutrisiran for the treatment of adults with ATTR amyloidosis with
cardiomyopathy by the March 23, 2025 PDUFA target action date.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250112711585/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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