− Achieved Fourth Quarter and Full Year 2024
Global Net Product Revenues of $451 Million and $1,646 Million,
Respectively, Representing 30% and 33% Growth Compared to Same
Periods in 2023 –
− Supplemental NDA for Vutrisiran for the
Treatment of ATTR Amyloidosis with Cardiomyopathy Accepted by FDA,
with March 23, 2025 PDUFA Date –
− Reiterates Product Sales and Profitability
Guidance and Provides Additional 2025 Financial Guidance –
− Company to Host and Webcast R&D Day on
February 25, 2025 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the fourth quarter and full year ended December 31,
2024 and reviewed recent business highlights.
“2024 was another year of impressive execution for Alnylam,
generating product revenues of over $1.6 billion, reflecting growth
of 33% compared to 2023, and highlighting the strength of our base
business in hATTR-PN and Rare in both the U.S. and international
markets. We look forward to potential global launches of vutrisiran
in ATTR-CM this year, which will mark an inflection point for our
TTR franchise and put us on a path to achieve the financial
guidance we’ve provided,” said Yvonne Greenstreet, MBChB, Chief
Executive Officer of Alnylam. “Furthermore, driven by our proven
RNAi platform, we anticipate 2025 will bring major advancements in
our pipeline and expect to have over 25 high-value programs in the
clinic across diverse indications by the end of the year. We’ve
seen a remarkable pace of progress toward our Alnylam P5x25 goals
and believe we are well positioned for the next half of the decade
to continue delivering sustainable innovation to patients.”
Fourth Quarter 2024 and Recent Significant Business
Highlights
Commercial Performance
Total TTR: ONPATTRO® (patisiran) & AMVUTTRA®
(vutrisiran)
- Achieved global net product revenues for ONPATTRO and AMVUTTRA
for the fourth quarter of $56 million and $287 million,
respectively, and $343 million combined, representing 35% total TTR
growth compared to Q4 2023, and full year 2024 revenues of $253
million and $970 million, respectively, and $1,223 million
combined, representing 34% total TTR growth compared to full year
2023.
Total Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Achieved global net product revenues for GIVLAARI and OXLUMO
for the fourth quarter of $65 million and $44 million,
respectively, and $108 million combined, representing 18% total
Rare growth compared to Q4 2023, and full year 2024 revenues of
$256 million and $167 million, respectively, and $423 million
combined, representing 29% total Rare growth compared to full year
2023.
R&D Highlights
- Submitted a supplemental New Drug Application (sNDA) to the
U.S. Food and Drug Administration (FDA) using a Priority Review
Voucher for vutrisiran for the treatment of ATTR amyloidosis
with cardiomyopathy. Parallel filings for regulatory approval have
also been submitted in all major regions, including Europe and
Japan.
- The FDA has accepted the sNDA and set an action goal date of
March 23, 2025, under the Prescription Drug User Fee Act
(PDUFA).
- Received United States Food & Drug Administration (FDA)
Orphan Drug Designation for nucresiran (ALN-TTRsc04) and
announced positive interim Phase 1 data in patients with ATTR
amyloidosis.
- Announced positive initial results from the multiple dose
portion of the Phase 1 study of mivelsiran in patients with
Alzheimer’s disease.
- Initiated a Phase 1 study of ALN-HTT02 in adult patients
with Huntington’s disease.
- Completed enrollment in the KARDIA-3 Phase 2 study of
zilebesiran, and initiated a Phase 1 study of zilebesiran
+ REVERSIR for hypertension.
- Initiated Phase 1 studies of ALN-6400 for a bleeding
disorder, and ALN-4324 for type 2 diabetes mellitus.
- Alnylam's partner, Regeneron Pharmaceuticals, reported positive
updated Phase 3 data from an exploratory cohort in the ACCESS-1
trial investigating its first-in-class pozelimab and
cemdisiran combination treatment compared to
standard-of-care ravulizumab in patients with paroxysmal nocturnal
hemoglobinuria (PNH) at the American Society of Hematology 2024
Annual Meeting.
Upcoming Events
Alnylam will host an R&D Day on February 25, 2025 at 9:00 am
ET in New York City. This event, which will be webcast live on the
Investors section of the Company’s website, www.alnylam.com, will
showcase Alnylam’s robust pipeline comprising numerous
multi-billion-dollar opportunities and highlight RNAi platform
innovation driving the next era of growth. A replay will be
available on the Company's website within 48 hours after the
event.
The PDUFA target action date for the sNDA for vutrisiran
is March 23, 2025.
Alnylam intends to initiate a Phase 3 study of nucresiran
in patients with ATTR amyloidosis with cardiomyopathy in the first
half of 2025.
The PDUFA target action date for fitusiran, an
investigational RNAi therapeutic in development for the treatment
of hemophilia A and B, with or without inhibitors, is March 28,
2025. Fitusiran is being advanced by Alnylam’s partner Sanofi.
Assuming approval, Alnylam is eligible to receive tiered royalties
of between 15 and 30 percent on global net sales of fitusiran.
Alnylam’s partner, Vir Biotechnology, expects to initiate a
Phase 3 chronic hepatitis delta registrational study of
elebsiran and to report functional cure results from a Phase
2 chronic hepatitis B study of elebsiran in 2025.
Financial Highlights for the Fourth Quarter and Year End
2024
Three Months Ended December
31,
Twelve Months Ended December
31,
(In thousands)
2024
2023
2024
2023
Net product revenues
$
450,831
$
346,288
$
1,646,228
$
1,241,474
Net revenues from collaborations
106,948
76,407
510,221
546,185
Royalty revenue
35,387
17,023
91,794
40,633
Total revenues
593,166
439,718
2,248,243
1,828,292
Total operating costs and expenses
698,325
556,122
2,425,128
2,110,467
Loss from operations
(105,159
)
(116,404
)
(176,885
)
(282,175
)
Total other expense, net
(88,799
)
(21,283
)
(200,490
)
(151,342
)
Benefit from (provision for) income
taxes
110,195
(183
)
99,218
(6,725
)
Net loss
$
(83,763
)
$
(137,870
)
$
(278,157
)
$
(440,242
)
Non-GAAP Operating (loss) income*
$
(13,514
)
$
(74,410
)
$
95,199
$
(60,495
)
Non-GAAP Net income (loss)*
$
8,048
$
(96,643
)
$
(3,051
)
$
(201,618
)
*For an explanation of our use of non-GAAP
financial measures refer to the "Use of Non-GAAP Financial
Measures" section later in this press release and for a
reconciliation of each non-GAAP financial measure to the most
comparable GAAP measures, see the table at the end of this press
release.
Net Product Revenues
Three Months Ended December
31,
Twelve Months Ended December
31,
(In thousands)
2024
2023
2024
2023
ONPATTRO net product revenues
$
56,103
$
79,006
$
252,857
$
354,546
AMVUTTRA net product revenues
286,510
175,254
970,450
557,838
Total TTR net product revenues
342,613
254,260
1,223,307
912,384
GIVLAARI net product revenues
64,645
59,298
255,871
219,251
OXLUMO net product revenues
43,573
32,730
167,050
109,839
Total Rare net product revenues
108,218
92,028
422,921
329,090
Total net product revenues
$
450,831
$
346,288
$
1,646,228
$
1,241,474
Year over Year %
Growth
Three Months Ended December
31, 2024
Twelve Months Ended December
31, 2024
As Reported
At CER*
As Reported
At CER*
Total TTR net product revenues
35
%
34
%
34
%
34
%
Total Rare net product revenues
18
%
17
%
29
%
28
%
Total net product revenues
30
%
29
%
33
%
33
%
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in 2023. CER is a non-GAAP
measure. For an explanation of our use of non-GAAP financial
measures refer to the "Use of Non-GAAP Financial Measures" section
later in this press release and for a reconciliation of each
non-GAAP financial measure to the most comparable GAAP measures,
see the table at the end of this press release.
- Net product revenues increased 30% and 33% at actual currency
during the three and twelve months ended December 31, 2024,
respectively, compared to the same periods in 2023, and 29% and 33%
at CER, respectively. The increases are primarily due to growth
from sales of AMVUTTRA driven by increased patient demand,
partially offset by a decrease in sales of ONPATTRO due to patient
switches to AMVUTTRA, as well as increased patients on GIVLAARI and
OXLUMO therapies.
Net Revenues from Collaborations
- Net revenues from collaborations increased $30.5 million during
the three months ended December 31, 2024, as compared to the same
period in 2023, primarily due to the timing of manufacturing
activities under our collaboration with Regeneron, as well as
revenue recognized under our license agreement with Novartis
associated with the achievement of specified commercialization
milestones.
- Net revenues from collaborations decreased by $36.0 million
during the twelve months ended December 31, 2024, as compared to
the same period in 2023, primarily due to differences in certain
revenue items between 2023 and 2024. During 2023, we recognized
$310.0 million of revenue from the upfront payment received from
Roche in connection with execution of our zilebesiran
collaboration. In comparison, during 2024, we recognized $185.0
million in revenues under our collaboration with Regeneron as we
modified the collaboration in June 2024 and provided Regeneron with
an exclusive license to develop, manufacture and commercialize
cemdisiran as a monotherapy.
Operating Expenses
Three Months Ended December
31,
Twelve Months Ended December
31,
(In thousands)
2024
2023
2024
2023
Cost of goods sold
$
102,649
$
71,975
$
306,513
$
268,216
Cost of goods sold as a percentage of net
product revenues
22.8
%
20.8
%
18.6
%
21.6
%
Cost of collaborations and royalties
$
168
$
13,883
$
16,857
$
42,190
GAAP Research and development expenses
$
300,169
$
272,141
$
1,126,232
$
1,004,415
Non-GAAP Research and development
expenses
$
259,544
$
253,056
$
998,483
$
907,142
GAAP Selling, general and administrative
expenses
$
295,339
$
198,123
$
975,526
$
795,646
Non-GAAP Selling, general and
administrative expenses
$
244,319
$
175,214
$
831,191
$
671,239
Cost of Goods Sold
- Cost of goods sold as a percentage of net product revenues
increased during the three months ended December 31, 2024, as
compared to the same period in the prior year, primarily due to
higher royalty rates payable on net sales of AMVUTTRA.
- Cost of goods sold as a percentage of net product revenues
decreased during the twelve months ended December 31, 2024, as
compared to the same period in the prior year. Approximately 5.0%
of the 21.6% of cost of goods sold as a percentage of net product
revenues for the year ended December 31, 2023 was attributable to
cancelled manufacturing commitments and the impairment of ONPATTRO
inventory that had been manufactured for future demand associated
with the use of ONPATTRO for the treatment of patients with ATTR
amyloidosis with cardiomyopathy, for which we did not receive
regulatory approval in the U.S. These one-time charges in 2023 did
not recur in 2024, resulting in the decrease in cost of goods sold
as a percentage of net product revenues in 2024, which was
partially offset by higher volume and royalty rates payable on net
sales of AMVUTTRA in 2024.
Research & Development (R&D) Expenses
- GAAP and non-GAAP R&D expenses increased during the three
and twelve months ended December 31, 2024, compared to the same
periods in 2023, primarily due to increased costs associated with
our preclinical activities as we develop our clinical pipeline of
RNAi therapeutics targeting multiple tissues, increased clinical
trial expenses associated with increased Phase 2 activities for the
zilebesiran KARDIA-3 and mivelsiran cAPPRicorn-1 clinical trials,
and increased employee compensation expenses. GAAP R&D expenses
further increased during the three and twelve months ended December
31, 2024, compared to the same periods in 2023, due to higher
stock-based compensation expense in 2024.
Selling, General & Administrative (SG&A) Expenses
- GAAP and non-GAAP SG&A expenses increased during the three
and twelve months ended December 31, 2024, compared to the same
periods in 2023, primarily due to higher costs associated with
marketing investments to promote our TTR therapies and prepare for
the potential launch of AMVUTTRA for the treatment of ATTR
amyloidosis with cardiomyopathy and increased employee compensation
expenses.
Benefit from (provision for) income taxes
- During the year ended December 31, 2024, we recorded a net
benefit from income taxes of $99.2 million. This is primarily
comprised of $106.8 million of foreign deferred benefit, partially
offset by $1.6 million of domestic state current provision and $5.9
million of foreign current provision.
Other Financial Highlights
- Cash, cash equivalents and marketable securities were $2.69
billion as of December 31, 2024, as compared to $2.44 billion as of
December 31, 2023, with the increase primarily due to improved
operating performance and increased net proceeds from the issuance
of common stock in connection with stock option exercises.
A reconciliation of our GAAP to non-GAAP results for the three
and twelve months ended December 31, 2024 and 2023, is included in
the tables of this press release.
2025 Financial Guidance
Our full-year 2025 financial guidance is summarized below:
Total TTR net product revenues (ONPATTRO,
AMVUTTRA)1
$1,600 million - $1,725
million
Total Rare net product revenues (GIVLAARI,
OXLUMO)
$450 million - $525 million
Total net product revenues
$2,050 million - $2,250
million
Net product revenues growth vs. 2024 at
currency exchange rates as of December 31, 20242
25% - 37%
Net product revenues growth vs. 2024 at
constant exchange rates3
26% - 39%
Net revenues from collaborations and
royalties4
$650 million - $750 million
Non-GAAP R&D and SG&A
expenses5
$2,100 million - $2,200
million
Non-GAAP Operating income5
Achieve profitability
1 Assumes U.S. sNDA approval of AMVUTTRA
for ATTR-CM by the March 23, 2025 PDUFA action date and approvals
and launches in Germany and Japan in the second half of 2025
2 Full-year 2025 guidance utilizing
currency exchange rates as of December 31, 2024: 1 EUR = 1.04 USD
and 1 USD = 157 JPY
3 Representing growth calculated as if the
exchange rates had remained unchanged from those used in 2024,
which is a non-GAAP financial measure
4 Collaboration revenues assume
achievement of $300 million milestone from Roche for initiation of
a Phase 3 cardiovascular outcomes trial for zilebesiran. Royalty
revenues assume approval of fitusiran by Sanofi by the March 28,
2025 PDUFA action date
5 Primarily excludes $270-$330 million of
stock-based compensation expense from estimated GAAP R&D and
SG&A expenses
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures,
including adjustments to exclude certain non-cash items and
non-recurring transactions or events outside the ordinary course of
the Company’s business. These measures are not in accordance with,
or an alternative to, GAAP, and may be different from non-GAAP
financial measures used by other companies.
The items included in GAAP presentations but excluded for
purposes of determining non-GAAP financial measures for the periods
presented in this press release are stock-based compensation
expenses and realized and unrealized gains and losses on marketable
equity securities. The Company has excluded the impact of
stock-based compensation expense, which 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 Company has excluded the impact
of the realized and unrealized gains and losses on marketable
equity securities because the Company does not believe these
adjustments accurately reflect the performance of the Company’s
ongoing operations for the period in which such gains or losses are
reported, as their sole purpose is to adjust amounts on the balance
sheet.
Percentage changes in revenue growth at CER are presented
excluding the impact of changes in foreign currency exchange rates
for investors to understand the underlying business performance.
The current period’s foreign currency revenue values are converted
into U.S. dollars using the average exchange rates from the prior
period.
The Company believes the presentation of non-GAAP financial
measures provides useful information to management and investors
regarding the Company’s financial condition and results of
operations. When GAAP financial measures are viewed in conjunction
with non-GAAP financial measures, investors are provided with a
more meaningful understanding of the Company’s ongoing operating
performance and are better able to compare the Company’s
performance between periods. Non-GAAP financial measures are not
intended to be considered in isolation or as a substitute for GAAP
financial measures. A reconciliation between GAAP and non-GAAP
measures is provided later in this press release.
Conference Call Information
Management will provide an update on the Company and discuss
fourth quarter and full year 2024 results as well as expectations
for the future via conference call on Thursday, February 13, 2025
at 8:30 am ET. A live audio webcast of the call will be available
on the Investors section of the Company’s website at
www.alnylam.com/events. An archived webcast will be available on
the Alnylam website approximately two hours after the event.
About ONPATTRO® (patisiran)
ONPATTRO is an RNAi therapeutic that is approved in the United
States and Canada for the treatment of adults with hATTR
amyloidosis with polyneuropathy. ONPATTRO is also approved in the
European Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with
polyneuropathy. ONPATTRO is an intravenously administered RNAi
therapeutic targeting transthyretin (TTR). It is designed to target
and silence TTR messenger RNA, thereby reducing the production of
TTR protein before it is made. Reducing the pathogenic protein
leads to a reduction in amyloid deposits in tissues. For more
information about ONPATTRO, including full Prescribing Information,
visit ONPATTRO.com.
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers
rapid knockdown of mutant and wild-type transthyretin (TTR),
addressing the underlying cause of transthyretin (ATTR)
amyloidosis. Administered quarterly via subcutaneous injection,
AMVUTTRA is approved and marketed in more than 15 countries for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran
is also in development for the treatment of ATTR amyloidosis with
cardiomyopathy (ATTR-CM), which encompasses both wild-type and
hereditary forms of the disease. For more information about
AMVUTTRA, including the full U.S. Prescribing Information, visit
AMVUTTRA.com.
About GIVLAARI® (givosiran)
GIVLAARI (givosiran) is an RNAi therapeutic targeting
aminolevulinic acid synthase 1 (ALAS1) approved in the United
States and Brazil for the treatment of adults with acute hepatic
porphyria (AHP). GIVLAARI is also approved in the European Union
for the treatment of AHP in adults and adolescents aged 12 years
and older. In the pivotal study, GIVLAARI was shown to
significantly reduce the rate of porphyria attacks that required
hospitalizations, urgent healthcare visits or intravenous hemin
administration at home compared to placebo. GIVLAARI is Alnylam’s
first commercially available therapeutic based on its Enhanced
Stabilization Chemistry ESC-GalNAc conjugate technology to increase
potency and durability. GIVLAARI is administered via subcutaneous
injection once monthly at a dose based on actual body weight and
should be administered by a healthcare professional. GIVLAARI works
by specifically reducing elevated levels of ALAS1 messenger RNA
(mRNA), leading to reduction of toxins associated with attacks and
other disease manifestations of AHP. For more information about
GIVLAARI, including the full U.S. Prescribing Information, visit
GIVLAARI.com.
About OXLUMO® (lumasiran)
OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid
oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by
silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits
production of oxalate – the metabolite that directly contributes to
the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced
Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which
enables subcutaneous dosing with increased potency and durability
and a wide therapeutic index. OXLUMO has received regulatory
approvals from the U.S. Food and Drug Administration (FDA) for the
treatment of primary hyperoxaluria type 1 (PH1) to lower urinary
and plasma oxalate levels in pediatric and adult patients and from
the European Medicines Agency (EMA) for the treatment of PH1 in all
age groups. In the pivotal ILLUMINATE-A study, OXLUMO was shown to
significantly reduce levels of urinary oxalate relative to placebo,
with the majority of patients reaching normal or near-normal
levels. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO
demonstrated an efficacy and safety profile consistent to that
observed in ILLUMINATE-A. In the ILLUMINATE-C study, OXLUMO
resulted in substantial reductions in plasma oxalate in patients
with advanced PH1. Across all three studies, injection site
reactions (ISRs) were the most common drug-related adverse
reaction. OXLUMO is administered via subcutaneous injection once
monthly for three months, then once quarterly beginning one month
after the last loading dose at a dose based on actual body weight.
For patients who weigh less than 10 kg, ongoing dosing remains
monthly. OXLUMO should be administered by a healthcare
professional. For more information about OXLUMO, including the full
U.S. Prescribing Information, visit OXLUMO.com.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma lipid nanoparticle
(LNP) intellectual property for use in RNAi therapeutic products
using LNP technology.
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 revolutionary approach has transformed 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 for 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 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 countries later in 2025; Alnylam’s advancement towards its
“Alnylam P5x25” goals and positioning to deliver sustainable
innovation to patients for the next half of the decade; the
potential for Alnylam to advance its research and development
programs, including the number of high-value programs Alnylam will
have in the clinic by the end of 2025 and the timing of Alnylam’s
initiation of a Phase 3 clinical trial of nucresiran in ATTR-CM;
the advancement of fitusiran through regulatory review and approval
and Alnylam’s receipt of any royalties on sales of fitusiran; and
Alnylam’s projected commercial and financial performance, including
the expected range of net product revenues and net revenues from
collaborations and royalties for 2025 and the expected range of
aggregate annual GAAP and non-GAAP R&D and SG&A expenses
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,
zilebesiran, nucresiran and mivelsiran; 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; 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 most recent Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (SEC), as may be updated
from time to time 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.
ALNYLAM PHARMACEUTICALS,
INC.
CONSOLIDATED BALANCE
SHEETS
(In thousands, except per
share amounts)
December 31, 2024
December 31, 2023
ASSETS
Current assets:
Cash and cash equivalents
$
966,428
$
812,688
Marketable debt securities
1,719,920
1,615,516
Marketable equity securities
8,156
11,178
Accounts receivable, net
405,308
327,787
Inventory
78,509
89,146
Prepaid expenses and other current
assets
116,964
126,382
Total current assets
3,295,285
2,982,697
Property, plant and equipment, net
502,784
526,057
Operating lease right-of-use assets
191,148
199,732
Deferred tax assets
116,863
10,101
Restricted investments
68,593
49,391
Other assets
65,310
61,902
Total assets
$
4,239,983
$
3,829,880
LIABILITIES AND STOCKHOLDERS’
EQUITY (DEFICIT)
Current liabilities:
Accounts payable
$
88,415
$
55,519
Accrued expenses
887,472
713,013
Operating lease liabilities
41,886
41,510
Deferred revenue
55,481
102,753
Liability related to the sale of future
royalties
113,018
54,991
Total current liabilities
1,186,272
967,786
Operating lease liabilities, net of
current portion
229,541
243,101
Deferred revenue, net of current
portion
—
188,175
Convertible debt
1,024,621
1,020,776
Liability related to the sale of future
royalties, net of current portion
1,334,353
1,322,248
Other liabilities
398,108
308,438
Total liabilities
4,172,895
4,050,524
Stockholders’ equity (deficit):
Preferred stock, $0.01 par value per
share, 5,000 shares authorized and no shares issued and outstanding
as of December 31, 2024 and December 31, 2023
—
—
Common stock, $0.01 par value per share,
250,000 shares authorized as of December 31, 2024 and December 31,
2023, respectively; 129,294 shares issued and outstanding as of
December 31, 2024; 125,794 shares issued and outstanding as of
December 31, 2023
1,293
1,259
Additional paid-in capital
7,388,061
6,811,063
Accumulated other comprehensive loss
(34,518
)
(23,375
)
Accumulated deficit
(7,287,748
)
(7,009,591
)
Total stockholders’ equity (deficit)
67,088
(220,644
)
Total liabilities and stockholders’ equity
(deficit)
$
4,239,983
$
3,829,880
This selected financial information should be read in
conjunction with the consolidated financial statements and notes
thereto included in Alnylam’s Annual Report on Form 10-K which
includes the audited financial statements for the year ended
December 31, 2024.
ALNYLAM PHARMACEUTICALS,
INC.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(In thousands, except per
share amounts)
Three Months Ended December
31,
Twelve Months Ended December
31,
2024
2023
2024
2023
(Unaudited)
(Unaudited)
Revenues:
Net product revenues
$
450,831
$
346,288
$
1,646,228
$
1,241,474
Net revenues from collaborations
106,948
76,407
510,221
546,185
Royalty revenue
35,387
17,023
91,794
40,633
Total revenues
593,166
439,718
2,248,243
1,828,292
Operating costs and expenses:
Cost of goods sold
102,649
71,975
306,513
268,216
Cost of collaborations and royalties
168
13,883
16,857
42,190
Research and development
300,169
272,141
1,126,232
1,004,415
Selling, general and administrative
295,339
198,123
975,526
795,646
Total operating costs and expenses
698,325
556,122
2,425,128
2,110,467
Loss from operations
(105,159
)
(116,404
)
(176,885
)
(282,175
)
Other (expense) income:
Interest expense
(38,971
)
(31,338
)
(141,858
)
(121,221
)
Interest income
31,019
30,406
121,992
95,561
Other expense, net
(80,847
)
(20,351
)
(180,624
)
(125,682
)
Total other expense, net
(88,799
)
(21,283
)
(200,490
)
(151,342
)
Loss before income taxes
(193,958
)
(137,687
)
(377,375
)
(433,517
)
Benefit from (provision for) income
taxes
110,195
(183
)
99,218
(6,725
)
Net loss
$
(83,763
)
$
(137,870
)
$
(278,157
)
$
(440,242
)
Net loss per common share — basic and
diluted
$
(0.65
)
$
(1.10
)
$
(2.18
)
$
(3.52
)
Weighted-average common shares used to
compute basic and diluted net loss per common share
129,116
125,613
127,651
124,906
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended December
31,
Twelve Months Ended December
31,
2024
2023
2024
2023
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP Research and development expenses
$
300,169
$
272,141
$
1,126,232
$
1,004,415
Less: Stock-based compensation
expenses
(40,625
)
(19,085
)
(127,749
)
(97,273
)
Non-GAAP Research and development
expenses
$
259,544
$
253,056
$
998,483
$
907,142
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP Selling, general and administrative
expenses
$
295,339
$
198,123
$
975,526
$
795,646
Less: Stock-based compensation
expenses
(51,020
)
(22,909
)
(144,335
)
(124,407
)
Non-GAAP Selling, general and
administrative expenses
$
244,319
$
175,214
$
831,191
$
671,239
Reconciliation of GAAP to Non-GAAP
operating (loss) income:
GAAP Loss from operations
$
(105,159
)
$
(116,404
)
$
(176,885
)
$
(282,175
)
Add: Stock-based compensation expenses
91,645
41,994
272,084
221,680
Non-GAAP Operating (loss) income
$
(13,514
)
$
(74,410
)
$
95,199
$
(60,495
)
Reconciliation of GAAP to Non-GAAP
other expense, net:
GAAP Total other expense, net
$
(88,799
)
$
(21,283
)
$
(200,490
)
$
(151,342
)
Add (Less): Realized and unrealized losses
(gains) on marketable equity securities
166
(767
)
3,022
16,944
Non-GAAP Other expense, net
$
(88,633
)
$
(22,050
)
$
(197,468
)
$
(134,398
)
Reconciliation of GAAP to Non-GAAP net
income (loss):
GAAP Net loss
$
(83,763
)
$
(137,870
)
$
(278,157
)
$
(440,242
)
Add: Stock-based compensation expenses
91,645
41,994
272,084
221,680
Add (Less): Realized and unrealized losses
(gains) on marketable equity securities
166
(767
)
3,022
16,944
Non-GAAP Net income (loss)
$
8,048
$
(96,643
)
$
(3,051
)
$
(201,618
)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP Net loss per common share - basic and
diluted
$
(0.65
)
$
(1.10
)
$
(2.18
)
$
(3.52
)
Add: Stock-based compensation expenses
0.71
0.33
2.13
1.77
Add (Less): Realized and unrealized losses
(gains) on marketable equity securities
—
(0.01
)
0.02
0.14
Non-GAAP Net loss per common share - basic
and diluted
$
0.06
$
(0.77
)
$
(0.02
)
$
(1.61
)
Please note that the figures presented above
may not sum exactly due to rounding
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF GAAP TO
NON-GAAP
PRODUCT REVENUE GROWTH AT
CONSTANT EXCHANGE RATE
December 31, 2024
Three Months Ended
Twelve Months Ended
Total TTR net product revenue growth*, as
reported
35
%
34
%
Add: Impact of foreign currency
translation
(1
)
—
Total TTR net product revenue growth at
constant exchange rate
34
%
34
%
Total Rare net product revenue growth*, as
reported
18
%
29
%
Add: Impact of foreign currency
translation
(1
)
(1
)
Total Rare net product revenue growth at
constant exchange rate
17
%
28
%
Total net product revenue growth*, as
reported
30
%
33
%
Add: Impact of foreign currency
translation
(1
)
—
Total net product revenue growth at
constant exchange rate
29
%
33
%
Total revenue growth*, as reported
35
%
23
%
Add: Impact of foreign currency
translation
(1
)
—
Total revenue growth at constant exchange
rate
34
%
23
%
*As compared to the three and twelve
months ended December 31, 2023, respectively.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20250213911700/en/
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
617-682-4340
Josh Brodsky (Investors) 617-551-8276
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