− Achieved Fourth Quarter and Full Year 2023
Global Net Product Revenues of $346 Million and $1,241 Million,
Respectively, Representing 39% Annual Growth Compared to 2022 –
− Company Announces Updated Statistical
Analysis Plan and Timing for HELIOS-B Phase 3 Study of Vutrisiran
–
− Announces U.S. FDA Clearance to Initiate
Multiple-Dosing in ALN-APP Phase 1 Study –
− Provides 2024 Combined Net Product Revenue
Guidance of $1,400 Million to $1,500 Million, in Addition to
Collaboration and Royalty Revenue and Operating Expense Guidance
–
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,
2023 and reviewed recent business highlights.
“2023 was a year of strong execution at Alnylam. We delivered
robust product revenue growth across our four wholly-owned
commercial medicines, with $1.24 billion in global net product
revenues, and achieved over 5,000 patients now being treated with
an Alnylam commercial medicine. We also extended our leadership in
RNAi with the first clinical demonstration of gene silencing in the
human brain using an RNAi therapeutic, strengthened our business
for the future through a landmark partnership with Roche and
delivered solid financial performance,” said Yvonne Greenstreet,
MBChB, Chief Executive Officer of Alnylam. “Looking ahead to 2024,
we are excited for a number of important milestones across the
pipeline, including results from the HELIOS-B Phase 3 study of
vutrisiran as well as the KARDIA-2 Phase 2 study of zilebesiran,
and six clinical study starts. This progress sets us up for
delivering on our Alnylam P5x25 goals of becoming a top-tier
biotech company delivering sustained innovation and exceptional
financial results.”
Fourth Quarter 2023 and Recent Significant Corporate
Highlights
Commercial Performance
Total TTR: ONPATTRO® (patisiran) & AMVUTTRA®
(vutrisiran)
- Achieved global net product revenues for ONPATTRO and AMVUTTRA
for the fourth quarter of $79 million and $175 million,
respectively, representing 10% total TTR quarterly growth compared
to Q3 2023, and full year 2023 revenues of $355 million and $558
million, respectively, representing 40% total TTR annual growth
compared to full year 2022.
- Attained over 4,060 hATTR amyloidosis patients with
polyneuropathy worldwide on commercial treatment with ONPATTRO or
AMVUTTRA as of December 31, 2023.
Total Ultra-Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Achieved global net product revenues for GIVLAARI and OXLUMO
for the fourth quarter of $59 million and $33 million,
respectively, representing 11% total Ultra-Rare quarterly growth
compared to Q3 2023, and full year 2023 revenues of $219 million
and $110 million, respectively, representing 35% total Ultra-Rare
annual growth compared to full year 2022.
- Attained over 650 patients on commercial GIVLAARI and over 430
patients on commercial OXLUMO worldwide as of December 31,
2023.
R&D Highlights
Alnylam announces today updates to the statistical analysis plan
for the HELIOS-B Phase 3 study of vutrisiran in patients
with ATTR amyloidosis with cardiomyopathy. These will include
updates to the primary and secondary endpoint structure, as well as
study exposure. Topline results are expected to be available in
late June or early July. Details will be discussed on the
conference call this morning.
Published results from Phase 3 APOLLO-B study of
patisiran in the New England Journal of Medicine.
Presented positive initial Phase 1 results with
ALN-TTRsc04 demonstrating rapid knockdown achieved by a
single dose with mean serum TTR reduction up to 97%, with
durability supporting potential for annual dosing and an
encouraging safety profile.
Presented positive results from the KARDIA-1 Phase 2
dose-ranging study of zilebesiran, an investigational RNAi
therapeutic in development to treat hypertension patients at high
cardiovascular risk, during the American Heart Association (AHA)
Scientific Sessions.
Announces today that the U.S. Food and Drug Administration (FDA)
has provided clearance to initiate the multiple-dose part (Part B)
of the ongoing Phase 1 study of ALN-APP, an investigational
RNAi therapeutic targeting amyloid precursor protein (APP) in
development for the treatment of Alzheimer’s disease and cerebral
amyloid angiopathy. The FDA has confirmed that multiple-dosing in
the Phase 1 study may proceed at doses up to 180 mg given every six
months, which covers all dose regimens planned to be explored in
Part B. A partial clinical hold remains for higher or more frequent
dosing regimens.
- Reported updated positive interim results for the ongoing
single ascending dose portion of the Phase 1 study of ALN-APP in
patients with early-onset Alzheimer’s disease at the 2023
Alzheimer’s Association International Conference and at the 16th
Clinical Trials in Alzheimer’s Disease conference.
Presented positive initial Phase 1 results with ALN-KHK
demonstrating robust target engagement and an encouraging safety
profile, supporting continued development as a novel treatment for
type 2 diabetes mellitus.
Filed an Investigational New Drug (IND) application for
ALN-BCAT, an investigational RNAi therapeutic targeting
β-catenin in development for the treatment of hepatocellular
carcinoma.
Sanofi presented positive results from the ATLAS-OLE Phase 3
extension study of fitusiran, demonstrating a substantially
improved safety profile and consistent bleed protection in people
with hemophilia A or B, with or without inhibitors. Specifically,
the risk of thrombosis was reduced, with rates comparable to those
reported in the general hemophilia population.
- Sanofi expects to submit a New Drug Application (NDA) to the
FDA in 2024.
Additional Business Updates
- Ranked #1 on Boston Globe’s Top Places to Work list for 2023 in
the “Largest Employer” category.
- Recognized by Science magazine as a Top Employer for the fifth
consecutive year.
Upcoming Events
Alnylam announces today that results from the KARDIA-2 Phase 2
study of zilebesiran will be presented in a Late-Breaker
presentation at the American College of Cardiology Scientific
Sessions 2024 on April 7, 2024 in Atlanta, Georgia.
In early 2024, Alnylam intends to:
- Report topline results from the HELIOS-B Phase 3 study of
vutrisiran in late June or early July.
- Initiate the KARDIA-3 Phase 2 study of zilebesiran.
- Initiate a Phase 2 study of ALN-APP in patients with
cerebral amyloid angiopathy.
- Initiate Part B of the Phase 1 study of ALN-KHK.
- Initiate a Phase 1 study of ALN-BCAT.
Financial Highlights for the Fourth Quarter and Year End
2023
Three Months Ended December
31,
Twelve Months Ended December
31,
(In thousands, except per share
amounts)
2023
2022
2023
2022
Net product revenues
$
346,288
$
261,675
$
1,241,474
$
894,329
Net revenue from collaborations
$
76,407
$
70,645
$
546,185
$
134,912
Royalty revenue
$
17,023
$
2,715
$
40,633
$
8,177
GAAP Operating loss
$
(116,404
)
$
(188,614
)
$
(282,175
)
$
(785,072
)
Non-GAAP Operating loss
$
(74,410
)
$
(145,847
)
$
(60,495
)
$
(554,423
)
GAAP Net loss
$
(137,870
)
$
(207,493
)
$
(440,242
)
$
(1,131,156
)
Non-GAAP Net loss
$
(96,643
)
$
(171,522
)
$
(201,618
)
$
(790,609
)
GAAP Net loss per common share - basic and
diluted
$
(1.10
)
$
(1.68
)
$
(3.52
)
$
(9.30
)
Non-GAAP Net loss per common share - basic
and diluted
$
(0.77
)
$
(1.39
)
$
(1.61
)
$
(6.50
)
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)
2023
2022
2023
2022
ONPATTRO net product revenues
$
79,006
$
122,221
$
354,546
$
557,608
AMVUTTRA net product revenues
175,254
68,566
557,838
93,795
Total TTR net product revenues
254,260
190,787
912,384
651,403
GIVLAARI net product revenues
59,298
47,058
219,251
173,144
OXLUMO net product revenues
32,730
23,830
109,839
69,782
Total net product revenues
$
346,288
$
261,675
$
1,241,474
$
894,329
Year over Year %
Growth
Three Months Ended December
31, 2023
Twelve Months Ended December
31, 2023
As Reported
At CER*
As Reported
At CER*
Total TTR net product revenues
33
%
31
%
40
%
40
%
GIVLAARI net product revenues
26
%
24
%
27
%
26
%
OXLUMO net product revenues
37
%
32
%
57
%
55
%
Total net product revenues
32
%
30
%
39
%
39
%
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in 2022. CER is a non-GAAP
measure.
- Net product revenues increased 32% and 39% at actual currency
during the three and twelve months ended December 31, 2023,
respectively, compared to the same periods in 2022, and 30% and 39%
at CER, respectively. The increases are primarily due to increased
patients on our commercial TTR therapies driven by the launch of
AMVUTTRA in the third quarter of 2022 as well as increased patients
on GIVLAARI and OXLUMO therapies.
Net Revenues from Collaborations
- Net revenues from collaborations increased 8% and 305% during
the three and twelve months ended December 31, 2023, respectively,
as compared to the same periods in 2022, primarily due to revenue
recognized under our Collaboration and License Agreement with
Roche, as executed in July 2023, and revenue recognized under our
Novartis Collaboration Agreement associated with the achievement of
specified commercialization and regulatory milestones.
Operating Expenses
Three Months Ended December
31,
Twelve Months Ended December
31,
(In thousands)
2023
2022
2023
2022
Cost of goods sold
$
71,975
$
46,172
$
268,216
$
140,174
Cost of goods sold as a percentage of net
product revenues
20.8
%
17.6
%
21.6
%
15.7
%
Cost of collaborations and royalties
$
13,883
$
5,094
$
42,190
$
28,643
GAAP research and development expenses
$
272,141
$
262,039
$
1,004,415
$
883,015
Non-GAAP research and development
expenses
$
253,056
$
245,095
$
907,142
$
790,854
GAAP selling, general and administrative
expenses
$
198,123
$
210,344
$
795,646
$
770,658
Non-GAAP selling, general and
administrative expenses
$
175,214
$
184,521
$
671,239
$
632,170
Cost of Goods Sold
- Cost of goods sold as a percentage of net product revenues
increased during the three and twelve months ended December 31,
2023, respectively, as compared to the same periods in 2022,
primarily due to increased volume and rate of royalties payable on
net sales of AMVUTTRA associated with tiered royalty percentages,
in addition to increased excess and obsolete charges primarily due
to canceling manufacturing commitments and the impairment of
ONPATTRO inventory that had been manufactured for future demand
associated with the ATTR amyloidosis with cardiomyopathy indication
for patisiran for which we did not receive regulatory
approval.
Research & Development (R&D) Expenses
- GAAP and non-GAAP R&D expenses increased during the three
and twelve months ended December 31, 2023, compared to the same
periods in 2022, primarily due to increased headcount and
infrastructure expenses to support our R&D pipeline,
development expenses associated with the KARDIA-1 and KARDIA-2
zilebesiran Phase 2 studies, and manufacturing and research related
expenses associated with our pre-clinical and developmental
activities. GAAP R&D expenses further increased during the
twelve month period due to increased stock-based compensation
expense related to the accounting for certain performance-based
awards during the period.
Selling, General & Administrative (SG&A) Expenses
- GAAP and non-GAAP SG&A expenses decreased during the three
months ended December 31, 2023, compared to the same period in
2022, primarily due to increased legal expenses in 2022 associated
with the Patent Infringement Lawsuits we filed in March 2022 and
the Department of Justice investigation, which closed in August
2023, and increased expenses in support of the global launch of
AMVUTTRA in the third quarter of 2022.
- GAAP and non-GAAP SG&A expenses increased during the twelve
months ended December 31, 2023, compared to the same period in
2022, primarily due to increased headcount and other strategic
investments in support of the global launch of AMVUTTRA and other
expenses to support our strategic growth.
Other Financial Highlights
- Cash, cash equivalents and marketable securities were $2.44
billion as of December 31, 2023, compared to $2.19 billion as of
December 31, 2022, with the increase primarily due to the receipts
of a $310 million upfront payment from Roche in connection with our
partnership to co-develop and co-commercialize zilebesiran, a $100
million payment from Regeneron in connection with the achievement
of certain criteria during early clinical development for our CNS
program, ALN-APP, and nearly $150 million from employee option
award exercises, offset by our operating loss for the year.
A reconciliation of our GAAP to non-GAAP results for the current
quarter is included in the tables at the end of this press
release.
2024 Financial Guidance
Full year December 31, 2024 financial guidance consists of the
following:
Combined net product revenues for
AMVUTTRA, ONPATTRO, GIVLAARI and OXLUMO1
$1,400 million – $1,500
million
Net Product Revenue Growth vs. 2023 at
reported Fx rates1
13% – 21%
Net Product Revenue Growth vs. 2023 at
constant exchange rates*
13% – 21%
Net revenues from collaborations and
royalties
$325 million – $425 million
GAAP R&D and SG&A expenses
$1,900 million - $2,050
million
Non-GAAP R&D and SG&A
expenses2
$1,675 million – $1,775
million
1 Uses January 31, 2024 FX rates
including: 1 EUR = 1.08 USD and 1 USD = 147 JPY
2 Primarily excludes $225-$275 million of
stock-based compensation expense from estimated GAAP R&D and
SG&A expenses.
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in the twelve months ended
December 31, 2023. CER is a non-GAAP measure.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures,
including expenses adjusted to exclude certain non-cash expenses
and non-recurring gains 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, as applicable, stock-based
compensation expenses, realized and unrealized (gains) losses on
marketable equity securities and loss on the extinguishment of
debt. 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) 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. The Company has excluded the loss on the extinguishment of
debt because the Company believes the item is a non-recurring
transaction outside the ordinary course of the Company’s
business.
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. In addition, these non-GAAP financial
measures are among those indicators the Company uses as a basis for
evaluating performance, allocating resources and planning and
forecasting future 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 2023 results as well as expectations
for the future via conference call on Thursday, February 15, 2024
at 8:30 am ET. To access the call, please register online at
https://register.vevent.com/register/BI94c4772acd31430480d414497e322a2f.
Participants are requested to register at least 15 minutes before
the start of the call. A replay of the call will be available two
hours after the call and archived on the same web page for six
months.
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 approved in the
United States for the treatment of adults with hATTR amyloidosis
with polyneuropathy. It is a double‑stranded small interfering RNA
(siRNA) that targets mutant and wild‑type transthyretin (TTR)
messenger RNA (mRNA). Using Alnylam’s Enhanced Stabilization
Chemistry (ESC)-GalNAc-conjugate delivery platform, AMVUTTRA is
designed for increased potency and high metabolic stability to
allow for subcutaneous injection once every three months
(quarterly). Results from the pivotal HELIOS-A Phase 3 study
demonstrate AMVUTTRA rapidly reduces serum TTR levels, has the
potential to reverse neuropathy impairment relative to baseline and
improves other key measures of disease burden relative to external
placebo in patients with the polyneuropathy of hATTR amyloidosis.
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, givosiran 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 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 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, statements 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
commercial products, and Alnylam’s projected commercial and
financial performance, including the expected range of net product
revenues and net revenues from collaborations and royalties for
2024, the expected range of aggregate annual GAAP and non-GAAP
R&D and SG&A expenses for 2024, the expected timing of
topline data from the HELIOS-B Phase 3 clinical study, 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,
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,
and ALN-APP; 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; 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 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)
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. 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, 2023
December 31, 2022
ASSETS
Current assets:
Cash and cash equivalents
$
812,688
$
866,394
Marketable debt securities
1,615,516
1,297,890
Marketable equity securities
11,178
28,122
Accounts receivable, net
327,787
237,963
Inventory
89,146
128,962
Prepaid expenses and other current
assets
126,382
132,916
Total current assets
2,982,697
2,692,247
Property, plant and equipment, net
526,057
523,494
Operating lease right-of-use assets
199,732
215,136
Restricted investments
49,391
49,390
Other assets
72,003
66,092
Total assets
$
3,829,880
$
3,546,359
LIABILITIES AND STOCKHOLDERS’
DEFICIT
Current liabilities:
Accounts payable
$
55,519
$
98,094
Accrued expenses
713,013
545,460
Operating lease liability
41,510
41,967
Deferred revenue
102,753
42,105
Liability related to the sale of future
royalties
54,991
40,289
Total current liabilities
967,786
767,915
Operating lease liability, net of current
portion
243,101
261,339
Deferred revenue, net of current
portion
188,175
193,791
Convertible debt
1,020,776
1,016,942
Liability related to the sale of future
royalties, net of current portion
1,322,248
1,252,015
Other liabilities
308,438
212,580
Total liabilities
4,050,524
3,704,582
Commitments and contingencies (Note
13)
Stockholders’ deficit:
Preferred stock, $0.01 par value per
share, 5,000 shares authorized and no shares issued and outstanding
as of December 31, 2023 and December 31, 2022
—
—
Common stock, $0.01 par value per share,
250,000 shares authorized as of December 31, 2023 and December 31,
2022, respectively; 125,794 shares issued and outstanding as of
December 31, 2023; 123,925 shares issued and outstanding as of
December 31, 2022
1,259
1,240
Additional paid-in capital
6,811,063
6,454,540
Accumulated other comprehensive loss
(23,375
)
(44,654
)
Accumulated deficit
(7,009,591
)
(6,569,349
)
Total stockholders’ deficit
(220,644
)
(158,223
)
Total liabilities and stockholders’
deficit
$
3,829,880
$
3,546,359
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, 2023.
ALNYLAM PHARMACEUTICALS,
INC.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(In thousands, except per
share amounts)
Three Months Ended December
31,
Twelve Months Ended December
31,
2023
2022
2023
2022
(Unaudited)
(Unaudited)
Revenues:
Net product revenues
$
346,288
$
261,675
$
1,241,474
$
894,329
Net revenues from collaborations
76,407
70,645
546,185
134,912
Royalty revenue
17,023
2,715
40,633
8,177
Total revenues
439,718
335,035
1,828,292
1,037,418
Operating costs and expenses:
Cost of goods sold
71,975
46,172
268,216
140,174
Cost of collaborations and royalties
13,883
5,094
42,190
28,643
Research and development
272,141
262,039
1,004,415
883,015
Selling, general and administrative
198,123
210,344
795,646
770,658
Total operating costs and expenses
556,122
523,649
2,110,467
1,822,490
Loss from operations
(116,404
)
(188,614
)
(282,175
)
(785,072
)
Other (expense) income:
Interest expense
(31,338
)
(29,913
)
(121,221
)
(155,968
)
Interest income
30,406
14,077
95,561
24,808
Other expense, net
(20,351
)
(2,571
)
(125,682
)
(134,175
)
Loss on the extinguishment of debt
—
—
—
(76,586
)
Total other expense, net
(21,283
)
(18,407
)
(151,342
)
(341,921
)
Loss before income taxes
(137,687
)
(207,021
)
(433,517
)
(1,126,993
)
Provision for income taxes
(183
)
(472
)
(6,725
)
(4,163
)
Net loss
$
(137,870
)
$
(207,493
)
$
(440,242
)
$
(1,131,156
)
Net loss per common share — basic and
diluted
$
(1.10
)
$
(1.68
)
$
(3.52
)
$
(9.30
)
Weighted-average common shares used to
compute basic and diluted net loss per common share
125,613
123,266
124,906
121,689
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,
2023
2022
2023
2022
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP Research and development
$
272,141
$
262,039
$
1,004,415
$
883,015
Less: Stock-based compensation
expenses
(19,085
)
(16,944
)
(97,273
)
(92,161
)
Non-GAAP Research and development
$
253,056
$
245,095
$
907,142
$
790,854
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP Selling, general and
administrative
$
198,123
$
210,344
$
795,646
$
770,658
Less: Stock-based compensation
expenses
(22,909
)
(25,823
)
(124,407
)
(138,488
)
Non-GAAP Selling, general and
administrative
$
175,214
$
184,521
$
671,239
$
632,170
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
$
(116,404
)
$
(188,614
)
$
(282,175
)
$
(785,072
)
Add: Stock-based compensation expenses
41,994
42,767
221,680
230,649
Non-GAAP Operating loss
$
(74,410
)
$
(145,847
)
$
(60,495
)
$
(554,423
)
Reconciliation of GAAP to Non-GAAP
Other (expense) income:
GAAP Total other expense, net
$
(21,283
)
$
(18,407
)
$
(151,342
)
$
(341,921
)
(Less) Add: Realized and unrealized (gain)
loss on marketable equity securities
(767
)
(6,796
)
16,944
33,312
Add: Loss on the extinguishment of
debt
—
—
—
76,586
Non-GAAP Other expense, net
$
(22,050
)
$
(25,203
)
$
(134,398
)
$
(232,023
)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP Net loss
$
(137,870
)
$
(207,493
)
$
(440,242
)
$
(1,131,156
)
Add: Stock-based compensation expenses
41,994
42,767
221,680
230,649
(Less) Add: Realized and unrealized (gain)
loss on marketable equity securities
(767
)
(6,796
)
16,944
33,312
Add: Loss on the extinguishment of
debt
—
—
—
76,586
Non-GAAP Net loss
$
(96,643
)
$
(171,522
)
$
(201,618
)
$
(790,609
)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP Net loss per common share - basic and
diluted
$
(1.10
)
$
(1.68
)
$
(3.52
)
$
(9.30
)
Add: Stock-based compensation expenses
0.33
0.35
1.77
1.90
(Less) Add: Realized and unrealized (gain)
loss on marketable equity securities
(0.01
)
(0.06
)
0.14
0.27
Add: Loss on the extinguishment of
debt
—
—
—
0.63
Non-GAAP Net loss per common share - basic
and diluted
$
(0.77
)
$
(1.39
)
$
(1.61
)
$
(6.50
)
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 CURRENCY
(Unaudited)
December 31, 2023
Three Months Ended
Twelve Months Ended
Total TTR net product revenue growth, as
reported
33
%
40
%
Add: Impact of foreign currency
translation
(2
)
—
Total TTR net product revenue growth at
constant currency
31
%
40
%
GIVLAARI net product revenue growth, as
reported
26
%
27
%
Add: Impact of foreign currency
translation
(2
)
(1
)
GIVLAARI net product revenue growth at
constant currency
24
%
26
%
OXLUMO net product revenue growth, as
reported
37
%
57
%
Add: Impact of foreign currency
translation
(5
)
(2
)
OXLUMO net product revenue growth at
constant currency
32
%
55
%
Total net product revenue growth, as
reported
32
%
39
%
Add: Impact of foreign currency
translation
(2
)
—
Total net product revenue growth at
constant currency
30
%
39
%
Total revenue growth, as reported
31
%
76
%
Add: Impact of foreign currency
translation
(2
)
—
Total revenue growth at constant
currency
29
%
76
%
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240215594931/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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