− Achieved Third Quarter 2023 Global Net
Product Revenues of $313 Million, Representing 35% Year-Over-Year
Growth Compared to Q3 2022 –
− Received Complete Response Letter from U.S.
FDA for Supplemental New Drug Application for Patisiran for the
Treatment of the Cardiomyopathy of ATTR Amyloidosis –
− Company Remains On Track to Report Topline
Results from HELIOS-B Phase 3 Study in Early 2024 –
– Reported Positive Topline Results from
KARDIA-1 Phase 2 Study of Zilebesiran –
− Updated 2023 Guidance for Revenues from
Collaborations and Royalties to $575 Million to $625 Million;
Reiterated 2023 Guidance for Combined Net Product Revenues of
$1,200 Million to $1,285 Million –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY),
the leading RNAi therapeutics company, today reported its
consolidated financial results for the third quarter ended
September 30, 2023 and reviewed recent business highlights.
“We continued to deliver strong commercial results in the third
quarter, with the successful launch of AMVUTTRA contributing to 35%
year-over-year growth in net product revenues,” said Yvonne
Greenstreet, MBChB, Chief Executive Officer of Alnylam. “While we
were disappointed by the recent Complete Response Letter for
ONPATTRO for the treatment of the cardiomyopathy of ATTR
amyloidosis, we remain confident in the HELIOS-B Phase 3 study of
vutrisiran, which is on track to deliver topline results in early
2024. We’re also excited about advancements across our broad
pipeline of RNAi therapeutics, including recent positive topline
results from the KARDIA-1 Phase 2 study of zilebesiran, as well as
upcoming Phase 1 results for ALN-TTRsc04 and ALN-KHK by year-end.
With this continued execution we remain on track to meet our
Alnylam P5x25 goals of becoming a top-tier biotech company
delivering sustained innovation and exceptional financial
results.”
Third 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 third quarter of $82 million and $149 million,
respectively, representing 35% total TTR reported year-over-year
growth compared to Q3 2022.
- Attained over 3,790 hATTR amyloidosis patients with
polyneuropathy worldwide on commercial treatment with ONPATTRO or
AMVUTTRA as of September 30, 2023.
Total Ultra-Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Achieved global net product revenues for GIVLAARI and OXLUMO
for the third quarter of $54 million and $29 million, respectively,
representing 33% total Ultra-Rare reported year-over-year growth
compared to Q3 2022.
- Attained over 625 patients worldwide on commercial GIVLAARI
treatment as of September 30, 2023.
- Attained over 375 patients worldwide on commercial OXLUMO
treatment as of September 30, 2023.
Leqvio® (inclisiran)
- Leqvio launch in the U.S. and other markets is ongoing, with
focus on patient on-boarding, removing access hurdles and enhancing
medical education. In the U.S., Leqvio is covered at or near label
for 76% of patients. More than 55% of Leqvio source of business in
the U.S. is now through "Buy and Bill" acquisition mode. FDA
expanded the label to include primary hyperlipidemia (patients at
increased risk of ASCVD) and the removal of four adverse reactions
from the safety section as well as Limitations of Use. In Q3 2023,
Leqvio was approved in China and in Japan and is now approved in 93
countries.
R&D Highlights
Reported positive topline results from the KARDIA-1 Phase 2
dose-ranging study of zilebesiran, an investigational RNAi
therapeutic in development to treat hypertension in patients at
high cardiovascular risk.
Published results from Phase 1 study of zilebesiran in the New
England Journal of Medicine.
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 on Alzheimer’s Disease conference.
- Based on the achievement of specified clinical
proof-of-principle criteria for the Phase 1 ALN-APP program,
Alnylam received a $100 million milestone payment from
Regeneron.
Presented new 24-month results from an interim analysis of the
open-label extension period of the APOLLO-B Phase 3 study of
patisiran in patients with the cardiomyopathy of
transthyretin-mediated amyloidosis at the Heart Failure Society of
America Annual Scientific Meeting 2023.
Published results from Phase 3 APOLLO-B study of patisiran in
the New England Journal of Medicine.
Sanofi reported positive data for fitusiran, in
development for the treatment of hemophilia A or B, with or without
inhibitors, from the Phase 3 open-label extension study
(ATLAS-OLE).
- Sanofi is currently in discussions with the FDA regarding
filing in 2024.
Additional Business Updates
- Entered into a global strategic collaboration with Roche for
the co-development and co-commercialization of
zilebesiran.
- Recognized by Science magazine as a Top Employer for the fifth
consecutive year.
Upcoming Events
Alnylam announces today that it will present a review of its
RNAi platform and pipeline progress at a virtual R&D Day on
December 13, 2023.
Alnylam announces today that results from the KARDIA-1 Phase 2
dose-ranging study of zilebesiran will be presented as a
Late-Breaker at the American Heart Association Scientific Sessions
2023 on November 11, 2023, in Philadelphia, Pennsylvania.
In addition, in late 2023:
- Alnylam intends to report topline results from the Phase 1
study of ALN-TTRsc04.
- Alnylam intends to report topline results from the Phase 1
study of ALN-KHK.
- Vir is conducting multiple trials evaluating the potential for
ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional
cure for chronic hepatitis B. Phase 2 data readouts are on track
for Q4 2023.
- Vir also announced that initial Phase 2 data readouts for the
SOLSTICE trial evaluating ALN-HBV02 (VIR-2218) and VIR-3434
as monotherapy and in combination for the treatment of people
living with chronic hepatitis delta, the most aggressive form of
viral hepatitis, are expected in Q4 2023.
Financial Results for the Quarter Ended September 30,
2023
Three Months Ended September
30,
(in thousands, except per share
amounts)
2023
2022
Net product revenues
$
313,153
$
232,267
Net revenue from collaborations
$
427,472
$
29,297
Royalty revenue
$
9,905
$
2,742
GAAP Operating income (loss)
$
213,867
$
(258,040
)
Non-GAAP Operating income (loss)
$
277,804
$
(129,922
)
GAAP Net income (loss)
$
147,753
$
(405,920
)
Non-GAAP Net income (loss)
$
228,534
$
(193,366
)
GAAP net income (loss) per common share -
basic
$
1.18
$
(3.32
)
Non-GAAP net income (loss) per common
share - basic
$
1.83
$
(1.58
)
GAAP net income (loss) per common share -
diluted
$
1.15
$
(3.32
)
Non-GAAP net income (loss) per common
share - diluted
$
1.74
$
(1.58
)
* 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 September
30,
Year over Year %
Growth
(in thousands, except percentages)
2023
2022
As Reported
At CER*
ONPATTRO net product revenues
$
81,589
$
144,950
(44
)%
(46
)%
AMVUTTRA net product revenues
148,680
25,229
489
%
488
%
Total TTR net product revenues
$
230,269
$
170,179
35
%
34
%
GIVLAARI net product revenues
54,148
45,659
19
%
17
%
OXLUMO net product revenues
28,736
16,429
75
%
69
%
Total net product revenues
$
313,153
$
232,267
35
%
33
%
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in the third quarter 2022. 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 35% during the third quarter
2023, as compared to the prior year, and 33% at CER. The increase
is primarily related to growth in our TTR product revenues driven
by the launch of AMVUTTRA in the third quarter of 2022 as well as
increased patients on GIVLAARI and OXLUMO therapies. The increase
was offset by a decrease of demand for ONPATTRO due to patient
switched to AMVUTTRA.
Net Revenues from Collaborations
- The increase in net revenues from collaborations, as compared
to the prior year, is due to revenue related to our collaborations
with Roche and Regeneron. During the quarter, we recognized $310
million of revenue from the upfront payment received from Roche and
$65 million in connection with our Regeneron Collaboration. The $65
million of revenue is a cumulative adjustment from the $100 million
milestone received for achieving certain criteria during early
clinical development for our CNS program, ALN-APP.
Operating Expenses
Three Months Ended September
30,
2023 vs 2022
(in thousands, except percentages)
2023
2022
% Change*
Cost of goods sold
$
79,473
$
36,507
118
%
Cost of goods sold as a percentage of net
product revenues
25.4
%
15.7
%
9.7
%
Cost of collaborations
$
4,836
$
4,609
5
%
GAAP research and development expenses
$
253,179
$
245,371
3
%
Non-GAAP research and development
expenses
$
224,024
$
192,409
16
%
GAAP selling, general and administrative
expenses
$
199,175
$
235,859
(16
)%
Non-GAAP selling, general and
administrative expenses
$
164,393
$
160,703
2
%
*For dollar values, we calculate the
percentage of change during Q3 2023 compared to Q3 2022. For cost
of goods sold as a percentage of net product revenues, we calculate
the basis point change during Q3 2023 compared to Q3 2022.
Cost of Goods Sold
- Cost of goods sold as a percent of net product revenues
increased during the third quarter 2023, as compared to the prior
year, primarily due to recording an impairment of ONPATTRO
inventory that had been manufactured for future demand associated
with the ATTR 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 third
quarter 2023, as compared to the prior year, primarily due to
increased costs related to clinical activities and increased
headcount to support our R&D pipeline and an expense for the
achievement of a certain clinical milestone payable to a partner.
Increased GAAP R&D expenses were offset by decreased
stock-based compensation expense related to the accounting for
certain performance-based awards that vested in 2022.
Selling, General & Administrative (SG&A) Expenses
- GAAP SG&A expenses decreased during the third quarter 2023,
as compared to the prior year, primarily due to decreased
stock-based compensation expense related to the accounting for
certain performance-based awards that vested in 2022. Non-GAAP
SG&A expenses increased in the third quarter of 2023, compared
to 2022, primarily due to increased headcount and other investments
supporting our strategic growth including the global launch of
AMVUTTRA.
Other Financial Highlights
- Cash, cash equivalents and marketable securities were $2.41
billion as of September 30, 2023 compared to $2.19 billion as of
December 31, 2022 with the increase primarily related to the $310
million upfront payment received from Roche in connection with our
partnership to co-develop and co-commercialize zilebesiran, offset
by our operating loss year-to-date.
The adjustments to the non-GAAP measures provided in the
financial results above and in the financial guidance below are
described under “Use of Non-GAAP Financial Measures” later in this
press release. A reconciliation of our GAAP to non-GAAP results
presented in this release is included in the tables at the end of
this press release.
2023 Financial Guidance Full year 2023 financial
guidance has been updated as follows:
Provided 2/23/20231
Updated 11/2/2023
Combined net product revenues for
ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO1
$1,200 million - $1,285
million
Unchanged
Net Product Revenue Growth vs. 2022 at
reported FX rates1
34% to 44%
Unchanged
Net Product Revenue Growth vs. 2022 at
CER*
34% to 44%
Unchanged
Net revenues from collaborations and
royalties
$100 million -
$175 million
$575 million -
$625 million
GAAP R&D and SG&A expenses
$1,790 million - $1,885
million
Unchanged
Non-GAAP R&D and SG&A
expenses2
$1,575 million - $1,650
million
Unchanged
1 Uses December 31, 2022 FX rates
including: 1 EUR = 1.07 USD and 1 USD = 131 JPY
2 Excludes $215 - $235 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, 2022. 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 certain losses
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 non-GAAP financial measures we present include Non-GAAP
Operating income (loss), Non-GAAP Net income (loss), Non-GAAP Net
income (loss) per common share - basic, Non-GAAP Net income (loss)
per common share - diluted and Non-GAAP R&D and SG&A
expenses. 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 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 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, also a non-GAAP
financial measure, 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 historical GAAP and
non-GAAP measures presented in this release is provided later in
this press release.
Conference Call Information Management will provide
an update on the Company and discuss third quarter 2023 results as
well as expectations for the future via conference call on
Thursday, November 2, 2023 at 8:30 am ET. To access the call,
please register online at
https://register.vevent.com/register/BI29582eb5fd274b758554baecef8f03fa.
Participants are requested to register at a minimum 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 the polyneuropathy of hATTR amyloidosis in adults.
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 the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. 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 on Instagram.
Alnylam Forward Looking Statements This press
release contains forward-looking statements within the meaning of
Section 27A of the Securities Act of 1933 and Section 21E of the
Securities Exchange Act of 1934. All statements other than
historical statements of fact regarding Alnylam’s expectations,
beliefs, goals, plans or prospects including, without limitation,
expectations regarding Alnylam’s aspiration to become a leading
biotech company and the planned achievement of its “Alnylam P5x25”
strategy, the potential for Alnylam to identify new potential drug
development candidates and advance its research and development
programs, Alnylam’s ability to obtain approval for new commercial
products or additional indications for its existing products, and
Alnylam’s projected commercial and financial performance, should be
considered forward-looking statements. Actual results and future
plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation: the
direct or indirect impact of the COVID-19 global pandemic or any
future pandemic on Alnylam’s business, results of operations and
financial condition; 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; 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, including as may arise from recent disruptions in the
supply of non-human primates; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
indication 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 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. Patisiran 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.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
Nine Months Ended
September 30,
2023
September 30,
2022
September 30,
2023
September 30,
2022
Statements of Operations
Revenues:
Net product revenues
$
313,153
$
232,267
$
895,186
$
632,654
Net revenues from collaborations
427,472
29,297
469,778
64,267
Royalty revenue
9,905
2,742
23,610
5,462
Total revenues
750,530
264,306
1,388,574
702,383
Operating costs and expenses:
Cost of goods sold
79,473
36,507
196,241
94,002
Cost of collaborations and royalties
4,836
4,609
28,307
23,549
Research and development
253,179
245,371
732,274
620,976
Selling, general and administrative
199,175
235,859
597,523
560,314
Total operating costs and expenses
536,663
522,346
1,554,345
1,298,841
Income (loss) from operations
213,867
(258,040
)
(165,771
)
(596,458
)
Other (expense) income:
Interest expense
(30,893
)
(41,084
)
(89,883
)
(126,055
)
Interest income
25,425
7,820
65,155
10,731
Other expense, net
(57,658
)
(38,053
)
(105,331
)
(131,604
)
Loss on the extinguishment of debt
—
(76,586
)
—
(76,586
)
Total other expense, net
(63,126
)
(147,903
)
(130,059
)
(323,514
)
Income (loss) before income taxes
150,741
(405,943
)
(295,830
)
(919,972
)
(Provision for) benefit from income
taxes
(2,988
)
23
(6,542
)
(3,691
)
Net income (loss)
$
147,753
$
(405,920
)
$
(302,372
)
$
(923,663
)
Net income (loss) per common share -
basic
$
1.18
$
(3.32
)
$
(2.43
)
$
(7.62
)
Net income (loss) per common share -
diluted
$
1.15
$
(3.32
)
$
(2.43
)
$
(7.62
)
Weighted-average common shares- basic
125,220
122,166
124,667
121,158
Weighted-average common shares-
diluted
131,337
122,166
124,667
121,158
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
(In thousands, except per
share amounts)
(Unaudited)
September 30, 2023
December 31, 2022
ASSETS
Current assets:
Cash and cash equivalents
$
1,033,024
$
866,394
Marketable debt securities
1,362,843
1,297,890
Marketable equity securities
10,411
28,122
Accounts receivable, net
325,445
237,963
Inventory
95,771
128,962
Prepaid expenses and other current
assets
157,958
132,916
Total current assets
2,985,452
2,692,247
Property, plant and equipment, net
525,591
523,494
Operating lease right-of-use assets
203,485
215,136
Restricted investments
49,390
49,390
Other assets
75,155
66,092
Total assets
$
3,839,073
$
3,546,359
LIABILITIES AND STOCKHOLDERS’
DEFICIT
Current liabilities:
Accounts payable
$
73,840
$
98,094
Accrued expenses
713,094
545,460
Operating lease liability
41,516
41,967
Deferred revenue
77,140
42,105
Liability related to the sale of future
royalties
44,195
40,289
Total current liabilities
949,785
767,915
Operating lease liability, net of current
portion
247,711
261,339
Deferred revenue, net of current
portion
196,086
193,791
Convertible debt
1,019,809
1,016,942
Liability related to the sale of future
royalties, net of current portion
1,310,814
1,252,015
Other liabilities
280,734
212,580
Total liabilities
4,004,939
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 September 30, 2023 and December 31, 2022
—
—
Common stock, $0.01 par value per share,
250,000 shares authorized; 125,454 shares issued and outstanding as
of September 30, 2023; 123,925 shares issued and outstanding as of
December 31, 2022
1,255
1,240
Additional paid-in capital
6,736,939
6,454,540
Accumulated other comprehensive loss
(32,339
)
(44,654
)
Accumulated deficit
(6,871,721
)
(6,569,349
)
Total stockholders’ deficit
(165,866
)
(158,223
)
Total liabilities and stockholders’
deficit
$
3,839,073
$
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, 2022.
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
Nine Months Ended
September 30,
2023
September 30,
2022
September 30,
2023
September 30,
2022
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP research and development
$
253,179
$
245,371
$
732,274
$
620,976
Less: Stock-based compensation
expenses
(29,155
)
(52,962
)
(78,188
)
(75,217
)
Non-GAAP research and development
$
224,024
$
192,409
$
654,086
$
545,759
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP selling, general and
administrative
$
199,175
$
235,859
$
597,523
$
560,314
Less: Stock-based compensation
expenses
(34,782
)
(75,156
)
(101,498
)
(112,665
)
Non-GAAP selling, general and
administrative
$
164,393
$
160,703
$
496,025
$
447,649
Reconciliation of GAAP to Non-GAAP
operating income (loss):
GAAP operating income (loss)
$
213,867
$
(258,040
)
$
(165,771
)
$
(596,458
)
Add: Stock-based compensation expenses
63,937
128,118
179,686
187,882
Non-GAAP operating income (loss)
$
277,804
$
(129,922
)
$
13,915
$
(408,576
)
Reconciliation of GAAP to Non-GAAP net
income (loss):
GAAP net income (loss)
$
147,753
$
(405,920
)
$
(302,372
)
$
(923,663
)
Add: Stock-based compensation expenses
63,937
128,118
179,686
187,882
Add: Realized and unrealized loss on
marketable equity securities
16,844
7,850
17,711
40,108
Add: Loss on the extinguishment of
debt
—
76,586
—
76,586
Non-GAAP net income (loss)
$
228,534
$
(193,366
)
$
(104,975
)
$
(619,087
)
Reconciliation of GAAP to Non-GAAP net
income (loss) per common share- basic:
GAAP net income (loss) per common share -
basic
$
1.18
$
(3.32
)
$
(2.43
)
$
(7.62
)
Add: Stock-based compensation expenses
0.51
1.05
1.44
1.55
Add: Realized and unrealized loss on
marketable equity securities
0.13
0.06
0.14
0.33
Add: Loss on the extinguishment of
debt
—
0.63
—
0.63
Non-GAAP net income (loss) per common
share - basic
$
1.83
$
(1.58
)
$
(0.84
)
$
(5.11
)
Reconciliation of GAAP to Non-GAAP net
income (loss) per common share- diluted:
GAAP net income (loss) per common share -
diluted
$
1.15
$
(3.32
)
$
(2.43
)
$
(7.62
)
Add: Stock-based compensation expenses
0.49
1.05
1.44
1.55
Add: Realized and unrealized loss on
marketable equity securities
0.13
0.06
0.14
0.33
Add: Loss on the extinguishment of
debt
—
0.63
—
0.63
Non-GAAP net income (loss) per common
share - diluted
$
1.74
$
(1.58
)
$
(0.84
)
$
(5.11
)
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)
September 30, 2023
Three Months Ended
Nine Months Ended
ONPATTRO net product revenue growth, as
reported
(44
)%
(37
)%
Add: Impact of foreign currency
translation
(2
)
—
ONPATTRO net product revenue growth at
constant currency
(46
)%
(37
)%
AMVUTTRA net product revenue growth, as
reported
489
%
1416
%
Add: Impact of foreign currency
translation
(1
)
18
AMVUTTRA net product revenue growth at
constant currency
488
%
1434
%
Total TTR net product revenue growth, as
reported
35
%
43
%
Add: Impact of foreign currency
translation
(1
)
1
Total TTR net product revenue growth at
constant currency
34
%
44
%
GIVLAARI net product revenue growth, as
reported
19
%
27
%
Add: Impact of foreign currency
translation
(2
)
—
GIVLAARI net product revenue growth at
constant currency
17
%
27
%
OXLUMO net product revenue growth, as
reported
75
%
68
%
Add: Impact of foreign currency
translation
(6
)
(1
)
OXLUMO net product revenue growth at
constant currency
69
%
67
%
Total net product revenue growth, as
reported
35
%
41
%
Add: Impact of foreign currency
translation
(2
)
1
Total net product revenue growth at
constant currency
33
%
42
%
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231102700234/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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