− Achieved First Quarter 2023 Global Net
Product Revenues of $276 Million, Representing 48% Year-Over-Year
Growth Compared to Q1 2022 –
− Reported Positive Interim Phase 1 Clinical
Data on ALN-APP, an Investigational RNAi Therapeutic for
Alzheimer’s Disease and Cerebral Amyloid Angiopathy, Establishing
First-Ever Human Translation of RNAi Therapeutics in CNS –
− Reiterated 2023 Financial Guidance, Including
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 first quarter ended March 31, 2023 and reviewed
recent business highlights.
“The first quarter of 2023 saw continued strong commercial
execution, driven by the ongoing launch of AMVUTTRA, delivering
robust 48% growth in total product sales compared with the first
quarter 2022. We also made significant progress with our broad
pipeline, delivering positive first-in-human results for an RNAi
therapeutic in the CNS from an interim analysis of the Phase 1
study of ALN-APP in patients with early-onset Alzheimer’s disease.
We believe this proof of concept is a significant catalyst for
future potential growth for Alnylam with the expansion of our
product engine to include tissues beyond the liver,” said Yvonne
Greenstreet, MBChB, Chief Executive Officer of Alnylam. “We’re also
excited for other key upcoming milestones from our pipeline,
including topline Phase 2 results from the KARDIA-1 study of
zilebesiran, which are expected mid-year. With this continued
execution, we believe we are tracking well toward our Alnylam P5x25
goals of becoming a top-tier biotech company delivering sustained
innovation and exceptional financial results.”
First 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 first quarter of $102 million and $102 million,
respectively, representing 49% total TTR reported year-over-year
growth compared to Q1 2022 and 54% growth on a CER* basis.
- Attained over 3,160 hATTR amyloidosis patients with
polyneuropathy worldwide on commercial treatment with ONPATTRO or
AMVUTTRA as of March 31, 2023.
Total Ultra-Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Achieved global net product revenues for GIVLAARI and OXLUMO
for the first quarter of $48 million and $24 million, respectively,
representing 45% total Ultra-Rare reported year-over-year growth
compared to Q1 2022 and 48% on a CER* basis.
- Attained over 550 patients worldwide on commercial GIVLAARI
treatment as of March 31, 2023.
- Attained over 300 patients worldwide on commercial OXLUMO
treatment as of March 31, 2023.
Leqvio® (inclisiran)
- 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 14 months after launch. More than 50% of Leqvio source
of business in the U.S. is now through “Buy and Bill” acquisition
model.
R&D Highlights
Reported positive interim results from the ongoing single
ascending dose (SAD) part of the Phase 1 study of ALN-APP in
patients with early-onset Alzheimer’s disease (EOAD). These results
establish the first human translation of Alnylam’s proprietary
C16-siRNA conjugate platform for central nervous system (CNS)
delivery and are the first clinical demonstration of gene silencing
in the human brain using an RNAi therapeutic.
The Company announces today that it has submitted 18-month data
from the APOLLO-B Phase 3 study to the U.S. FDA as part of the sNDA
review for ONPATTRO (patisiran) for the treatment of the
cardiomyopathy of ATTR amyloidosis.
Sanofi announced that results from the ATLAS-INH and ATLAS-A/B
studies evaluating the efficacy and safety of fitusiran were
published respectively in The Lancet and The Lancet Haematology,
reinforcing the potential of investigational fitusiran to transform
the current standard of care and address unmet needs for all types
of hemophilia, regardless of inhibitor status.
Additional Key Pipeline Progress:
- Submitted an sNDA to the Brazilian Health Regulatory Agency
(ANVISA) for ONPATTRO (patisiran) for the treatment of the
cardiomyopathy of ATTR amyloidosis.
- Presented post-hoc analyses from the Phase 3 ENVISION study of
givosiran in patients with AHP through Month 36 at the
Hemostasis & Thrombosis Research Society (HTRS) Scientific
Symposium.
- Presented preclinical data for ALN-HTT, for the
potential treatment of Huntington’s disease, at the CHDI 18th
Annual Huntington’s Disease Therapeutics Conference, in
collaboration with Regeneron.
- Announced today that dosing has been initiated in a Phase 1
study of ALN-KHK, for the potential treatment of type 2
diabetes.
- Regeneron announced that it has initiated a Phase 2 study of
ALN-HSD in patients with NASH.
- Vir initiated the Phase 2 PREVAIL platform trial and its
THRIVE/STRIVE sub-protocols evaluating combinations of
ALN-HBV02 (VIR-2218) and VIR-3434 and/or PEG-IFN-α in two
hepatitis B virus (HBV) patient populations, with the potential to
evaluate other populations in the future. Initial data are expected
in the first half of 2024.
Additional Business Updates
- Appointed Peter Kellogg to its Board of Directors.
- Published 2022 Corporate Responsibility Report.
- Named Fast Company Top Workplace for Innovators.
- Ranked #6 for gender diversity among executive leadership and
#1 for gender diversity among board of directors in the Eos
Foundation’s Women’s Power Gap study.
Upcoming Events
Alnylam announces today that it intends to present 18-month
results from the APOLLO-B Phase 3 study of patisiran at
ESC-HF 2023, being held May 20-23, 2023 in Prague.
Alnylam also announces today that it intends to present results
from the Randomized Treatment Extension (RTE) of the HELIOS-A Phase
3 study evaluating a biannual dosing regimen of vutrisiran
at the Italian Association for the Study of the Peripheral Nervous
System (ASNP) conference, being held May 25-27, 2023 in Naples.
In addition, in early and mid-2023:
- Alnylam intends to report topline results from the KARDIA-1
Phase 2 study of zilebesiran.
- Alnylam also intends to complete enrollment in the KARDIA-2
Phase 2 study of zilebesiran.
- Vir is conducting multiple trials evaluating the potential for
ALN-HBV02 (VIR-2218) and VIR-3434 to achieve a functional
cure for chronic HBV, which remain ongoing with readouts expected
in 2023.
- Vir also announced that the Phase 2 SOLSTICE trial evaluating
ALN-HBV02 (VIR-2218) and VIR-232 as monotherapy and in
combination for the treatment of people living with chronic
hepatitis D virus, the most aggressive form of viral hepatitis,
remains ongoing with data expected in the second half of 2023.
Financial Results for the Quarter Ended March 31,
2023
Three Months Ended March
31,
(in thousands, except per share
amounts)
2023
2022
Net product revenues
$
276,328
$
186,872
Net revenue from collaborations
$
36,462
$
25,945
Royalty revenue
$
6,500
$
442
GAAP Operating loss
$
(149,807
)
$
(146,732
)
Non-GAAP Operating loss
$
(109,860
)
$
(117,439
)
GAAP Net loss
$
(174,101
)
$
(240,341
)
Non-GAAP Net loss
$
(131,887
)
$
(179,887
)
GAAP Net loss per common share – basic and
diluted
$
(1.40
)
$
(2.00
)
Non-GAAP Net loss per common share – basic
and diluted
$
(1.06
)
$
(1.49
)
Net Product Revenues
Three Months Ended March
31,
Year over Year %
Growth
(in thousands, except percentages)
2023
2022
As Reported
At CER*
ONPATTRO net product revenues
$
102,493
$
137,009
(25
)%
(23
)%
AMVUTTRA net product revenues
101,768
----
N/A
N/A
Total TTR net product revenues
$
204,261
$
137,009
49
%
54
%
GIVLAARI net product revenues
47,906
35,277
36
%
39
%
OXLUMO net product revenues
24,161
14,586
66
%
70
%
Total net product revenues
$
276,328
$
186,872
48
%
52
%
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in the first quarter 2022. CER
is a Non-GAAP measure.
- Net product revenues increased 48% at actual currency during
the first quarter 2023, as compared to the prior year, and 52% at
CER. The increase is primarily related to growth in our TTR product
revenues driven by the launch of AMVUTTRA in the U.S. 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 41% during the first
quarter 2023, as compared to the prior year, primarily due to an
increase in revenue from our collaboration arrangements with
Regeneron attributed to an increase in reimbursable activities
under our research services arrangement in addition to an increase
in revenue recognized associated with licensed programs within the
collaboration.
Operating Expenses
Three Months Ended March
31,
2023 vs. 2022
(in thousands, except percentages)
2023
2022
% Change*
Cost of goods sold
$
41,432
$
23,457
77
%
Cost of goods sold as a percentage of net
product revenues
15.0
%
12.6
%
2.4
%
Cost of collaborations
$
13,437
$
12,170
10
%
GAAP research and development expenses
$
230,569
$
169,893
36
%
Non-GAAP research and development
expenses
$
214,337
$
158,276
35
%
GAAP selling, general and administrative
expenses
$
183,659
$
154,471
19
%
Non-GAAP selling, general and
administrative expenses
$
159,944
$
136,795
17
%
* For dollar values, we calculate the
percentage of change during Q1 2023 compared to Q1 2022. For cost
of goods sold as a percentage of net product revenues, we calculate
the basis point change during Q1 2023 compared to Q1 2022.
Cost of Goods Sold
- Cost of goods sold as a percent of product sales increased
during the first quarter 2023, as compared to the prior year,
primarily due to increased royalties due to third parties driven by
sales of AMVUTTRA partially offset by lower manufacturing costs for
AMVUTTRA compared with ONPATTRO.
Research & Development (R&D) Expenses
- GAAP and non-GAAP R&D expenses increased during the first
quarter 2023, as compared to the prior year, primarily due to
increases in headcount to support our R&D pipeline, development
expenses associated with the APOLLO-B (patisiran), HELIOS-B
(vutrisiran), and KARDIA-1/KARDIA-2 (zilebesiran) clinical studies,
and manufacturing related expenses associated with our pre-clinical
activities.
Selling, General & Administrative (SG&A) Expenses
- GAAP and non-GAAP SG&A expenses increased during the first
quarter 2023, as compared to the prior year, 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 $2.07
billion as of March 31, 2023 compared to $2.19 billion as of
December 31, 2022 with the decrease primarily due to our operating
loss in the first quarter 2023.
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 Guidance1
Full year 2023 financial guidance is reiterated and consists of
the following:
Combined net product revenues for
ONPATTRO, AMVUTTRA, GIVLAARI and OXLUMO1,2
$1,200 million – $1,285
million
Net Product Revenue Growth vs. 2022 at
reported Fx rates1
34% to 44%
Net Product Revenue Growth vs. 2022 at
constant exchange rates*
34% to 44%
Net revenues from collaborations and
royalties
$100 million – $175 million
GAAP R&D and SG&A expenses
$1,790 million – $1,885
million
Non-GAAP R&D and SG&A
expenses3
$1,575 million – $1,650
million
1 Uses December 31, 2022 Fx rates
including: 1 EUR = 1.07 USD and 1 USD = 131 JPY
2 Assumes U.S. sNDA approval of patisiran
for ATTR amyloidosis with cardiomyopathy by the PDUFA date on
October 8, 2023
3 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 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
first quarter 2023 results as well as expectations for the future
via conference call on Thursday, May 4, 2023 at 8:30 am ET. To
access the call, please register online at
https://register.vevent.com/register/BId2c9a28af23d4f119b97d77675d085dc.
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 20 years ago, 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 Twitter at @Alnylam, on LinkedIn, or 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 patisiran and
vutrisiran; actions or advice of regulatory agencies and Alnylam’s
ability to obtain and maintain regulatory approval for its product
candidates, including patisiran and 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 the recent disruptions in the supply of non-human primates;
obtaining, maintaining and protecting intellectual property;
Alnylam’s ability to successfully expand the indication for
ONPATTRO or 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 Novartis, Sanofi,
Regeneron and Vir; the outcome of litigation; the potential impact
of a current government investigation and the risk 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.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
March 31, 2023
March 31, 2022
Statements of Operations
Revenues:
Net product revenues
$
276,328
$
186,872
Net revenues from collaborations
36,462
25,945
Royalty revenue
6,500
442
Total revenues
319,290
213,259
Operating costs and expenses:
Cost of goods sold
41,432
23,457
Cost of collaborations and royalties
13,437
12,170
Research and development
230,569
169,893
Selling, general and administrative
183,659
154,471
Total operating costs and expenses
469,097
359,991
Loss from operations
(149,807
)
(146,732
)
Other (expense) income:
Interest expense
(28,955
)
(42,362
)
Interest income
18,655
1,012
Other expense, net
(12,255
)
(51,274
)
Total other expense, net
(22,555
)
(92,624
)
Loss before income taxes
(172,362
)
(239,356
)
Provision for income taxes
(1,739
)
(985
)
Net loss
$
(174,101
)
$
(240,341
)
Net loss per common share - basic and
diluted
$
(1.40
)
$
(2.00
)
Weighted-average common shares used to
compute basic and diluted net loss per common share
124,111
120,393
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
March 31, 2023
March 31, 2022
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP research and development
$
230,569
$
169,893
Less: Stock-based compensation
expenses
(16,232
)
(11,617
)
Non-GAAP research and development
$
214,337
$
158,276
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP selling, general and
administrative
$
183,659
$
154,471
Less: Stock-based compensation
expenses
(23,715
)
(17,676
)
Non-GAAP selling, general and
administrative
$
159,944
$
136,795
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
$
(149,807
)
$
(146,732
)
Add: Stock-based compensation expenses
39,947
29,293
Non-GAAP operating loss
$
(109,860
)
$
(117,439
)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
$
(174,101
)
$
(240,341
)
Add: Stock-based compensation expenses
39,947
29,293
Add: Realized and unrealized loss on
marketable equity securities
2,267
31,161
Non-GAAP net loss
$
(131,887
)
$
(179,887
)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP net loss per common share - basic and
diluted
$
(1.40
)
$
(2.00
)
Add: Stock-based compensation expenses
0.32
0.24
Add: Realized and unrealized loss on
marketable equity securities
0.02
0.26
Non-GAAP net loss per common share - basic
and diluted
$
(1.06
)
$
(1.49
)
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)
Three Months Ended March 31,
2023
ONPATTRO net product revenue growth, as
reported
(25
)%
Add: Impact of foreign currency
translation
2
ONPATTRO net product revenue growth at
constant currency
(23
)%
AMVUTTRA net product revenue growth, as
reported
N/A
Add: Impact of foreign currency
translation
N/A
AMVUTTRA net product revenue growth at
constant currency
—
%
Total TTR net product revenue growth, as
reported
49
%
Add: Impact of foreign currency
translation
5
Total TTR net product revenue growth at
constant currency
54
%
GIVLAARI net product revenue growth, as
reported
36
%
Add: Impact of foreign currency
translation
3
GIVLAARI net product revenue growth at
constant currency
39
%
OXLUMO net product revenue growth, as
reported
66
%
Add: Impact of foreign currency
translation
4
OXLUMO net product revenue growth at
constant currency
70
%
Total Ultra Rare net product revenue
growth, as reported
45
%
Add: Impact of foreign currency
translation
3
Total Ultra Rare net product revenue
growth at constant currency
48
%
Total net product revenue growth, as
reported
48
%
Add: Impact of foreign currency
translation
4
Total net product revenue growth at
constant currency
52
%
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
(In thousands, except per
share amounts)
(Unaudited)
March 31, 2023
December 31, 2022
ASSETS
Current assets:
Cash and cash equivalents
$
672,245
$
866,394
Marketable debt securities
1,372,574
1,297,890
Marketable equity securities
25,856
28,122
Accounts receivable, net
219,434
237,963
Inventory
131,879
128,962
Prepaid expenses and other current
assets
119,030
132,916
Total current assets
2,541,018
2,692,247
Property, plant and equipment, net
523,032
523,494
Operating lease right-of-use assets
215,049
215,136
Restricted investments
49,388
49,390
Other assets
63,453
66,092
Total assets
$
3,391,940
$
3,546,359
LIABILITIES AND STOCKHOLDERS’
DEFICIT
Current liabilities:
Accounts payable
$
62,237
$
98,094
Accrued expenses
508,599
545,460
Operating lease liability
43,055
41,967
Deferred revenue
35,794
42,105
Liability related to the sale of future
royalties
23,706
40,289
Total current liabilities
673,391
767,915
Operating lease liability, net of current
portion
259,399
261,339
Deferred revenue, net of current
portion
191,257
193,791
Convertible debt
1,017,887
1,016,942
Liability related to the sale of future
royalties, net of current portion
1,285,200
1,252,015
Other liabilities
224,046
212,580
Total liabilities
3,651,180
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 March 31, 2023 and December 31, 2022
—
—
Common stock, $0.01 par value per share,
250,000 shares authorized; 124,241 shares issued and outstanding as
of March 31, 2023; 123,925 shares issued and outstanding as of
December 31, 2022
1,243
1,240
Additional paid-in capital
6,522,091
6,454,540
Accumulated other comprehensive loss
(39,124
)
(44,654
)
Accumulated deficit
(6,743,450
)
(6,569,349
)
Total stockholders’ deficit
(259,240
)
(158,223
)
Total liabilities and stockholders’
deficit
$
3,391,940
$
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230504005271/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340 Josh Brodsky (Investors)
617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024