− Achieved Second Quarter 2023 Global Net
Product Revenues of $306 Million, Representing 43% Year-Over-Year
Growth Compared to Q2 2022 –
−Submitted 18-Month APOLLO-B Data to the U.S.
Food and Drug Administration as Amendment to Supplemental New Drug
Application for Patisiran –
−Presented Updated Positive Interim Results
from Phase 1 Study of ALN-APP in Patients with Early-Onset
Alzheimer’s Disease –
−Entered into Global Strategic Collaboration
with Roche for Co-Development and Co-Commercialization of
Zilebesiran –
−U.S. Attorney’s Office for District of
Massachusetts Concluded and Closed Investigation Regarding
Marketing and Promotion of ONPATTRO, with no Action Taken –
−Akshay Vaishnaw, M.D., Ph.D., Named Alnylam’s
First Chief Innovation Officer –
− 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 second quarter ended June 30, 2023 and reviewed
recent business highlights.
“The second quarter of 2023 was a very productive one at
Alnylam, with commercial execution delivering 43% year-over-year
growth in net product revenues, supported by the strong ongoing
launch of AMVUTTRA,” said Yvonne Greenstreet, MBChB, Chief
Executive Officer of Alnylam. “We also had some very important data
readouts in the quarter and recent period, including updated
interim results from the Phase 1 trial of ALN-APP in patients with
early onset Alzheimer’s disease, as well as positive 18-month
results from the APOLLO-B Phase 3 study of patisiran, that we
believe reaffirm the potential of patisiran in ATTR amyloidosis
with cardiomyopathy. Furthermore, we entered into an exciting new
partnership with Roche for the development and commercialization of
zilebesiran. This progress underscores our commitment to executing
across all areas of the business in order to meet our Alnylam P5x25
goals of becoming a top-tier biotech company delivering sustained
innovation and exceptional financial results.”
Second 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 second quarter of $91 million and $132 million,
respectively, representing 46% total TTR reported year-over-year
growth compared to Q2 2022. The acceleration of growth in the U.S.
market is particularly noteworthy, as Q2 2023 growth was 72%
compared to Q2 2022, marking the fourth consecutive quarter of
greater than 70% total TTR growth in the U.S. since the launch of
AMVUTTRA in mid-2022.
- Attained over 3,490 hATTR amyloidosis patients with
polyneuropathy worldwide on commercial treatment with ONPATTRO or
AMVUTTRA as of June 30, 2023.
Total Ultra-Rare: GIVLAARI® (givosiran) & OXLUMO®
(lumasiran)
- Achieved global net product revenues for GIVLAARI and OXLUMO
for the second quarter of $58 million and $24 million,
respectively, representing 37% total Ultra-Rare reported
year-over-year growth compared to Q2 2022.
- Attained over 570 patients worldwide on commercial GIVLAARI
treatment as of June 30, 2023.
- Attained over 350 patients worldwide on commercial OXLUMO
treatment as of June 30, 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.
- Novartis announced that the FDA approved a label update for
Leqvio to enable earlier use in patients with elevated LDL-C who
have an increased risk of heart disease, as an adjunct to diet and
statin therapy.
R&D Highlights
Presented new results from an interim analysis of data from the
open-label extension (OLE) period of the APOLLO-B Phase 3 study of
patisiran, demonstrating continued evidence of sustained
benefit across measures of functional capacity and health status
and quality of life, as well as cardiac stress and injury.
Patisiran demonstrated a safety profile consistent with that
observed in the 12-month double-blind period, with no new safety
findings.
- Submitted these 18-month data from the APOLLO-B Phase 3 study
to the U.S. FDA as part of the sNDA review for patisiran for the
treatment of the cardiomyopathy of ATTR amyloidosis.
- Announced that the U.S. FDA has set a date of September 13,
2023 for the meeting of the Cardiovascular and Renal Drugs Advisory
Committee to review the sNDA for patisiran. As previously
announced, the FDA has set an action date of October 8, 2023 under
the Prescription Drug User Fee Act.
The Company announces today that a U.S. Expanded Access Program
(EAP) for patisiran that was initiated in August 2022 has fully
enrolled, with 200 patients across 20 centers in less than a year.
The patisiran EAP aims to provide access to patisiran for patients
with ATTR amyloidosis with cardiomyopathy who have had an
inadequate response to or cannot tolerate currently available
treatment.
Published results from Phase 1 study of zilebesiran in
the New England Journal of Medicine, showing that, compared to
placebo, zilebesiran was associated with dose-dependent reductions
in serum AGT, achieving tonic blood pressure control with
consistent and durable blood pressure reduction throughout a
24-hour period, sustained up to six months after single doses of
≥200 mg of zilebesiran. Zilebesiran also demonstrated an acceptable
safety profile supporting continued clinical development.
Completed enrollment in the KARDIA-2 Phase 2 study, evaluating
the safety and efficacy of zilebesiran in patients with
uncontrolled hypertension when added on top of another
antihypertensive medication.
- Topline results are expected in early 2024.
Reported updated positive interim results for the ongoing single
ascending dose (SAD) portion of the Phase 1 study of ALN-APP
in patients with early-onset Alzheimer’s disease (EOAD) at the 2023
Alzheimer’s Association International Conference (AAIC).
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:
- Presented nine-month results from the randomized treatment
extension period of the HELIOS-A study of vutrisiran in
patients with they polyneuropathy of hATTR amyloidosis at the
Italian Association for the Study of the Peripheral Nervous
System
- Presented findings from a Phase 1 study of ALN-HSD in
healthy adults and patients with nonalcoholic steatohepatitis at
the European Association for the Study of the Liver (EASL) Congress
2023.
Additional Business Updates
- Announced today that the U.S. Attorney’s Office for the
District of Massachusetts has concluded and closed its
investigation regarding the marketing and promotion of ONPATTRO,
with no action being taken against the company.
- Announced today that Akshay Vaishnaw, M.D., Ph.D., Alnylam’s
President and key scientific leader, will be transitioning to a new
role within the organization, serving as Alnylam’s first Chief
Innovation Officer. In this new role Akshay will become the
Company’s key innovation leader, focused on the future of its
R&D engine.
- Entered into a global strategic collaboration with Roche for
the co-development and co-commercialization of
zilebesiran.
- As announced earlier today, Alnylam has entered into an
exclusive worldwide license agreement with Agios Pharmaceuticals to
develop and commercialize a novel preclinical siRNA targeting
TMPRSS6 as a potential disease-modifying treatment for
patients with polycythemia vera and related iron-overload
disorders.
- Published 2022 Corporate Responsibility Report.
Upcoming Events
In mid- and late 2023:
- Alnylam intends to report topline results from the KARDIA-1
Phase 2 study of zilebesiran.
- 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 June 30, 2023
Three Months Ended June
30,
(in thousands, except per share
amounts)
2023
2022
Net product revenues
$
305,705
$
213,515
Net revenue from collaborations
$
5,844
$
9,025
Royalty revenue
$
7,205
$
2,278
GAAP Operating loss
$
(229,831
)
$
(191,686
)
Non-GAAP Operating loss*
$
(154,029
)
$
(161,215
)
GAAP Net loss
$
(276,024
)
$
(277,402
)
Non-GAAP Net loss*
$
(201,622
)
$
(245,834
)
GAAP Net loss per common share - basic and
diluted
$
(2.21
)
$
(2.29
)
Non-GAAP Net loss per common share - basic
and diluted*
$
(1.62
)
$
(2.03
)
* For an explanation of our use of
non-GAAP financial measures see page 8 and for a reconciliation of
each non-GAAP financial measure to the most comparable GAAP
measures, see page 15.
Net Product Revenues
Three Months Ended
June 30,
Year over Year %
Growth
(in thousands, except percentages)
2023
2022
As Reported
At CER*
ONPATTRO net product revenues
$
91,458
$
153,428
(40
)%
(40
)%
AMVUTTRA net product revenues
132,136
—
N/A
N/A
Total TTR net product revenues
$
223,594
$
153,428
46
%
47
%
GIVLAARI net product revenues
57,899
45,150
28
%
28
%
OXLUMO net product revenues
24,212
14,937
62
%
62
%
Total net product revenues
$
305,705
$
213,515
43
%
44
%
* CER = Constant Exchange Rate,
representing growth calculated as if the exchange rates had
remained unchanged from those used in the second quarter 2022. CER
is a Non-GAAP measure.
- Net product revenues increased 43% at actual currency during
the second quarter 2023, as compared to the prior year, and 44% 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.
Net Revenues from Collaborations
- Net revenues from collaborations decreased 35% during the
second quarter 2023, as compared to the prior year, primarily due
to operating variability, such as the level of work reimbursed, in
our collaboration with Regeneron.
Operating Expenses
Three Months Ended June
30,
2023 vs 2022
(in thousands, except percentages)
2023
2022
% Change*
Cost of goods sold
$
75,336
$
34,038
121
%
Cost of goods sold as a percentage of net
product revenues
24.6
%
15.9
%
8.7
%
Cost of collaborations
$
10,034
$
6,770
48
%
GAAP research and development expenses
$
248,526
$
205,712
21
%
Non-GAAP research and development
expenses
$
215,725
$
195,074
11
%
GAAP selling, general and administrative
expenses
$
214,689
$
169,984
26
%
Non-GAAP selling, general and
administrative expenses
$
171,688
$
150,151
14
%
*For dollar values, we calculate the
percentage of change during Q2 2023 compared to Q2 2022. For cost
of goods sold as a percentage of net product revenues, we calculate
the basis point change during Q2 2023 compared to Q2 2022.
Cost of Goods Sold
- Cost of goods sold as a percent of product sales increased
during the second quarter 2023, as compared to the prior year,
primarily due to cancelling manufacturing commitments for ONPATTRO
and other adjustments to inventory as ongoing patients continue to
switch to AMVUTTRA. Costs of goods sold as a percentage of net
product revenues also increased as royalties owed to third parties
increased driven by the growth of AMVUTTRA following its launch in
Q3 2022. These increases were 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 second
quarter 2023, as compared to the prior year, primarily due to
increased development expenses associated with our KARDIA-1 /
KARDIA-2 (zilebesiran) clinical studies and increased compensation
and related expenses as a result of increased headcount to support
our R&D pipeline. GAAP R&D expenses further increased due
to increased stock-based compensation expense primarily related to
certain performance-based awards.
Selling, General & Administrative (SG&A) Expenses
- GAAP and non-GAAP SG&A expenses increased during the second
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. GAAP SG&A
expenses further increased due to increased stock-based
compensation expense primarily related to certain performance-based
awards.
Other Financial Highlights
- Cash, cash equivalents and marketable securities were $2.06
billion as of June 30, 2023 compared to $2.19 billion as of
December 31, 2022 with the decrease primarily due to our operating
loss in the six months ended June 30, 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 Guidance
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 non-GAAP financial measures we present include Non-GAAP
Operating loss, Non-GAAP Net loss, Non-GAAP Net loss per common
share – basic and 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
second quarter 2023 results as well as expectations for the future
via conference call on Thursday, August 3, 2023 at 8:30 am ET. To
access the call, please register online at
https://register.vevent.com/register/BI8e6d8bfa374f41278e284759aa762e9e.
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, 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, 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 Roche, Novartis,
Sanofi, Regeneron and Vir; the outcome of litigation; the potential
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.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
Six Months Ended
June 30, 2023
June 30, 2022
June 30, 2023
June 30, 2022
Statements of Operations
Revenues:
Net product revenues
$
305,705
$
213,515
$
582,033
$
400,387
Net revenues from collaborations
5,844
9,025
42,306
34,970
Royalty revenue
7,205
2,278
13,705
2,720
Total revenues
318,754
224,818
638,044
438,077
Operating costs and expenses:
Cost of goods sold
75,336
34,038
116,768
57,495
Cost of collaborations and royalties
10,034
6,770
23,471
18,940
Research and development
248,526
205,712
479,095
375,605
Selling, general and administrative
214,689
169,984
398,348
324,455
Total operating costs and expenses
548,585
416,504
1,017,682
776,495
Loss from operations
(229,831
)
(191,686
)
(379,638
)
(338,418
)
Other (expense) income:
Interest expense
(30,035
)
(42,609
)
(58,990
)
(84,971
)
Interest income
21,075
1,899
39,730
2,911
Other expense, net
(35,418
)
(42,277
)
(47,673
)
(93,551
)
Total other expense, net
(44,378
)
(82,987
)
(66,933
)
(175,611
)
Loss before income taxes
(274,209
)
(274,673
)
(446,571
)
(514,029
)
Provision for income taxes
(1,815
)
(2,729
)
(3,554
)
(3,714
)
Net loss
$
(276,024
)
$
(277,402
)
$
(450,125
)
$
(517,743
)
Net loss per common share - basic and
diluted
$
(2.21
)
$
(2.29
)
$
(3.62
)
$
(4.29
)
Weighted-average common shares used to
compute basic and diluted net loss per common share
124,659
120,896
124,387
120,646
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
(In thousands, except per
share amounts)
(Unaudited)
June 30, 2023
December 31, 2022
ASSETS
Current assets:
Cash and cash equivalents
$
657,800
$
866,394
Marketable debt securities
1,372,451
1,297,890
Marketable equity securities
27,256
28,122
Accounts receivable, net
220,635
237,963
Inventory
100,453
128,962
Prepaid expenses and other current
assets
145,452
132,916
Total current assets
2,524,047
2,692,247
Property, plant and equipment, net
527,474
523,494
Operating lease right-of-use assets
208,801
215,136
Restricted investments
49,388
49,390
Other assets
92,686
66,092
Total assets
$
3,402,396
$
3,546,359
LIABILITIES AND STOCKHOLDERS’
DEFICIT
Current liabilities:
Accounts payable
$
59,746
$
98,094
Accrued expenses
598,530
545,460
Operating lease liability
42,074
41,967
Deferred revenue
35,377
42,105
Liability related to the sale of future
royalties
33,650
40,289
Total current liabilities
769,377
767,915
Operating lease liability, net of current
portion
253,416
261,339
Deferred revenue, net of current
portion
213,391
193,791
Convertible debt
1,018,843
1,016,942
Liability related to the sale of future
royalties, net of current portion
1,298,446
1,252,015
Other liabilities
257,054
212,580
Total liabilities
3,810,527
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
1,250
1,240
Additional paid-in capital
6,647,173
6,454,540
Accumulated other comprehensive loss
(37,080
)
(44,654
)
Accumulated deficit
(7,019,474
)
(6,569,349
)
Total stockholders’ deficit
(408,131
)
(158,223
)
Total liabilities and stockholders’
deficit
$
3,402,396
$
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
Six Months Ended
June 30, 2023
June 30, 2022
June 30, 2023
June 30, 2022
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP research and development
$
248,526
$
205,712
$
479,095
$
375,605
Less: Stock-based compensation
expenses
(32,801
)
(10,638
)
(49,033
)
(22,255
)
Non-GAAP research and development
$
215,725
$
195,074
$
430,062
$
353,350
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP selling, general and
administrative
$
214,689
$
169,984
$
398,348
$
324,455
Less: Stock-based compensation
expenses
(43,001
)
(19,833
)
(66,716
)
(37,509
)
Non-GAAP selling, general and
administrative
$
171,688
$
150,151
$
331,632
$
286,946
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
$
(229,831
)
$
(191,686
)
$
(379,638
)
$
(338,418
)
Add: Stock-based compensation expenses
75,802
30,471
115,749
59,764
Non-GAAP operating loss
$
(154,029
)
$
(161,215
)
$
(263,889
)
$
(278,654
)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
$
(276,024
)
$
(277,402
)
$
(450,125
)
$
(517,743
)
Add: Stock-based compensation expenses
75,802
30,471
115,749
59,764
(Less) Add: Realized and unrealized (gain)
loss on marketable equity securities
(1,400
)
1,097
867
32,258
Non-GAAP net loss
$
(201,622
)
$
(245,834
)
$
(333,509
)
$
(425,721
)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP net loss per common share - basic and
diluted
$
(2.21
)
$
(2.29
)
$
(3.62
)
$
(4.29
)
Add: Stock-based compensation expenses
0.61
0.25
0.93
0.50
(Less) Add: Realized and unrealized (gain)
loss on marketable equity securities
(0.01
)
0.01
0.01
0.27
Non-GAAP net loss per common share - basic
and diluted
$
(1.62
)
$
(2.03
)
$
(2.68
)
$
(3.53
)
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)
June 30, 2023
Three Months Ended
Six Months Ended
ONPATTRO net product revenue growth, as
reported
(40) %
(33) %
Add: Impact of foreign currency
translation
—
1
ONPATTRO net product revenue growth at
constant currency
(40) %
(32) %
AMVUTTRA net product revenue growth, as
reported
N/A
N/A
Add: Impact of foreign currency
translation
N/A
N/A
AMVUTTRA net product revenue growth at
constant currency
— %
— %
Total TTR net product revenue growth, as
reported
46 %
47 %
Add: Impact of foreign currency
translation
1
3
Total TTR net product revenue growth at
constant currency
47 %
50 %
GIVLAARI net product revenue growth, as
reported
28 %
32 %
Add: Impact of foreign currency
translation
—
1
GIVLAARI net product revenue growth at
constant currency
28 %
33 %
OXLUMO net product revenue growth, as
reported
62 %
64 %
Add: Impact of foreign currency
translation
—
2
OXLUMO net product revenue growth at
constant currency
62 %
66 %
Total net product revenue growth, as
reported
43 %
45 %
Add: Impact of foreign currency
translation
1
3
Total net product revenue growth at
constant currency
44 %
48 %
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230803655131/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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