− Achieved First Quarter 2021 Combined Net
Product Revenues of $136 Million for ONPATTRO®, GIVLAARI®, and
OXLUMO® –
− Advanced Vutrisiran toward Market with
Positive Results from HELIOS-A Phase 3 Study and Submission of New
Drug Application (NDA) with U.S. Food and Drug Administration (FDA)
–
– Launched “Alnylam P5x25” Strategy to Deliver
Transformative Rare and Prevalent Disease Medicines for Patients
Around the World Through Sustainable Innovation and Exceptional
Financial Performance –
– Provides Enrollment Update on HELIOS-B Phase
3 Study of Vutrisiran and Now Expects to Complete Enrollment in
Late 2021, Ahead of Previous Expectations –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the first quarter ended March 31, 2021 and reviewed
recent business highlights.
“We are extremely pleased with the commercial performance of our
marketed products in the first quarter, reflecting strong execution
by our global commercial teams. In particular, we achieved steady
and continued growth for ONPATTRO with approximately 13% quarterly
growth and we observed strong initial demand for OXLUMO in its
first full quarter of launch. We also presented positive results
from the HELIOS-A Phase 3 study of vutrisiran, and with our recent
NDA filing we are one step closer to potentially bringing this
transformative medicine to patients. Given the strong pace of
enrollment in the HELIOS-B Phase 3 study of vutrisiran, we are
announcing today that we expect to complete study enrollment in
late 2021, earlier than previously anticipated,” said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Finally,
early in the quarter we launched our new five-year vision, ‘Alnylam
P5x25,’ marking our strategy for a planned transition to a top five
biotech company in market capitalization by the end of 2025. With
Alnylam P5x25, we aim to deliver transformative medicines for rare
and prevalent diseases to patients around the world, while
advancing a robust and high-yielding pipeline of first and/or
best-in-class clinical programs from our organic product engine,
while delivering strong topline growth and profitability within the
period.”
First Quarter 2021 and Recent Significant Corporate
Highlights
Commercial Performance
ONPATTRO®
- Achieved global net product revenues for the first quarter of
2021 of $102 million, representing 13% growth compared to Q4
2020.
- Attained over 1,500 patients worldwide on commercial ONPATTRO
treatment as of March 31, 2021.
- Secured additional market access with over 30 countries now
selling ONPATTRO through direct reimbursement, named patient sales,
or reimbursed expanded access.
GIVLAARI®
- Achieved global net product revenues for the first quarter of
2021 of $25 million, representing 11% growth compared to Q4
2020.
- Attained approximately 225 patients worldwide on commercial
GIVLAARI treatment as of March 31, 2021.
- Received marketing authorization approval for GIVLAARI in
Switzerland for the treatment of acute hepatic porphyria in adults
and adolescents.
- Continued strong progress toward establishing value-based
agreements (VBAs), with over 10 VBAs finalized to date with
commercial payers and confirmed access for over 98% of covered U.S.
lives.
- Maintained steady progress with market access efforts across
the CEMEA region, with recent launch in Italy, ongoing launch in
Germany, Temporary Authorization for Use (ATU) supply in France,
and named patient sales in other countries.
OXLUMO®
- Achieved global net product revenues for the first quarter of
2021 of $9 million, representing strong initial demand in the first
full quarter of the OXLUMO launch.
- Received over 30 Start Forms in the U.S. and attained
approximately 50 patients on commercial OXLUMO treatment in the
U.S. and EU from launch through March 31, 2021.
- Continued strong progress toward establishing VBAs, with over 5
VBAs finalized to date with commercial payers and confirmed access
for about two thirds of covered U.S. lives.
- Continued progress with market access efforts across the CEMEA
region, with recent launch in Germany, ATU supply in France, and
named patient sales in other countries.
R&D Highlights
Patisiran (the non-proprietary name for ONPATTRO), in
development for the treatment of the cardiomyopathy of both
hereditary and wild-type ATTR amyloidosis
- Continued enrollment in the APOLLO-B Phase 3 study in ATTR
amyloidosis patients with cardiomyopathy and remain on track to
complete enrollment in early 2021.
Vutrisiran, a subcutaneously administered investigational
RNAi therapeutic in development for the treatment of ATTR
amyloidosis
- Filed a NDA with the FDA.
- Presented positive 9-month results from the HELIOS-A Phase 3
study.
- Announces today that due to strong pace of enrollment in the
HELIOS-B Phase 3 study, the Company now expects to complete study
enrollment in late 2021, earlier than previously anticipated.
Lumasiran (the non-proprietary name for OXLUMO), for the
treatment of primary hyperoxaluria type 1 (PH1)
- Continued dosing PH1 patients with advanced renal disease in
the ILLUMINATE-C Phase 3 study, and remain on track to report
topline results in mid-2021.
Inclisiran (the non-proprietary name for Leqvio®)
for the treatment of hypercholesterolemia or mixed dyslipidemia, in
collaboration with Novartis
- Response to U.S. Complete Response Letter to be submitted Q2-Q3
2021.
- ORION-4 readout expected 2026 due to COVID-19.
Fitusiran, in development for the treatment of hemophilia
A or B with and without inhibitors, in collaboration with
Sanofi
- The amended protocol for all ongoing adult and adolescent
fitusiran clinical studies, aimed at further enhancing the
benefit-risk profile, was presented at the 14th Annual Congress of
the European Association for Haemophilia and Allied Disorders
(EAHAD).
Early- and mid-stage RNAi therapeutic pipeline programs
- Continued enrollment and dosing in the Phase 2 study of
cemdisiran monotherapy in IgA nephropathy, and continued
dosing in a Phase 1 study of combination therapy with pozelimab, an
anti-C5 monoclonal antibody, in collaboration with Regeneron.
- Alnylam’s partner Vir Biotechnology continued enrollment and
dosing in a Phase 2 combination trial of ALN-HBV02
(VIR-2218) with pegylated interferon-alpha (PEG-IFN-α).
- Presented updated positive interim results from the Phase 1
study of ALN-AGT, in development for the treatment of
hypertension.
- Continued enrollment and dosing in the Phase 1 study of
ALN-HSD, in development for the treatment of non-alcoholic
steatohepatits (NASH) , in collaboration with Regeneron.
- Continued progress with investigational RNAi therapeutics for
CNS and ocular diseases, including advancement of ALN-APP,
in development for the treatment of autosomal dominant Alzheimer’s
Disease (ADAD) and cerebral amyloid angiopathy (CAA), with an
expected CTA filing in mid-2021, in collaboration with
Regeneron.
Additional Business Updates
- Launched Alnylam P5x25 strategy.
- Issued first ever Corporate Responsibility Summary.
Upcoming Events
In mid-2021, Alnylam intends to:
- Complete enrollment in the APOLLO-B Phase 3 study of
patisiran
- Initiate a study of vutrisiran administered biannually
- File a CTA for ALN-APP
- Achieve marketing authorization for GIVLAARI in Japan
- Achieve marketing authorization for OXLUMO in Brazil
- Report topline results from the ILLUMINATE-C Phase 3 study of
lumasiran
- Initiate KARDIA Phase 2 studies of ALN-AGT
Financial Results for the Quarter Ended March 31,
2021
“We continued to see strong performance from our commercial
products in the first quarter of 2021, and are pleased with the
impact that our three wholly owned products are having on patients
around the world,” said Jeff Poulton, Chief Financial Officer of
Alnylam. “We are reiterating our guidance that we expect to achieve
between $610 million and $660 million in combined net product
revenues across our three wholly owned commercial brands for the
full year 2021. Through strong topline growth, and by continuing to
demonstrate disciplined investment in our operations, we believe
that we are effectively transitioning toward achieving a
self-sustainable financial profile in line with our Alnylam P5x25
strategy.”
Financial highlights
(in thousands, except per share
amounts)
Three Months Ended March
31,
2021
2020
Net product revenues
$
135,769
$
71,938
ONPATTRO net product revenues
$
101,951
$
66,664
GIVLAARI net product revenues
$
24,673
$
5,274
OXLUMO net product revenues
$
9,145
$
—
Net revenue from collaborations
$
41,797
$
27,538
GAAP operating loss
$
(186,254)
$
(210,158)
Non-GAAP operating loss
$
(130,564)
$
(175,580)
GAAP net loss
$
(200,291)
$
(182,221)
Non-GAAP net loss
$
(191,617)
$
(171,754)
GAAP net loss per common share - basic and
diluted
$
(1.71)
$
(1.62)
Non-GAAP net loss per common share - basic
and diluted
$
(1.64)
$
(1.52)
Net Product Revenues
- Combined net product revenues increased 89% compared to the
first quarter of 2020, primarily due to increased ONPATTRO demand
in the U.S. and Europe, the ongoing launch of GIVLAARI, and the
initial launch of OXLUMO in the first quarter of 2021.
Net Revenues from Collaborations
- Net revenues from collaborations increased 52% compared to the
first quarter of 2020, primarily due to an increase in revenue from
our collaborations with Regeneron and Novartis.
First Quarter 2021 Expenses
Three Months Ended March
31,
2021
2020
GAAP research and development expenses
$
185,899
$
169,571
Non-GAAP research and development
expenses
$
161,524
$
153,522
GAAP selling, general and administrative
expenses
$
146,859
$
126,761
Non-GAAP selling, general and
administrative expenses
$
115,544
$
108,232
Research & Development (R&D) Expenses
- GAAP and Non-GAAP R&D expenses increased compared to the
first quarter of 2020 primarily due to increased investment in
clinical activities in our late stage programs, and GAAP R&D
expenses also increased due to higher performance-based stock
expense.
Selling, General & Administrative (SG&A) Expenses
- GAAP and Non-GAAP SG&A expenses increased compared to the
first quarter of 2020 primarily due to increased investment to
support the global growth of our three commercial products,
including the initial launch of OXLUMO, and GAAP SG&A expenses
also increased due to higher performance-based stock expense.
Other Financial Highlights
Total Other (Expense) Income
- Interest expense was $32.5 million in the first quarter 2021
which included $28.2 million associated with the sale of future
royalties and $4.3 million from our long-term debt following the
initial $200 million drawdown of our Blackstone credit facility at
year-end 2020.
- Change in the fair value of our development derivative
liability associated with our R&D funding arrangement with
Blackstone on vutrisiran and ALN-AGT was $22.5 million in the first
quarter 2021.
Cash and Investments
- Cash, cash equivalents and marketable securities were $1.71
billion as of March 31, 2021 compared to $1.87 billion as of
December 31, 2020 with the decrease primarily due to our operating
loss in the first quarter of 2021.
A reconciliation of our GAAP to non-GAAP results for the current
quarter is included in the tables of this press release.
2021 Financial Guidance
Full year 2021 financial guidance is reiterated and consists of
the following:
Combined net product revenues for
ONPATTRO, GIVLAARI and OXLUMO
$610 million - $660 million
Net revenues from collaborations and
royalties
$150 million - $200 million
GAAP R&D and SG&A expenses
$1,335 million - $1,455
million
Non-GAAP R&D and SG&A
expenses*
$1,175 million - $1,275
million
*Excludes $160-$180 million of stock-based
compensation expenses from estimated GAAP R&D and SG&A
expenses.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures,
including expenses adjusted to exclude certain non-cash expenses
and non-recurring gains outside the ordinary course of the
Company’s business. These measures are not in accordance with, or
an alternative to, GAAP, and may be different from non-GAAP
financial measures used by other companies.
The items included in GAAP presentations but excluded for
purposes of determining non-GAAP financial measures for the periods
presented in this press release are stock-based compensation
expenses and unrealized gains 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 unrealized gains
on marketable equity securities because the Company does not
believe these adjustments accurately reflect the performance of the
Company’s ongoing operations for the period in which such gains or
losses are reported, as their sole purpose is to adjust amounts on
the balance sheet.
The Company believes the presentation of non-GAAP financial
measures provides useful information to management and investors
regarding the Company’s financial condition and results of
operations. When GAAP financial measures are viewed in conjunction
with non-GAAP financial measures, investors are provided with a
more meaningful understanding of the Company’s ongoing operating
performance and are better able to compare the Company’s
performance between periods. In addition, these non-GAAP financial
measures are among those indicators the Company uses as a basis for
evaluating performance, allocating resources and planning and
forecasting future periods. Non-GAAP financial measures are not
intended to be considered in isolation or as a substitute for GAAP
financial measures. A reconciliation between GAAP and non-GAAP
measures is provided later in this press release.
Conference Call Information
Management will provide an update on the Company and discuss
first quarter 2021 results as well as expectations for the future
via conference call on Thursday, April 29, 2021 at 8:30 am ET. To
access the call, please dial 877-312-7507 (domestic) or
+1-631-813-4828 (international) five minutes prior to the start
time and refer to conference ID 7986608. A replay of the call will
be available beginning at 11:30 am ET on the day of the call. To
access the replay, please dial 855-859-2056 (domestic) or
+1-404-537-3406 (international) and refer to conference ID
7986608.
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 was 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 blocking the production of
TTR protein before it is made. ONPATTRO blocks the production of
TTR in the liver, reducing its accumulation in the body’s tissues
in order to halt or slow down the progression of the polyneuropathy
associated with the disease. For more information about ONPATTRO,
visit ONPATTRO.com.
ONPATTRO Important Safety Information
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with ONPATTRO® (patisiran). In a controlled clinical study,
19% of ONPATTRO-treated patients experienced IRRs, compared to 9%
of placebo-treated patients. The most common symptoms of IRRs with
ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea,
and headache.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, acetaminophen, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
ONPATTRO infusion. Monitor patients during the infusion for signs
and symptoms of IRRs. If an IRR occurs, consider slowing or
interrupting the infusion and instituting medical management as
clinically indicated. If the infusion is interrupted, consider
resuming at a slower infusion rate only if symptoms have resolved.
In the case of a serious or life-threatening IRR, the infusion
should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended
Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A
levels. Supplementation at the recommended daily allowance (RDA) of
vitamin A is advised for patients taking ONPATTRO. Higher doses
than the RDA should not be given to try to achieve normal serum
vitamin A levels during treatment with ONPATTRO, as serum levels do
not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they
develop ocular symptoms suggestive of vitamin A deficiency (e.g.
night blindness).
Adverse Reactions
The most common adverse reactions that occurred in patients
treated with ONPATTRO were upper respiratory tract infections (29%)
and infusion-related reactions (19%).
For additional information about ONPATTRO, please see the full
Prescribing Information.
About GIVLAARI® (givosiran)
GIVLAARI 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 aminolevulinic acid synthase 1 (ALAS1) messenger
RNA (mRNA), leading to reduction of toxins associated with attacks
and other disease manifestations of AHP. For more information about
GIVLAARI, visit GIVLAARI.com.
GIVLAARI Important Safety Information
Contraindications
GIVLAARI is contraindicated in patients with known severe
hypersensitivity to givosiran. Reactions have included
anaphylaxis.
Anaphylactic Reaction
Anaphylaxis has occurred with GIVLAARI treatment (<1% of
patients in clinical trials). Ensure that medical support is
available to appropriately manage anaphylactic reactions when
administering GIVLAARI. Monitor for signs and symptoms of
anaphylaxis. If anaphylaxis occurs, immediately discontinue
administration of GIVLAARI and institute appropriate medical
treatment.
Hepatic Toxicity
Transaminase elevations (ALT) of at least 3 times the upper
limit of normal (ULN) were observed in 15% of patients receiving
GIVLAARI in the placebo-controlled trial. Transaminase elevations
primarily occurred between 3 to 5 months following initiation of
treatment.
Measure liver function tests prior to initiating treatment with
GIVLAARI, repeat every month during the first 6 months of
treatment, and as clinically indicated thereafter. Interrupt or
discontinue treatment with GIVLAARI for severe or clinically
significant transaminase elevations. In patients who have dose
interruption and subsequent improvement, reduce the dose to 1.25
mg/kg once monthly. The dose may be increased to the recommended
dose of 2.5 mg/kg once monthly if there is no recurrence of severe
or clinically significant transaminase elevations at the 1.25 mg/kg
dose.
Renal Toxicity
Increases in serum creatinine levels and decreases in estimated
glomerular filtration rate (eGFR) have been reported during
treatment with GIVLAARI. In the placebo-controlled study, 15% of
patients receiving GIVLAARI experienced a renally-related adverse
reaction. The median increase in creatinine at Month 3 was 0.07
mg/dL. Monitor renal function during treatment with GIVLAARI as
clinically indicated.
Injection Site Reactions
Injection site reactions were reported in 25% of patients
receiving GIVLAARI in the placebo-controlled trial. Symptoms
included erythema, pain, pruritus, rash, discoloration, or swelling
around the injection site. One (2%) patient experienced a single,
transient, recall reaction of erythema at a prior injection site
with a subsequent dose administration.
Drug Interactions
Concomitant use of GIVLAARI increases the concentration of
CYP1A2 or CYP2D6 substrates, which may increase adverse reactions
of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2
or CYP2D6 substrates for which minimal concentration changes may
lead to serious or life-threatening toxicities. If concomitant use
is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in
accordance with approved product labeling.
Adverse Reactions
The most common adverse reactions that occurred in patients
receiving GIVLAARI were nausea (27%) and injection site reactions
(25%).
For additional information about GIVLAARI, please see full
Prescribing Information.
About OXLUMO® (lumasiran)
OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1
(HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to
lower urinary oxalate levels in pediatric and adult patients. HAO1
encodes glycolate oxidase (GO), an enzyme upstream of the
disease-causing defect in PH1. OXLUMO works by degrading HAO1
messenger RNA and reducing the synthesis of GO, which inhibits
hepatic production of oxalate – the toxic metabolite responsible
for the clinical manifestations of PH1. 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. Injection site reactions (ISRs) were
the most common drug-related adverse reaction. In the ILLUMINATE-B
pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety
profile consistent to that observed in ILLUMINATE-A. OXLUMO
utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc
conjugate technology designed to increase potency and durability.
OXLUMO is administered via subcutaneous injection once monthly for
three months, then once quarterly thereafter 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, visit
OXLUMO.com.
OXLUMO Important Safety Information
Adverse Reactions
The most common adverse reaction that occurred in patients
treated with OXLUMO was injection site reaction (38%). Symptoms
included erythema, pain, pruritus, and swelling.
Pregnancy and Lactation
No data are available on the use of OXLUMO in pregnant women. No
data are available on the presence of OXLUMO in human milk or its
effects on breastfed infants or milk production. Consider the
developmental and health benefits of breastfeeding along with the
mother’s clinical need for OXLUMO and any potential adverse effects
on the breastfed child from OXLUMO or the underlying maternal
condition.
For additional information about OXLUMO, please see the full
Prescribing Information.
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 (Nasdaq: ALNY) is leading 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 genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning
science, RNAi therapeutics represent a powerful, clinically
validated approach for the treatment of a wide range of severe and
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust RNAi therapeutics platform. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), GIVLAARI®
(givosiran), and OXLUMO® (lumasiran), as well as Leqvio®
(inclisiran), which is being developed and commercialized by
Alnylam’s partner Novartis. Alnylam has a deep pipeline of
investigational medicines, including six 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
Various statements in this release concerning Alnylam’s
expectations, plans and prospects, including, without limitation,
its plans for additional global regulatory filings and the
continuing product launches of its approved products and
expectations regarding reimbursement for those products in various
territories, expectations regarding FDA review of inclisiran,
conditions at the third party manufacturer where inclisiran is
manufactured and the expected timing of resubmission of the
inclisiran NDA by Novartis, the achievement of additional pipeline
milestones, including relating to ongoing clinical studies of
patisiran and vutrisiran and the expected timing for completion of
study enrollment in the APOLLO-B and HELIOS-B Phase 3 studies,
lumasiran and the timing of topline results from the ILLUMINATE-C
Phase 3 study, the expected timing for filing a CTA for ALN-APP,
expectations relating to continued revenue growth for its approved
products and the expected range of net product revenues and net
revenues from collaborations and royalties for 2021, the expected
range of aggregate annual GAAP and non-GAAP R&D and SG&A
expenses, and expectations regarding Alnylam’s ability to achieve
its “Alnylam P5x25” strategy, constitute forward-looking statements
for the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. 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 and the effectiveness or timeliness of
Alnylam’s efforts to mitigate the impact of the pandemic; 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
its product candidates; actions or advice of regulatory agencies
and Alnylam’s ability to obtain and maintain regulatory approval
for its product candidates, as well as favorable pricing and
reimbursement; successfully launching, marketing and selling its
approved products globally; delays, interruptions or failures in
the manufacture and supply of its product candidates or its
marketed products; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
indication for ONPATTRO 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,
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 most recent Annual Report on Form 10-K filed with the
Securities and Exchange Commission (SEC) 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. 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
March 31, 2021
March 31, 2020
Statements of Operations
Revenues:
Net product revenues
$
135,769
$
71,938
Net revenues from collaborations
41,797
27,538
Total revenues
177,566
99,476
Operating costs and expenses:
Cost of goods sold
23,023
13,302
Cost of collaborations
8,039
—
Research and development
185,899
169,571
Selling, general and administrative
146,859
126,761
Total operating costs and expenses
363,820
309,634
Loss from operations
(186,254)
(210,158)
Other (expense) income:
Interest expense
(32,515)
—
Interest income
450
5,480
Other income, net
19,044
23,032
Total other (expense) income
(13,021)
28,512
Loss before income taxes
(199,275)
(181,646)
Provision for income taxes
(1,016)
(575)
Net loss
$
(200,291)
$
(182,221)
Net loss per common share - basic and
diluted
$
(1.71)
$
(1.62)
Weighted-average common shares used to
compute basic and diluted net loss per common share
117,080
112,748
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
Three Months Ended
March 31, 2021
March 31, 2020
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP Research and development
$
185,899
$
169,571
Less: Stock-based compensation
expenses
(24,375)
(16,049)
Non-GAAP Research and development
$
161,524
$
153,522
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP Selling, general and
administrative
$
146,859
$
126,761
Less: Stock-based compensation
expenses
(31,315)
(18,529)
Non-GAAP Selling, general and
administrative
$
115,544
$
108,232
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
$
(186,254)
$
(210,158)
Add: Stock-based compensation expenses
55,690
34,578
Non-GAAP operating loss
$
(130,564)
$
(175,580)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
$
(200,291)
$
(182,221)
Add: Stock-based compensation expenses
55,690
34,578
Less: Unrealized gain on marketable equity
securities
(47,016)
(24,111)
Non-GAAP net loss
$
(191,617)
$
(171,754)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP net loss per common share - basic and
diluted
$
(1.71)
$
(1.62)
Add: Stock-based compensation expenses
0.48
0.31
Less: Unrealized gain on marketable equity
securities
(0.40)
(0.21)
Non-GAAP net loss per common share - basic
and diluted
$
(1.64)
$
(1.52)
Please note that the figures
presented above may not sum exactly due to rounding
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
(In thousands, except share
amounts)
(Unaudited)
March 31, 2021
December 31, 2020
Cash, cash equivalents, and marketable
debt and equity securities
$
1,709,537
$
1,874,395
Restricted investments
40,725
40,725
Accounts receivable, net
110,626
102,413
Inventory
91,040
92,302
Prepaid expenses and other assets
101,556
90,712
Property, plant and equipment, net
464,572
465,029
Operating lease right-of-use lease
assets
237,213
241,485
Receivable related to the sale of future
royalties
500,000
500,000
Total assets
$
3,255,269
$
3,407,061
Accounts payable, accrued expenses and
other liabilities
$
386,369
$
445,783
Total deferred revenue
324,463
352,301
Operating lease liability
326,932
329,911
Liability related to the sale of future
royalties
1,099,725
1,071,541
Long-term debt
191,590
191,278
Total stockholders’ equity (117.3 million
shares issued and outstanding at March 31, 2021; 116.4 million
shares issued and outstanding at December 31, 2020)
926,190
1,016,247
Total liabilities and stockholders'
equity
$
3,255,269
$
3,407,061
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, 2020.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210429005161/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340 Josh Brodsky (Investors)
617-551-8276
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