− Achieved First Quarter 2020 ONPATTRO® Net
Product Revenues of $66.7 Million with More Than 950 Patients on
Commercial Product Worldwide –
− Achieved First Quarter 2020 GIVLAARI® Net
Product Revenues of $5.3 Million with More Than 50 Patients on
Commercial Product in the U.S. –
– Completed New Drug Application and Filed
Marketing Authorisation Application for Lumasiran, Advancing
Another Alnylam Investigational RNAi Therapeutic toward Market
–
− Announces New Positive Initial Topline
Results from Ongoing Phase 1 Study of ALN-AGT in Hypertension with
Over 90 Percent Knockdown of Angiotensinogen and an Over 10 mmHg
Mean Reduction of 24-hour Systolic Blood Pressure Relative to
Placebo –
– Secured Bridge Enabling Self-Sustainable
Financial Profile without Need for Future Equity Offerings via $2
Billion Strategic Financing Collaboration with Blackstone –
– Lowers 2020 Guidance Range for ONPATTRO
Revenue from $285-$315 Million to $270-$300 Million due to COVID-19
Pandemic; Lowers Operating Expense Guidance –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the first quarter 2020 and reviewed recent business
highlights.
“The first quarter of 2020 was an unprecedented period, as the
world confronted the beginnings of an ongoing public health crisis
with the COVID-19 pandemic. It’s also a moment in time that
underscores how particularly proud we are at Alnylam to be part of
an industry advancing science and innovation to address the
coronavirus crisis at hand, while also maintaining our focus on
continuing to bring important medicines to patients in need. To
that end, we are very pleased with our first full quarter as a
multi-product, global commercial company where we delivered
continued and steady worldwide growth of ONPATTRO and impressive
early demand for GIVLAARI in the U.S.,” said John Maraganore,
Ph.D., Chief Executive Officer of Alnylam. “In addition, we also
continued to drive progress across our late-stage pipeline,
including submission of NDA and MAA filings for lumasiran,
completion of enrollment in the ILLUMINATE-B and HELIOS-A Phase 3
studies of lumasiran and vutrisiran, respectively, and continued
enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of
patisiran and vutrisiran, respectively. We’re also thrilled to
highlight today the achievement of initial human proof of concept
for ALN-AGT in development for the treatment of hypertension, an
area of prevalent unmet need that cries out for innovation.”
Dr. Maraganore continued: “Notwithstanding these accomplishments
and our strong underlying confidence for Alnylam in 2020 and
beyond, the trajectory of the COVID-19 pandemic is dynamic and
remains uncertain in its scope, timeline, and impact to healthcare
systems. While we are implementing what we believe are appropriate
mitigation steps across our operations, we are lowering our 2020
revenue guidance for ONPATTRO by 5 percent and taking thoughtful
steps to reduce our expense profile. Notwithstanding the challenges
and uncertainties caused by the pandemic, we remain confident that
we are on track to exceed our Alnylam 2020 strategic plan announced
in 2015, building a multi-product, global biopharma company with a
deep clinical pipeline to fuel future growth and a robust, organic
product engine for sustainable innovation, a profile rarely
achieved in our industry.”
First Quarter 2020 and Recent Significant Corporate
Highlights
Commercial Performance
ONPATTRO®
- Achieved net product revenues for the first quarter of 2020 of
$66.7 million.
- Attained over 950 patients worldwide on commercial ONPATTRO
treatment as of March 31, 2020.
- Received marketing authorization approval for ONPATTRO in
Brazil for the treatment of hereditary transthyretin-mediated
(hATTR) amyloidosis in adults with stage 1 or stage 2
polyneuropathy, with patients now on commercial product.
- Continued progress with market access efforts across the
Canada, Europe, Middle East, and Africa (CEMEA) region, with recent
launches in Italy, Sweden, Israel, Turkey, and Spain.
- Maintained momentum in Asia with a strong launch in Japan and
submission of a New Drug Application (NDA) in Taiwan.
- In the U.S., Alnylam has now completed definitive value-based
agreements (VBAs) covering ONPATTRO with over 15 commercial payers,
including 9 of the top 10 commercial payers, with signed VBAs now
covering over 135 million U.S. lives in the aggregate.
GIVLAARI®
- Achieved net product revenues for the first quarter of 2020 of
$5.3 million, representing strong initial demand in the first full
quarter of the GIVLAARI launch in the U.S.
- Received over 60 Start Forms in the U.S. and attained over 50
patients on commercial GIVLAARI treatment from launch through March
31, 2020.
- Made significant progress toward establishing VBAs, including a
Prevalence-Based Adjustment feature, with multiple ongoing
discussions with U.S. payers; the Company announces today
finalization of the first VBA for GIVLAARI.
- Continued progress with market access efforts across the CEMEA
region, with initial launch underway in Germany.
- Continued work with physicians in multiple regions to provide
pre-approval access to GIVLAARI in an Expanded Access Program (EAP)
in accordance with local requirements.
R&D Highlights
- Advanced the development of patisiran (the
non-proprietary name for ONPATTRO) for the potential 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.
- The Company announces today that due to the impact of the
COVID-19 pandemic, enrollment delays in APOLLO-B will result in a
shift in the enrollment completion date from late 2020 into
2021.
- Received approval of GIVLAARI (givosiran) in the
European Union for the treatment of acute hepatic porphyria (AHP)
in adults and adolescents aged 12 years and older, with patients
now on commercial product.
- Advanced lumasiran, an investigational RNAi therapeutic
in development for the treatment of primary hyperoxaluria type 1
(PH1).
- Initiated and completed the rolling submission of an NDA to the
Food and Drug Administration (FDA) and submitted a Marketing
Authorisation Application (MAA) to the European Medicines Agency
(EMA), completing the filings in less than four months after
announcing topline results from the ILLUMINATE-A Phase 3
study.
- Completed enrollment in the ILLUMINATE-B Phase 3 study of
lumasiran in PH1 patients less than six years of age with preserved
renal function, and remains on track to report topline results in
mid-2020.
- Continued enrollment in the ILLUMINATE-C Phase 3 study of
lumasiran for the treatment of advanced PH1 in patients of all
ages.
- Advanced vutrisiran, a subcutaneously administered
investigational RNAi therapeutic in development for the treatment
of ATTR amyloidosis.
- Completed enrollment in the HELIOS-A Phase 3 study in patients
with hereditary ATTR amyloidosis with polyneuropathy, and remains
on track to report topline results in early 2021.
- Received Fast Track Designation from the FDA for the treatment
of the polyneuropathy of hATTR amyloidosis.
- Continued enrollment in the HELIOS-B Phase 3 study in patients
with hereditary and wild-type ATTR amyloidosis with
cardiomyopathy.
- Inclisiran, potentially the first and only siRNA
cholesterol-lowering treatment, continues to advance under
Alnylam’s partner, Novartis.
- Acceptance of NDA and MAA by FDA and EMA, respectively.
- Findings published in The New England Journal of Medicine from
the completed ORION Phase 3 pivotal trials showing durable and
potent LDL-C reduction achieved with inclisiran, with a safety
profile similar to placebo.
- New analysis of pooled data from the completed ORION Phase 3
clinical trials presented at the American College of Cardiology’s
Annual Scientific Session.
- Alnylam’s partner, Sanofi, continues enrollment in the ATLAS
Phase 3 program for fitusiran in patients with hemophilia A
or B with and without inhibitors, with topline results expected in
H1 2021.
- Advanced early- and mid-stage RNAi therapeutic pipeline
programs.
- The Company announces today positive initial topline results
from the ongoing Phase 1 study (N=48) of ALN-AGT in
hypertension, providing initial human proof of concept with over 90
percent mean knockdown of angiotensinogen (AGT) and an over 10 mmHg
reduction of mean 24-hour systolic blood pressure at week 8
relative to placebo, with a durability that supports once quarterly
or less frequent dose administration. In addition, ALN-AGT
administration was associated with an encouraging safety and
tolerability profile including no drug-related serious adverse
events (SAEs).
- Alnylam’s partner, Vir Biotechnology, presented positive
interim data from the ongoing Phase 2 trial in patients and results
from the Phase 1 trial in healthy volunteers of ALN-HBV02
(VIR-2218), an investigational RNAi therapeutic for the
treatment of chronic hepatitis B virus (HBV) infection.
- Under expanded collaboration with Vir, selected a Development
Candidate (DC), ALN-COV (VIR-2703), for SARS-CoV-2 – the
virus that causes COVID-19 – with potent and highly cross-reactive
activity, with a plan for accelerated filing of an IND at or around
year-end 2020.
- The Company announces today that it will shift filing of its
ALN-LEC IND into 2021 to focus on its COVID-19
investigational RNAi therapeutic program.
Additional Business Updates
- Entered into a broad strategic financing collaboration with
Blackstone under which Alnylam will receive up to $2 billion that
is expected to enable Alnylam to achieve a self-sustainable
financial profile without the need for future equity
financing.
- Expanded collaboration with Vir to include the development and
commercialization of RNAi therapeutics targeting SARS-CoV-2 and up
to three additional targets focused on host factors for SARS-CoV-2,
including angiotensin converting enzyme-2 (ACE2), transmembrane
protease, serine 2 (TMPRSS2), and potentially a third host factor
target to emerge from Vir’s functional genomics work.
- Entered into an agreement with Dicerna to develop and
commercialize investigational RNAi therapeutics for the treatment
of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease,
and completed a non-exclusive cross-licensing agreement with
Dicerna regarding the companies’ respective intellectual property
for Alnylam’s lumasiran and Dicerna’s nedosiran investigational
programs for the treatment of primary hyperoxaluria.
- Appointed Dr. Olivier Brandicourt, former CEO of Sanofi, to the
Company’s Board of Directors, and announced the retirement of Dr.
Paul Schimmel, an Alnylam co-founder, in May 2020 after nearly 18
years of service; Dr. Schimmel will remain a member of the
Company’s Scientific Advisory Board.
Upcoming Events
In mid-2020, Alnylam intends to:
- Continue global commercialization of ONPATTRO and
GIVLAARI.
- Present full results from the ILLUMINATE-A Phase 3 study of
lumasiran at the European Renal Association-European Dialysis and
Transplant Association (ERA-EDTA) Congress, currently scheduled for
June 7, 2020 as a virtual event.
- Report topline results from the ILLUMINATE-B Phase 3 study of
lumasiran.
- File a Clinical Trial Application (CTA) for ALN-HSD, an
investigational RNAi therapeutic targeting HSD17B13 for the
treatment of non-alcoholic steatohepatitis (NASH), in collaboration
with Regeneron.
Financial Results for the Quarter Ended March 31,
2020
“We believe our results for the first quarter demonstrate the
strength of our commercial teams in challenging circumstances,
delivering solid quarterly growth for ONPATTRO and robust uptake
for GIVLAARI in the U.S. in its first full quarter of launch. While
we have confidence in our business continuity plans, we expect an
impact from the COVID-19 pandemic and are lowering our 2020 revenue
guidance range for ONPATTRO by 5 percent from $285-$315 million to
$270-$300 million. In parallel, we are implementing further
discipline in our operations to moderate our spend and are lowering
our operating expense guidance range for the year,” said Jeff
Poulton, Chief Financial Officer of Alnylam. “Of course, a business
highlight for the recent period was the completion of our $2
billion strategic financing collaboration with Blackstone, the
largest ever financing of a pre-profitable biotechnology company.
We believe this new financing, together with our expectations for
continued topline growth and disciplined expense management,
bridges our transition toward a self-sustainable financial profile
without the need for future equity offerings. In sum, we believe
Alnylam is taking appropriate measures to operate through the
pandemic and recovery phases in 2020, into the ‘new normal’ and
beyond, continuing to deliver on the promise of RNAi therapeutics
and our commitments to patients and our shareholders.”
Financial Highlights (in thousands, except
per share amounts)
Three Months Ended March
31,
2020
2019
Net product revenues
$
71,938
$
26,291
ONPATTRO net product revenues
$
66,664
$
26,291
GIVLAARI net product revenues
$
5,274
$
—
Net revenue from collaborations
$
27,538
$
7,003
GAAP combined research and development and
selling, general and administrative expenses
$
296,332
$
218,735
Non-GAAP combined research and development
and selling, general and administrative expenses
$
261,754
$
186,703
GAAP operating loss
$
(210,158)
$
(188,788)
Non-GAAP operating loss
$
(175,580)
$
(156,756)
GAAP net loss
$
(182,221)
$
(181,915)
Non-GAAP net loss
$
(171,754)
$
(149,883)
GAAP net loss per common share - basic and
diluted
$
(1.62)
$
(1.73)
Non-GAAP net loss per common share - basic
and diluted
$
(1.52)
$
(1.42)
March 31, 2020
December 31, 2019
Cash, cash equivalents, marketable debt
and equity securities and restricted investments
$
1,366,928
$
1,550,987
Net Product Revenues
- Net product revenues were $71.9 million in the first quarter
2020 representing 174 percent growth from the first quarter 2019 as
a result of the addition of new patients on therapy and expansion
into new markets for ONPATTRO, as well as the U.S. commercial
launch of GIVLAARI.
Net Revenues from Collaborations
- Net revenues from collaborations were $27.5 million in the
first quarter 2020, an increase from $7.0 million in the first
quarter 2019, primarily due to revenues recognized from our
Regeneron and Vir collaborations.
Combined Research & Development (R&D) and Selling,
General & Administrative (SG&A) Expenses
- Combined R&D and SG&A expenses increased on a GAAP and
non-GAAP basis primarily due to increased activity related to the
advancement of our late stage R&D programs and increased
activity in commercial and medical affairs to support the ongoing
launches of ONPATTRO and GIVLAARI.
Cash and Investments
- Cash, cash equivalents, marketable debt and equity securities,
and restricted investments were $1.37 billion at the end of the
first quarter 2020 compared to $1.55 billion at the end of 2019.
The decrease was due to cash used in our operations to support
overall growth. Cash at the end of the first quarter excludes
$600.0 million in proceeds from the Blackstone strategic financing
collaboration, which closed in the second quarter.
A reconciliation of our GAAP to non-GAAP results for the current
quarter is included in the tables of this press release.
2020 Updated Financial Guidance
Full year 2020 financial guidance consists of the following:
Item
Provided 2/6/2020 ($
millions)
Updated 5/6/2020 ($
millions)
ONPATTRO net product revenues
$285 - $315
$270 - $300
GIVLAARI net product revenues
No guidance provided
Unchanged
Net revenues from collaborations
$100 - $150
Unchanged
GAAP R&D and SG&A expenses
$1,180 - $1,300
$1,155 - $1,250
Non-GAAP R&D and SG&A
expenses*
$1,025 - $1,125
$1,000 - $1,075
The $2 billion strategic financing collaboration with Blackstone
is expected to enable Alnylam’s achievement of a self-sustainable
financial profile without need for future equity financings.
*Excludes $155 million - $175 million of stock-based
compensation 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 the press release are stock-based compensation
expenses and unrealized gain 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 impact the fair value of these awards.
The Company has excluded the impact of the unrealized gain on
marketable equity securities because the Company believes this item
is a one-time event occurring outside the ordinary course of the
Company’s business.
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 2020 results as
well as expectations for the future via conference call on
Wednesday, May 6, 2020 at 8:30 am ET. To access the call, please
dial 800-239-9838 (domestic) or +1-323-794-2551 (international)
five minutes prior to the start time and refer to conference ID
6976021. 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
888-203-1112 (domestic) or +1-719-457-0820 (international) and
refer to conference ID 6976021.
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%).
Indication ONPATTRO is indicated
for the treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults.
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) for
the treatment of adults and adolescents with acute hepatic
porphyria (AHP). 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 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 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), approved in the U.S., EU,
Canada, Japan, Switzerland and Brazil, and GIVLAARI® (givosiran),
approved in the U.S. and EU. Alnylam has a deep pipeline of
investigational medicines, including six product candidates that
are in late-stage development. Alnylam is executing on its “Alnylam
2020” strategy of building a multi-product, commercial-stage
biopharmaceutical company with a sustainable pipeline of RNAi-based
medicines to address the needs of patients who have limited or
inadequate treatment options. 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 or on LinkedIn.
Alnylam Forward Looking Statements Various statements in
this release concerning Alnylam’s expectations, plans and
prospects, including, without limitation, expectations regarding
the direct or indirect effects on Alnylam’s business, activities
and prospects as a result of the COVID-19 pandemic, or delays or
interruptions resulting therefrom and the success of Alnylam’s
mitigation efforts, Alnylam's views and plans with respect to the
potential for RNAi therapeutics, including ONPATTRO, GIVLAARI,
lumasiran, patisiran, vutrisiran, inclisiran, fitusiran, ALN-AAT02,
ALN-HBV02, ALN-AGT, ALN-COV and ALN-HSD, its expectations for
ALN-AGT as a potential treatment of hypertension, an area of
prevalent unmet need, its plans for additional global regulatory
filings and the continuing product launches of ONPATTRO and
GIVLAARI, expectations regarding reimbursement for ONPATTRO and
GIVLAARI in various territories and the status of VBA negotiations
and executed agreements, the advancement of lumasiran and
inclisiran through regulatory review and toward the market, the
expected presentation of full ILLUMINATE-A Phase 3 results and
topline ILLUMINATE-B Phase 3 results for lumasiran, the achievement
of additional pipeline milestones, including relating to ongoing
clinical studies of vutrisiran and the delay in completion of
enrollment in its APOLLO-B study of patisiran in ATTR amyloidosis
with cardiomyopathy due to the impact of COVID-19, the expected
timing for filing INDs for ALN-COV and ALN-HSD, as well as the
delayed filing of an IND for ALN-LEC, its expectations relating to
continued ONPATTRO and GIVLAARI revenue growth and the revised
expected range of ONPATTRO net product revenues for 2020, the
expected range for net revenues from collaborations for 2020, the
expected range of 2020 aggregate annual non-GAAP and GAAP R&D
and SG&A expenses after reductions expected from the
implementation of further discipline in operations to moderate
spend, Alnylam’s belief that the funding provided by Blackstone
should enable Alnylam to achieve a self-sustainable profile without
the need for future equity financing, and expectations regarding
the achievement or potential to exceed its “Alnylam 2020” strategic
plan announced in 2015 for the advancement and commercialization of
RNAi therapeutics, 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 a future
pandemic, such as the scope and duration of the outbreak,
government actions and restrictive measures implemented in
response, material delays in diagnoses of rare diseases, initiation
or continuation of treatment for diseases addressed by Alnylam
products, or in patient enrollment in clinical trials, potential
supply chain disruptions, and other potential impacts to Alnylam’s
business, the effectiveness or timeliness of steps taken by Alnylam
to mitigate the impact of the pandemic, and Alnylam’s ability to
execute business continuity plans to address disruptions caused by
the COVID-19 or a future 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, including vutrisiran, ALN-AGT, ALN-HSD and ALN-COV; the
pre-clinical and clinical results for its product candidates, which
may not be replicated or continue to occur in other subjects or in
additional studies or otherwise support further development of
product candidates for a specified indication or at all; actions or
advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional pre-clinical and/or clinical
testing; delays, interruptions or failures in the manufacture and
supply of its product candidates or its marketed products,
including ONPATTRO, GIVLAARI, inclisiran, lumasiran and vutrisiran;
obtaining, maintaining and protecting intellectual property;
intellectual property matters including potential patent litigation
relating to its platform, products or product candidates; obtaining
regulatory approval for its product candidates, including lumasiran
and inclisiran, and maintaining regulatory approval and obtaining
pricing and reimbursement for its products, including ONPATTRO and
GIVLAARI; progress in continuing to establish a commercial and
ex-United States infrastructure; successfully launching, marketing
and selling its approved products globally, including ONPATTRO and
GIVLAARI and achieving net product revenues for ONPATTRO within its
revised expected range during 2020; Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future;
competition from others using technology similar to Alnylam's and
others developing products for similar uses; Alnylam's ability to
manage its growth and operating expenses within the reduced ranges
of guidance provided by Alnylam through the implementation of
further discipline in operations to moderate spend and its ability
to achieve a self-sustainable financial profile in the future
without the need for future equity financing; Alnylam’s ability to
establish and maintain strategic business alliances and new
business initiatives, including completing an agreement for funding
by Blackstone of certain R&D activities for vutrisiran and
ALN-AGT; Alnylam's dependence on third parties, including
Regeneron, for development, manufacture and distribution of certain
products, including eye and CNS products, and Ironwood, for
assistance with the education about and promotion of GIVLAARI, and
Vir for the development of ALN-COV and other potential RNAi
therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2;
the outcome of litigation; the risk of government investigations;
and unexpected expenditures, as well as those risks more fully
discussed in the “Risk Factors” filed with Alnylam's most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) and in other filings that Alnylam makes
with the SEC. In addition, any forward-looking statements represent
Alnylam's views only as of today and should not be relied upon as
representing its views as of any subsequent date. Alnylam
explicitly disclaims any obligation, except to the extent required
by law, to update any forward-looking statements.
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except per share amounts) (Unaudited)
Three Months Ended March
31,
2020
2019
Statements of Operations
Revenues:
Net product revenues
$
71,938
$
26,291
Net revenues from collaborations
27,538
7,003
Total revenues
99,476
33,294
Operating costs and expenses:
Cost of goods sold
$
13,302
$
3,347
Research and development
169,571
129,127
Selling, general and administrative
126,761
89,608
Total operating costs and expenses
309,634
222,082
Loss from operations
(210,158)
(188,788)
Other income:
Interest income
5,480
7,525
Other income
23,032
43
Total other income
28,512
7,568
Loss before income taxes
(181,646)
(181,220)
Provision for income taxes
(575)
(695)
Net loss
$
(182,221)
$
(181,915)
Net loss per common share - basic and
diluted
$
(1.62)
$
(1.73)
Weighted-average common shares used to
compute basic and diluted net loss per common share
112,748
105,400
Statements of Comprehensive
Loss
Net loss
$
(182,221)
$
(181,915)
Unrealized gain on marketable debt
securities
4,045
360
Foreign currency translation
340
—
Defined benefit pension plans, net of
tax
74
—
Comprehensive loss
$
(177,762)
$
(181,555)
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP MEASURES TO NON-GAAP MEASURES (In
thousands, except per share amounts)
Three Months Ended March
31,
2020
2019
Reconciliation of GAAP to Non-GAAP
R&D and SG&A expenses:
GAAP R&D and SG&A expenses
296,332
218,735
Less: Stock-based compensation
expenses
(34,578)
(32,032)
Non-GAAP R&D and SG&A expenses
261,754
186,703
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
(210,158)
(188,788)
Add: Stock-based compensation expenses
34,578
32,032
Non-GAAP operating loss
(175,580)
(156,756)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
(182,221)
(181,915)
Add: Stock-based compensation expenses
34,578
32,032
Less: Unrealized gain on marketable equity
securities
(24,111)
—
Non-GAAP net loss
(171,754)
(149,883)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP net loss per common share - basic and
diluted
(1.62)
(1.73)
Add: Stock-based compensation expenses
0.31
0.31
Less: Unrealized gain on marketable equity
securities
(0.21)
—
Non-GAAP net loss per common share - basic
and diluted
(1.52)
(1.42)
Please note that the figures presented
above may not sum exactly due to rounding
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands,
except share amounts) (Unaudited)
March 31, 2020
December 31, 2019
Cash, cash equivalents and marketable debt
and equity securities
$
1,342,203
$
1,536,162
Restricted investments
24,725
14,825
Accounts receivable, net
75,572
43,011
Inventory
68,300
56,348
Prepaid expenses and other assets
117,730
98,412
Property, plant and equipment, net
429,814
425,179
Operating lease right-of-use lease
assets
221,280
221,197
Total assets
$
2,279,624
$
2,395,134
Accounts payable, accrued expenses and
other liabilities
$
232,903
$
256,415
Total deferred revenue
392,167
396,204
Operating lease liability
304,825
303,823
Total stockholders’ equity (113.2 million
shares issued and outstanding at March 31, 2020; 112.2 million
shares issued and outstanding at December 31, 2019)
1,349,729
1,438,692
Total liabilities and stockholders'
equity
$
2,279,624
$
2,395,134
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, 2019.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200506005169/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
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