− Achieved Second Quarter 2020 ONPATTRO® Global
Net Product Revenues of $66.5 Million, with More Than 1,050
Patients on Commercial Product Worldwide –
− Achieved Second Quarter 2020 GIVLAARI® Global
Net Product Revenues of $11.0 Million with More Than 100 Patients
on Commercial Product Worldwide –
– Presented Complete Results from ILLUMINATE-A
Phase 3 Study of Lumasiran and Completed Filings of New Drug
Application and Marketing Authorisation Application –
− Completed $2 Billion Strategic Financing
Collaboration with Blackstone, Enabling Achievement of
Self-Sustainable Financial Profile without Need for Future Equity
Offerings –
– Increased Midpoint of Guidance Range for 2020
ONPATTRO Revenue, Narrowing Range from $270-$300 million to
$280-$300 million –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the second quarter 2020 and reviewed recent business
highlights.
“In the second quarter of 2020, the world experienced
unprecedented challenges as it continued to confront the COVID-19
pandemic. While the pandemic continues in some countries and
states, our global commercialization strategy is now enabling some
customer-facing activities to resume in most markets as we enter
the third quarter. We are very pleased with our ONPATTRO and
GIVLAARI commercial performance in the second quarter, in the face
of the ongoing pandemic, and believe it reflects strong demand for
our products as well as our team’s unwavering commitment to assure
access to these RNAi therapeutics for patients around the world,”
said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
“In the quarter, we also continued advancing our robust late-stage
pipeline of investigational RNAi therapeutics, where, notably, we
presented full results from the ILLUMINATE-A Phase 3 study and
completed regulatory filings for lumasiran, and continued
enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of
patisiran and vutrisiran, respectively. Further, a key business
highlight in the second quarter was our completion of a landmark
strategic financing collaboration with Blackstone, which we believe
will secure our ability to achieve a self-sustainable financial
profile without the need for future equity financing. These and
other achievements position us to realize our Alnylam 2020 vision
of building a multi-product, global biopharma company with a deep
clinical pipeline to fuel continued growth and a robust, organic
product engine to drive sustainable innovation and further value
creation.”
Second Quarter 2020 and Recent Significant Corporate
Highlights
Commercial Performance
ONPATTRO®
- Achieved global net product revenues for the second quarter of
2020 of $66.5 million.
- Attained over 1,050 patients worldwide on commercial ONPATTRO
treatment as of June 30, 2020.
- Continued strong market access in the U.S., with over 15
value-based agreements (VBAs) to date with commercial payers and
confirmed access for over 98% of covered U.S. lives.
- Continued progress with market access efforts across the CEMEA
region (Canada, Europe, Middle East,
and Africa), with recent launches in Spain and Italy.
- The Company announces today that it has achieved agreement on
pricing and reimbursement in France, completing patient access for
ONPATTRO in all major European markets in under 2 years following
approval.
GIVLAARI®
- Achieved global net product revenues for the second quarter of
2020 of $11.0 million.
- Since launch, received over 85 Start Forms in the U.S. and
attained over 100 patients globally on commercial GIVLAARI
treatment from launch through June 30, 2020.
- Received marketing authorization approval for GIVLAARI in
Brazil for the treatment of acute hepatic porphyria in adults.
- Continued strong progress with market access in the U.S., with
seven completed VBAs with commercial payers, and confirmed access
for over 75% of covered U.S. lives.
- Continued progress with market access efforts across the CEMEA
region, with a successful launch in Germany, commencement of cohort
ATU supply in France, and named patient sales in other countries.
- Received an Improvement of Medical Benefit (ASMR) score of II
from Haute Autorité de Santé (HAS) in France, concluding that
GIVLAARI offers significant additional therapeutic value. Only two
new commercial medicines received a similar ASMR score in
2019.
- Continued work with physicians in multiple regions to support
requests for pre-approval access to GIVLAARI in an Expanded Access
Program (EAP) in accordance with local requirements.
R&D Highlights
- Advanced 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.
- Presented additional clinical data with patisiran – including
24-month results from the global open-label extension (OLE) study
and results from an open-label study in post-orthotopic liver
transplant hATTR amyloidosis patients – and published findings from
an evaluation of patisiran with concomitant use of TTR
stabilizers.
- Presented new 12-month interim data from the ENVISION Phase 3
study of givosiran (the non-proprietary name for GIVLAARI)
in acute hepatic porphyria (AHP).
- In addition, published pivotal results from the ENVISION Phase
3 study in The New England Journal of Medicine.
- Submitted a Marketing Authorization Application (MAA) for
givosiran in Switzerland and Israel.
- Advanced lumasiran, an investigational RNAi therapeutic
in development for the treatment of primary hyperoxaluria type 1
(PH1).
- Completed the rolling submission of a New Drug Application
(NDA) to the Food and Drug Administration (FDA) and submitted an
MAA to the European Medicines Agency (EMA), with both applications
now accepted.
- The FDA also granted Priority Review for the NDA and set an
action date of December 3, 2020 under the Prescription Drug User
Fee Act (PDUFA).
- The EMA granted an accelerated assessment for the lumasiran
MAA.
- Received a positive scientific opinion from the UK’s Medicines
and Healthcare Products Regulatory Agency (MHRA) through the Early
Access to Medicines Scheme (EAMS).
- Presented complete results from the ILLUMINATE-A Phase 3
study.
- Continued treating patients in ILLUMINATE-B, a global Phase 3
pediatric 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.
- Continued treating patients in the fully enrolled HELIOS-A
Phase 3 study of vutrisiran in hATTR amyloidosis patients with
polyneuropathy, and remain 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 in adults.
- Continued enrollment in the HELIOS-B Phase 3 study in ATTR
amyloidosis patients with cardiomyopathy.
- Alnylam’s partner, Novartis, continued advancing
inclisiran, potentially the first and only siRNA
cholesterol-lowering treatment, which is undergoing review for
approval in the U.S. and EU.
- Published results from the completed ORION Phase 3 pivotal
trials in The New England Journal of Medicine, showing durable and
potent LDL-C reduction achieved with inclisiran, with a safety
profile similar to placebo.
- Alnylam’s partner, Sanofi, continued 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.
- Presented updated interim results at the World Federation of
Hemophilia meeting from a Phase 2 OLE study of fitusiran in
patients with hemophilia A and B with and without inhibitors.
- Advanced early- and mid-stage RNAi therapeutic pipeline
programs.
- In collaboration with Regeneron, advanced cemdisiran, an
investigational RNAi therapeutic for the treatment of
complement-mediated diseases.
- Continued enrollment in a Phase 2 clinical trial of cemdisiran
monotherapy in patients with IgA nephropathy, with topline results
expected in 2021.
- Regeneron filed a Clinical Trial Application (CTA) in The
Netherlands to initiate a Phase 1 study of cemdisiran in
combination with pozelimab, an anti-C5 monoclonal antibody,
in normal healthy volunteers and patients with paroxysmal nocturnal
hemoglobinuria (PNH).
- 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.
- Reported positive initial topline results from the ongoing
Phase 1 study of ALN-AGT in hypertension, demonstrating
acceptable safety, and clinically significant blood pressure
lowering with durability enabling a quarterly or less frequent
dosing regimen. Additional Phase 1 results are expected to be
presented at a scientific meeting later in 2020, pending abstract
acceptance.
- Filed a Clinical Trial Application (CTA) for ALN-HSD, an
investigational RNAi therapeutic targeting HSD17B13 in development
for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD
is being advanced in collaboration with Regeneron.
- Selected a Development Candidate (DC), ALN-COV
(VIR-2703), for SARS-CoV-2 – the virus that causes COVID-19 –
with a plan for accelerated filing of an IND around year-end
2020.
- Continued progress advancing investigational RNAi therapeutics
for CNS and ocular diseases, including ALN-APP, in
development for the treatment of hereditary cerebral amyloid
angiopathy (hCAA) and autosomal dominant Alzheimer’s Disease
(ADAD), which remains on track for a CTA filing in 2021. The
Company announces today that Regeneron has exercised its
co-development/co-commercialization option on the ALN-APP program,
which Alnylam will lead.
Additional Business Updates
- Barry Greene, President of Alnylam, announced his intention to
transition from Alnylam to pursue new opportunities at the end of
the third quarter. Yvonne Greenstreet, Chief Operating Officer,
will assume an expanded role as President and Chief Operating
Officer on October 1, 2020.
- Entered into a 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.
- 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.
- Expanded infectious disease collaboration with Vir to include
the development and commercialization of RNAi therapeutics
targeting up to three host factor targets 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.
- Formed a Distribution Agreement with taiba Middle East to
commercialize Alnylam’s RNAi therapeutics in the Gulf states.
Upcoming Events
In mid- and late 2020, Alnylam intends to:
- Achieve regulatory approval for ONPATTRO in Israel.
- File a new drug application for GIVLAARI in Japan and achieve
regulatory approval for GIVLAARI in Canada.
- Report topline results from the ILLUMINATE-B Phase 3 study of
lumasiran in PH1 patients less than six years of age with preserved
renal function.
- Present additional clinical results from the ongoing Phase 1
trial of ALN-AGT.
- Initiate a Phase 1 trial of ALN-HSD.
- Achieve regulatory approvals for lumasiran in U.S. and EU.
- Alnylam’s partner Novartis expects an FDA action date for
inclisiran in late 2020.
- Alnylam’s partner Regeneron plans to initiate a Phase 1 study
of cemdisiran in combination with pozelimab.
Financial Results for the Quarter Ended June 30, 2020
“We are extremely pleased with our financial results for the
second quarter, particularly in the face of these challenging
circumstances. Our team’s ability to swiftly adapt to virtual
engagement with stakeholders and to support the transition of
patients, where needed, to alternate sites of care, in addition to
strong international results, led to commercial performance for
both ONPATTRO and GIVLAARI that exceeded the initial expectations
we communicated in May 2020. As a result, we are further revising
our full-year revenue guidance for ONPATTRO, with an increase in
the midpoint of our guidance as we narrow the range from $270-$300
million to $280-$300 million,” said Jeff Poulton, Chief Financial
Officer of Alnylam. “Outside of our commercial performance, a key
business highlight in the second quarter was our achievement of
year-over-year improvement in our non-GAAP operating loss versus
2019. Driven by strong top-line year-over-year growth and
disciplined investment in our operations, we continue to believe
2019 represents our peak operating loss year as we begin our
journey towards a self-sustainable financial profile. In summary,
we believe that we’re well positioned to effectively navigate
through the pandemic and continue to deliver on the promise of RNAi
therapeutics, bringing transformative therapies to patients with
serious diseases around the world.”
Financial Highlights
(in thousands, except per share amounts)
Three Months Ended June
30,
2020
2019
Net product revenues
$
77,533
$
38,231
ONPATTRO net product revenues
$
66,535
$
38,231
GIVLAARI net product revenues
$
10,998
$
—
Net revenue from collaborations
$
26,429
$
6,483
Cost of goods sold
$
19,929
$
4,326
GAAP research and development expenses
$
154,996
$
163,890
Non-GAAP research and development
expenses
$
139,206
$
148,608
GAAP selling, general and administrative
expenses
$
127,896
$
112,769
Non-GAAP selling, general and
administrative expenses
$
109,611
$
97,448
GAAP operating loss
$
(198,859)
$
(236,271)
Non-GAAP operating loss
$
(164,784)
$
(205,668)
GAAP net loss
$
(179,229)
$
(219,481)
Non-GAAP net loss
$
(191,328)
$
(198,300)
GAAP net loss per common share - basic and
diluted
$
(1.56)
$
(2.02)
Non-GAAP net loss per common share - basic
and diluted
$
(1.67)
$
(1.83)
June 30, 2020
December 31, 2019
Cash, cash equivalents, marketable debt
and equity securities and restricted investments
$
1,950,289
$
1,550,987
Net Product Revenues
- Net product revenues were $77.5 million in the second quarter
2020 representing 103% growth from the second quarter 2019 as a
result of the addition of new patients on therapy and expansion
into new markets for ONPATTRO, as well as the ongoing U.S.
commercial launch and initial European launch of GIVLAARI.
Net Revenues from Collaborations
- Net revenues from collaborations were $26.4 million in the
second quarter 2020, an increase from $6.5 million in the second
quarter 2019, primarily due to increases in revenues recognized
from our Regeneron and Vir collaborations.
Research & Development (R&D) and Selling, General &
Administrative (SG&A) Expenses
- R&D expenses decreased in the second quarter 2020 compared
to the same period in 2019 on a GAAP and non-GAAP basis primarily
due to nonrecurring expenses in 2019 from license fees related to
the execution of our collaboration agreement with Regeneron, as
well as a decrease in expenses associated with material
manufactured for clinical trials.
- SG&A expenses increased in the second quarter 2020 compared
to the same period in 2019 on a GAAP and non-GAAP basis primarily
due to increased investment in commercial and medical affairs
activity to support the ongoing launches of ONPATTRO and GIVLAARI
and initial launch preparation activities for lumasiran.
Cash and Investments
- Cash, cash equivalents, marketable debt and equity securities,
and restricted investments were $1.95 billion at the end of the
second quarter 2020 compared to $1.55 billion at the end of 2019.
The increase was primarily due to $600.0 million in proceeds
received in the second quarter of 2020 from the sale of future
royalties and issuance of common stock to Blackstone and its
affiliates, partially offset by cash used in our operations to
support overall growth.
A reconciliation of our GAAP to non-GAAP results for the current
quarter is included in the tables of this press release.
2020 Updated Financial Guidance
Full year 2020 financial guidance consists of the following:
Item
Provided 5/6/2020 ($
millions)
Updated 8/6/2020 ($
millions)
ONPATTRO net product revenues
$270 - $300
$280 - $300
GIVLAARI net product revenues
No guidance provided
Unchanged
Net revenues from collaborations
$100 - $150
Unchanged
GAAP R&D and SG&A expenses
$1,155 - $1,250
$1,130 - $1,225
Non-GAAP R&D and SG&A
expenses*
$1,000 - $1,075
Unchanged
*Excludes $130-$150 million (previously
$155-175 million) of stock-based compensation and costs associated
with the strategic financing collaboration from estimated GAAP
R&D and SG&A expenses.
The strategic financing collaboration with Blackstone under
which Alnylam will receive up to $2 billion is expected to enable
Alnylam’s achievement of a self-sustainable financial profile
without need for future equity financings.
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, unrealized gain on marketable equity securities, costs
associated with our strategic financing collaboration, a gain on
contractual settlement, and a gain on the change in fair value of a
liability obligation. 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 gain on marketable equity securities, costs
associated with our strategic financing collaboration, the gain on
contractual settlement, and a gain on the change in fair value of a
liability obligation because the Company believes these items are
non-recurring transactions 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
second quarter 2020 results as well as expectations for the future
via conference call on Thursday, August 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%).
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 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, 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., EU and Brazil. 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, cemdisiran, ALN-HBV02, ALN-AGT, ALN-HSD, ALN-COV and
ALN-APP, 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, its expectations regarding continued progress
supporting pre-approval access to GIVLAARI in its EAP program, the
advancement of lumasiran and inclisiran through regulatory review
and toward the market, the expected timing for the presentation of
topline ILLUMINATE-B Phase 3 results for lumasiran, the achievement
of additional pipeline milestones, including relating to ongoing
clinical studies of vutrisiran, the expected timing for filing INDs
or CTAs for ALN-COV and ALN-APP, the initiation of Phase 1 clinical
studies of ALN-HSD by Alnylam and cemdisiran and pozelimab by
Regeneron, its expectations relating to continued ONPATTRO and
GIVLAARI revenue growth and the further revised expected range of
ONPATTRO net product revenues for 2020, the expected range for net
revenues from collaborations for 2020, the revised expected range
of 2020 aggregate annual non-GAAP and GAAP R&D and SG&A
expenses, 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 of 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 any 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 any 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, ALN-APP 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
further 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 ALN-APP, 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.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In
thousands, except per share amounts) (Unaudited)
Three Months Ended
Six Months Ended
June 30, 2020
June 30, 2019
June 30, 2020
June 30, 2019
Statements of Operations
Revenues:
Net product revenues
$
77,533
$
38,231
$
149,471
$
64,522
Net revenues from collaborations
26,429
6,483
53,967
13,486
Total revenues
103,962
44,714
203,438
78,008
Operating costs and expenses:
Cost of goods sold
$
19,929
$
4,326
$
33,231
$
7,673
Research and development
154,996
163,890
324,567
293,017
Selling, general and administrative
127,896
112,769
254,657
202,377
Total operating costs and expenses
302,821
280,985
612,455
503,067
Loss from operations
(198,859)
(236,271)
(409,017)
(425,059)
Other income:
Interest expense
(27,248)
—
(27,248)
—
Interest income
3,165
8,781
8,645
16,306
Other income (expense)
45,039
(453)
68,071
(410)
Change in fair value of liability
obligation
—
9,422
—
9,422
Total other income
20,956
17,750
49,468
25,318
Loss before income taxes
(177,903)
(218,521)
(359,549)
(399,741)
Provision for income taxes
(1,326)
(960)
(1,901)
(1,655)
Net loss
$
(179,229)
$
(219,481)
$
(361,450)
$
(401,396)
Net loss per common share - basic and
diluted
$
(1.56)
$
(2.02)
$
(3.18)
$
(3.75)
Weighted-average common shares used to
compute basic and diluted net loss per common share
114,911
108,576
113,830
106,997
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP MEASURES TO NON-GAAP
MEASURES (In thousands, except per share amounts)
Three Months Ended
June 30, 2020
June 30, 2019
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP Research and development
154,996
163,890
Less: Stock-based compensation
expenses
(15,790)
(15,282)
Non-GAAP Research and development
139,206
148,608
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP Selling, general and
administrative
127,896
112,769
Less: Stock-based compensation
expenses
(17,965)
(15,321)
Less: Costs associated with the strategic
financing collaboration
(320)
—
Non-GAAP Selling, general and
administrative
109,611
97,448
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
(198,859)
(236,271)
Add: Stock-based compensation expenses
33,755
30,603
Add: Costs associated with the strategic
financing collaboration
320
—
Non-GAAP operating loss
(164,784)
(205,668)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
(179,229)
(219,481)
Add: Stock-based compensation expenses
33,755
30,603
Add: Costs associated with the strategic
financing collaboration
320
—
Less: Unrealized gain on marketable equity
securities
(45,532)
—
Less: Gain on contractual settlement
(642)
—
Less: Change in Fair value of liability
contribution
—
(9,422)
Non-GAAP net loss
(191,328)
(198,300)
Reconciliation of GAAP to Non-GAAP net
loss per common share- basic and diluted:
GAAP net loss per common share - basic and
diluted
(1.56)
(2.02)
Add: Stock-based compensation expenses
0.29
0.28
Add: Costs associated with the strategic
financing collaboration
—
—
Less: Unrealized gain on marketable equity
securities
(0.39)
—
Less: Gain on contractual settlement
(0.01)
—
Less: Change in Fair value of liability
contribution
—
(0.09)
Non-GAAP net loss per common share - basic
and diluted
(1.67)
(1.83)
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)
June 30, 2020
December 31, 2019
Cash, cash equivalents and marketable debt
and equity securities
$
1,925,564
$
1,536,162
Restricted investments
24,725
14,825
Accounts receivable, net
69,115
43,011
Inventory
77,418
56,348
Prepaid expenses and other assets
108,745
98,412
Property, plant and equipment, net
439,126
425,179
Operating lease right-of-use lease
assets
229,674
221,197
Receivable related to the sale of future
royalties
500,000
—
Total assets
$
3,374,367
$
2,395,134
Accounts payable, accrued expenses and
other liabilities
$
256,166
$
256,415
Total deferred revenue
389,117
396,204
Operating lease liability
315,065
303,823
Liability related to the sale of future
royalties
1,014,293
—
Total stockholders’ equity (115.6 million
shares issued and outstanding at June 30, 2020; 112.2 million
shares issued and outstanding at December 31, 2019)
1,399,726
1,438,692
Total liabilities and stockholders'
equity
$
3,374,367
$
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/20200806005302/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
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