Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced
its first quarter 2024 financial results and business highlights.
“We made excellent progress against our strategic priorities
during the quarter, including bringing SYFOVRE to more patients in
the U.S., advancing EMPAVELI within multiple high unmet need areas,
and progressing our earlier-stage pipeline,” said Cedric Francois,
M.D., Ph.D., co-founder and chief executive officer of Apellis.
Dr. Francois continued, “SYFOVRE performance continues to exceed
expectations in the U.S., with 20% revenue growth in the first
quarter, and we are working closely with the EU regulators towards
our goal of bringing pegcetacoplan to the GA community in Europe.
EMPAVELI continues to elevate the standard of care in PNH, and we
are excited about the upcoming topline readout from our Phase 3
VALIANT trial with EMPAVELI for C3G and IC-MPGN. With two
commercial products and an emerging pipeline, we are making
tremendous progress on our vision to develop life-changing
medicines for people living with some of the most challenging
diseases.”
First Quarter 2024 Business Highlights and Upcoming
Milestones
Ophthalmology Highlights
- SYFOVRE for the
treatment of geographic atrophy (GA) secondary to age-related
macular degeneration (AMD):
- Generated $137.5 million in SYFOVRE U.S. net product revenue in
the first quarter of 2024.
- SYFOVRE is the #1 chosen treatment for GA, delivering
approximately 77,000 SYFOVRE doses to physician practices in the
first quarter of 2024 (72,000 commercial doses and 5,000
samples).
- Approximately 250,000 SYFOVRE
injections are estimated to have been administered through March
2024, including clinical trials.
- Launched a branded
direct-to-consumer (DTC) awareness campaign in April 2024. The
branded campaign builds on the previous disease state awareness
campaigns and is aimed at educating GA patients about SYFOVRE.
- Announced that the European
Medicines Agency (EMA) has reset the pegcetacoplan marketing
authorization application (MAA) review to day 180, the last phase
of the initial assessment.
- The update follows the judgment by
the Court of Justice of the European Union (CJEU) on March 14,
2024, which ruled on the organization of EMA’s expert groups. This
decision by EMA is strictly procedural in response to the CJEU
judgment and is not related to the pegcetacoplan data package.
- Apellis now anticipates a CHMP
opinion no later than July 2024.
- Leading retina specialist Dr.
Philip Ferrone joined Apellis in March 2024 as chief medical retina
advisor.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Highlights
- EMPAVELI for the
treatment of PNH:
- Generated $25.6 million in EMPAVELI
U.S. net product revenue in the first quarter of 2024.
- Continued high patient compliance
rates of 97%.
R&D Highlights
- C3 glomerulopathy (C3G) and immune
complex glomerulonephritis (IC-MPGN): Topline data from the Phase 3
VALIANT study of systemic pegcetacoplan is expected in mid-2024.
- VALIANT is a randomized,
placebo-controlled, double-blinded, multi-center study designed to
evaluate the safety and efficacy of systemic pegcetacoplan in 124
patients aged 12 and up with either C3G or IC-MPGN, and either pre-
or post-kidney transplant.
- The primary endpoint is the log
transformed ratio of urine protein-to-creatinine ratio (uPCR) at
week 26 compared to baseline.
- Hematopoietic stem cell
transplantation-associated thrombotic microangiopathy (HSCT-TMA):
Sobi continues to enroll patients in its Phase 2 study evaluating
the efficacy and safety of systemic pegcetacoplan in patients with
HSCT-TMA.
- APL-3007 (small
interfering RNA silencing C3): Continue to expect to report topline
data from the Phase 1 dose escalation study in 2024.
First Quarter 2024 Financial Results
Total Revenue.
- Total revenue was $172.3 million for the first quarter of 2024,
which consisted of $137.5 million of U.S. net product revenue of
SYFOVRE, $25.6 million of U.S. net product revenue of EMPAVELI, and
$9.3 million in licensing and other revenue associated with the
Sobi collaboration.
- Total revenue was $44.8 million for the first quarter of 2023,
which consisted of, $18.4 million of U.S. net product revenue of
SYFOVRE, $20.4 million in net product revenue of EMPAVELI and $6.0
million in revenue associated with the Sobi collaboration.
Cost of Sales.
- Cost of sales was $20.2 million for the first quarter of 2024,
compared to $7.8 million for same period in 2023.
- The increase in cost of sales was primarily driven by an
increase due to higher volume from commercial sales and product
provided under our patient assistance programs, an increase in
royalty expense, and an increase in expenses incurred related to
excess or obsolete inventory.
R&D Expenses.
- R&D expenses were $84.7 million for the first quarter of
2024, compared to $110.0 million for the same period in 2023.
- The decrease in R&D expenses for the first quarter of 2024
was primarily attributable to a decrease in program specific
external costs, a decrease in compensation and related personnel
costs, and a decrease in non-program specific external costs, which
were partially offset by a $15.0 million one-time expense in the
first quarter related to the discontinuation of systemic
pegcetacoplan for cold agglutinin disease (CAD).
Selling, General and Administrative (SG&A) Expenses.
- SG&A expenses were $129.5 million for the first quarter of
2024, compared to $102.1 million for the same period in 2023.
- The increase in SG&A expenses for the first quarter of 2024
was primarily attributable to an increase in personnel related
costs, an increase in professional and consulting fees and general
commercial preparation activities, and higher office costs, which
were partially offset by a decrease in travel expenses and a
decrease in insurance expenses.
Net Loss. Apellis reported a net loss of $66.4 million for the
first quarter 2024, compared to a net loss of $177.8 million for
the same period in 2023.
Cash. As of March 31, 2024, Apellis had $325.9 million in cash
and cash equivalents, compared to $351.2 million in cash and cash
equivalents as of December 31, 2023.
- In February 2024, Apellis received $98.8 million in
non-dilutive, cash proceeds related to the unwind of its capped
call transactions in connection with the issuance of the Company’s
3.500% Senior Convertible Notes due 2026.
- Apellis anticipates its cash balance, combined with projected
sales of EMPAVELI and SYFOVRE, will be sufficient to fund
operations for the foreseeable future.
Conference Call and WebcastApellis will host a
conference call and webcast to discuss its first quarter 2024
financial results and business highlights today, May 7, 2024, at
8:30 a.m. ET. To access the live call by phone, please pre-register
for the call here. A live audio webcast of the event and
accompanying slides may also be accessed through the “Events and
Presentations” page of the “Investors and Media” section of the
company’s website. A replay of the webcast will be available
for 30 days following the event.
About SYFOVRE® (pegcetacoplan
injection)SYFOVRE® (pegcetacoplan injection) is the
first-ever approved therapy for geographic atrophy (GA). By
targeting C3, SYFOVRE is designed to provide comprehensive control
of the complement cascade, part of the body’s immune system.
SYFOVRE is approved in the United States for the treatment of GA
secondary to age-related macular degeneration.
About
EMPAVELI®/Aspaveli® (pegcetacoplan)EMPAVELI®/Aspaveli®
(pegcetacoplan) is a targeted C3 therapy designed to regulate
excessive activation of the complement cascade, part of the body’s
immune system, which can lead to the onset and progression of many
serious diseases. It is approved for the treatment of paroxysmal
nocturnal hemoglobinuria (PNH) in the United States, European
Union, and other countries globally. The therapy is also under
investigation for several other rare diseases across hematology and
nephrology.
U.S. Important Safety Information for
SYFOVRE®(pegcetacoplan injection)
CONTRAINDICATIONS
- SYFOVRE is
contraindicated in patients with ocular or periocular infections,
and in patients with active intraocular inflammation
WARNINGS AND PRECAUTIONS
- Endophthalmitis and
Retinal Detachments
- Intravitreal
injections, including those with SYFOVRE, may be associated with
endophthalmitis and retinal detachments. Proper aseptic injection
technique must always be used when administering SYFOVRE to
minimize the risk of endophthalmitis. Patients should be instructed
to report any symptoms suggestive of endophthalmitis or retinal
detachment without delay and should be managed appropriately.
- Retinal Vasculitis
and/or Retinal Vascular Occlusion
- Retinal vasculitis
and/or retinal vascular occlusion, typically in the presence of
intraocular inflammation, have been reported with the use of
SYFOVRE. Cases may occur with the first dose of SYFOVRE and may
result in severe vision loss. Discontinue treatment with SYFOVRE in
patients who develop these events. Patients should be instructed to
report any change in vision without delay.
- Neovascular AMD
- In clinical trials,
use of SYFOVRE was associated with increased rates of neovascular
(wet) AMD or choroidal neovascularization (12% when administered
monthly, 7% when administered every other month and 3% in the
control group) by Month 24. Patients receiving SYFOVRE should be
monitored for signs of neovascular AMD. In case anti-Vascular
Endothelial Growth Factor (anti-VEGF) is required, it should be
given separately from SYFOVRE administration.
- Intraocular
Inflammation
- In clinical trials,
use of SYFOVRE was associated with episodes of intraocular
inflammation including: vitritis, vitreal cells, iridocyclitis,
uveitis, anterior chamber cells, iritis, and anterior chamber
flare. After inflammation resolves, patients may resume treatment
with SYFOVRE.
- Increased
Intraocular Pressure
- Acute increase in
IOP may occur within minutes of any intravitreal injection,
including with SYFOVRE. Perfusion of the optic nerve head should be
monitored following the injection and managed as needed.
ADVERSE REACTIONS
- Most common adverse reactions (incidence ≥5%) are ocular
discomfort, neovascular age-related macular degeneration, vitreous
floaters, conjunctival hemorrhage.
Please see accompanying full Prescribing
Information for more information.
U.S. Important Safety Information for
EMPAVELI® (pegcetacoplan)
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED
BACTERIA
EMPAVELI, a complement inhibitor, increases the risk of
serious infections, especially those caused by encapsulated
bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis,
and Haemophilus influenzae type B. Life-threatening and fatal
infections with encapsulated bacteria have occurred in patients
treated with complement inhibitors. These infections may become
rapidly life-threatening or fatal if not recognized and treated
early.
- Complete or update vaccination for encapsulated
bacteria at least 2 weeks prior to the first dose of EMPAVELI,
unless the risks of delaying therapy with EMPAVELI outweigh the
risks of developing a serious infection. Comply with the most
current Advisory Committee on Immunization Practices (ACIP)
recommendations for vaccinations against encapsulated bacteria in
patients receiving a complement inhibitor.
- Patients receiving EMPAVELI are at increased risk for
invasive disease caused by encapsulated bacteria, even if they
develop antibodies following vaccination. Monitor patients for
early signs and symptoms of serious infections and evaluate
immediately if infection is suspected.
Because of the risk of serious infections caused by
encapsulated bacteria, EMPAVELI is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy
(REMS) called the EMPAVELI REMS.
CONTRAINDICATIONS
- Hypersensitivity to
pegcetacoplan or to any of the excipients
- For initiation in
patients with unresolved serious infection caused by encapsulated
bacteria including Streptococcus pneumoniae, Neisseria
meningitidis, and Haemophilus influenzae type B
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated
Bacteria
EMPAVELI, a complement inhibitor, increases a patient’s
susceptibility to serious, life-threatening, or fatal infections
caused by encapsulated bacteria including Streptococcus pneumoniae,
Neisseria meningitidis (caused by any serogroup, including
non-groupable strains), and Haemophilus influenzae type B.
Life-threatening and fatal infections with encapsulated bacteria
have occurred in both vaccinated and unvaccinated patients treated
with complement inhibitors. The initiation of EMPAVELI treatment is
contraindicated in patients with unresolved serious infection
caused by encapsulated bacteria.
Complete or update vaccination against encapsulated bacteria at
least 2 weeks prior to administration of the first dose of
EMPAVELI, according to the most current ACIP recommendations for
patients receiving a complement inhibitor. Revaccinate patients in
accordance with ACIP recommendations considering the duration of
therapy with EMPAVELI. Note that, ACIP recommends an administration
schedule in patients receiving complement inhibitors that differs
from the administration schedule in the vaccine prescribing
information. If urgent EMPAVELI therapy is indicated in a patient
who is not up to date with vaccines against encapsulated bacteria
according to ACIP recommendations, provide the patient with
antibacterial drug prophylaxis and administer these vaccines as
soon as possible. The benefits and risks of treatment with
EMPAVELI, as well as the benefits and risks of antibacterial drug
prophylaxis in unvaccinated or vaccinated patients, must be
considered against the known risks for serious infections caused by
encapsulated bacteria.
Vaccination does not eliminate the risk of serious encapsulated
bacterial infections, despite development of antibodies following
vaccination. Closely monitor patients for early signs and symptoms
of serious infection and evaluate patients immediately if an
infection is suspected. Inform patients of these signs and symptoms
and instruct patients to seek immediate medical care if these signs
and symptoms occur. Promptly treat known infections. Serious
infection may become rapidly life-threatening or fatal if not
recognized and treated early. Consider interruption of EMPAVELI in
patients who are undergoing treatment for serious infections.
EMPAVELI is available only through a restricted program under a
REMS.
EMPAVELI REMS
EMPAVELI is available only through a restricted program under a
REMS called EMPAVELI REMS, because of the risk of serious
infections caused by encapsulated bacteria. Notable requirements of
the EMPAVELI REMS include the following:
Under the EMPAVELI REMS, prescribers must enroll in the program.
Prescribers must counsel patients about the risks, signs, and
symptoms of serious infections caused by encapsulated bacteria,
provide patients with the REMS educational materials, ensure
patients are vaccinated against encapsulated bacteria at least 2
weeks prior to the first dose of EMPAVELI, prescribe antibacterial
drug prophylaxis if patients’ vaccine status is not up to date and
treatment must be started urgently, and provide instructions to
always carry the Patient Safety Card both during treatment, as well
as for 2 months following last dose of EMPAVELI. Pharmacies that
dispense EMPAVELI must be certified in the EMPAVELI REMS and must
verify prescribers are certified.
Further information is available at www.empavelirems.com or
1-888-343-7073.
Infusion-Related Reactions
Systemic hypersensitivity reactions (e.g., facial swelling,
rash, urticaria) have occurred in patients treated with EMPAVELI.
One patient (less than 1% in clinical studies) experienced a
serious allergic reaction which resolved after treatment with
antihistamines. If a severe hypersensitivity reaction (including
anaphylaxis) occurs, discontinue EMPAVELI infusion immediately,
institute appropriate treatment, per standard of care, and monitor
until signs and symptoms are resolved.
Monitoring PNH Manifestations after Discontinuation of
EMPAVELI
After discontinuing treatment with EMPAVELI, closely monitor for
signs and symptoms of hemolysis, identified by elevated LDH levels
along with sudden decrease in PNH clone size or hemoglobin, or
reappearance of symptoms such as fatigue, hemoglobinuria, abdominal
pain, dyspnea, major adverse vascular events (including
thrombosis), dysphagia, or erectile dysfunction. Monitor any
patient who discontinues EMPAVELI for at least 8 weeks to detect
hemolysis and other reactions. If hemolysis, including elevated
LDH, occurs after discontinuation of EMPAVELI, consider restarting
treatment with EMPAVELI.
Interference with Laboratory Tests
There may be interference between silica reagents in coagulation
panels and EMPAVELI that results in artificially prolonged
activated partial thromboplastin time (aPTT); therefore, avoid the
use of silica reagents in coagulation panels.
ADVERSE REACTIONS
Most common adverse reactions in patients with PNH (incidence
≥10%) were injection site reactions, infections, diarrhea,
abdominal pain, respiratory tract infection, pain in extremity,
hypokalemia, fatigue, viral infection, cough, arthralgia,
dizziness, headache, and rash.
USE IN SPECIFIC POPULATIONS
Females of Reproductive Potential
EMPAVELI may cause embryo-fetal harm when administered to
pregnant women. Pregnancy testing is recommended for females of
reproductive potential prior to treatment with EMPAVELI. Advise
female patients of reproductive potential to use effective
contraception during treatment with EMPAVELI and for 40 days after
the last dose.
Please see full Prescribing Information, including Boxed
WARNING regarding serious infections caused by encapsulated
bacteria, and Medication Guide.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical
company that combines courageous science and compassion to develop
life-changing therapies for some of the most challenging diseases
patients face. We ushered in the first new class of complement
medicine in 15 years and now have two approved medicines targeting
C3. These include the first-ever therapy for geographic atrophy, a
leading cause of blindness around the world. We believe we have
only begun to unlock the potential of targeting C3 across serious
retinal, rare, and neurological diseases. For more information,
please visit http://apellis.com or follow us
on Twitter and LinkedIn.
Apellis Forward-Looking StatementStatements
in this press release about future expectations, plans and
prospects, as well as any other statements regarding matters that
are not historical facts, may constitute “forward-looking
statements” within the meaning of The Private Securities Litigation
Reform Act of 1995. These statements include, but are not limited
to, statements regarding the expected timing of clinical data, the
review of the marketing authorization application of SYFOVRE by the
EMA. The words “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “intend,” “may,” “plan,” “potential,”
“predict,” “project,” “should,” “target,” “will,” “would” and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Actual results may differ materially from
those indicated by such forward-looking statements as a result of
various important factors, including whether the benefit/risk
profile of SYFOVRE following the events of retinal vasculitis will
impact the Company’s commercialization efforts; whether SYFOVRE
will receive approval from foreign regulatory agencies for GA when
expected or at all, including the impact of the reported events of
retinal vasculitis on the likelihood and timing of such approvals;
whether the Company’s clinical trials will be completed when
anticipated; whether results obtained in clinical trials will be
indicative of results that will be generated in future clinical
trials; whether pegcetacoplan will successfully advance through the
clinical trial process on a timely basis, or at all; whether the
results of the Company’s clinical trials will warrant regulatory
submissions and whether systemic pegcetacoplan will receive
approval from the FDA or equivalent foreign regulatory agencies for
C3G and IC-MPGN or any other indication when expected or at all;
the period for which the Company believes that its cash resources
will be sufficient to fund its operations; and other factors
discussed in the “Risk Factors” section of Apellis’ Annual Report
on Form 10-K with the Securities and Exchange Commission on
February 27, 2024 and the risks described in other filings that
Apellis may make with the Securities and Exchange Commission. Any
forward-looking statements contained in this press release speak
only as of the date hereof, and Apellis specifically disclaims any
obligation to update any forward-looking statement, whether as a
result of new information, future events or otherwise.
Media Contact:Lissa
Pavlukmedia@apellis.com617.977.6764
Investor Contact:Meredith
Kayameredith.kaya@apellis.com617.599.8178
|
|
|
APELLIS PHARMACEUTICALS, INC. |
CONDENSED CONSOLIDATED BALANCE SHEETS |
(Amounts in thousands, except per share
amounts) |
|
March 31, |
|
December 31, |
|
|
2024 |
|
|
|
2023 |
|
Assets |
(Unaudited) |
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
325,923 |
|
|
$ |
351,185 |
|
Accounts receivable |
|
267,837 |
|
|
|
206,442 |
|
Inventory |
|
161,283 |
|
|
|
146,362 |
|
Prepaid assets |
|
43,163 |
|
|
|
38,820 |
|
Restricted cash |
|
1,103 |
|
|
|
1,114 |
|
Other current assets |
|
12,119 |
|
|
|
22,408 |
|
Total current assets |
|
811,428 |
|
|
|
766,331 |
|
Non-current assets: |
|
|
|
Right-of-use assets |
|
14,994 |
|
|
|
16,745 |
|
Property and equipment, net |
|
4,195 |
|
|
|
4,345 |
|
Other assets |
|
1,313 |
|
|
|
1,309 |
|
Total assets |
$ |
831,930 |
|
|
$ |
788,730 |
|
Liabilities and Stockholders' Equity |
|
|
|
Current liabilities: |
|
|
|
Accounts payable |
$ |
26,788 |
|
|
|
37,516 |
|
Accrued expenses |
|
101,399 |
|
|
|
127,806 |
|
Current portion of development liability |
|
77,287 |
|
|
|
75,830 |
|
Current portion of lease liabilities |
|
6,257 |
|
|
|
6,441 |
|
Deferred Revenue |
|
3,560 |
|
|
|
— |
|
Total current liabilities |
|
215,291 |
|
|
|
247,593 |
|
Long-term liabilities: |
|
|
|
Long-term development liability |
|
244,426 |
|
|
|
239,817 |
|
Convertible senior notes |
|
93,109 |
|
|
|
93,033 |
|
Lease liabilities |
|
9,770 |
|
|
|
11,454 |
|
Other liabilities |
|
2,658 |
|
|
|
2,312 |
|
Total liabilities |
|
565,254 |
|
|
|
594,209 |
|
Stockholders' equity: |
|
|
|
Preferred stock, $0.0001 par value; 10,000 shares authorized and
zero shares issued and outstanding at March 31, 2024 and December
31, 2023 |
|
— |
|
|
|
— |
|
Common stock, $0.0001 par value; 200,000 shares authorized at March
31, 2024 and December 31, 2023; 121,267 shares issued and
outstanding at March 31, 2024, and 119,556 shares issued and
outstanding at December 31, 2023 |
|
12 |
|
|
|
12 |
|
Additional paid-in capital |
|
3,174,100 |
|
|
|
3,035,539 |
|
Accumulated other comprehensive loss |
|
(3,525 |
) |
|
|
(3,542 |
) |
Accumulated deficit |
|
(2,903,911 |
) |
|
|
(2,837,488 |
) |
Total stockholders' equity |
|
266,676 |
|
|
|
194,521 |
|
Total liabilities and stockholders' equity |
$ |
831,930 |
|
|
$ |
788,730 |
|
|
|
|
|
APELLIS PHARMACEUTICALS, INC. |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS |
(Amounts in thousands, except per share
amounts) |
|
|
|
|
|
For the Three Months Ended March 31, |
|
|
2024 |
|
|
|
2023 |
|
|
(Unaudited) |
Revenue: |
|
|
|
Product revenue, net |
$ |
163,075 |
|
|
$ |
38,800 |
|
Licensing and other revenue |
|
9,250 |
|
|
|
6,046 |
|
Total revenue: |
|
172,325 |
|
|
|
44,846 |
|
Operating expenses: |
|
|
|
Cost of sales |
|
20,209 |
|
|
|
7,809 |
|
Research and development |
|
84,701 |
|
|
|
110,027 |
|
Selling, general and administrative |
|
129,505 |
|
|
|
102,093 |
|
Operating expenses: |
|
234,415 |
|
|
|
219,929 |
|
Net operating loss |
|
(62,090 |
) |
|
|
(175,083 |
) |
Interest income |
|
3,303 |
|
|
|
5,393 |
|
Interest expense |
|
(6,967 |
) |
|
|
(7,529 |
) |
Other (expense) /income, net |
|
(499 |
) |
|
|
(277 |
) |
Net loss before taxes |
|
(66,253 |
) |
|
|
(177,496 |
) |
Income tax expense |
|
170 |
|
|
|
282 |
|
Net loss |
$ |
(66,423 |
) |
|
$ |
(177,778 |
) |
Other comprehensive gain/(loss): |
|
|
|
Foreign currency translation |
|
17 |
|
|
|
100 |
|
Total other comprehensive income |
|
17 |
|
|
|
100 |
|
Comprehensive loss, net of tax |
$ |
(66,406 |
) |
|
$ |
(177,678 |
) |
Net loss per common share, basic and diluted |
$ |
(0.54 |
) |
|
$ |
(1.56 |
) |
Weighted-average number of common shares used in net loss per
common share, basic and diluted |
|
122,957 |
|
|
|
113,872 |
|
|
|
|
|
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