Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global
biopharmaceutical company and leader in targeted C3 therapies,
today announced its fourth quarter and full year 2020 financial
results and business highlights.
“2020 was a defining year for Apellis, marked by positive Phase
3 PEGASUS data, which highlighted both the potential of
pegcetacoplan to elevate the standard of care in PNH and the broad
platform potential of targeting C3 for complement-driven diseases,”
said Cedric Francois M.D., Ph.D., co-founder and chief executive
officer of Apellis. “In 2021, we look forward to a transformational
year for Apellis as we further build on our global leadership in
complement.
“With a potential U.S. product approval and commercial launch in
PNH in the first half of the year, Phase 3 study readouts in
geographic atrophy in the third quarter, and multiple clinical
development milestones across our systemic pipeline, we are
positioned to deliver on the full potential of targeting C3 across
multiple diseases with high unmet need,” Dr. Francois continued.
“Importantly, we have a unique opportunity to advance the first
potential medicine for geographic atrophy, a leading cause of
blindness that affects approximately five million people around the
world.”
Fourth Quarter Business Highlights and Upcoming
Milestones:
Systemic Pegcetacoplan
- In December 2020, Apellis and Sobi announced positive top-line
results at Week 48 from the Phase 3 PEGASUS study, which found that
treatment with pegcetacoplan, an investigational targeted C3
therapy, resulted in sustained hematological and clinical
improvements in patients with paroxysmal nocturnal hemoglobinuria
(PNH). The safety profile of pegcetacoplan was consistent with
previously reported data.
- In December 2020, the company reported additional analyses from
the Phase 3 PEGASUS study, demonstrating statistically significant
improvements in overall treatment response and substantial
quality-of-life improvements with pegcetacoplan for PNH compared to
Soliris® (eculizumab) at 16 weeks. Additionally, a
matching-adjusted indirect comparison (MAIC) suggested improvements
in clinical, hematological, and quality-of-life outcomes in
patients treated with pegcetacoplan compared to Ultomiris®
(ravulizumab), a longer-acting C5 inhibitor. The data were
presented at the virtual American Society of Hematology Annual
Meeting (ASH).
- In November 2020, Apellis and Sobi announced that the first
patient had been dosed in the potentially registrational Phase 2
MERIDIAN study of pegcetacoplan, which is expected to enroll more
than 200 adults with sporadic amyotrophic lateral sclerosis (ALS).
The companies expect to complete study enrollment in the second
half of 2021.
- In November 2020, the company announced that the FDA accepted
and granted Priority Review designation for the New Drug
Application (NDA) for pegcetacoplan for the treatment of PNH, with
a Prescription Drug User Fee Act (PDUFA) target action date of May
14, 2021. A Marketing Authorization Application (MAA) is also under
review by the EMA with the potential for a European Commission
decision on the MAA in the second half of 2021.
- Apellis and Sobi expect to report top-line results from the
Phase 3 PRINCE study in PNH patients who are treatment-naïve in the
second quarter of 2021.
- In the second half of 2021, Apellis expects to initiate a Phase
3 study in immune complex membranoproliferative glomerulonephritis
(IC-MPGN) and C3 glomerulopathy (C3G), and Sobi plans to initiate a
Phase 3 study in cold agglutinin disease (CAD) and a potentially
registrational Phase 2 study in hematopoietic stem cell
transplantation-associated thrombotic microangiopathy
(HSCT-TMA).
Intravitreal Pegcetacoplan
- In November 2020, Apellis announced findings from the largest
retrospective study in GA, which underscored the high unmet need
for a treatment in geographic atrophy. Conducted in partnership
with Verana Health, the analysis of the American Academy of
Ophthalmology (AAO) IRIS® Registry was presented in a late-breaking
oral session at AAO 2020. Results showed that visual acuity
substantially worsened over a brief time, with a significant
percentage of GA patients becoming ineligible to drive and legally
blind within one to two years of diagnosis. The data also
reiterated that wet AMD is an expected occurrence in people with
GA, which happens nearly three times more frequently in people with
wet AMD in the fellow eye than in people with bilateral GA (13.3%
vs. 4.7%, respectively, over 12 months).
- The company expects to announce top-line results from the Phase
3 DERBY and OAKS studies in the third quarter of 2021.
COVID-19 Clinical Program with APL-9
- Apellis expects to report top-line data from its Phase 1/2
clinical study of APL-9, a targeted C3 therapy designed for acute
conditions, in patients with severe COVID-19 in the second quarter
of 2021.
Pipeline Expansion
- Apellis plans to advance three new product candidates into
clinical development by the end of 2022.
Corporate Highlights
- In January 2021, Apellis announced that it entered into
separate, privately negotiated exchange agreements with certain
holders of its 3.500% Convertible Senior Notes due 2026 issued in
September 2019. Under the terms of the exchange agreements, the
holders agreed to exchange with Apellis approximately $126.1
million in aggregate principal amount of Notes held by them for an
aggregate of 3,906,869 shares of its common stock. The exchange
transactions closed on January 25, 2021.
Fourth Quarter and Full Year 2020 Financial
Results:
As of December 31, 2020, Apellis had $877.6 million in cash,
cash equivalents, and short-term marketable securities, compared to
$352.0 million in cash and cash equivalents as of December 31,
2019. This increase primarily reflects the addition of cash from
the company’s follow-on offering for gross proceeds of $404.2
million in January 2020, a convertible offering for gross proceeds
of $328.9 million in May 2020, and the receipt of $250.0 million in
the upfront proceeds for the Sobi transaction in October 2020, less
cash used in operations.
Apellis reported net income of $78.3 million for the fourth
quarter of 2020, compared to a net loss of $113.2 million for the
fourth quarter of 2019. This change was due to the Sobi upfront
payment, which was recognized as revenue in the fourth quarter. For
the full year ending December 31, 2020, Apellis reported a net loss
of $344.9 million, compared to a net loss of $304.7 million for the
full year ending December 31, 2019.
Research and development expenses were $75.4 million in the
fourth quarter of 2020, compared to $78.5 million for the same
period in 2019. For the full year ending December 31, 2020,
research and development expenses were $325.0 million compared to
$221.0 million for the full year ending December 31, 2019. The
increase in R&D expense for full year 2020 was primarily
attributable to an increase in manufacturing expenses in connection
with the supply of pegcetacoplan for the company’s Phase 3 clinical
trials and potential commercial launch, clinical trial costs
associated with the ongoing Phase 3 trials and planned clinical
trials in other indications, compensation and related personnel
costs primarily due to the hiring of additional personnel,
regulatory and quality expenses, licensee fee to Penn related to
the Sobi transaction, research and innovation expense, and device
development expenses. We expect our research and development
expenses to continue to increase as the number of patients in our
trials increases and the number of ongoing trials increases.
General and administrative expenses were $44.5 million in the
fourth quarter of 2020, compared to $27.5 million for the same
period in 2019. For the full year ending December 31, 2020, general
and administrative expenses were $139.4 million, compared to $67.0
million for the full year ending December 31, 2019. The increase in
general and administrative expenses for the full year 2020 was
primarily attributable to an increase in professional and
consulting fees, employee-related costs due to the hiring of
additional personnel, directors stock compensation expense,
insurance costs, and office, travel and related costs, offset by a
decrease in information technology expenses.
Conference Call and WebcastApellis will host a
conference call and webcast to discuss its fourth quarter and full
year 2020 financial results and business highlights today, February
25, 2021, at 4:30 p.m. ET. To access the conference call, please
dial (866) 774-0323 (local) or (602) 563-8683 (international) at
least 10 minutes prior to the start time and refer to conference ID
6956712. 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 at
http://investors.apellis.com/events-and-presentations. A replay of
the webcast will be available for 30 days following the event.
About Pegcetacoplan Pegcetacoplan is an
investigational, 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. Pegcetacoplan is a synthetic cyclic peptide conjugated to
a polyethylene glycol polymer that binds specifically to C3 and
C3b. Pegcetacoplan is being evaluated in several clinical studies
across hematology, ophthalmology, nephrology, and neurology.
Marketing applications for pegcetacoplan for paroxysmal nocturnal
hemoglobinuria (PNH) are under review by the U.S. Food and Drug
Administration (FDA), which has granted the application Priority
Review designation, and the European Medicines Agency (EMA).
Pegcetacoplan was granted Fast Track designation by the FDA for the
treatment of geographic atrophy and received orphan drug
designation for the treatment of C3G by the FDA and EMA. For
additional information regarding pegcetacoplan clinical trials,
visit https://apellis.com/our-science/clinical-trials.
About APL-9APL-9 is an investigational,
targeted C3 therapy designed to control the complement cascade
centrally and may have the potential to treat a range of diseases
caused by excessive activation of complement. APL-9 leverages the
same mechanism of action as Apellis’ lead compound, pegcetacoplan,
but has a lower molecular weight and shorter half-life. APL-9 is
designed to be intravenously administered for acute use.
About ApellisApellis Pharmaceuticals, Inc. is a
global biopharmaceutical company that is committed to leveraging
courageous science, creativity, and compassion to deliver
life-changing therapies. Leaders in targeted C3 therapies, we aim
to develop transformative therapies for a broad range of
debilitating diseases that are driven by excessive activation of
the complement cascade, including those within hematology,
ophthalmology, nephrology, and neurology. For more information,
please visit www.apellis.com.
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 in respect of the expected closing of the exchanges. 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 conditions for the closing
of the exchanges will be satisfied and other factors discussed in
the “Risk Factors” section of Apellis’ Annual Report on Form 10-K
filed with the Securities and Exchange Commission on
February 25, 2021 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.Investor Contact:Argot
Partnersapellis@argotpartners.com +1 212.600.1902
Media Contact:Tracy
Vineismedia@apellis.com 617.420.4839
|
|
APELLIS
PHARMACEUTICALS, INC. |
CONDENSED
CONSOLIDATED BALANCE SHEETS |
(Amounts in
thousands, except per share amounts) |
|
|
December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
565,779 |
|
|
$ |
351,985 |
|
Marketable securities |
|
|
311,869 |
|
|
|
- |
|
Prepaid assets |
|
|
11,400 |
|
|
|
19,802 |
|
Restricted cash |
|
|
1,266 |
|
|
|
- |
|
Other current assets |
|
|
26,878 |
|
|
|
1,308 |
|
Total current assets |
|
|
917,192 |
|
|
|
373,095 |
|
Non-current
assets: |
|
|
|
|
Right-of-use assets |
|
|
17,719 |
|
|
|
14,110 |
|
Property and equipment, net |
|
|
6,803 |
|
|
|
1,655 |
|
Other assets |
|
|
18,855 |
|
|
|
385 |
|
Total
assets |
|
$ |
960,569 |
|
|
$ |
389,245 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
8,477 |
|
|
$ |
8,361 |
|
Accrued expenses |
|
|
111,935 |
|
|
|
54,783 |
|
Current portion of development derivative liability |
|
|
4,230 |
|
|
|
- |
|
Current portion of right-of-use liabilities |
|
|
3,685 |
|
|
|
2,609 |
|
Total current liabilities |
|
|
128,327 |
|
|
|
65,753 |
|
Long-term
liabilities: |
|
|
|
|
Convertible senior notes |
|
|
358,830 |
|
|
|
142,567 |
|
Development derivative liability |
|
|
253,638 |
|
|
|
134,839 |
|
Right-of-use liabilities |
|
|
15,217 |
|
|
|
11,857 |
|
Total
liabilities |
|
|
756,012 |
|
|
|
355,016 |
|
Commitments
and contingencies (note 15) |
|
|
- |
|
|
|
- |
|
Stockholders' equity: |
|
|
|
|
Preferred stock, $0.0001 par value; 10,000 shares authorized and
zero shares issued and outstanding at December 31, 2020 and
2019 |
|
|
- |
|
|
|
- |
|
Common stock, $0.0001 par value; 200,000 shares authorized at
December 31, 2020 and 2019; 76,130 and 63,938 shares issued and
outstanding at December 31, 2020 and 2019, respectively |
|
|
8 |
|
|
|
6 |
|
Additional paid-in capital |
|
|
1,131,013 |
|
|
|
615,850 |
|
Accumulated other comprehensive loss |
|
|
(117 |
) |
|
|
(154 |
) |
Accumulated deficit |
|
|
(926,347 |
) |
|
|
(581,473 |
) |
Total
stockholders' equity |
|
|
204,557 |
|
|
|
34,229 |
|
Total
liabilities and stockholders' equity |
|
$ |
960,569 |
|
|
$ |
389,245 |
|
|
|
|
|
|
|
|
|
|
APELLIS
PHARMACEUTICALS, INC. |
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE
LOSS |
(Amounts in
thousands, except per share amounts) |
|
|
|
|
|
|
|
|
|
For the three months ended December 31, |
|
Year Ended December 31, |
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
Revenue: |
|
|
|
|
|
|
|
Licensing revenue |
$ |
250,000 |
|
|
$ |
— |
|
|
$ |
250,494 |
|
|
$ |
— |
|
Collaboration revenue |
|
— |
|
|
|
— |
|
|
|
152 |
|
|
|
Total
Revenue: |
|
250,000 |
|
|
|
— |
|
|
|
250,646 |
|
|
|
— |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
50,337 |
|
|
|
78,471 |
|
|
|
299,921 |
|
|
|
220,969 |
|
License expense |
|
25,050 |
|
|
|
— |
|
|
|
25,050 |
|
|
|
- |
|
General and administrative |
|
44,492 |
|
|
|
27,469 |
|
|
|
139,401 |
|
|
|
67,046 |
|
Operating
expenses: |
|
119,879 |
|
|
|
105,940 |
|
|
|
464,372 |
|
|
|
288,015 |
|
Operating
loss |
|
130,121 |
|
|
|
(105,940 |
) |
|
|
(213,726 |
) |
|
|
(288,015 |
) |
Loss on
extinguishment of debt |
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(1,501 |
) |
Loss from
remeasurement of development derivative liability |
|
(40,090 |
) |
|
|
(4,736 |
) |
|
|
(103,029 |
) |
|
|
(14,839 |
) |
Interest
income |
|
194 |
|
|
|
1,478 |
|
|
|
4,164 |
|
|
|
5,108 |
|
Interest
expense |
|
(9,610 |
) |
|
|
(3,930 |
) |
|
|
(29,937 |
) |
|
|
(5,285 |
) |
Other income
(expense), net |
|
(505 |
) |
|
|
(89 |
) |
|
|
(501 |
) |
|
|
(175 |
) |
Net loss
before taxes |
|
80,110 |
|
|
|
(113,217 |
) |
|
|
(343,029 |
) |
|
|
(304,707 |
) |
Income tax
expense |
|
1,845 |
|
|
|
— |
|
|
|
1,845 |
|
|
|
- |
|
Net
income/(loss) |
|
78,265 |
|
|
|
(113,217 |
) |
|
|
(344,874 |
) |
|
|
(304,707 |
) |
Other
comprehensive loss: |
|
|
|
|
|
|
|
Unrealized gain loss |
|
(130 |
) |
|
|
— |
|
|
|
(8 |
) |
|
|
— |
|
Foreign currency gain/(loss) |
|
1,772 |
|
|
|
51 |
|
|
|
45 |
|
|
|
(31 |
) |
Total other
comprehensive loss |
|
1,642 |
|
|
|
51 |
|
|
|
37 |
|
|
|
(31 |
) |
Comprehensive income/(loss), net of tax |
$ |
79,907 |
|
|
$ |
(113,166 |
) |
|
$ |
(344,837 |
) |
|
$ |
(304,738 |
) |
Net
income/(loss) per common share, basic |
$ |
1.03 |
|
|
$ |
(1.77 |
) |
|
$ |
(4.59 |
) |
|
$ |
(4.90 |
) |
Net
income/(loss) per common share, diluted |
$ |
0.93 |
|
|
$ |
(1.77 |
) |
|
$ |
(4.59 |
) |
|
$ |
(4.90 |
) |
Weighted-average number of common shares used in net loss per
common share, basic |
|
75,875 |
|
|
|
63,901 |
|
|
|
75,163 |
|
|
|
62,229 |
|
Weighted-average number of common shares used in net loss per
common share, diluted |
|
94,321 |
|
|
|
63,901 |
|
|
|
75,163 |
|
|
|
62,229 |
|
|
|
|
|
|
|
|
|
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