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Marketing application for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) is under review by the U.S. Food and Drug Administration (FDA) with a PDUFA target action date of May 14, 2021
Top-line results from the Phase 3 PRINCE study in treatment-naïve PNH patients expected in the second quarter of 2021
Top-line results from Phase 3 geographic atrophy (GA) studies expected in the third quarter of 2021
Three new product candidates advancing into clinical development by the end of 2022
Cash and investments of $723.7 million as of March 31, 2021 support cash runway into the second half of 2022
Conference call scheduled today at 4:30 p.m. ET
WALTHAM, Mass., April 28, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its first quarter 2021 financial results and business highlights.
“With a potential U.S. approval for pegcetacoplan just a couple of weeks away, we are at the beginning of a transformational year for Apellis. Our commercial team is prepared to successfully execute our first product launch and meet the needs of PNH patients,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “At the same time, we are focused on advancing registrational programs of pegcetacoplan across several debilitating rare diseases and delivering on our goal to become number one in the retina. Geographic atrophy is the most significant remaining unmet need in the retina, and we are excited to see top-line results from our Phase 3 studies of pegcetacoplan in GA in the third quarter of this year.
“For more than a decade, our team has built the foundation for Apellis’ leadership in complement, and we look forward to seeing the results of those efforts come together this year for patients living with serious, complement-driven diseases,” Dr. Francois continued.
First Quarter 2021 Business Highlights and Upcoming Milestones:
Systemic Pegcetacoplan in Rare Disease
In March 2021, Apellis and Sobi announced that the New England Journal of Medicine published results from the Phase 3 PEGASUS study. The data at 16 weeks showed that pegcetacoplan, an investigational targeted C3 therapy, demonstrated statistically superior increases in mean hemoglobin levels compared with the C5 inhibitor Soliris® (eculizumab) and provided substantial improvements in other key markers of disease in adults with PNH who had persistent anemia following treatment with Soliris.
Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The New Drug Application was granted Priority Review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of May 14, 2021. The Marketing Authorization Application has the potential for a European Commission decision in the second half of 2021.
In the second quarter 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 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 Ophthalmology
In April 2021, Apellis announced 24-month data from the Phase 1b study of intravitreal pegcetacoplan in patients with advanced geographic atrophy (GA) and low vision. Data from a post hoc analysis demonstrated a 46% decrease in the growth rate of GA lesions in the treated eye compared to the untreated eye in eight patients with bilateral GA (disease in both eyes) at 24 months (p=0.007).
In March 2021, Apellis announced that two leading journals, Ophthalmology and the American Journal of Ophthalmology, published post hoc analyses from the Phase 2 FILLY study of intravitreal pegcetacoplan for GA secondary to age-related macular degeneration. The published data underscore the potential of pegcetacoplan for GA.
The company expects to announce top-line results from the Phase 3 DERBY and OAKS studies in the third quarter of 2021.
APL-9 in COVID-19
In March 2021, Apellis announced that the company will not pursue additional development of APL-9, an investigational targeted C3 therapy designed for acute interventions, for the treatment of severe COVID-19. The decision followed an interim review of mortality data from the Phase 1/2 study by an independent data monitoring committee (DMC), which found no meaningful reduction in the overall mortality rate in patients treated with APL-9 in combination with standard of care therapy compared to standard of care alone. No safety signals were observed by the DMC.
Apellis plans to advance three new product candidates into clinical development by the end of 2022.
First Quarter 2021 Financial Results:
As of March 31, 2021, Apellis had $723.7 million in cash, cash equivalents, and short-term marketable securities, compared to $646.7 million in cash, cash equivalents, and short-term marketable securities as of March 31, 2020. This increase primarily reflects the addition of cash from the company’s convertible offering for gross proceeds of $328.9 million in May 2020, the receipt of $250.0 million in the upfront proceeds for the Sobi transaction in October 2020, and an additional $25.0 million receipt from Sobi in January 2021 less cash used in operations.
Apellis reported a net loss of $183.7 million for the first quarter of 2021, compared to a net loss of $168.8 million for the same period in 2020.
Research and development expenses were $84.0 million in the first quarter of 2021, compared to $69.3 million for the same period in 2020. The increase in R&D expense for first quarter 2021 was primarily attributable to an increase in clinical trial costs associated with the ongoing Phase 3 trials and the preparation and commencement of our clinical trials in other indications, personnel-related costs primarily due to the hiring of additional personnel, and increased quality and medical affairs 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 $40.6 million in the first quarter of 2021, compared to $29.5 million for the same period in 2020. The increase in general and administrative expenses for the first quarter 2021 was primarily attributable to an increase in employee-related costs, professional and consulting fees, general commercial preparation activities, director stock compensation expense, and insurance.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its first quarter 2021 financial results and business highlights today, April 28, 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 7883183. 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.
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 U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.
APL-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.
Apellis 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 Statement
Statements 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’ Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 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.
APELLIS PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(Amounts in thousands, except per share amounts)
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Other current assets
Total current assets
Property and equipment, net
Liabilities and Stockholders' Equity
Current portion of development derivative liability
Current portion of right-of-use liabilities
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Convertible senior notes
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Commitments and contingencies (note 13)
Preferred stock, $0.0001 par value; 10,000 shares authorized, and zero shares issued and outstanding at March 31, 2021 and December 31, 2020
Common stock, $0.0001 par value; 200,000 shares authorized at March 31, 2021 and December 31, 2020; 80,438 shares issued and outstanding at March 31, 2021, and 76,130 shares issued and outstanding at December 31, 2020
Additional paid-in capital
Accumulated other comprehensive loss
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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,
Research and development
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Loss from remeasurement of development derivative liability
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Weighted-average number of common shares used in net loss per common share, basic and diluted