• Received U.S. FDA approval for EMPAVELI^® (pegcetacoplan) for treatment of patients 12 years and older with C3G and primary IC-MPGN
  
• Announced capped royalty purchase agreement with Sobi under which Apellis receives up to $300 million for 90% of ex-U.S. royalties of Aspaveli^® (pegcetacoplan)
  
• Generated $178 million in 2Q 2025 revenues, including $171 million in U.S. net product sales
  
• SYFOVRE^® (pegcetacoplan injection) injection demand grew 6% quarter-over-quarter, with U.S. net product revenue of $151 million
  
• Cash and cash equivalents of $370 million as of June 30, 2025; existing cash, $275 million payment from Sobi, and future product sales expected to be sufficient to fund business to sustainable profitability.
  
• Management to host conference call today at 8:30 a.m. ET

WALTHAM, Mass., July 31, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its second quarter 2025 financial results and business highlights.

“We are incredibly proud of the recent FDA approval of EMPAVELI in C3G and primary IC-MPGN and are now focused on bringing this transformational therapy to patients. With two C3-targeting medicines approved across four serious diseases, we have further cemented our position as a leader in complement,” said Cedric Francois, M.D., Ph.D., chief executive officer at Apellis. “On top of our regulatory success, we were encouraged to see SYFOVRE’s continued market leadership in GA.”

Second Quarter 2025 Business Highlights and Upcoming Milestones

Maximizing EMPAVELI’s impact in rare diseases

• C3 glomerulopathy (C3G) and primary immune complex glomerulonephritis (IC-MPGN):
  • EMPAVELI was approved by the U.S. Food and Drug Administration (FDA) as the first treatment for C3G and primary IC-MPGN for patients 12 and older. The approval was based on the Phase 3 VALIANT study results, which demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo.
    • EMPAVELI is the first and only U.S. FDA approved treatment for primary IC-MPGN, adolescent patients with C3G, and post-transplant C3G disease recurrence
    • New 52-week data from the VALIANT study, presented at the European Renal Association (ERA) Congress in June, demonstrated sustained efficacy, including robust proteinuria reduction and stable kidney function across a broad patient population. Safety data remained favorable and continued to show an acceptable profile.
    • Sobi, the Company’s exclusive ex-U.S. commercialization partner, expects an opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for its indication extension application for Aspaveli (the brand name for EMPAVELI outside the U.S.) before year-end 2025
      
• Paroxysmal nocturnal hemoglobinuria (PNH):
  • Recorded $20.8 million in EMPAVELI U.S. net product revenue for the second quarter 2025
  • Continued high patient compliance rates of 97%
    
• Delayed graft function (DGF) and Focal segmental glomerulosclerosis (FSGS)
  • On track to initiate pivotal studies in 2H 2025, one in DGF and one in FSGS, two rare kidney diseases in which the complement pathway plays a significant role and there are no approved therapies

Transforming the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD)

• SYFOVRE:
  • Generated $150.6 million in SYFOVRE U.S. net product revenue in the second quarter of 2025
  • Total injections grew 6% quarter-over-quarter
  • SYFOVRE remains the clear market leader in GA with 55% of new patient starts and total market share exceeding 60% during the quarter
  • Utilization of SYFOVRE free goods remained high during the quarter and impacted revenue by approximately $13 million
  • Delivered approximately 95K SYFOVRE doses to physician offices, including ~82K commercial doses and ~13K free goods doses
  • Initiated the Phase 2 study of APL-3007 + SYFOVRE; potential next generation treatment aimed at comprehensively blocking complement activity in the retina and choroid

Advancing our innovative pipeline and leveraging our complement expertise

• Advancing investigational pre-clinical research for one-time neonatal Fc receptor (FcRn) treatment using gene editing technology from Beam Therapeutics
  

Business Update

• Apellis and Sobi announced a capped royalty purchase agreement in July in which Apellis will receive up to $300 million in exchange for 90% of Apellis’ future ex-U.S. royalties for Aspaveli.
  • Per the companies’ 2020 collaboration agreement, Apellis is eligible for tiered royalties on ex-U.S. sales of Aspaveli ranging from high teens to high twenties.
  • Under the terms of the newly announced royalty purchase agreement, Sobi acquired 90% of Apellis’ ex-U.S. royalties for Aspaveli for $275 million in cash. Apellis is also eligible for up to $25 million in milestone payments upon EMA approval of Aspaveli for C3G and IC-MPGN.
  • The agreement is subject to defined caps tied to Aspaveli’s performance. Sobi retains 90% of ex-U.S. royalties until these caps are achieved, after which 100% of all ex-U.S. royalties revert to Apellis.

Organizational Updates

• Leslie Meltzer, Ph.D., has been named chief research and development officer, effective August 25. An experienced biopharmaceutical leader, she brings a proven track record of advancing programs from discovery through global approvals and commercialization. Most recently, Leslie served as chief medical officer at Orchard Therapeutics, where she led the company’s development functions and was instrumental in securing global regulatory approvals. She previously held senior roles at Keryx, Biogen, and Actelion Pharmaceuticals. Leslie holds a Ph.D. in neuroscience from Stanford University and a bachelor’s degree in biology and neuroscience from Brandeis University.
• Kelley Boucher recently joined Apellis as chief people officer, bringing more than 20 years of human resources experience across the biotechnology and medical device industries. Most recently, she served as chief human resources officer at Alnylam Pharmaceuticals, where she led the global HR organization during a period of significant growth and industry recognition. She previously held senior HR roles at Abiomed and Shire. Kelley holds a bachelor’s degree in government relations from Cornell University.

Second Quarter 2025 Financial Results

Total Revenue

Total revenue was $178.5 million for the second quarter of 2025, which consisted of $150.6 million of SYFOVRE U.S. net product revenue, $20.8 million of EMPAVELI U.S. net product revenue, and $7.1 million in licensing and other revenue associated with the Sobi collaboration. Total revenue was $199.7 million for the second quarter of 2024, which consisted of $154.6 million of SYFOVRE U.S. net product revenue, $24.5 million in EMPAVELI U.S. net product revenue, and $20.5 million in licensing and other revenue associated with the Sobi collaboration.

Cost of Sales

Cost of sales was $13.6 million for the second quarter 2025, compared to $23.1 million for the same period in 2024. The decrease in cost of sales was primarily driven by lower volumes of product supplied to Sobi and a decrease in expenses incurred related to excess, obsolete or scrapped inventory. The decreases were partially offset by higher volume from commercial sales and product provided under our patient assistance programs, as well as increased costs incurred in connection with cancellable purchase commitments.

R&D Expenses

R&D expenses were $67.0 million for the second quarter of 2025, compared to $78.0 million for the same period in 2024. The decrease in R&D expenses was primarily driven by lower program-specific and non-program-specific external costs, reduced compensation and related personnel costs, and lower other expenses.

Selling, General and Administrative (SG&A) Expenses

SG&A expenses were $131.1 million for the second quarter of 2025, compared to $128.1 million for the same period in 2024. The increase in SG&A was primarily driven by higher office and travel expenses, professional and consulting fees, factoring fees, and insurance expenses, partially offset by lower personnel costs.

Net Loss

Apellis reported a net loss of $42.2 million for the second quarter 2025, compared to a net loss of $37.7 million for the same period in 2024.

Cash 

As of June 30, 2025, Apellis had $370.0 million in cash and cash equivalents, compared to $411.3 million in cash and cash equivalents as of December 31, 2024. Apellis anticipates its cash, combined with expected product revenues and funds from the Sobi royalty purchase agreement, will fund the business to profitability.

Conference Call and Webcast

Apellis will host a conference call and webcast to discuss its second quarter 2025 financial results and business highlights today, July 31, 2025, 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 90 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 and Australia.  

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 C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the United States and paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for other rare diseases.

About the Apellis and Sobi Collaboration

Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialization. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

U.S. Important Safety Information for SYFOVRE^® (pegcetacoplan injection)

CONTRAINDICATIONS

• SYFOVRE is contraindicated in patients with ocular or periocular infections, in patients with active intraocular inflammation, and in patients with hypersensitivity to pegcetacoplan or any of the excipients in SYFOVRE. Systemic hypersensitivity reactions (e.g., anaphylaxis, rash, urticaria) have occurred.
  

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 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 (eg, facial swelling, rash, urticaria, pyrexia) have occurred in patients treated with EMPAVELI, which may resolve after treatment with antihistamines. Cases of anaphylaxis leading to treatment discontinuation have been reported. 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 Paroxysmal Nocturnal Hemoglobinuria (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 adult 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.

Most common adverse reactions in adult and pediatric patients 12 years of age and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) (incidence ≥10%) were injection-site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea.

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 leading the way in complement science 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 C3-targeting medicines approved to treat four serious diseases. Breakthroughs for patients include the first-ever therapy for geographic atrophy, a leading cause of blindness, and the first treatment for patients 12 and older with C3G or primary IC-MPGN, two severe, rare kidney diseases. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on LinkedIn and X.

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. 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 results of the Company’s clinical trials for EMPAVELI, SYFOVRE, or any of its future products will warrant regulatory submissions to the FDA or equivalent foreign regulatory agencies; whether systemic pegcetacoplan will receive approval from foreign regulatory agencies for C3G and primary IC-MPGN; rate and degree of market acceptance and clinical utility of EMPAVELI, SYFOVRE and any future products for which we receive marketing approval will impact our commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all; 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 or in the real world setting; whether 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 28, 2025 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:
Tracy Vineis
media@apellis.com
617.420.4839

Investor Contact:
Neil Carnahan, CFA
neil.carnahan@apellis.com
617.977.5703