Apellis Pharmaceuticals, Inc. operates as a commercial-stage biopharmaceutical company. The company focuses on the discovery, development and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through the inhibition of the complement system, which is an integral component of the immune system.
The company has two marketed drugs that target C3, the central protein in the complement cascade: SYFOVRE (pegcetacoplan injection), approved by the U.S. Food and Dru...
Apellis Pharmaceuticals, Inc. operates as a commercial-stage biopharmaceutical company. The company focuses on the discovery, development and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through the inhibition of the complement system, which is an integral component of the immune system.
The company has two marketed drugs that target C3, the central protein in the complement cascade: SYFOVRE (pegcetacoplan injection), approved by the U.S. Food and Drug Administration, or FDA, in February 2023 for the treatment of geographic atrophy secondary to age-related macular degeneration, or GA; and EMPAVELI (pegcetacoplan), approved by the FDA in May 2021 for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH.
The company has exclusive, worldwide commercialization rights for intravitreal pegcetacoplan, it intends to focus its commercialization efforts on the U.S. and explore international expansion in select markets, including Australia, where the company received marketing approval in January 2025.
The company has exclusive the U.S. commercialization rights for EMPAVELI, and its collaboration partner, Swedish Orphan Biovitrum AB (Publ), or Sobi, has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan outside of the United States.
The next indications the company is pursuing with EMPAVELI are C3 glomerulopathy, or C3G, and primary immune complex membranoproliferative glomerulonephritis, or IC-MPGN.
Finally, the company is developing new product candidates to further advance its pipeline. Through the company’s collaboration with Beam Therapeutics, Inc., or Beam, it has commenced pre-clinical studies for a treatment targeting the neonatal Fc receptor, orFcRn, which has the potential to be a first-in-class gene editing treatment for future target indications with one-time dosing. The company is also developing other programs with its proprietary in-house capabilities.
Programs
Pegcetacoplan targets C3, the central protein of the complement cascade. Pegcetacoplan is a conjugate of a compstatin analogue, formulated both for intravitreal administration by injections directly into the eye, and systemic administration by subcutaneous injection, which is an injection into the tissue under the skin. The company has developed and are developing pegcetacoplan and other product candidates through various routes of administration.
Ophthalmology
The commercializing SYFOVRE as a monotherapy for patients with GA.
Regulatory Matters
In February 2023, the FDA approved intravitreal pegcetacoplan with the brand name SYFOVRE for the treatment of adult patients with GA secondary to AMD.
Commercial and Medical Activities for GA
The company launched SYFOVRE, the first approved treatment for GA, in the United States in March 2023. SYFOVRE is the market-leading treatment for GA, a disease that affects an estimated 1.5 million people in the United States.
The company’s U.S. field sales team has been engaging with eyecare professionals, or ECPs, focusing specifically on retina specialists and treating ophthalmologists. Field teams are focused on SYFOVRE brand messaging, highlighting key advantages, such as increasing effects over time, its strong clinical profile, and dosing flexibility. The company also has a thought leader liaison team, which is focused on building advocacy with key opinion leaders in the retina space, and a strategic account team, which identifies and develops working relationships with key decision makers within targeted private equity groups and large accounts. The company’s marketing efforts are designed to reach ECPs through digital and print media. The company seeks to reach patients through direct-to-consumer (TV, print and digital media) disease state education and branded SYFOVRE messaging encouraging them to see their eye doctor if they have symptoms or a previous diagnosis. Additionally, the company’s efforts have focused on increasing awareness of GA and SYFOVRE with general ophthalmologists and optometrists to ensure GA patients are able to connect with a retina specialist or ophthalmologist who can treat them. The company has launched a practice finder tool to help physicians and patients identify practices near them that have recently treated GA.
The company’s market access team has been engaging with primary and secondary payers representing a significant percentage of GA patients. The company has also established a robust distribution network by partnering with key specialty distributors and specialty pharmacies to maximize product access by retina specialists. Finally, the company has a field reimbursement team to educate practices and address access issues to fully support the reimbursement journey for SYFOVRE.
The company’s medical affairs team is engaging with ECPs through its presence at medical meetings and other in-person engagements. Throughout 2024, the company participated in key scientific meetings, including the American Academy of Ophthalmology, Retina Society, FLORETINA, and Macula Society.
Clinical Development
The company is conducting two post-marketing studies: GALE and GARLAND. The company’s registrational Phase 3 DERBY and OAKS trials evaluated the efficacy and safety of SYFOVRE in patients with GA secondary to AMD. The DERBY and OAKS were initiated in September 2018, and the company presented reported 24-month results from its DERBY and OAKS trials in August 2022, following 18-month results presented in March 2022 and 12-month results presented in September 2021. Prior to DERBY and OAKS, the company completed the Phase 2 FILLY trial in August 2017.
Post-Marketing Studies
The company is conducting a 36-month, open-label extension study (GALE) to evaluate the long-term safety and efficacy of intravitreal pegcetacoplan in patients with GA secondary to AMD.
In February 2025, the company presented data from GALE following four years of continuous treatment with SYFOVRE. The company is conducting a 36-month, open label, Phase 4 study (GARLAND) to evaluate the safety, tolerability and treatment patterns of SYFOVRE in patients over the age of 60 with GA in a clinical practice setting.
Phase 3 Clinical Trials
The company’s Phase 3 clinical program in GA consisted of two prospective, multicenter, randomized, double-masked, sham-injection controlled trials (DERBY and OAKS) conducted at more than 200 sites worldwide to assess the efficacy and safety of multiple intravitreal injections of pegcetacoplan in patients with GA. The company enrolled 621 patients in DERBY and 637 patients in OAKS.
Patients in each Phase 3 trial received a dose of 15 mg of pegcetacoplan injected intravitreally in a 0.1 cc volume, monthly or every other month for 24 months. In the sham-injection cohorts, patients received a simulated injection. As with the company’s Phase 2 FILLY clinical trial, the primary endpoint of each trial was the change in total area of GA lesions in the study eye compared to sham. The measurements of change in lesion size were analyzed at 12 months, 18 months, and 24 months. Patients who develop new onset exudation in the study eye continued to be treated with pegcetacoplan along with anti-VEGF injections, the standard of care for wet AMD.
The company completed the primary analysis for the 24-month treatment period in August 2022. Monthly and every-other-month, or EOM, treatment with SYFOVRE showed increased effects over time. In OAKS, monthly and EOM treatment with SYFOVRE reduced GA lesion growth by 22% (p<0.0001) and 18% (p=0.0002), respectively. In DERBY, monthly and EOM treatment with SYFOVRE reduced GA lesion growth by 19% (p=0.0004) and 16% (p=0.0030), respectively. All p-values are nominal and were calculated using the same methodologies as the 12-month primary endpoint analysis.
The company used a liquid formulation of pegcetacoplan in its Phase 3 trials instead of the freeze-dried formulation that it used in the Phase 2 FILLY trial.
SYFOVRE and APL-3007
In January 2025, the company shared Phase 1 data with APL-3007, its siRNA, in healthy volunteers showing greater than 90% knockdown of C3 as measured by the remaining levels of protein in the blood. Based on the results of this study, the company is developing a next generation treatment for GA by combining SYFOVRE plus APL-3007.
Rare Diseases
EMPAVELI in PNH
The company launched EMPAVELI in the United States for patients with PNH following its approval by the FDA in May 2021. The company believes that EMPAVELI elevated the standard of care for patients with PNH.
Commercialization
The company’s sales efforts are focused on the health care professionals, or HCPs, and key treatment centers, who have patients that continue to experience breakthrough hemolysis, have persistently low hemoglobin, high fatigue, and require transfusions despite being on C5 inhibitors.
The company also has Apellis Assist, a patient-focused program specifically designed to assist patients with onboarding, product training and ongoing support with pegcetacoplan treatment, and it has built a care educator team to connect directly with PNH patients and their caregivers to provide education and training on the use of pegcetacoplan.
The company’s medical affairs team is engaging with physicians through its presence at medical meetings and other in-person engagements. In December 2024, the company participated in the American Hematology Society, or ASH, annual meeting.
Regulatory Matters and Clinical Development
The company submitted a sNDA to the FDA in early 2025 following the positive results from the Phase 3 VALIANT trial investigating EMPAVELI in adolescent and adult patients with naive and post-transplant recurrence C3G and IC-MPGN. EMPAVELI received orphan drug designation from the FDA for the treatment of C3G in December 2018.
In August 2024, the company announced positive results from its VALIANT study, a randomized, placebo-controlled, double-blinded, multi-center Phase 3 trial in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN.
In October 2023, the company announced positive results from its NOBLE trial, a randomized, placebo-controlled Phase 2 trial in post-transplant recurrence of C3G and IC-MPGN.
Beam Research Collaboration
In June 2021, the company entered an exclusive five-year research collaboration with Beam focused on the use of Beam’s proprietary base editing technology to discover new treatments for complement-driven diseases. Under the collaboration agreement, the company is collaborating on up to six research programs focused on C3 and other complement targets in the eye, liver and brain. The company has commenced pre-clinical studies for a FcRn treatment, which has the potential to be a first-in-class gene editing treatment for future target indications with one-time dosing.
Collaboration and License Agreement with Sobi
On October 27, 2020, the company and its subsidiaries, Apellis International GmbH (f/k/a Apellis Switzerland GmbH) and APL DEL Holdings, LLC, entered into a Collaboration and License Agreement (the ‘Sobi collaboration agreement’) with Sobi, concerning the development and commercialization of pegcetacoplan and specified other structurally and functionally similar compstatin analogues or derivatives for use systemically or for local non-ophthalmological administration (collectively referred to as the ‘Licensed Products’).
Under the Sobi collaboration agreement, the company granted Sobi an exclusive (subject to certain retained rights of the company), sublicensable license of certain patent rights and know-how to develop and commercialize Licensed Products in all countries outside of the United States.
The company retains the right to commercialize Licensed Products in the United States and, subject to specified limitations, to develop Licensed Products worldwide for commercialization in the United States.
Under the Sobi collaboration agreement, the company and Sobi agreed to collaborate to develop Licensed Products for certain indications, including PNH, C3G, IC-MPGN and HSCT-TMA, and any other indications subsequently agreed upon by the parties, for commercialization by or on behalf of the company in the United States and by or on behalf of Sobi outside of the United States.
The company and Sobi have formed several governance committees to oversee the development and manufacture, and to review and discuss the commercialization, of Licensed Products.
The company has agreed to supply Licensed Products to Sobi for development and for commercialization outside of the United States in accordance with a supply agreement to be negotiated by the parties.
The company has entitled to receive tiered, double-digit royalties (ranging from high teens to high twenties) on sales of Licensed Products outside of the United States, subject to customary deductions and third-party payment obligations, until the latest to occur of: (i) expiration of the last-to-expire of specified licensed patent rights; (ii) expiration of regulatory exclusivity; and (iii) ten (10) years after the first commercial sale of the applicable Licensed Product, in each case on a Licensed Product-by-Licensed Product and country-by-country basis. Under the Sobi collaboration agreement, the company remains responsible for its license fee obligations (including royalty obligations) to the Trustees of the University of Pennsylvania (‘Penn’), as a licensor of Apellis.
Research Collaboration with Beam
In June 2021, the company entered an exclusive five-year research collaboration (the ‘Beam collaboration agreement’) with Beam focused on the use of Beam’s proprietary base editing technology to discover new treatments for complement-driven diseases. The company and Beam agreed to collaborate on up to six research programs focused on C3 and other complement targets in the eye, liver and brain. Under the terms of the Beam collaboration agreement, the company is responsible for selecting specific genes within the complement system in various organs including the eye, liver and brain (the ‘Target List’) and providing analytical support while Beam will apply its base editing technology and conduct preclinical research on up to six base editing programs for the Target List. The company will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development and commercialization.
Intellectual Property
As of December 31, 2024, the company owned a total of 27 U.S. patents and 35 pending U.S. patent applications, including original filings, continuations, and divisional applications, as well as numerous foreign counterparts of many of these patents and patent applications.
The intellectual property in-licensed under the company’s two license agreements with Penn includes four U.S. patents and numerous foreign counterparts, with claims granted in Europe, Japan and elsewhere.
The company granted worldwide rights to use and license the intellectual property that it holds with respect to pegcetacoplan to its wholly owned subsidiaries, APL DEL Holdings, LLC and Apellis International GmbH (f/k/a Apellis Switzerland GmbH).
Patent License Agreement with The Trustees of the University of Pennsylvania (Non-ophthalmic Fields of Use)
The company’s party to an agreement with Penn for an exclusive worldwide license, under specified patent rights controlled by Penn, to develop and commercialize products covered by the licensed patent rights for all fields except the treatment of ophthalmic indications. The company has the right to grant sublicenses under this license.
The patent rights licensed to the company by Penn include patents with claims that recite a class of compounds generically covering pegcetacoplan, and specifically recite the active component. Three of these patents are listed for EMPAVELI in the FDA’s Orange Book.
Amended and Restated Patent License Agreement with The Trustees of the University of Pennsylvania (Ophthalmic Field of Use)
The company’s party to an agreement with Penn for an exclusive worldwide license, under specified patent rights controlled by Penn, to develop and commercialize products covered by the licensed patent rights for the treatment of ophthalmic indications. Three of the licensed patents are listed for SYFOVRE in the FDA’s orange book. The company has the right to grant sublicenses under the license.
Competition
The principal competitors for EMPAVELI, and possibly other indications in the company’s hematology and nephrology programs are eculizumab (marketed as Soliris) and ravulizumab (marketed as Ultomiris), which are C5 inhibitors marketed by AstraZeneca.
Sales and Marketing
The company retains the U.S. commercialization rights for systemic pegcetacoplan and worldwide commercialization rights for intravitreal pegcetacoplan. The company is conducting commercialization efforts for EMPAVELI and SYFOVRE in the United States and plan to conduct commercial development for EMPAVELI in the United States if it is approved in other indications. Sobi has global co-development and exclusive ex-U.S. commercialization rights for Aspaveli. The company plans to conduct commercial development for intravitreal pegcetacoplan in select countries outside of the U.S. The company has developed focused capabilities to commercialize development programs for certain indications where it believes that the medical specialists for the indications are sufficiently concentrated to allows the company to effectively promote the product with a targeted sales team.
For EMPAVELI and SYFOVRE the company has defined its marketing, disease education, patient support and distribution strategies, identified primary and secondary payers representing a significant percentage of patients with PNH and GA, has built the company’s field market access team and its sales team.
Commercial Supply Agreement with Bachem
In December 2020, the company entered into a commercial supply agreement, or the Bachem Agreement, with Bachem to supply the drug substance for the finished dosage form of systemic pegcetacoplan and intravitreal pegcetacoplan.
Under the Bachem Agreement, the company agreed to purchase from Bachem a significant portion of its requirements for the drug substance during the term of the agreement, and to purchase all the company’s requirements for drug substance for commercial sale, subject to certain exceptions, for a period after the effective date of the agreement.
Amended and Restated Commercial Supply Agreement with NOF
In March 2021, the company entered into an amended and restated commercial supply agreement, or the NOF Agreement, with NOF to purchase PEG, which is a component of each of systemic pegcetacoplan and intravitreal pegcetacoplan. Under the NOF Agreement, NOF’s affiliate, NOF America Corporation, supplies PEG to the company on a non-exclusive basis.
Research and Development
The company's research and development expenses included $ 327.6 million for the year ended December 31, 2024.
History
Apellis Pharmaceuticals, Inc. was founded in 2009. The company was incorporated under the laws of the state of Delaware in 2009.
