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Here are 1-3 brief analogies to describe Apellis Pharmaceuticals (APLS):

• An emerging Alexion (now AstraZeneca Rare Disease) for complement-mediated diseases.
• A specialized biotech company, much like a focused Regeneron for specific eye diseases and rare blood disorders.
• Like a Vertex Pharmaceuticals, focused on becoming a leader in complement-driven diseases and a major eye condition.

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• SYFOVRE: An injectable treatment approved for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
• EMPAVELI: A targeted C3 complement inhibitor approved for adults with paroxysmal nocturnal hemoglobinuria (PNH).

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Apellis Pharmaceuticals (APLS) sells primarily to other companies, specifically major pharmaceutical wholesalers and specialty distributors, rather than directly to individuals.

The company has identified the following as its major customers:

• Cencora, Inc. (COR) - formerly AmerisourceBergen Corporation
• Cardinal Health, Inc. (CAH)
• McKesson Corporation (MCK)

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Cedric Francois, MD, PhD Co-Founder & Chief Executive Officer/President

Dr. Cedric Francois co-founded Apellis Pharmaceuticals in 2009. He also co-founded Potentia Pharmaceuticals, Inc. in 2001, serving as its President and CEO. Potentia Pharmaceuticals was transacted with Alcon Research Ltd in 2009, with Dr. Francois noting he avoided bankruptcy by selling the company that year. He is also a founder of Revon Systems and Liberate Medical. Prior to his entrepreneurial endeavors, Dr. Francois had a career as a surgeon and was part of the research team that performed the first successful hand transplantation and the Louisville Face Transplant Team.

Timothy Sullivan Chief Financial Officer

Timothy Sullivan has served as Chief Financial Officer of Apellis Pharmaceuticals since October 2017. Before joining Apellis, he was a Partner at AJU IB Investment, a venture capital firm, from January 2014 to October 2017, where he led investments in life sciences companies. This role, along with his prior experience as an investment banker, indicates a pattern of managing companies backed by private equity firms. He previously held positions as Managing Director, head of life sciences banking at RBS Citizens, Senior Vice President at Jefferies & Company, and Vice President at Bear Stearns. Sullivan also served as an observer on Apellis' board of directors for nearly three years prior to his CFO appointment.

Pascal Deschatelets, PhD Co-Founder & Chief Scientific Officer

Pascal Deschatelets is a co-founder of Apellis Pharmaceuticals and serves as the Chief Scientific Officer. He previously held the role of COO/Co-Founder at Potentia Pharmaceuticals Inc., which was co-founded by Cedric Francois and transacted with Alcon in 2009.

Caroline Baumal, MD Chief Medical Officer

Dr. Caroline Baumal is the Chief Medical Officer at Apellis Pharmaceuticals, a position she assumed in 2023.

Leslie Meltzer, PhD Chief Research & Development Officer

Dr. Leslie Meltzer holds the title of Chief Research & Development Officer at Apellis Pharmaceuticals.

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Key Risks to Apellis Pharmaceuticals (APLS)

1. Safety Concerns and Regulatory Rejections for SYFOVRE: Apellis' flagship product, SYFOVRE (pegcetacoplan injection) for geographic atrophy (GA), has faced significant safety setbacks and regulatory hurdles. Rare events of retinal vasculitis were reported in real-world treatment with SYFOVRE, leading to a safety warning added to its label in December 2023. Furthermore, the European Medicines Agency (EMA) has repeatedly declined to endorse Apellis' marketing authorization application for SYFOVRE in the EU, with negative opinions issued in January, June, and September 2024, significantly hindering the drug's potential market expansion outside the U.S. These issues have contributed to persistent revenue stagnation for SYFOVRE.
2. Intensifying Competition in the Geographic Atrophy (GA) Market: While SYFOVRE was the first FDA-approved treatment for GA, the competitive landscape is rapidly evolving. Astellas Pharma's Izervay (avacincaptad pegol) received FDA approval in August 2023, posing direct competition to SYFOVRE. The market's adaptation to new drug availability, and how their efficacy, safety profiles, and pricing compare to SYFOVRE, remains uncertain, potentially impacting SYFOVRE's market share and revenue generation.
3. Financial Pressures and Profitability Challenges: Apellis Pharmaceuticals faces ongoing financial pressures and challenges in achieving consistent profitability. The company has reported persistent revenue stagnation for SYFOVRE and experienced a decline in Q1 2025 revenues compared to the prior year, partly due to issues with co-pay assistance programs, resulting in an estimated $10 million in lost revenue. The company has recorded negative earnings per share and operating/net margins, indicating operational inefficiencies. Additionally, an Altman Z-Score of -2.15 places the company in a financial distress zone, and a relatively high debt-to-equity ratio of 2.86 further highlights financial leverage. Apellis reported net losses in 2023, 2024, and Q1 2025.

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The approval and market entry of Iolanda (avacincaptad pegol) developed by Astellas Pharma (via its acquisition of Iveric Bio) poses a clear emerging threat. Iolanda received FDA approval in August 2023 for geographic atrophy (GA) secondary to age-related macular degeneration, directly competing with Apellis's SYFOVRE (pegcetacoplan), which was approved in February 2023 for the same indication. This creates a direct head-to-head competition in a newly established market where Apellis initially held a first-mover advantage, potentially splitting market share and limiting SYFOVRE's growth.

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Apellis Pharmaceuticals (APLS) has two main commercialized products with identified addressable markets:

• SYFOVRE (pegcetacoplan injection) for Geographic Atrophy (GA):
  • Over 1 million people in the U.S. are affected by GA.
  • Globally, approximately 5 million people are affected by GA.
• EMPAVELI (pegcetacoplan) for Paroxysmal Nocturnal Hemoglobinuria (PNH):
  • The total PNH market size in the 7 Major Markets (7MM), which includes the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, was valued at approximately USD 1.4 billion in 2023, with projections to grow to about USD 2.5 billion by 2034.
  • In 2023, there were approximately 12,000 diagnosed cases of PNH in the 7MM, with projections suggesting this number could rise to around 13,000 by 2034.
• EMPAVELI (pegcetacoplan) for C3 Glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN):
  • The approval of EMPAVELI for these indications expands its addressable market by approximately 5,000 patients. (Region not explicitly specified in all snippets, but context often implies U.S. market in these discussions.)

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1. Continued Growth of SYFOVRE in Geographic Atrophy (GA): SYFOVRE (pegcetacoplan injection) is the leading treatment for geographic atrophy (GA) secondary to age-related macular degeneration. The company reported strong U.S. net product revenue of $611.9 million for SYFOVRE in full-year 2024, representing over 120% year-over-year growth. Apellis anticipates steady, measured injection growth for SYFOVRE in the near term, with a continued focus on reaching more GA patients and maintaining its market leadership, which accounted for over 60% of the overall market in Q3 2025. The company also plans to leverage robust data from SYFOVRE trials and real-world evidence to strengthen its market position.
2. Expansion of EMPAVELI into New Rare Kidney Diseases (C3G and IC-MPGN): EMPAVELI (pegcetacoplan) received U.S. FDA approval in July 2025 for treating patients aged 12 and older with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). This approval significantly expands EMPAVELI's addressable market by approximately 5,000 patients, offering a breakthrough therapy with meaningful results in proteinuria reduction, eGFR stabilization, and substantial clearance of C3 deposits. Apellis is actively launching EMPAVELI for these new indications, targeting 225 cumulative patient start forms by year-end 2025.
3. Geographic Expansion of SYFOVRE and EMPAVELI: Apellis is actively pursuing the global expansion of its products. SYFOVRE is approved in the United States and Australia, and the company is preparing for an initial launch in Germany within the European Union. For EMPAVELI (marketed as Aspaveli in Europe), the European Commission approved it for adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Furthermore, Apellis is eligible for up to $25 million in milestone payments upon EMA approval of Aspaveli for C3G and IC-MPGN, signaling potential future revenue from international markets for this indication.
4. Advancement of Pipeline Programs (FSGS and DGF, APL-3007): Apellis is investing in its pipeline to fuel long-term growth. The company is on track to initiate two Phase 3 clinical trials for EMPAVELI in focal segmental glomerulosclerosis (FSGS) and in delayed graft function (DGF) in the second half of 2025. Additionally, Apellis expects to initiate a Phase 2 multi-dose study of APL-3007 in combination with SYFOVRE in Q2 2025, which represents a potential next-generation treatment aimed at comprehensively blocking complement activity in the retina and choroid. Successful progression and potential future approvals of these pipeline assets could provide new revenue streams.
5. Strategic Collaborations and Licensing Agreements: Apellis has demonstrated its ability to generate revenue through strategic partnerships. In July 2025, Apellis and Sobi announced a capped royalty purchase agreement where Apellis receives up to $300 million for 90% of its future ex-U.S. royalties for Aspaveli. This agreement included a $275 million upfront payment, which contributed significantly to Q3 2025 revenue. Such collaborations provide non-dilutive capital and diversified revenue streams, reinforcing the company's financial position and supporting its growth initiatives.

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Share Issuance

• Apellis Pharmaceuticals announced inducement grants to new employees on December 2, 2024, consisting of options to purchase 27,445 shares and 28,630 restricted stock units (RSUs).
• Proceeds from the issuance of common stock under employee share purchase plans were $4.5 million in 2024, $5.4 million in 2023, and $4.2 million in 2022.

Inbound Investments

• In October 2020, Apellis entered into a Collaboration and License Agreement with Sobi for the development and commercialization of pegcetacoplan and similar compstatin analogues for systemic or local non-ophthalmological administration.
• Apellis received a $275.0 million upfront payment from Sobi in connection with a capped ex-U.S. royalty purchase agreement on Aspaveli in the third quarter of 2025.
• Apellis strengthened its balance sheet through a royalty purchase agreement for ex-U.S. royalties of Aspaveli, receiving $275 million upfront in exchange for 90% of future ex-U.S. royalties, with potential for an additional $25 million in milestones upon EMA approval for C3G and primary IC-MPGN.

Capital Expenditures

• Capital expenditures were approximately $0.4 million in 2024, $0.77 million in 2023, $1.52 million in 2022, $1.1 million in 2021, and $5.42 million in 2020.