Apellis Pharmaceuticals

Overview

• Sector: Commercial-stage biopharmaceutical company
• Strategy: Discover, develop and commercialize novel therapeutics that inhibit the complement system
• Focus: Diseases driven by excessive complement activation with high unmet need
• Marketing rights:
  • SYFOVRE (pegcetacoplan): exclusive, worldwide commercialization rights
  • EMPAVELI (pegcetacoplan): exclusive U.S. commercialization rights; Sobi holds ex-U.S. rights for systemic pegcetacoplan

Key products and programs

• SYFOVRE (pegcetacoplan)
  • Route: Intravitreal injection
  • Indication: Geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
  • Market status: Commercially marketed; U.S. launch in March 2023
• EMPAVELI (pegcetacoplan)
  • Route: Systemic subcutaneous administration
  • Indication: Paroxysmal nocturnal hemoglobinuria (PNH)
  • Regulatory: FDA-approved May 2021
  • Rights: Apellis retains U.S. commercialization; Sobi retains ex-U.S. rights for systemic pegcetacoplan
• Aspaveli
  • Indication/territory: C3G and primary IC-MPGN outside the U.S. (Sobi holds ex-U.S. rights)
  • Regulatory milestone: EMA approval for C3G and primary IC-MPGN (January 2026)
• APL-3007
  • Type: siRNA targeting complement in the retina
  • Status: In development; Phase 2 multi-dose trial in GA initiated June 2025
  • Rationale: Combined therapy with SYFOVRE to extend complement inhibition in the retina and choroid
• Beam collaboration and APL-9099
  • 2021: Exclusive five-year collaboration with Beam for base editing in complement targets
  • 2025: Opted in to Beam License Agreement for FcRn in the liver; upfront milestone paid
  • Ongoing: Additional programs under in-house and Beam collaborations

Financial highlights (reported)

• SYFOVRE — U.S. net product revenue
  • 2025: $586.9 million
  • 2024: $611.9 million
• EMPAVELI — U.S. net product revenue
  • 2025: $102.4 million
  • 2024: $98.1 million
• Royalties from Sobi
  • 2025: $13.2 million
  • 2024: $18.4 million
• Royalty Buy-Down with Sobi
  • July 1, 2025: Upfront payment of $275.0 million
  • Structure: 90% reduction in Sobi’s royalty obligations under the collaboration agreement, subject to caps (initial cap of 1.45x the upfront amounts); if the cap is met, royalties revert to 100%
• Additional 2025–2026 cash events
  • January 2026: EMA approval of Aspaveli
  • February 2026: Sobi paid a $25.0 million milestone under the Royalty Agreement
• Beam collaboration payments (highlights)
  • Upfronts: $50.0 million (July 2021); $25.0 million (June 2022)
  • August 2025: Opt-in for FcRn in the liver; $3.8 million upfront milestone paid in September 2025
  • Potential future payments: Up to $168.8 million in regulatory/development milestones; up to $215.0 million in sales milestones; low single-digit royalties on annual net sales (territory-dependent)

Partnerships and commercial arrangements

• Sobi collaboration
  • Territory: Ex-U.S. commercialization of systemic pegcetacoplan
  • Apellis retains U.S. commercialization rights for pegcetacoplan
  • Aspaveli: Sobi holds ex-U.S. rights; a royalty buy-down reduces Sobi royalties subject to defined caps
• Royalty Agreement with Sobi
  • Upfront: $275.0 million (July 2025)
  • Milestones: $25.0 million upon EMA approval of Aspaveli (paid February 2026)
  • Economics: Caps tied to Aspaveli performance; royalties revert to 100% if cap conditions are met
• Beam collaboration and license
  • Initial collaboration (2021–2025): Base editing programs targeting complement pathologies
  • Opt-in license (August 2025): FcRn in the liver
  • Financials: Historical upfronts totaling $53.8 million; potential milestones and royalties as described above

Pipeline and R&D focus

• Target areas: Complement pathway modulation across eye diseases, systemic complementopathies, and rare kidney diseases
  • Ophthalmology: GA with SYFOVRE; APL-3007 siRNA program in GA
  • Hematology: PNH with EMPAVELI
  • Nephrology: C3G and primary IC-MPGN (Aspaveli ex-U.S.); programs in FSGS and delayed graft function (DGF)
• Innovative approaches: siRNA, injectable biologics, and base editing via the Beam collaboration (including FcRn targets)
• Selected clinical programs:
  • GA: DERBY and OAKS Phase 3 showed slowing of GA lesion growth with monthly and every-other-month dosing options
  • PNH: PEGASUS and PRINCE pivotal trials showed efficacy versus comparator
  • C3G/transplant: VALIANT and NOBLE trials addressed transplant recurrence and proteinuria endpoints
• Delivery and administration: Continued work on co-pack and single-dose prefilled syringe formats for SYFOVRE (single-dose prefilled syringe planned for regulatory submission in H1 2026)

Intellectual property

• Portfolio: 31 U.S. patents and 29 U.S. patent applications, plus foreign counterparts
• Core technologies: Pegylated compstatin analogs (pegcetacoplan) and dosing/methods for ophthalmic and non-ophthalmic indications
• Licenses:
  • Penn: Exclusive worldwide license for ophthalmic and non-ophthalmic fields
  • Beam: Non-exclusive license for base editing technology; Apellis holds exclusive rights for therapeutic programs covered by the collaboration where applicable
• Patent terms: Expirations range into the 2030s–2040s; patent term extensions and regulatory exclusivities are being pursued where available

Manufacturing and operations

• Model: Outsourced manufacturing with contract manufacturers
• Key suppliers:
  • Bachem Americas, Inc.: Peg drug substance for systemic and intravitreal pegcetacoplan
  • NOF Corporation: PEG derivative supply
• Supply agreements:
  • Bachem supply agreement: Initial term through 2025 with automatic renewal
  • NOF supply agreement: Extended through 2030; minimum purchase obligations were adjusted for 2025
• Packaging and device development:
  • Single-package co-pack for SYFOVRE introduced in Q4 2025
  • Single-dose prefilled syringe development for SYFOVRE planned for regulatory submission in H1 2026

Regulatory and market environment

• Approvals:
  • U.S. FDA approvals for SYFOVRE (GA) and EMPAVELI (PNH)
  • EMA approval of Aspaveli for C3G and primary IC-MPGN (January 2026)
• Post-approval activities: Ongoing pharmacovigilance, post-approval studies, REMS considerations, and marketing authorization maintenance
• Data privacy: Compliance with HIPAA, GDPR/DPAs, and evolving global privacy laws affects trial data and operations

Summary

Apellis is a specialized biotech focused on complement inhibition with two marketed pegcetacoplan products in the U.S. (SYFOVRE and EMPAVELI), an international commercialization strategy through Sobi, and a pipeline spanning ophthalmology, hematology, and nephrology. The company has structured collaboration and licensing arrangements that include a substantial royalty buy-down with Sobi and an active research and optioned-licensing relationship with Beam for base editing approaches. Financially, disclosed 2025 product revenues and collaboration royalties demonstrate commercial traction in U.S. markets, and milestone and license payments provide near-term cash events.