Subcutaneous (SC) administration of anti-C5 antibodies for treatment of complement-associated conditions
Inventors
Miano, Dino C. • Wang, Hweirung Amy • MEZHEBOVSKY, Tatyana
Assignees
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Publication Number
US-12617846-B2
Publication Date
2026-05-05
Expiration Date
Abstract
Provided are methods for clinical treatment of complement-associated conditions comprising administering to the patient an anti-C5 antibody, or antigen binding fragment thereof, wherein the anti-C5 antibody, or antigen binding fragment thereof, is administered (or is for administration) subcutaneously according to a particular clinical dosage regimen (i.e., at a particular dose amount and according to a specific dosing schedule). In one embodiment, the patient has previously been treated with eculizumab (SOLIRIS®) or ravulizumab (ULTOMIRIS®); particularly intravenously administered SOLIRIS® or ULTOMIRIS®.
Core Innovation
The document describes a method of treating a human patient with a complement-associated condition by administering an effective amount of an anti-C5 antibody or antigen binding fragment thereof subcutaneously using an on-body delivery system. The method uses an anti-C5 antibody or antigen binding fragment comprising CDR1, CDR2 and CDR3 heavy chain sequences as set forth in SEQ ID NOs: 19, 18 and 3, respectively, and CDR1, CDR2 and CDR3 light chain sequences as set forth in SEQ ID NOs: 4, 5 and 6, respectively. The on-body delivery system includes a pharmaceutical formulation comprising ravulizumab (70 mg/mL) in defined excipients and water for injection.
The patent centers on ravulizumab (ULTOMIRIS®) as the administered complement inhibitor, delivered during an administration cycle by subcutaneous on-body delivery system. It provides clinical evidence through a Phase 3 randomized noninferiority study comparing ULTOMIRIS® SC versus ULTOMIRIS® IV in eculizumab-pretreated adults, including PK noninferiority based on Day 71 serum ravulizumab trough concentrations. The approach evaluates in vivo C5 inhibition performance using serum ravulizumab trough concentrations and free C5 inhibition thresholds, alongside efficacy endpoints associated with complement activity and patient outcomes.
The document further describes a clinical study comparing subcutaneous ravulizumab administered via an on-body delivery system with intravenous ravulizumab in a complement-associated condition. The study includes defined visit/time windows and endpoints intended to assess pharmacokinetics and pharmacodynamics, including measurements related to terminal complement inhibition, pharmacokinetics, pharmacodynamics, immunogenicity, antidrug antibodies, breakthrough hemolysis, safety outcomes, and quality-of-life questionnaires.
Claims Coverage
The provided excerpt includes two independent claims: one directed to a subcutaneous treatment method using a defined anti-C5 antibody and on-body delivery system with a defined ravulizumab formulation, and another directed to a device for subcutaneous administration comprising the on-body delivery system and the same defined subcutaneous formulation.
Subcutaneous on-body delivery system treatment of a complement-associated condition with a defined anti-C5 antibody
Administering during an administration cycle to a human patient an effective amount of an anti-C5 antibody or antigen binding fragment thereof having specified CDR heavy and light chain sequences, wherein the antibody or fragment is administered subcutaneously by an on-body delivery system and the on-body delivery system comprises a pharmaceutical formulation comprising ravulizumab (70 mg/mL) in defined excipients and water for injection.
On-body delivery system device for subcutaneous administration of ravulizumab formulation
A device for subcutaneous administration of an anti-C5 antibody or antigen binding fragment thereof comprising an on-body delivery system and a subcutaneous formulation of the anti-C5 antibody or antigen binding fragment thereof, wherein the subcutaneous formulation comprises ravulizumab (70 mg/mL) in defined excipients and water for injection.
Across the independent claims, coverage is directed to subcutaneous administration of an anti-C5 ravulizumab treatment for complement-associated conditions using an on-body delivery system, with the independent-claim limitation anchored by the specified CDR sequence-defined antibody and a quantitative ravulizumab formulation composition; the second independent claim covers the corresponding device comprising the on-body delivery system and that formulation.
Stated Advantages
PK noninferiority based on Day 71 serum ravulizumab trough concentrations.
Noninferior subcutaneous versus intravenous trough exposure while complement inhibition is maintained.
Documented Applications
Treatment of a human patient with a complement-associated condition, including paroxysmal nocturnal hemoglobinuria (PNH) and eculizumab-pretreated adults evaluated in a Phase 3 randomized noninferiority study comparing ULTOMIRIS® SC versus ULTOMIRIS® IV.
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