Methods for increasing serum concentration of phosphorous and/or 1,25-hydroxy vitamin d

Inventors

Kakkis, Emil D. • San Martin, Javier • SUDO, Tomohiro

Assignees

Kyowa Kirin Co Ltd • Ultragenyx Pharmaceutical Inc

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Publication Number

US-12358976-B2

Patent

Publication Date

2025-07-15

Expiration Date


Abstract

The present invention provides compositions and methods for treating a hypophosphatemic disorder, such as X-linked hypophosphatemia (XLH). The method entails administering to a subject a pharmaceutical composition containing an anti-FGF23 ligand, wherein the dosing regimen of the pharmaceutical is designed to reach effective and efficient control of FGF23 activity.

Core Innovation

The invention relates to methods of treating a subject having a hypophosphatemic disorder by administering an anti-FGF23 antibody about every two weeks. The method increases serum phosphorus levels and/or serum 1,25-dihydroxy vitamin D levels in a subject having a hypophosphatemic disorder, and the anti-FGF23 antibody comprises the CDR sequences of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, and SEQ ID NO: 6.

The document describes KRN23 clinical-study pharmacodynamics and pharmacokinetics in adult subjects with XLH. In adult XLH, serum phosphorus increases with each dosing interval, peaking around Day 7/14, while TmP/GFR increases in parallel, supporting renal phosphate reabsorption as the mechanism, and serum 1,25-dihydroxyvitamin D also increases with similar timing.

The pharmacokinetic analysis supports dose-proportional exposure and a linear correlation between KRN23 exposure (AUC) and changes in serum phosphorus, TmP/GFR, and 1,25(OH)2D. The document also describes an elimination half-life of about 16.4 days, correlations between exposure and biomarker changes, longer-term/extension design elements, and preliminary pediatric Phase 2 design comparing Q4 versus Q2 dosing.

Claims Coverage

The independent claim set comprises three independent methods that use an anti-FGF23 antibody with a defined CDR sequence composition and an administration frequency of about every two weeks. The inventive features are directed to increasing serum phosphorus levels, increasing serum 1,25-dihydroxy vitamin D levels, and increasing serum concentrations of phosphorus and/or 1,25-dihydroxy vitamin D in a subject with a hypophosphatemic disorder.

Anti-FGF23 antibody for increasing serum phosphorus

A method of increasing serum phosphorus levels in a subject having a hypophosphatemic disorder, comprising administering an effective amount of an anti-FGF23 antibody about every two weeks, wherein the anti-FGF23 antibody comprises the CDR sequences of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, and SEQ ID NO: 6.

Anti-FGF23 antibody for increasing serum 1,25-dihydroxy vitamin D

A method of increasing serum 1,25-dihydroxy vitamin D levels in a subject having a hypophosphatemic disorder, comprising administering an effective amount of an anti-FGF23 antibody about every two weeks, wherein the anti-FGF23 antibody comprises the CDR sequences of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, and SEQ ID NO: 6.

Anti-FGF23 antibody for increasing serum phosphorus and/or 1,25-dihydroxy vitamin D

A method of increasing serum concentrations of phosphorus and/or 1,25-dihydroxy vitamin D in a subject having a hypophosphatemic disorder, comprising administering an effective amount of an anti-FGF23 antibody about every two weeks, wherein the anti-FGF23 antibody comprises the CDR sequences of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, and SEQ ID NO: 6.

Overall, the claim coverage focuses on using a specifically defined anti-FGF23 antibody administered about every two weeks to increase serum phosphorus and/or serum 1,25-dihydroxy vitamin D in subjects with hypophosphatemic disorders.

Stated Advantages

Increases serum phosphorus levels in a subject having a hypophosphatemic disorder.

Increases serum 1,25-dihydroxy vitamin D levels in a subject having a hypophosphatemic disorder.

Increases serum concentrations of phosphorus and/or 1,25-dihydroxy vitamin D in a subject having a hypophosphatemic disorder.

Supports renal phosphate reabsorption as the mechanism.

Includes dose-proportional exposure and a linear correlation between KRN23 exposure (AUC) and changes in serum phosphorus, TmP/GFR, and 1,25(OH)2D.

Documented Applications

Clinical-study evaluation of KRN23 (anti-FGF23) pharmacodynamics and pharmacokinetics in adult XLH, including PK-PD relationships and changes in serum phosphorus, TmP/GFR, 1,25(OH)2D, and bone biomarkers.

Preliminary pediatric Phase 2 design comparing Q4 versus Q2 dosing, including preliminary data indicating steadier, less fluctuating increases under Q2 dosing and general tolerability.

Treatment of hypophosphatemic disorders, including X-linked hypophosphatemia (XLH), where the goal is increasing serum phosphorus levels and/or increasing serum 1,25-dihydroxy vitamin D levels.

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