# US 2022/0016103 A1 · Methods of using factor b inhibitors

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- Number: US 2022/0016103 A1
- Document ID: US20220016103A1
- Status: Published application
- Kind: A1
- Application number: 17/376,483
- Filed: 2021-07-15
- Published: 2022-01-20
- Earliest claimed: 2020-07-16
- Assignees: Novartis AG
- Applicants: Novartis AG
- Inventors: Irina BALTCHEVA; Peter END; Julie Marie-Pomme Gabrielle MILOJEVIC
- Web page: https://patentgrep.com/patent/US20220016103A1

## CPC

- A61K 31/454: containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61P 7/06: Antianaemics
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Described herein are methods of treating paroxysmal nocturnal hemoglobinuria (PNH) with the Factor B inhibitor LNP023 or a pharmaceutically acceptable salt thereof, e.g. LNP023 hydrochloride.

## Claims

### Claim 1

A method of treating paroxysmal nocturnal hemoglobinuria (PNH) in a subject in need thereof, the method comprising orally administering to the subject LNP023 or a pharmaceutically acceptable salt thereof, at a dose of about 200 mg twice daily (b.i.d.), to thereby treat the subject (wherein the dosing amount refers to the anhydrous free base of LNP023 hydrochloride).

### Claim 2 (depends on 1)

The method of claim 1, wherein the subject has been previously treated with an anti-C5 therapy prior to administration of LNP023 or a pharmaceutically acceptable salt thereof.

### Claim 3 (depends on 2)

The method of claim 2, wherein the anti-C5 therapy is an anti-C5 monoclonal antibody therapy.

### Claim 4 (depends on 1)

The method of claim 1, wherein the subject has residual anemia.

### Claim 5 (depends on 1)

The method of claim 1, wherein hemoglobin levels, haptoglobin levels, reticulocyte levels, and/or bilirubin levels of the subject, are evaluated prior to administration of LNP023 or a pharmaceutically acceptable salt thereof.

### Claim 6 (depends on 5)

The method of claim 5, wherein the hemoglobin levels of the subject are less than or equal to about 10 g/dL, prior to administration of LNP023 or a pharmaceutically acceptable salt thereof.

### Claim 7 (depends on 1)

The method of claim 1, wherein treating PNH comprises increasing hemoglobin levels in the subject as compared to hemoglobin levels in the subject prior to administration of LNP023 or a pharmaceutically acceptable salt thereof.

### Claim 8 (depends on 1)

The method of claim 1, wherein treating PNH comprises normalizing intravascular hemolysis (IVH) and/or extravascular hemolysis (EVH) in the subject.

### Claim 9 (depends on 8)

The method of claim 8, wherein normalizing IVH and/or EVH hemolysis comprises increasing the haptoglobin level, decreasing reticulocytes level, or decreasing bilirubin level, in the subject as compared to baseline.

### Claim 10 (depends on 1)

The method of claim 1, wherein treating PNH comprises treating PNH associated hemolysis.

### Claim 11 (depends on 1)

The method of claim 1, wherein treating PNH comprises reducing C3 deposition in the subject.

### Claim 12 (depends on 1)

The method of claim 1, wherein treating PNH comprises increasing red blood cell (RBC) survival in the subject.

### Claim 13 (depends on 1)

The method of claim 1, wherein treating PNH comprises reducing Fragment Bb level in the subject.

### Claim 14 (depends on 1)

The method of claim 1, wherein treating PNH comprises increasing hemoglobin levels in the subject by about 2 g/dL or more as compared to baseline.

### Claim 15 (depends on 1)

The method of claim 1, wherein treating PNH comprises achieving a sustained increase in hemoglobin level.

### Claim 16 (depends on 1)

The method of claim 1, wherein treating PNH comprises achieving a sustained increase in hemoglobin level to greater than or equal to about 12 g/dL.

### Claim 17 (depends on 1)

The method of claim 1, wherein treating PNH comprises increasing hemoglobin levels in the subject in the absence of receiving one or more red blood cell (RBC) transfusion(s).

### Claim 18 (depends on 1)

The method of claim 1, wherein the subject has received RBC transfusions.

### Claim 19 (depends on 1)

The method of claim 1, wherein the subject has been vaccinated prior to administration of LNP023 or a pharmaceutically acceptable salt thereof prior vaccination against Neisseria meningitidis (types A, C, Y and W-135).

### Claim 20 (depends on 1)

The method of claim 1, wherein efficacy of treatment is determined by measuring the hemoglobin levels in the subject as compared to baseline.