# US 2025/0205346 A1 · Anti-c5 agent for treatment of dry age-related macular degeneration (amd) or geographic atrophy secondary to dry amd

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- Number: US 2025/0205346 A1
- Document ID: US20250205346A1
- Status: Published application
- Kind: A1
- Application number: 19/079,359
- Filed: 2025-03-13
- Published: 2025-06-26
- Earliest claimed: 2019-10-27
- Assignees: Astellas US LLC
- Applicants: Astellas US LLC
- Inventors: Kourous A. REZAEI
- Web page: https://patentgrep.com/patent/US20250205346A1

## CPC

- A61K 47/60: the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 9/0048: {Eye, e.g. artificial tears}
- A61P 27/02: Ophthalmic agents
- C12N 15/115: Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith {; Nucleic acids binding to non-nucleic acids, e.g. aptamers}
- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- C12N 2310/16: Aptamers
- C12N 2310/317: with an inverted bond, e.g. a cap structure
- C12N 2310/351: Conjugate
- 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
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

This invention relates to methods and compositions useful for treatment of subjects with dry age-related macular degeneration or geographic atrophy secondary to dry age-related macular degeneration. The methods involve administration of a pharmaceutical composition comprising an anti-C5 agent ARC1905, which comprises a C5-specific aptamer conjugated to a polyethylene glycol moeity via a linker, in an amount effective for slowing or inhibiting loss of low luminance visual acuity in the subject. The aptamer consists of the sequence fCmGfCfCGfCmGmGfUfCfUfCmAmGmGfCGfCfUmGmAmGfUfCfUmGmAmGfUfUfUAfCf CfUmGfCmG-3T, wherein fC and fU=2′ fluoro nucleotides, mG and mA=2′-OMe nucleotides, all other nucleotides are 2′-OH, and 3T indicates an inverted deoxythymidine. Dosages and administration schedules are disclosed.

## Claims

### Claim 1

A method for treating a subject with geographic atrophy secondary to dry age-related macular degeneration, the method comprising administering to the subject an anti-C5 agent,
wherein the anti-C5 agent comprises a C5-specific aptamer comprising the following structure:
or a salt thereof,
wherein Aptamer=fCmGfCfCGfCmGmGfUfCfUfCmAmGmGfCGfCfUmGmAmGfUfCfUmGmAmGfUfU fUAfCfCfUmGfCmG-3T (SEQ ID NO: 1), wherein fC and fU=2′ fluoronucleotides, mG and mA=2′-OMe nucleotides, all other nucleotides are 2′-OH, and 3T indicates an inverted deoxythymidine;
wherein the anti-C5 agent is administered to the subject in a loading phase followed by a maintenance phase, wherein the loading phase comprises administering the anti-C5 agent at a dose of about 2 mg/eye for a duration of twelve months at a frequency in which the duration between doses is one month, and wherein the maintenance phase comprises administering the anti-C5 agent at a dose of about 2 mg/eye thereafter at a frequency in which the duration between doses is two months; and
wherein the anti-C5 agent is administered intraocularly to the subject via intravitreal injection into the eye.

### Claim 2

(canceled)

### Claim 3 (depends on 1)

The method according to claim 1, wherein the administration of the anti-C5 agent to the subject slows or inhibits the decrease in low luminance best corrected visual acuity as compared to a subject who is not administered the anti-C5 agent.

### Claim 4 (depends on 1, 3)

The method according to claim 1 or claim 3, wherein the administration of the anti-C5 agent to the subject increases the low luminance best corrected visual acuity as compared to a subject who is not administered the anti-C5 agent.

### Claim 5 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 1 month.

### Claim 6 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 6 months.

### Claim 7 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 8 months.

### Claim 8 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 12 months.

### Claim 9 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 18 months.

### Claim 10 (depends on 4)

The method according to claim 4, wherein the increase is measured between baseline and at least or about 24 months.

### Claim 11 (depends on 3)

The method according to claim 3, wherein the low luminance best corrected visual acuity is measured using ETDRS letters.

### Claim 12

(canceled)

### Claim 13

(canceled)

### Claim 14

(canceled)

### Claim 15 (depends on 1, 3, 4, 5, 6, 7, 8, 9, 10, 11)

The method according to any one of claims 1 and 3-11, wherein subject is human.

### Claim 16

(canceled)