# US 2021/0330685 A1 · Inhalation formulations of 1'-cyano substituted carba-nucleoside analogs

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- Number: US 2021/0330685 A1
- Document ID: US20210330685A1
- Status: Published application
- Kind: A1
- Application number: 17/222,125
- Filed: 2021-04-05
- Published: 2021-10-28
- Earliest claimed: 2020-04-06
- Assignees: Gilead Sciences, Inc.
- Applicants: Gilead Sciences, Inc.
- Inventors: Scott Ellis; Davin S. Rautiola; Dustin S. Siegel; Maria M. Toteva; Adelle A. Vandersteen
- Web page: https://patentgrep.com/patent/US20210330685A1

## CPC

- A61K 31/685: one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
- 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 9/0078: {for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions}
- A61K 9/08: Solutions {(composition of solutions A61K47/00)}
- A61K 9/10: Dispersions; Emulsions
- A61K 9/19: lyophilised {, i.e. freeze-dried, solutions or dispersions (lyophilised products with subsequent particle size reduction A61K9/14; granules or pellets made by lyphilisation A61K9/1682; solid oral dosage forms made by lyophilisation A61K9/2095; lyophilisation additives A61K47/00)}
- A61K 31/675: having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
- A61K 47/40: Cyclodextrins; Derivatives thereof
- A61P 31/12: Antivirals
- A61P 31/14: for RNA viruses
- A61P 31/16: for influenza or rhinoviruses
- A61P 31/18: for HIV
- Y02A 50/30: Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
- 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
- Y02A: TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE

## Abstract

The present disclosure provides pharmaceutical formulations of the compound of Formula I, Formula Ia, or Formula Ib, or a pharmaceutically acceptable salt thereof, and an aqueous vehicle. The pharmaceutical formulations of the disclosure are useful in treatment and prevention of viral infections in subjects in need thereof and are for administration by inhalation.

## Claims

### Claim 1

A pharmaceutical formulation comprising:
i. a compound of Formula I, Formula Ia, or Formula Ib:
or a pharmaceutically acceptable salt thereof; and
ii. an aqueous vehicle;
wherein the pharmaceutical formulation is suitable for administration via inhalation.

### Claim 2

A pharmaceutical formulation comprising:
i. a compound of Formula I:
or a pharmaceutically acceptable salt thereof; and
ii. an aqueous vehicle;
wherein the pharmaceutical formulation is suitable for administration via inhalation.

### Claim 3

A pharmaceutical formulation comprising:
i. a compound of Formula Ia:
or a pharmaceutically acceptable salt thereof; and
ii. an aqueous vehicle;
wherein the pharmaceutical formulation is suitable for administration via inhalation.

### Claim 4

A pharmaceutical formulation comprising:
i. a compound of Formula Ib:
or a pharmaceutically acceptable salt thereof; and
ii. an aqueous vehicle;
wherein the pharmaceutical formulation is suitable for administration via inhalation.

### Claim at position 5

5-49. (canceled)

### Claim 50

A pharmaceutical formulation comprising:
i. a compound of Formula I, Formula Ia, or Formula Ib:
or a pharmaceutically acceptable salt thereof;
ii. water; and
iii. cyclodextrin;
wherein the pharmaceutical formulation is suitable for administration via inhalation.

### Claim 51 (depends on 50)

The pharmaceutical formulation of claim 50, wherein the formulation comprises
i. a compound of Formula Ia:
or a pharmaceutically acceptable salt thereof;
ii. water; and
iii. cyclodextrin.

### Claim 52 (depends on 1)

The pharmaceutical formulation of claim 1, wherein the pharmaceutical formulation is suitable for administration via inhalation in a nebulized form.

### Claim 53 (depends on 1)

A kit comprising the pharmaceutical formulation of claim 1.

### Claim 54

(canceled)

### Claim 55 (depends on 1)

An inhalation device comprising the pharmaceutical formulation of claim 1.

### Claim 56

(canceled)

### Claim 57 (depends on 1)

A method of treating or preventing a viral infection in a human in need thereof, wherein the method comprises administering to the human the pharmaceutical formulation of claim 1.

### Claim 58

(canceled)

### Claim 59 (depends on 57)

The method of claim 57, wherein the method comprises administering to the human at least one additional therapeutic agent.

### Claim 60 (depends on 57)

The method of claim 57, wherein the viral infection is a coronavirus infection.

### Claim 61 (depends on 57)

The method of claim 57, wherein the viral infection is SARS-CoV-2 infection (COVID-19).

### Claim 62 (depends on 57)

The method of claim 57, wherein the viral infection is a SARS virus infection.

### Claim 63 (depends on 57)

The method of claim 57, wherein the viral infection is a MERS virus infection.

### Claim 64 (depends on 57)

The method of claim 57, wherein the viral infection is a pneumoviridae virus infection.

### Claim at position 21

-66. (canceled)

### Claim 67 (depends on 57)

The method of claim 57, wherein the viral infection is a picornaviridae virus infection.

### Claim 68

(canceled)

### Claim 69 (depends on 57)

The method of claim 57, wherein the viral infection is a flaviviridae virus infection.

### Claim 70

(canceled)

### Claim 71 (depends on 57)

The method of claim 57, wherein the viral infection is a filoviridae virus infection.

### Claim 72

(canceled)

### Claim 73 (depends on 57)

The method of claim 57, wherein the viral infection is an orthomyxovirus infection.

### Claim 74 (depends on 73)

The method of claim 73, wherein the viral infection is an influenza virus infection.

### Claim 75 (depends on 57)

The method of claim 57, wherein the viral infection is a paramyxoviridae virus infection.

### Claim at position 31

-81. (canceled)

### Claim 82

A method of treating or preventing a viral infection in a human in need thereof, comprising administering to the human a reconstituted lyophilized formulation by inhalation; wherein the lyophilized pharmaceutical formulation comprises:
i. a compound of Formula I, Formula Ia, or Formula Ib:
or a pharmaceutically acceptable salt thereof; and
ii. cyclodextrin.

### Claim 83 (depends on 82)

The method of claim 82, wherein the lyophilized formulation comprises:
i. the compound of Formula Ia; and
ii. cyclodextrin.

### Claim 84 (depends on 82)

The method of claim 82, wherein the lyophilized formulation comprises the compound of Formula I, Formula Ia, or Formula Ib in an amount of 1% to 10% w/w and cyclodextrin in an amount of from 90% to 99% w/w.

### Claim at position 35

-91. (canceled)

### Claim 92 (depends on 82)

The method of claim 82, wherein the cyclodextrin is a beta-cyclodextrin.

### Claim 93 (depends on 82)

The method of claim 82, wherein the cyclodextrin is a beta-cyclodextrin selected from a group consisting of sulfobutylalkylether-beta-cyclodextrin, betadex-sulfobutylether sodium, and hydroxypropyl-beta-cyclodextrin.

### Claim at position 38

-95. (canceled)

### Claim 96 (depends on 57)

The method of claim 57, wherein the human receives at least one dose of remdesivir by intravenous infusion.

### Claim 97

(canceled)

### Claim 98

A lyophilized formulation comprising:
(i) a compound of Formula I, Formula Ia, or Formula Ib in an amount of 5% to 10% w/w; and
(ii) cyclodextrin in an amount of from 90% to 95% w/w.

### Claim at position 42

-102. (canceled)

### Claim 103 (depends on 98)

The lyophilized formulation of claim 98, wherein the cyclodextrin is a beta-cyclodextrin.

### Claim at position 44

-105. (canceled)