# US 2019/0169138 A1 · Small molecule regulators of mitochondrial fusion and methods of use thereof

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- Number: US 2019/0169138 A1
- Document ID: US20190169138A1
- Status: Published application
- Kind: A1
- Application number: 16/152,172
- Filed: 2018-10-04
- Published: 2019-06-06
- Earliest claimed: 2017-04-23
- Assignees: Wahington University
- Applicants: Wahington University
- Inventors: Gerald W. Dorn, II; James Janetka
- Web page: https://patentgrep.com/patent/US20190169138A1

## CPC

- C07D 249/12: Oxygen or sulfur atoms
- A61K 31/4196: 1,2,4-Triazoles
- A61K 31/454: containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- C07D 405/12: linked by a chain containing hetero atoms as chain links
- C12N 9/16: acting on ester bonds (3.1)
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- 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

Compositions comprising small molecule mitofusin agonists are described. The mitofusin modulating agents are useful for treating diseases or disorders associated with a mitochondria-associated disease, disorder, or condition such as diseases or disorders associated with mitofusin 1 (Mfn1) and/or mitofusin 2 (Mfn2), or mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations, and screening methods for identifying compounds that regulate mitochondrial function are also described.

## Claims

### Claim 1

A compound of formula (I):
wherein, R1 is selected from the group consistina of
R2 is selected from the group consisting of
R3 is selected from the group consisting of hydrogen (H) and C1-8 alkyl;
R4 is selected form the group consisting of hydrogen (H) and C1-8 alkyl;
A is a SO, SO2;
X is N;
Y is N; and
Z is a linker group selected from the group consisting of a bond or C1-6 alkyl.

### Claim 2 (depends on 1)

The compound of claims 1, wherein, R1, R2, R3, or R4 are optionally substituted by one or more of:
acetamide, C1-8 alkoxy, amino, azo, Br, C1-8 alkyl, carbonyl, carboxyl, Cl, cyano, C3-8 cycloalkyl, C3-8 heteroaryl, C3-8 heterocyclyl, hydroxyl, F, halo, indole, N, nitrile, O, phenyl, S, sulfoxide, sulfur dioxide, or thiophene; and
optionally further substituted with one or more acetamide, alkoxy, amino, azo, Br, C1-8 alkyl, carbonyl, carboxyl, Cl, cyano, C3-8 cycloalkyl, C3-8 heteroaryl, C3-8 heterocyclyl, hydroxyl, F, halo, indole, N, nitrile, O, phenyl, S, sulfoxide, sulfur dioxide, or thiophene;
wherein,
the alkyl, cycloalkyl, heteroaryl, heterocyclyl, indole, or phenyl, is optionally further substituted with one or more selected from the group consisting of acetamide, alkoxy, amino, azo, Br, C1-8 alkyl, carbonyl, carboxyl, Cl, cyano, C3-8 cycloalkyl, C3-8 heteroaryl, C3-8 heterocyclyl, hydroxyl, F, halo, indole, N, nitrile, O, phenyl, S, sulfoxide, sulfur dioxide, or thiophene.

### Claim 3 (depends on 1)

The compound of claim 1, selected from the group consisting of:

### Claim 4 (depends on 1)

A pharmaceutical compostion comprising the compound of claim 1.