公开(公告)号：BR112022001123A2

公开(公告)日：2022-03-15

申请号：BR112022001123

申请日：2020-07-23

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： KEEFE DENNIS ,  ZHENG GUOZHU ,  BROCCOLI VANIA

IPC: A61K45/06 ,  A61K31/4245 ,  A61P25/16 ,  A61P25/28

Abstract: o composto peptidomimétido (r)-2-amino-n-((s-l-(((s)-5-amino-l-(3-benzil-1,2,4,-oxadiazol-5-il)pentil)amino-3-(4-hidroxi-2,6-dimetilfenil)-1-oxopropan-2-il)-5-guanidinopentanamida no tratamento de doenças neurodegenerativas. a presente divulgação fornece novos métodos para tratar ou prevenir a esclerose lateral amiotrófica (als), métodos para retardar o início dos sintomas neurológicos associados à als, aumentar a sobrevivência em sujeitos afetados por als e atenuar o declínio da força muscular associada à als em um sujeito precisando do mesmo. a presente divulgação também fornece métodos para tratar ou prevenir a-sinucleinopatia ou proteinopatia tdp-43. os métodos compreendem administrar ao sujeito uma quantidade eficaz de um composto peptidomimético direcionado a mitocôndrias, tal como (r)-2-amino-n-((s)-1-(((s)-5-amino-1-(3-benzil-1,2,4-oxadiazol-5-il)pentil)amino)-3-(4-hidroxi-2,6-dimetilfenil)-1-oxopropan-2-il)-5-guanidinopentanamida, ou um sal, estereoisômero, tautômero, hidrato e/ou solvato farmaceuticamente aceitável do mesmo.

公开(公告)号：CA3156499A1

公开(公告)日：2021-04-08

申请号：CA3156499

申请日：2020-10-02

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： KEEFE DENNIS ,  ZHENG GUOZHU ,  ARSENJANS PAVELS

IPC: C07F7/08 ,  C07C50/28 ,  C07D201/00

Abstract: The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g. Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).

公开(公告)号：AU2020358970A1

公开(公告)日：2022-04-14

申请号：AU2020358970

申请日：2020-10-02

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： KEEFE DENNIS ,  ZHENG GUOZHU ,  ARSENJANS PAVELS

IPC: C07F7/08 ,  C07C50/28 ,  C07D201/00

Abstract: The disclosure provides therapeutic compositions (

公开(公告)号：AU2020316449A8

公开(公告)日：2022-02-17

申请号：AU2020316449

申请日：2020-07-23

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： BROCCOLI VANIA ,  KEEFE DENNIS ,  ZHENG GUOZHU

IPC: A61K31/4245 ,  A61K45/06 ,  A61P25/16 ,  A61P25/28

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

公开(公告)号：CA3148090A1

公开(公告)日：2021-01-28

申请号：CA3148090

申请日：2020-07-23

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： KEEFE DENNIS ,  ZHENG GUOZHU ,  BROCCOLI VANIA

IPC: A61K38/05 ,  A61P25/16 ,  A61P25/28

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing a-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

公开(公告)号：AU2021246142A1

公开(公告)日：2022-10-20

申请号：AU2021246142

申请日：2021-04-02

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： KEEFE DENNIS ,  ZHENG GUOZHU

IPC: C07D319/18 ,  A61K31/122 ,  A61P9/00 ,  A61P27/02 ,  C07C39/08 ,  C07C43/23 ,  C07C50/06 ,  C07C50/24 ,  C07D317/46

Abstract: The disclosure provides therapeutic compounds, compositions (e.g., therapeutic agents or medicaments) and methods for preventing or treating mitochondrial disease such as Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with mitochondrial disease, such as Friedreich's ataxia, and/or reducing the likelihood or severity of mitochondrial disease such as Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions. In some instances, the intermediates may themselves by therapeutic agents or prodrugs of therapeutic agents (e.g. reduced forms of the therapeutic compounds).

公开(公告)号：AU2020316449A1

公开(公告)日：2022-02-10

申请号：AU2020316449

申请日：2020-07-23

Applicant: STEALTH BIOTHERAPEUTICS INC

Inventor： BROCCOLI VANIA ,  KEEFE DENNIS ,  ZHENG GUOZHU

IPC: A61K31/4245 ,  A61K45/06 ,  A61P25/16 ,  A61P25/28

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.