公开(公告)号：US20250009717A1

公开(公告)日：2025-01-09

申请号：US18672743

申请日：2024-05-23

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Vania Broccoli

IPC: A61K31/4245 ,  A61K31/135 ,  A61K31/197 ,  A61K31/4152 ,  A61K31/4178 ,  A61K31/4184 ,  A61K31/5513 ,  A61K38/30 ,  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：US12037350B2

公开(公告)日：2024-07-16

申请号：US17221463

申请日：2021-04-02

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng

IPC: C07F7/08 ,  C07C43/23 ,  C07C50/28

CPC classification number: C07F7/081 ,  C07C43/23 ,  C07C50/28

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：US12016843B2

公开(公告)日：2024-06-25

申请号：US17689666

申请日：2022-03-08

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Vania Broccoli

IPC: A61K31/4245 ,  A61K31/135 ,  A61K31/197 ,  A61K31/4152 ,  A61K31/4178 ,  A61K31/4184 ,  A61K31/5513 ,  A61K38/30 ,  A61P25/28

CPC classification number: A61K31/4245 ,  A61K31/135 ,  A61K31/197 ,  A61K31/4152 ,  A61K31/4178 ,  A61K31/4184 ,  A61K31/5513 ,  A61K38/30 ,  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：US20230303506A1

公开(公告)日：2023-09-28

申请号：US18087890

申请日：2022-12-23

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Pavels Arsenjans

IPC: C07D305/10 ,  C07C49/835

CPC classification number: C07D305/10 ,  C07C49/835

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：US20230256047A1

公开(公告)日：2023-08-17

申请号：US18010526

申请日：2021-06-16

Applicant: STEALTH BIOTHERAPEUTICS INC.

Inventor： David BROWN ,  Jim CARR ,  Dennis Keefe

IPC: A61K38/07 ,  A61P9/00 ,  A61P21/00 ,  A61K31/56 ,  A61K31/7125 ,  C12N15/113

CPC classification number: A61K38/07 ,  A61K31/56 ,  A61K31/7125 ,  A61P9/00 ,  A61P21/00 ,  C12N15/113 ,  C12N2310/11

Abstract: The disclosure provides methods for treating muscular dystrophy (MD), such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) in a mammalian subject. The methods are particularly useful for treating, inhibiting, reducing, ameliorating or delaying the onset of hypertrophic cardiomyopathy, dilated cardiomyopathy, heart failure and/or cardiac fibrosis in subjects diagnosed with, and/or being treated for, MD.The methods comprise administering to the subject an effective amount of a peptide such as H-D-Arg-2,6-Dmt-Lys-PHe-NH2 (a.k.a. elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, optionally in combination with at least one other active ingredient (i.e. drug) such a corticosteroid, ACE inhibitor, ARB(s), beta-blocker or drug that increases or corrects the expression of dystrophin in the subject (e.g. Eteplirsen (Exondys 51®), Golodirsen (Vyondys 53™)) or Casimersen (Amondys 45™).

公开(公告)号：US12049455B2

公开(公告)日：2024-07-30

申请号：US18087890

申请日：2022-12-23

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Pavels Arsenjans

IPC: C07D305/10 ,  C07C49/835

CPC classification number: C07D305/10 ,  C07C49/835

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：US20230405080A1

公开(公告)日：2023-12-21

申请号：US18460346

申请日：2023-09-01

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Martin Redmon ,  Brian Hotchkiss ,  Anthony Abbruscato

IPC: A61K38/05 ,  A61P27/02

CPC classification number: A61K38/05 ,  A61P27/02

Abstract: The disclosure generally relates to compounds (i.e. peptidomimetics), compositions (e.g. formulations or medicaments), methods and related uses for treating, preventing, inhibiting, amelioration or delaying the onset of ophthalmic diseases, disorders or conditions in a mammalian subject, such as a human. In some embodiments, the ophthalmic disease, disorder or condition may be associated with deterioration of the integrity of the ellipsoid zone of one or more eyes of the mammalian subject. The methods and uses comprise administering an effective amount of peptidomimetic (alone, as formulated and/or in combination with at least one additional therapeutic agent) to mammalian subjects in need thereof.

公开(公告)号：US20240400588A1

公开(公告)日：2024-12-05

申请号：US18672819

申请日：2024-05-23

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng

IPC: C07F7/08 ,  C07C43/23 ,  C07C50/28

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：US11584728B2

公开(公告)日：2023-02-21

申请号：US17062339

申请日：2020-10-02

Applicant: STEALTH BIOTHERAPEUTICS INC.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Pavels Arsenjans

IPC: C07C49/835 ,  C07D305/10

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：US20240400529A1

公开(公告)日：2024-12-05

申请号：US18761854

申请日：2024-07-02

Applicant: Stealth BioTherapeutics Inc.

Inventor： Dennis Keefe ,  Guozhu Zheng ,  Pavels Arsenjans

IPC: C07D305/10 ,  C07C49/835

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)).