Mitochondrial Diseases (Metabolic Disorders) – Drugs in Development, 2021

Mitochondrial Diseases (Metabolic Disorders) – Drugs in Development, 2021 provides an overview of the Mitochondrial Diseases pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mitochondrial Diseases, complete with analysis by Stage of Development, Drug Target, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type. The report also covers the descriptive Pharmacological Action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Mitochondrial Diseases and features dormant and discontinued projects.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note:

* Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the Global Therapeutic Landscape of Mitochondrial Diseases (Metabolic Disorders).

– The report reviews pipeline therapeutics for Mitochondrial Diseases (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

– The report covers pipeline products based on various stages of development ranging from discovery till pre-registration.

– The report features descriptive drug profiles for the pipeline products which includes, Product Description, Descriptive MoA, R&D Brief, Licensing and Collaboration details & Other Developmental Activities.

– The report reviews key players involved in the development of Mitochondrial Diseases (Metabolic Disorders) therapeutics and enlists all their major and minor projects.

– The report assesses Mitochondrial Diseases (Metabolic Disorders) therapeutics based on Drug Target, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type.

– The report summarizes all the dormant and discontinued pipeline projects.

– The report reviews latest news related to pipeline therapeutics for Mitochondrial Diseases (Metabolic Disorders).

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective Research and Development (R&D) strategies.

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Identify and understand important and diverse types of therapeutics under development for Mitochondrial Diseases (Metabolic Disorders).

- Identify potential new clients or partners in the target demographic.

- Develop strategic initiatives by understanding the focus areas of leading companies.

- Plan Mergers and Acquisitions (M&A) effectively by identifying key players and it’s most promising pipeline therapeutics.

- Devise corrective measures for pipeline projects by understanding Mitochondrial Diseases (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline.

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Mitochondrial Diseases – Overview

Mitochondrial Diseases – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Mitochondrial Diseases – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Mitochondrial Diseases – Companies Involved in Therapeutics Development

Abliva AB

Astellas Pharma Inc

Demuris Ltd

Energenesis Biomedical Co Ltd

Generian Pharmaceuticals Inc

Khondrion BV

Mission Therapeutics Ltd

Mitobridge Inc

OMEICOS Therapeutics GmbH

PTC Therapeutics Inc

SBI Pharmaceuticals Co Ltd

Triplex Therapeutics Inc

Yungjin Pharm Co Ltd

Mitochondrial Diseases – Drug Profiles

(aminolevulinic acid hydrochloride + sodium ferrous citrate) – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AH-901 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ENERGIF-707 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene-Modified Cell Therapy for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

KL-1333M – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ML-0426 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NV-354 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OMT-174 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

OMT-224 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule to Inhibit USP30 for Mitochondrial Diseases and Parkinson’s Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules 3 for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules 4 for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate AMPK for Glomerulonephritis, Polycystic Kidney Disease, Myotonic Dystrophy and Mitochondrial Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

sonlicromanol – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

vatiquinone – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Mitochondrial Diseases – Dormant Projects

Mitochondrial Diseases – Discontinued Products

Mitochondrial Diseases – Product Development Milestones

Featured News & Press Releases

Mar 16, 2021: Abliva reports completion of KL1333 Phase 1a/b study

Jan 04, 2021: Ablivas KL1333 heading for phase II/III in 2021

Dec 16, 2020: BioStock: Big investment sign of great interest in mitochondrial diseases

Nov 17, 2020: Abliva receives positive regulatory feedback from UK MHRA on KL1333 Phase II/III study plan

Nov 10, 2020: Abliva initiates a drug-drug interaction study and paves the way for a KL1333 pivotal study in 2021

Oct 07, 2020: Abliva doses the first patient in its KL1333 Phase Ia/b clinical study

Sep 09, 2020: Abliva intends to start a pivotal KL1333 study second half 2021

Jul 10, 2020: BioStock: Abliva’s KL1333 development plan receives OK from FDA

Jul 10, 2020: Abliva receives positive FDA feedback on its KL1333 development plan

Apr 05, 2020: Khondrion highlights potential role of inflammatory lipid modulator prostaglandin E2 in COVID-19 disease and proposes potential treatment pathway with sonlicromanol

Jan 27, 2020: Khondrion announces first patients dosed in Phase IIb study of Sonlicromanol for mitochondrial diseases

Dec 20, 2019: NeuroVive completes recruitment in the second part of its ongoing KL1333 clinical phase Ia/b study

Jul 04, 2019: NeuroVive initiates second part of its ongoing KL1333 Phase Ia/b clinical study

Mar 19, 2019: NeuroVive enrolls first subject in its KL1333 phase Ia/b clinical study

Nov 15, 2018: NeuroVive receives Vinnova funding to support development of NV354, a novel treatment for genetic mitochondrial disorders

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

Number of Products under Development for Mitochondrial Diseases, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Mitochondrial Diseases – Pipeline by Abliva AB, 2021

Mitochondrial Diseases – Pipeline by Astellas Pharma Inc, 2021

Mitochondrial Diseases – Pipeline by Demuris Ltd, 2021

Mitochondrial Diseases – Pipeline by Energenesis Biomedical Co Ltd, 2021

Mitochondrial Diseases – Pipeline by Generian Pharmaceuticals Inc, 2021

Mitochondrial Diseases – Pipeline by Khondrion BV, 2021

Mitochondrial Diseases – Pipeline by Mission Therapeutics Ltd, 2021

Mitochondrial Diseases – Pipeline by Mitobridge Inc, 2021

Mitochondrial Diseases – Pipeline by OMEICOS Therapeutics GmbH, 2021

Mitochondrial Diseases – Pipeline by PTC Therapeutics Inc, 2021

Mitochondrial Diseases – Pipeline by SBI Pharmaceuticals Co Ltd, 2021

Mitochondrial Diseases – Pipeline by Triplex Therapeutics Inc, 2021

Mitochondrial Diseases – Pipeline by Yungjin Pharm Co Ltd, 2021

Mitochondrial Diseases – Dormant Projects, 2021

Mitochondrial Diseases – Discontinued Products, 2021

List of Figures

Number of Products under Development for Mitochondrial Diseases, 2021

Number of Products under Development by Companies, 2021

Number of Products by Top 10 Targets, 2021

Number of Products by Stage and Top 10 Targets, 2021

Number of Products by Top 10 Mechanism of Actions, 2021

Number of Products by Stage and Top 10 Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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