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Mitochondrial Disease Therapies Market, 2019-2030

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    Report

  • 250 Pages
  • July 2019
  • Region: Global
  • Roots Analysis
  • ID: 4846369
Description Jump to:

Mitochondrial diseases are characterized by chronic, genetic and progressive medical symptoms that arise due to inherited or spontaneous mutations in mitochondrial DNA (a small, circular, double-stranded with ~16,500 base pairs that exist outside of the nucleus), or in some cases, nuclear DNA, which adversely alter the function of mitochondria. Such clinical conditions are rare and are known to affect multiple parts of the body, including neurons and nerves, kidneys, heart, liver, eyes, ears, and pancreas. It is worth highlighting that over 300 mutations have been identified and are associated with various mitochondrial diseases.  Examples of diseases that are caused due to mutations in mitochondrial DNA include Leber's hereditary optic neuropathy (LHON), Leigh syndrome, mitochondrial DNA depletion syndrome, mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), and myoclonic epilepsy with ragged red fibers (MERRF).

In the US, mitochondrial diseases are known to annually affect 1 in 5,000 individuals. It is also estimated that 1,000-4,000 children are born with a mitochondrial disease every year, in the same region.  Primary mitochondrial diseases are usually difficult to diagnose. In many cases, clinicians and medical professionals are compelled to use the term possible mitochondrial disease, indicating that based on the presenting symptoms they suspect a patient to be suffering from a mitochondrial disease. However, they are unable to confirm the condition via genetic diagnosis. Such instances are further complicated by the aforementioned uncertainty, leading to delays in treatment and subsequent deterioration in the patient’s quality of life.  Currently, several industry stakeholders are engaged in efforts to develop disease-modifying interventions for treating different mitochondrial diseases, in addition to drug/therapy candidates that offer symptomatic relief. There are several non-profit organizations that have been established across the globe to support individuals suspected/diagnosed with mitochondrial diseases and also fund research / clinical studies being conducted for the treatment of such conditions. In the coming years, the market is anticipated to grow at a significant pace as more novel therapy solutions are approved by regulatory authorities and enter the market.

Scope of the Report

The “Mitochondrial Disease Therapies Market, 2019-2030” report features an extensive study of the current market landscape, offering an informed opinion on the likely adoption of therapeutics designed to treat mitochondrial diseases. It features an in-depth analysis, highlighting the capabilities of stakeholder companies/organizations engaged in this domain. In addition to other elements, the study includes:


  • A detailed assessment of the current market landscape, providing information on drug developer(s), phase of development (marketed, clinical and preclinical / discovery stage) of lead candidates, type of molecule (small molecule and biologic), type of therapy (monotherapy and combination therapy), path to clinic (dedicated, repurposed and repositioned), target disease indication, and route of administration of the drugs / therapies that are being developed for the treatment of different mitochondrial diseases.
  • Detailed profiles of the players that are engaged in the development of therapies for mitochondrial diseases (shortlisted on the basis of the number/phase of products), featuring an overview of the company, its financial information (if available), a description of its product portfolio, recent collaborations and an informed future outlook.
  • An analysis of close to 200 peer-reviewed, scientific articles published during the period 2014-2019 (till February), highlighting the research focus within this niche industry segment. It includes an informed opinion on the key trends observed across the aforementioned publications, including information on target disease indications, target mutations across different indications, and analysis based on various relevant parameters, such as study type (review article, research article and case report), year of publication, and most popular journals (in terms of number of articles published in the given time period).
  • A list of key opinion leaders (KOLs) within this domain, featuring detailed 2X2 matrices to assess the relative experience of key individuals, who were shortlisted based on their contributions (in terms of involvement in various clinical studies) to this field. It also includes a schematic world map representation, highlighting the geographical locations of eminent scientists/researchers engaged in this domain. In addition, it presents an analysis assessing the credibility and (relative) level of expertise of different KOLs, based on the number of publications, number of citations, number of clinical trials, number of affiliations and strength of professional network (based on information available on LinkedIn).
  • A study of the various grants that have been awarded to research institutes engaged in projects related to different types of mitochondrial diseases, between 2015 and 2019 (till May), highlighting various important parameters, such as year of award, support period, amount awarded, funding institute, grant type, responsible study section, focus area and type of recipient organization.

One of the key objectives of the report was to estimate the existing market size and the future opportunity of therapies for mitochondrial diseases, over the next decade. Based on multiple parameters, such as disease prevalence, anticipated adoption of the forecasted therapies and the likely selling price of such therapeutic products, we have provided informed estimates on the evolution of the market for the period 2019-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] different target indications (Leigh syndrome, LHON, MELAS, mitochondrial DNA depletion syndrome, mitochondrial myopathy, and others), [B] type of therapy (combination and monotherapy), [C] route of administration (oral, intravenous, and others) and [D] key geographical regions (the US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.  

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD unless otherwise specified.


Table of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. An Overview of Mitochondria
3.1.1. Structure and Function of Mitochondria
3.1.2. Mitochondrial DNA
3.1.3. Mitochondrial Inheritance
3.2. Mitochondrial Genetic Diseases
3.2.1. Genetic Mutations and Clinical Manifestations of Mitochondrial Diseases
3.2.2. Diagnosis of Mitochondrial Diseases
3.3. Emerging Therapies for Mitochondrial Diseases
3.4. Initiatives Supporting Awareness and Research on Mitochondrial Diseases
3.5. Challenges and Future Perspectives in Treatment of Mitochondrial Diseases
4. CURRENT MARKET LANDSCAPE
4.1. Chapter Overview
4.2. Mitochondrial Disease Therapies: Marketed and Development Pipeline
4.2.1. Analysis by Phase of Development
4.2.2. Analysis by Target Disease Indication
4.2.3. Analysis by Type of Therapy
4.2.4. Analysis by Type of Molecule
4.2.5. Analysis by Route of Administration
4.2.6. Analysis by Path to Clinic
4.2.7. Analysis by Target Population
4.2.8 Analysis by Type of Maintenance Defect
4.2.9 Analysis by Common Clinical Manifestations
4.2.10 Analysis by Age of Disease Onset
4.2.11 Analysis by Molecular Target
4.2.12. Funnel Analysis: Analysis by Phase of Development, Type of Molecule and Path to Clinic
4.2.13 Grid Analysis: Analysis by Target Disease Indication, Path to Clinic and Type of Molecule
4.3. Mitochondrial Disease Therapies: List of Therapy Developers
4.3.1. Analysis by Year of Establishment
4.3.2. Analysis by Company Size and Location of Headquarters
4.3.3. Analysis by Geography
5. COMPANY PROFILE: MITOCHONDRIAL DISEASES
5.1. NeuroVive Pharmaceutical
5.1.1. Company Overview
5.1.2. Financial Information
5.1.3. Mitochondrial Disease Product Portfolio
5.1.4. Recent Collaborations
5.1.5. Future Outlook
5.2. Ixchel Pharma
5.2.1. Company Overview
5.2.2. Mitochondrial Diseases Product Portfolio
5.3. Stealth BioTherapeutics
5.3.1. Company Overview
5.3.2. Mitochondrial Disease Product Portfolio
5.3.3. Recent Collaborations
5.3.4. Future Outlook
5.4. Santhera Pharmaceuticals
5.4.1. Company Overview
5.4.2. Financial Information
5.4.3. Mitochondrial Disease Product Portfolio
5.4.4. Recent Collaborations
5.4.5. Future Outlook
5.5. GenSight Biologics
5.5.1. Company Overview
5.5.2. Mitochondrial Disease Product Portfolio
5.5.1. Recent Collaborations
5.5.2. Future Outlook
5.6. BioElectron Technology
5.6.1. Company Overview
5.6.2. Mitochondrial Disease Product Portfolio
5.6.4. Recent Collaborations
5.7. Reata Pharmaceuticals
5.7.1. Company Overview
5.7.2. Financial Information
5.7.3. Mitochondrial Disease Product Portfolio
5.7.4. Future Outlook
6. PUBLICATION ANALYSIS
6.1. Chapter Overview
6.2. Types of Study
6.3. Methodology
6.4. Mitochondrial Diseases: List of Publications
6.4.1. Analysis by Year of Publication
6.4.2. Analysis by Type of Study
6.4.3. Analysis by Type of Study Objective
6.4.4. Analysis by Mutated Gene / Phenotype of Disorder
6.4.5. Analysis by Type of Mutation
6.4.6. Analysis by Target Disease Indication
6.4.7. Key Journals: Analysis by Number of Publications
7. CASE STUDY: MITOCHONDRIAL DYSFUNCTION IN VARIOUS DISEASES
7.1. Chapter Overview
7.2. Huntington’s Disease
7.2.1. Mitochondrial Dysfunction in Huntington’s Disease
7.2.2. Treatment Strategies for Huntington’s Disease
7.3. Rett Syndrome
7.3.1. Mitochondrial Dysfunction in Rett Syndrome
7.3.2. Treatment Strategies for Rett Syndrome
7.4. Non-alcoholic Steatohepatitis (NASH)
7.4.1. Mitochondrial Dysfunction in NASH
7.4.2. Treatment Strategies for NASH
7.5. Type-2 Diabetes
7.5.1. Mitochondrial Dysfunction in Type-2 Diabetes
7.5.2. Treatment Strategies for Type-2 Diabetes
7.6. Friedreich’s Ataxia
7.6.1. Mitochondrial Dysfunction in Friedreich’s Ataxia
7.6.2. Treatment Strategies for Friedreich’s Ataxia
8. GRANT ANALYSIS
8.1. Chapter Overview
8.2. Scope and Methodology
8.3. Mitochondrial Diseases: List of Academic Grants
8.3.1. Analysis by Project Start Year
8.3.2. Analysis by Year-wise Trends
8.3.3. Analysis by Amount Awarded
8.3.4. Analysis by Support Period
8.3.5. Analysis by Project Terms
8.3.6. Analysis by Type of Grant Application
8.3.7. Analysis by Most Popular NIH Departments
8.3.8. Analysis by Study Section
8.3.9. Analysis by Focus Area of the Grant
8.3.10. Analysis by Popular Indications
8.3.11. Analysis by Type of Recipient Organization
8.3.12. Analysis by Most Popular Recipient Organization
8.3.13. Analysis by Administrative Institutes and Centers
8.3.14. Analysis by Activity of Grants
8.3.15. Analysis by Support Period by Administering Organizations
8.3.16. Analysis by Prominent Project Leaders
8.3.17. Analysis by Funding Institute Center
8.3.18. Analysis by Funding Mechanism
8.3.19. Regional Distribution of Recipient Organization
9. KOL ANALYSIS
9.1. Chapter Overview
9.2. Methodology
9.3. Principal Investigators / Involved in Clinical Trials
9.3.1. Analysis by Type of Organizations
9.3.2. Geographical Distribution of Key Opinion Leaders
9.4. Prominent Key Opinion Leaders
9.5. KOL Benchmarking: Roots Analysis versus Third Party Scoring (ResearchGate Score)
9.6. Most Active Key Opinion Leaders
9.6.1. Profile: KOL A (Merrimack College)
9.6.2. Profile: KOL B (McMaster University)
9.6.3. Profile: KOL C (Hospital Necker-Enfants Malades)
9.6.4. Profile: KOL D (Mayo Clinic)
9.6.5. Profile: KOL E (University of Texas)
9.6.6. Profile: KOL F (Stanford University)
9.6.7. Profile: KOL G (University of Iowa Department of Ophthalmology and Visual Sciences)
9.6.8. Profile: KOL H (Monraz Medical Complex)
9.6.9. Profile: KOL I (Herlev Hospital)
10. MARKET SIZING AND OPPORTUNITY ANALYSIS
10.1. Chapter Overview
10.2. Forecast Methodology and Key Assumptions
10.3. Global Mitochondrial Disease Therapies Market, 2019-2030
10.4. Global Mitochondrial Disease Therapies Market: Product-wise Sales Forecasts
10.4.1. Raxone (Santhera Pharmaceuticals)
10.4.1.1. Target Patient Population
10.4.1.2. Sales Forecast
10.4.2. GS010 (GenSight Biologics)
10.4.2.1. Target Patient Population
10.4.2.2. Sales Forecast
10.4.3. Taurine Powder (Taisho Toyama Pharmaceuticals)
10.4.3.1. Target Patient Population
10.4.3.2. Sales Forecast
10.4.4. Elamipretide (Stealth BioTherapeutics)
10.4.4.1. Target Patient Population
10.4.4.2. Sales Forecast
10.4.5. KH176 (Khondrion)
10.4.5.1. Target Patient Population
10.4.5.2. Sales Forecast
10.4.6. Omaveloxolone (Reata Pharmaceuticals)
10.4.6.1. Target Patient Population
10.4.6.2. Sales Forecast
10.4.7. KL1333 (NeuroVive Pharmaceutical)
10.4.7.1. Target Patient Population
10.4.7.2. Sales Forecast
10.4.8. MT-1621 (Modis Therapeutics)
10.4.8.1. Target Patient Population
10.4.8.2. Sales Forecast
10.4.9. ABI-009 (Aadi Bioscience)
10.4.9.1. Target Patient Population
10.4.9.2. Sales Forecast
10.5. Global Mitochondrial Disease Therapies Market: Distribution by Region
10.5.1. Mitochondrial Disease Therapies Market in the US, 2019-2030
10.5.2. Mitochondrial Disease Therapies Market in the UK, 2019-2030
10.5.3. Mitochondrial Disease Therapies Market in Germany, 2019-2030
10.5.4. Mitochondrial Disease Therapies Market in France, 2019-2030
10.5.5. Mitochondrial Disease Therapies Market in Italy, 2019-2030
10.5.6. Mitochondrial Disease Therapies Market in Spain, 2019-2030
10.6. Global Mitochondrial Disease Therapies Market: Distribution by Target Disease Indication
10.6.1. Global Mitochondrial Disease Therapies Market for LHON, 2019-2030
10.6.2. Global Mitochondrial Disease Therapies Market for MELAS, 2019-2030
10.6.3. Global Mitochondrial Disease Therapies Market for Mitochondrial Myopathy, 2019-2030
10.6.4. Global Mitochondrial Disease Therapies Market for Mitochondrial DNA Depletion Syndrome, 2019-2030
10.6.5. Global Mitochondrial Disease Therapies Market for Leigh syndrome, 2019-2030
10.7. Global Mitochondrial Disease Therapies Market: Distribution by Type of Molecule
10.7.1. Global Mitochondrial Disease Therapies Market for Small Molecules, 2019-2030
10.7.2. Global Mitochondrial Disease Therapies Market for Biologics, 2019-2030
10.8. Global Mitochondrial Disease Therapies Market: Distribution by Route of Administration
10.8.1. Global Mitochondrial Disease Therapies Market for Oral therapies, 2019-2030
10.8.2. Global Mitochondrial Disease Therapies Market for Intravenous Therapies, 2019-2030
10.8.3. Global Mitochondrial Disease Therapies Market for Therapies for Other Routes of Administration, 2019-2030
10.9. Global Mitochondrial Disease Therapies Market: Distribution by Type of Therapy
10.9.1. Global Mitochondrial Disease Therapies Market for Monotherapies, 2019-2030
10.9.2. Global Mitochondrial Disease Therapies Market for Combination Therapies, 2019-2030
11 CONCLUDING REMARKS12 APPENDIX 1: TABULATED DATA13 APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
List of Figures
Figure 3.1 Mitochondria: Powerhouse of the Cell
Figure 3.2 Maternal Inheritance of Mitochondrial DNA Mutations
Figure 3.3 Symptoms Associated with Mitochondrial Diseases
Figure 3.4 Methods / Techniques for the Diagnosis of Mitochondrial Diseases
Figure 4.1 Mitochondrial Diseases: Distribution by Phase of Development
Figure 4.2 Mitochondrial Disease Therapies: Distribution by Target Disease Indication
Figure 4.3 Mitochondrial Disease Therapies: Distribution by Type of Therapy
Figure 4.4 Mitochondrial Disease Therapies: Distribution by Type of Molecule
Figure 4.5 Mitochondrial Disease Therapies: Distribution by Route of Administration
Figure 4.6 Mitochondrial Disease Therapies: Distribution by Path to Clinic
Figure 4.7 Mitochondrial Disease Therapies: Distribution by Target Population
Figure 4.8 Mitochondrial Diseases Therapies: Analysis by Type of Maintenance Defect
Figure 4.9 Mitochondrial Diseases: Analysis by Common Clinical Manifestations
Figure 4.10 Mitochondrial Diseases: Analysis by Age of Disease Onset
Figure 4.11 Mitochondrial Diseases: Word Cloud Analysis by Molecular Target
Figure 4.12 Funnel Analysis: Distribution by Phase of Development, Type of Molecule and Path to Clinic
Figure 4.13 Grid Analysis: Distribution by Target Disease Indication, Path to Clinic and Type of Molecule
Figure 4.14 Mitochondrial Disease Therapy Developers: Distribution by Year of Establishment
Figure 4.15 Mitochondrial Disease Therapy Developers: Distribution by Company Size and Location of Headquarters
Figure 4.16 Mitochondrial Disease Therapy Developers: Distribution by Geography
Figure 5.1 Santhera Pharmaceuticals: Annual Revenues, FY2014-FY2018 (CHF Million)
Figure 5.2 Reata Pharmaceuticals: Annual Revenues, FY2014-FY2018 (USD Thousand)
Figure 6.1 Publications: Year-wise Trend, 2014-2019
Figure 6.2 Publications: Distribution by Type of Study
Figure 6.3 Publications: Year-wise Distribution by Type of Study
Figure 6.4 Publications: Distribution by Study Objective
Figure 6.5 Publications: Word Cloud on Mutated Gene / Phenotype of Disorder
Figure 6.6 Publications: Distribution by Type of Mutation
Figure 6.7 Publications: Distribution of Types of Mutation across Study Objectives
Figure 6.8 Publications: Distribution by Target Disease Indication
Figure 6.9 Publications: Distribution by Type of Mutation across Target Disease Indication
Figure 6.10 Publications: Most Popular Journals
Figure 8.1 Academic Grant Analysis: Cumulative Trend by Number of Grants, January 2015- May 2019
Figure 8.2 Academic Grant Analysis: Cumulative Trend by Amount Awarded (USD Million), January 2015- May 2019
Figure 8.3 Academic Grant Analysis: Distribution by Support Period
Figure 8.4 Academic Grant Analysis: Word Cloud Analysis
Figure 8.5 Academic Grant Analysis: Distribution by Application Type
Figure 8.6 Academic Grant Analysis: Distribution by Year and Type of Application
Figure 8.7 Popular NIH Departments: Distribution by Number of Grants Awarded
Figure 8.8 Academic Grant Analysis: Distribution by Responsible Study Section
Figure 8.9 Academic Grant Analysis: Distribution by Focus Area of the Grant
Figure 8.10 Academic Grant Analysis: Distribution by Popular Indications
Figure 8.11 Academic Grant Analysis: Distribution by Type of Recipient Organization
Figure 8.12 Popular Recipient Organizations: Distribution by Number of Grants Awarded
Figure 8.13 Academic Grant Analysis: Distribution by Administrative Institutes and Centers
Figure 8.14 Academic Grant Analysis: Distribution of Administrative Institutes and Centers within Different Focus Areas
Figure 8.15 Academic Grant Analysis: Distribution by Activity of Grants
Figure 8.16 Academic Grant Analysis: Distribution by Support Period within Administrative Institutes and Centers
Figure 8.17 Prominent Project Leaders: Distribution by Number of Grants Awarded
Figure 8.18 Academic Grant Analysis: Distribution by Funding Institute Center
Figure 8.19 Popular NIH Departments: Distribution of Grants by Funding Mechanism
Figure 8.20 Academic Grant Analysis: Regional Distribution of Recipient Organization
Figure 9.1 KOL Analysis: Distribution by Type of Organization
Figure 9.2 KOL Analysis: Geographical Distribution of Principal Investigators
Figure 9.3 KOL Analysis: Dot-Plot of Principal Investigators
Figure 9.4 KOL Benchmarking: Roots Analysis versus Third Party Scoring
Figure 9.5 KOL Analysis: Most Prominent Key Opinion Leaders
Figure 10.1 Global Mitochondrial Disease Therapies Market, 2019-2030 (USD million)
Figure 10.2 Raxone: Target Patient Population
Figure 10.3 Raxone: Sales Forecast (Till 2030)
Figure 10.4 GS010: Target Patient Population
Figure 10.5 GS010: Sales Forecast (Till 2030)
Figure 10.6 Taurine Powder: Target Patient Population
Figure 10.7 Taurine Powder: Sales Forecast (Till 2030)
Figure 10.8 Elamipredite: Target Patient Population
Figure 10.9 Elamipretide: Sales Forecast (Till 2030)
Figure 10.10 KH176: Target Patient Population
Figure 10.11 KH176: Sales Forecast (Till 2030)
Figure 10.12 Omaveloxolone: Target Patient Population
Figure 10.13 Omaveloxolone: Sales Forecast (Till 2030)
Figure 10.14 KL1333: Target Patient Population
Figure 10.15 KL1333: Sales Forecast (Till 2030)
Figure 10.16 MT1621: Target Patient Population
Figure 10.17 MT1621: Sales Forecast (Till 2030)
Figure 10.16 ABI-009: Target Patient Population
Figure 10.17 ABI-009: Sales Forecast (Till 2030)
Figure 10.18 Global Mitochondrial Disease Therapies Market: Distribution by Region, 2019-2030
Figure 10.19 Mitochondrial Disease Therapies Market in the US: 2019-2030 (USD Million)
Figure 10.20 Mitochondrial Disease Therapies Market in the UK: 2019-2030 (USD Million)
Figure 10.21 Mitochondrial Disease Therapies Market in Germany: 2019-2030 (USD Million)
Figure 10.22 Mitochondrial Disease Therapies Market in France: 2019-2030 (USD Million)
Figure 10.23 Mitochondrial Disease Therapies Market in Italy: 2019-2030 (USD Million)
Figure 10.24 Mitochondrial Disease Therapies Market in Spain: 2019-2030 (USD Million)
Figure 10.27 Global Mitochondrial Disease Therapies Market for LHON, 2019-2030 (USD Million)
Figure 10.28 Global Mitochondrial Disease Therapies Market for MELAS, 2019-2030 (USD Million)
Figure 10.29 Global Mitochondrial Disease Therapies Market for Mitochondrial Myopathy, 2019-2030 (USD Million)
Figure 10.30 Global Mitochondrial Disease Therapies Market for Mitochondrial DNA Depletion Syndrome, 2019-2030 (USD Million)
Figure 10.31 Global Mitochondrial Disease Therapies Market for Leigh syndrome, 2026-2030 (USD Million)
Figure 10.33 Global Mitochondrial Disease Therapies Market: Distribution by Type of Molecule, 2019-2030
Figure 10.34 Global Mitochondrial Disease Therapies Market for Small Molecules, 2019-2030 (USD Million)
Figure 10.35 Global Mitochondrial Disease Therapies Market for Biologics, 2019-2030 (USD Million)
Figure 10.36 Global Mitochondrial Disease Therapies Market: Distribution by Route of Administration, 2019-2030
Figure 10.37 Global Mitochondrial Disease Therapies Market for Oral Therapies, 2019-2030 (USD Million)
Figure 10.38 Global Mitochondrial Disease Therapies Market for Intravenous Therapies, 2019-2030 (USD Million)
Figure 10.40 Global Mitochondrial Disease Therapies Market for Therapies for Other Routes of Administration, 2019-2030 (USD Million)
Figure 10.41 Global Mitochondrial Disease Therapies Market: Distribution by Type of Therapy, 2019-2030
Figure 10.42 Global Mitochondrial Disease Therapies Market for Monotherapies, 2019-2030 (USD Million)
Figure 10.43 Global Mitochondrial Disease Therapies Market for Combination Therapies, 2019-2030 (USD Million)
List of Tables
Table 3.1 Comparison Between Nuclear and Mitochondrial Genome
Table 3.2 Mitochondrial Diseases and their Associated Mutations
Table 3.3 Emerging Therapies for Mitochondrial Diseases
Table 4.1 Mitochondrial Disease Therapies: Marketed and Development Pipeline
Table 4.2 Mitochondrial Disease Therapies: Developer Overview
Table 5.1 Mitochondrial Diseases: List of Companies Profiled
Table 5.2 NeuroVive Pharmaceutical: Company Snapshot
Table 5.3 Drug Profile: KL1333
Table 5.4 Drug Profile: NV354
Table 5.6 Drug Profile: NVP025
Table 5.7 NeuroVive Pharmaceuticals: Future Outlook
Table 5.8 Ixchel Pharma: Company Snapshot
Table 5.9 Drug Profile: IXC 103
Table 5.10 Drug Profile: IXC 201
Table 5.11 Drug Profile: IXC 203
Table 5.12 Stealth BioTherapeutics: Company Snapshot
Table 5.13 Drug Profile: Elamipretide
Table 5.15 Stealth BioTherapeutics: Future Outlook
Table 5.16 Santhera Pharmaceuticals: Company Snapshot
Table 5.17 Drug Profile: Raxone
Table 5.18 Santhera Pharmaceuticals: Future Outlook
Table 5.19 GenSight Biologics: Company Snapshot
Table 5.20 Drug Profile: GS010
Table 5.21 Future Outlook
Table 5.22 BioElectron Technology: Company Snapshot
Table 5.23 Drug Profile: BioE-743
Table 5.24 Reata pharmaceuticals: Company Snapshot
Table 5.25 Drug Profile: Omaveloxolone / RTA-408
Table 5.26 Reata Pharmaceuticals: Future Outlook
Table 6.1 Mitochondrial Diseases: List of Publications, 2014-2018
Table 7.1 Treatment Strategies for Huntington’s Disease
Table 7.2 Treatment Strategies for Rett Syndrome
Table 7.4 Treatment Strategies for NASH
Table 7.5 Treatment Strategies for Type-2 Diabetes
Table 7.6 Treatment Strategies for Friedreich Ataxia
Table 8.1 Mitochondrial Diseases: List of Academic Grants, January 2015- May 2019
Table 8.2 Mitochondrial Diseases: Academic Grants (Additional Information), January 2015- May 2019
Table 8.3 Most Prominent Project Leaders (Additional Information)
Table 9.1 KOL Analysis: List of Principal Investigators
Table 9.2 KOL Analysis: List of Universities and Hospitals
Table 9.3 KOL Analysis: Roots Analysis Proprietary Scoring System
Table 9.4 Profile: KOL A (Merrimack College)
Table 9.5 Profile: KOL B (Sidney Kimmel Medical College)
Table 9.6 Profile: KOL C (Hospital Necker-Enfants Malades)
Table 9.7 Profile: KOL D (Mayo Clinic)
Table 9.8 Profile: KOL E (University of Texas)
Table 9.9 Profile: KOL F (Stanford University)
Table 9.10 Profile: KOL G (University of Iowa Department of Ophthalmology and Visual Sciences )
Table 9.11 Profile: KOL H (Monraz Medical Complex)
Table 9.12 Profile: KOL I (Herlev Hospital)
Table 10.1 Mitochondrial Disease Therapies: List of Forecasted Drug Candidates
Table 12.1 Mitochondrial Disease Therapies: Distribution by Phase of Development
Table 12.2 Mitochondrial Disease Therapies: Distribution by Target Disease Indication
Table 12.3 Mitochondrial Disease Therapies: Distribution by Type of Therapy
Table 12.4 Mitochondrial Disease Therapies: Distribution by Type of Molecule
Table 12.5 Mitochondrial Disease Therapies: Distribution by Route of Administration
Table 12.6 Mitochondrial Disease Therapies: Distribution by Path to Clinic
Table 12.7 Mitochondrial Disease Therapies: Distribution by Target Population
Table 12.8 Mitochondrial Disease Therapies: Distribution by Type of Maintenance Defect
Table 12.9 Mitochondrial Disease Therapies: Distribution by Common Clinical Manifestation
Table 12.10 Mitochondrial Disease Therapies: Distribution by Age of Disease Onset
Table 12.11 Mitochondrial Disease Therapies: Distribution by Company Size and Headquarters of Developer
Table 12.12 Mitochondrial Disease Therapies: Distribution by Headquarters of Developer
Table 12.13 Mitochondrial Disease Therapy Developers: Distribution by Geography
Table 12.14 Santhera Pharmaceuticals: Annual Revenues, FY2014-FY2018 (CHF Million)
Table 12.15 Reata Pharmaceuticals: Annual Revenues, FY2014-FY2018 (USD Thousand)
Table 12.16 Publications: Year-wise Trend, 2014-2019
Table 12.17 Publications: Distribution by Type of Study
Table 12.18 Publications: Distribution by Study Objective
Table 12.19 Publications: Distribution by Type of Mutation
Table 12.20 Publications: Distribution by Target Disease Indication
Table 12.21 Publications: Most Popular Journals
Table 12.22 Academic Grant Analysis: Cumulative Trend by Number of Grants, January 2015- May 2019
Table 12.23 Academic Grant Analysis: Cumulative Trend by Amount Awarded (USD Million), January 2015- May 2019
Table 12.24 Academic Grant Analysis: Distribution by Support Period
Table 12.25 Academic Grant Analysis: Distribution by Application Type
Table 12.26 Academic Grant Analysis: Distribution by Year and Type of Application
Table 12.27 Popular NIH Departments: Distribution by Number of Grants Awarded
Table 12.28 Academic Grant Analysis: Distribution by Responsible Study Section
Table 12.29 Academic Grant Analysis: Distribution by Focus Area of the Grant
Table 12.30 Academic Grant Analysis: Distribution by Popular Indications
Table 12.31 Popular Recipient Organization: Distribution by Number of Grants Awarded
Table 12.32 Academic Grant Analysis: Distribution by Administrative Institutes and Centers
Table 12.33 Academic Grant Analysis: Distribution by Activity of Grants
Table 12.34 Prominent Project Leaders: Distribution by Number of Grants Awarded
Table 12.35 Popular NIH Departments: Distribution of Grants by Funding Mechanism
Table 12.36 Academic Grant Analysis: Regional Distribution of Recipient Organization
Table 12.37 KOL Analysis: Distribution by Type of Organization
Table 12.38 Global Mitochondrial Disease Therapies Market, Base, Conservative and Optimistic Scenario, 2019-2030 (USD million)
Table 12.39 Raxone: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.40 GS010: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.41 Taurine Powder: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.42 Elamipretide: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.43 KH176: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.44 Omaveloxolone: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.45 KL1333: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.46 MT1621: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.47 ABI-009: Sales Forecast (Till 2030), Base, Conservative and Optimistic Scenario (USD Million)
Table 12.48 Global Mitochondrial Disease Therapies Market: Distribution by Region, 2019-2030
Table 12.49 Mitochondrial Disease Therapies Market in the US: 2019-2030 (USD Million)
Table 12.50 Mitochondrial Disease Therapies Market in the UK: 2019-2030 (USD Million)
Table 12.51 Mitochondrial Disease Therapies Market in Germany: 2019-2030 (USD Million)
Table 12.52 Mitochondrial Disease Therapies Market in France: 2019-2030 (USD Million)
Table 12.53 Mitochondrial Disease Therapies Market in Italy: 2019-2030 (USD Million)
Table 12.54 Mitochondrial Disease Therapies Market in Spain: 2019-2030 (USD Million)
Table 12.55 Global Mitochondrial Disease Therapies Market for LHON, 2019-2030 (USD Million)
Table 12.56 Global Mitochondrial Disease Therapies Market for MELAS, 2019-2030 (USD Million)
Table 12.57 Global Mitochondrial Disease Therapies Market for Mitochondrial Myopathy, 2019-2030 (USD Million)
Table 12.58 Global Mitochondrial Disease Therapies Market for Mitochondrial DNA Depletion Syndrome, 2019-2030 (USD Million)
Table 12.59 Global Mitochondrial Disease Therapies Market for Leigh syndrome, 2019-2030 (USD Million)
Table 12.60 Global Mitochondrial Disease Therapies Market: Distribution by Type of Molecule, 2019-2030
Table 12.61 Global Mitochondrial Disease Therapies Market for Small Molecules, 2019-2030 (USD Million)
Table 12.62 Global Mitochondrial Disease Therapies Market for Biologics, 2019-2030 (USD Million)
Table 12.63 Global Mitochondrial Disease Therapies Market: Distribution by Route of Administration, 2019-2030
Table 12.64 Global Mitochondrial Disease Therapies Market for Oral Therapies, 2019-2030 (USD Million)
Table 12.65 Global Mitochondrial Disease Therapies Market for Intravenous Therapies, 2019-2030 (USD Million)
Table 12.66 Global Mitochondrial Disease Therapies Market for Therapies for Other Routes of Administration, 2019-2030 (USD Million)
Table 12.67 Global Mitochondrial Disease Therapies Market: Distribution by Type of Therapy, 2019-2030
Table 12.68 Global Mitochondrial Disease Therapies Market for Monotherapies, 2019-2030 (USD Million)
Table 12.69 Global Mitochondrial Disease Therapies Market for Combination Therapies, 2019-2030 (USD Million)

Executive Summary

Chapter 2 provides an executive summary of the insights captured in our research. It offers a high-level view of the state of the market for mitochondrial diseases, in the short-mid term and long term.

Chapter 3 provides a general overview of the structure and function of mitochondria and the pattern of mitochondrial inheritance. The chapter lays emphasis on genetic diseases caused due to mutations in the mitochondrial DNA and clinical conditions that are associated with mitochondrial dysfunction. It also highlights the diverse strategies that are available / being investigated for the treatment of mitochondrial diseases and key challenges/drivers associated with this segment of the pharmaceutical industry.

Chapter 4 includes information on nearly 60 molecules that are currently under development for the treatment of various mitochondrial diseases. It features a comprehensive analysis of pipeline molecules, highlighting phase of development (marketed, clinical and preclinical / discovery stage) of lead candidates, type of molecule (small molecule and biologic), type of therapy (monotherapy and combination therapy), path to clinic (dedicated, repurposed and repositioned), target indication, and route of administration of the drugs / therapies that are being developed for the treatment of different mitochondrial diseases. In addition, the chapter provides information on drug developer(s), highlighting year of their establishment, location of headquarters and employee strength.

Chapter 5 provides detailed profiles of some of the leading stakeholders in this field (shortlisted on the basis of number/stage of the products). Each profile presents a brief overview of the company, its financial information (if available), product portfolio, recent collaborations, and an informed future outlook.

Chapter 6 provides a detailed analysis of close to 200 research articles related to therapies for mitochondrial diseases, published in the period 2014-2019 (till February). The analysis takes into consideration several aspects, namely target disease indications, mutations studies across different indications, and analysis based on various relevant parameters, such as study type (review article, research article and case reports), year of publication, and most popular journals (in terms of number of articles published in the given time period), within this domain.

Chapter 7 features a discussion on the popular secondary mitochondrial diseases, which have not been included in the scope of the report but are important to highlight. It provides information on the role of mitochondria in disease pathogenesis, laying emphasis on the various treatment strategies available / under development, to treat such clinical conditions. The chapter also presents a list of companies that have undertaken initiatives to develop drug/therapy candidate for the treatment of the aforementioned disorders.

Chapter 8 provides information on the various grants that were awarded to research institutes conducting projects related to different types of mitochondrial diseases, between 2015 and 2019 (till May). The analysis also highlights important parameters associated with grants, such as year of the award, support period, the amount awarded, funding institute, grant type, responsible study section, focus area and type of recipient organization.

Chapter 9 provides an analysis of KOLs in the field of mitochondrial diseases. It features a comprehensive list of principal investigators/study directors of different clinical trials, along with the information related to the affiliated research institutes. The chapter features a schematic representation on a world map, highlighting the geographical locations of eminent scientists/researchers who are engaged in clinical research in this domain. It also presents a comparative analysis, highlighting those KOLs who have relatively more experience in this domain. The credibility and (relative) level of expertise of different KOLs defined by other analysts/industry experts were compared to the results obtained using proprietary scoring criteria, which was based on number of publications, number of citations, number of clinical trials, number of affiliations and extent of their professional network.

Chapter 10 features a detailed market forecast analysis, highlighting the likely growth of mitochondrial disease therapies till the year 2030. We have provided inputs on the likely distribution of the opportunity based on across different target indications (Leigh syndrome, LHON, MELAS, mitochondrial DNA depletion syndrome, mitochondrial myopathy, and others), type of therapy (combination and monotherapy), route of administration (oral, intravenous, and others) and key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.  

Chapter 11 summarizes the entire report. It presents a list of key takeaways and offers our independent opinion on the current market scenario. Further, it captures the evolutionary trends that are likely to determine the future of this segment of the mitochondrial disease therapeutics industry.

Chapter 12 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 13 is an appendix, which contains a list of companies and organizations mentioned in this report.


Companies Mentioned

  • Aadi Bioscience
  • Adelphi University
  • Advanced Imaging Research Center
  • Akron Children's Hospital
  • Al-Mumtaz Medical Complex
  • Albert Einstein College of Medicine
  • Alkeus Pharmaceuticals
  • Allergan
  • Anavex Life Sciences
  • Angers University Hospital
  • Animas
  • Asian Society for Mitochondrial Research and Medicine
  • Assistance publique – Hôpitaux de Paris
  • Astellas Pharma
  • Athena Diagnostics
  • AveXis
  • B-MoGen Biotechnologies
  • Bascom Palmer Eye Institute
  • Baylor College of Medicine
  • Beth Israel Deaconess Medical Center
  • BioElectron Technology
  • Biovista
  • Brescia University
  • BridgeBio Pharma
  • California State University
  • Case Western Reserve University
  • CENTOGENE
  • Cerecor
  • Chiang Mai University
  • Children's Hospital of Philadelphia
  • Cincinnati Children's Hospital Medical Center
  • Columbia University
  • Copenhagen Neuromuscular Center
  • Dana-Farber Cancer Institute
  • Duke University
  • ElexoPharm
  • Eloxx Pharmaceuticals
  • Emory School of Medicine
  • Erasme Hospital
  • Esousa Holdings
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development
  • Evotec
  • Ewopharma
  • Fight AIDS Foundation
  • Florida Hospital
  • Fortify Therapeutics
  • Foundation for the Promotion of Health and Biomedical Research of Valencia Region
  • Galmed Pharmaceuticals
  • GeneDx
  • GENFIT
  • GenSight Biologics
  • Ghent University Hospital
  • Gilead Sciences
  • Harvard Medical School
  • Henry M Jackson Foundation for the Advancement of Military Medicine
  • Herlev Hospital
  • Horizon Therapeutics
  • Hospices Civils de Lyon
  • Hospital Clinic of Barcelona
  • Huazhong University of Science & Technology
  • Illumina
  • Innocoll
  • Institut de la Vision
  • Insulet
  • Intercept Pharmaceuticals
  • International Rare Diseases Research Consortium
  • Inventiva Pharma
  • Ionis Pharmaceuticals
  • IRCCS Carlo Besta Neurological Institute Foundation
  • IRCCS Eugenio Medea
  • Ixchel Pharma
  • Janssen Pharmaceuticals
  • Joslin Diabetes Center
  • Jupiter Orphan Therapeutics
  • Karolinska Institutet
  • Khondrion
  • Les Hôpitaux Universitaires de Strasbourg
  • Maastricht University Medical Center
  • Mahidol University
  • Marquette University
  • Massachusetts Eye and Ear
  • Massachusetts General Hospital
  • Maverix Biomics
  • Mayo Clinic
  • McLean Hospital
  • McMaster University
  • MD Stem Cells
  • Medical Center of the University of Munich
  • Medosome Biotec
  • Medtronic
  • Merrimack College
  • Metro International Biotech
  • Michigan State University
  • Minoryx Therapeutics
  • Minovia Therapeutics
  • Mission Therapeutics
  • MitoAction
  • Mitobridge
  • MitoCanada Foundation
  • Mitochon Pharmaceuticals
  • Mitochondria Research and Medicine Society
  • Mitochondrial Medicine Society
  • Mitochondrial Research Guild
  • MitoEAGLE
  • Mitotech
  • Modis Therapeutics
  • Moorfields Eye Hospital
  • Murdoch Children's Research Institute
  • Nanna Therapeutics
  • National Center for Advancing Translational Sciences
  • National Eye Institute
  • National Heart, Lung, and Blood Institute
  • National Hospital for Neurology and Neurosurgery
  • National Human Genome Research Institute
  • National Institute of Allergy and Infectious Diseases
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases
  • National Institute of Dental and Craniofacial Research
  • National Institute of Diabetes and Digestive and Kidney Diseases
  • National Institute of Environmental Health Sciences
  • National Institute of General Medical Sciences
  • National Institute of Neurological Disorders and Stroke
  • National Institute on Aging
  • National Institute on Drug Abuse
  • Necker-Enfants Malades Hospital
  • Neuren Pharmaceuticals
  • NeuroVive Pharmaceutical
  • Novartis
  • Ochsner Health System
  • Oregon Health & Science University
  • Oroboros Instruments
  • Perlara
  • Pharmathen
  • Phoenix Children's Hospital
  • Postgraduate Institute of Medical Education and Research
  • Queen's University
  • Reata Pharmaceuticals
  • Relmada Therapeutics
  • Reneo Pharmaceuticals
  • Retrotope
  • Rowan University School of Osteopathic Medicine
  • Rutgers Robert Wood Johnson Medical School
  • San Carlos Clinical Hospital
  • Santhera Pharmaceuticals
  • Seattle Children’s Hospital
  • Seoul National University Hospital
  • Serbian Society for Mitochondrial and Free Radical Physiology
  • Shiyan Taihe Hospital
  • Sidney Kimmel Medical College
  • SOM Biotech
  • Spark Therapeutics
  • St George's, University of London
  • St Luke's Roosevelt Hospital
  • Stanford University
  • Statistical Analysis Center, Columbia University
  • Stealth BioTherapeutics
  • Stony Brook University
  • Sumitomo Dainippon Pharma
  • Taipei Veterans General Hospital
  • Taisho Pharmaceutical Holdings
  • Taisho Toyama Pharmaceuticals
  • Takeda Pharmaceutical
  • Tandem Diabetes Care
  • Texas A&M AgriLife Research
  • The Edmond de Rothschild Foundations
  • The George Washington University
  • The Hospital for Sick Children (SickKids)
  • The Medical University of South Carolina
  • The National Institute of Neurological Disorders and Stroke
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust
  • The Scripps Research Institute
  • The University of Texas
  • The University of Utah
  • uniQure
  • United Mitochondrial Disease Foundation
  • University College London
  • University of Alcalá
  • University of Arkansas
  • University of Bologna
  • University of British Columbia
  • University of California
  • University of Cambridge
  • University of Colorado
  • University of Florida
  • University of Guadalajara
  • University of Illinois at Urbana-Champaign
  • University of Iowa Health Care
  • University of Kansas Medical Center
  • University of Louisville
  • University of Miami
  • University of Michigan
  • University of Minnesota
  • University of Naples Federico II
  • University of Nebraska
  • University of Pittsburgh
  • University of Rochester
  • University of South Alabama
  • University of South Carolina
  • University of South Florida
  • University of Washington
  • Upstate Medical University
  • Use Sapienza University of Rome
  • UT Southwestern Medical Center
  • Vanderbilt University Medical Center
  • Voyager Therapeutics
  • Wave Life Sciences
  • Weill Cornell Medicine
  • Wellcome Centre for Mitochondrial Research
  • Whitehead Institute for Biomedical Research
  • Woodinville Psychiatric Associates
  • Yale University
  • YUNGJIN Pharmaceutical

Methodology

 

 

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