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Mitochondrial Disease Therapies Market, 2019-2030

Mitochondrial Disease Therapies Market, 2019-2030

  • July 2019
  • 253 pages
  • ID: 5821927
  • Format: PDF
  • By Roots Analysis Private Ltd.

Summary

Table of Contents

INTRODUCTION
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 exists 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 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 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.

RESEARCH METHODOLOGY
The data presented in this report has been gathered via secondary research. For most of our projects, we conduct interviews / surveys with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include:
• Annual reports
• Investor presentations
• SEC filings
• Industry databases
• News releases from company websites
• Government policy documents
• Industry analysts’ views

CHAPTER OUTLINES
Chapter 2 provides an executive summary of the insights captured in our research. It offers a high-level view on 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 treatment of mitochondrial diseases and key challenges / drivers associated within this segment o 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 award, support period, 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 a proprietary scoring criteria, which was based on number of publications, number of citations, number of clinical trials, number of affiliations and extent of 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.

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