DelveInsight
DelveInsight
Ataxia - Pipeline Insight, 2023
  • Published Date : Apr 2023

  • Pages : 120

  • Delivery Time : 72 Hours

  • Region : Global

Ataxia Pipeline Insight

DelveInsight’s, “Ataxia - Pipeline Insight, 2023,” report provides comprehensive insights about 32+ companies and 32+ pipeline drugs in Ataxia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

  • Global coverage

Ataxia Understanding

Ataxia: Overview

Ataxia is a degenerative disease of the nervous system. Many symptoms of Ataxia mimic those of being drunk, such as slurred speech, stumbling, falling, and incoordination. These symptoms are caused by damage to the cerebellum, the part of the brain that is responsible for coordinating movement. Ataxia treatment involves a combination of medication to treat symptoms and therapy to improve quality of life. People affected by Ataxia may experience problems with using their fingers and hands, arms, legs, walking, speaking or moving their eyes. Ataxia affects people of all ages. Age of symptom-onset can vary widely, from childhood to late-adulthood. Complications from the disease are serious and oftentimes debilitating. Some types of Ataxia can lead to an early death.

"Ataxia - Pipeline Insight, 2023" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Ataxia pipeline landscape is provided which includes the disease overview and Ataxia treatment guidelines. The assessment part of the report embraces, in depth Ataxia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Ataxia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

  • A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Ataxia.
  • In the coming years, the Ataxia market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics that are working to assess challenges and seek opportunities that could influence Ataxia R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • A detailed portfolio of major pharma players who are involved in fueling the Ataxia treatment market. Several potential therapies for Ataxia are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Ataxia market size in the coming years.
  • Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Ataxia) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Ataxia Emerging Drugs Chapters

This segment of the Ataxia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Ataxia Emerging Drugs

RT 001: Retrotope

RT001, is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) that is in development for a range of orphan neurodegenerative diseases, including infantile neuroaxonal dystrophy (INAD), Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and progressive supranuclear palsy (PSP). To date, RT001 has been safely administered orally on a daily basis to more than 100 patients, spanning more than 1,000 patient months. The compound is currently being evaluated in clinical trials across several neurodegenerative diseases with multiple data readouts expected by the end of 2021. These include a potentially pivotal Phase 2/3 trial in INAD, a pivotal Phase 2/3 study in FA and a Phase 2 trial in ALS. RT001 has received a variety of key regulatory designations from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In the U.S., these include rare pediatric disease designation for INAD and FA, Fast Track designation for FA, and orphan drug designation for FA, PSP, and PLA2G6-associated neurodegeneration, which includes INAD. Additionally, in Europe, RT001 has received orphan drug designation for the treatment of INAD.

Omaveloxolone: Reata Pharmaceuticals

Omaveloxolone is an investigational, oral, once-daily, activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug and Fast Track Designations to omaveloxolone for the treatment of Friedreich’s ataxia. The European Commission has granted Orphan Drug Designation in Europe to omaveloxolone for the treatment of Friedreich’s ataxia.

Further product details are provided in the report……..

Ataxia: Therapeutic Assessment

This segment of the report provides insights about the different Ataxia drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Ataxia

There are approx. 32+ key companies which are developing the therapies for Ataxia. The companies which have their Ataxia drug candidates in the most advanced stage, i.e. preregistration include Reata Pharmaceuticals.

Phases

DelveInsight’s report covers around 32+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates

Route of Administration

Ataxia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Inhalation
  • Inhalation/Intravenous/Oral
  • Intranasal
  • Intravenous
  • Intravenous/ Subcutaneous
  • NA
  • Oral
  • Oral/intranasal/subcutaneous
  • Parenteral
  • Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

  • Antibody
  • Antisense oligonucleotides
  • Immunotherapy
  • Monoclonal antibody
  • Peptides
  • Protein
  • Recombinant protein
  • Small molecule
  • Stem Cell
  • Vaccine

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Ataxia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Ataxia therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Ataxia drugs.

Ataxia Report Insights

  • Ataxia Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Ataxia Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Ataxia drugs?
  • How many Ataxia drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Ataxia?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Ataxia therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Ataxia and their status?
  • What are the key designations that have been granted to the emerging drugs?

Introduction

Executive Summary

Ataxia: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Ataxia – DelveInsight’s Analytical Perspective

Late Stage Products (Preregistration)

  • Comparative Analysis

Omaveloxolone: Reata Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

IB1001: IntraBio

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

DT-216: Design Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

VO 659: Vico Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Ataxia Key Companies

Ataxia Key Products

Ataxia- Unmet Needs

Ataxia- Market Drivers and Barriers

Ataxia- Future Perspectives and Conclusion

Ataxia Analyst Views

Ataxia Key Companies

Appendix

List of Tables:

  • List of Table
  • Table 1: Total Products for Ataxia
  • Table 2: Late Stage Products
  • Table 3: Mid Stage Products
  • Table 4: Early Stage Products
  • Table 5: Pre-clinical & Discovery Stage Products
  • Table 6: Assessment by Product Type
  • Table 7: Assessment by Stage and Product Type
  • Table 8: Assessment by Route of Administration
  • Table 9: Assessment by Stage and Route of Administration
  • Table 10: Assessment by Molecule Type
  • Table 11: Assessment by Stage and Molecule Type
  • Table 12: Inactive Products

List of Figures:

  • List of Figures
  • Figure 1: Total Products for Ataxia
  • Figure 2: Late Stage Products
  • Figure 3: Mid Stage Products
  • Figure 4: Early Stage Products
  • Figure 5: Preclinical and Discovery Stage Products
  • Figure 6: Assessment by Product Type
  • Figure 7: Assessment by Stage and Product Type
  • Figure 8: Assessment by Route of Administration
  • Figure 9: Assessment by Stage and Route of Administration
  • Figure 10: Assessment by Molecule Type
  • Figure 11: Assessment by Stage and Molecule Type
  • Figure 12: Inactive Products

List of Companies:

  • Retrotope
    Reata Pharmaceuticals
    PTC Therapeutics
    Metro International Biotech, LLC
    Design Therapeutics
    Larimar Therapeutics
    Minoryx Therapeutics
    EryDel
    Biogen
    Matrix Biomed
    IntraBio
    Biohaven Pharmaceuticals, Inc.
    Stealth BioTherapeutics Inc.
    Acasti Pharma
    Seelos Therapeutics
    Kissei Pharmaceutical
    Vico Therapeutics
    Q-State Biosciences
    Locanabio
    Lexeo Therapeutics
    Voyager Therapeutics
    CRISPR Therapeutics
    Capsida Biotherapeutics
    AavantiBio
    StrideBio
    Wave Life Sciences
    REPROCELL
    SHIONOGI & Co.
    CORESTEM
    Blade Therapeutics
    Exicure
    Lacerta Therapeutics
    Healx Ltd.
    Uniqure
    Ionis Pharmaceuticals
    Jupiter Neurosciences

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