Ataxia - Pipeline Insight, 2024

This “Ataxia - Pipeline Insight, 2024,” 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, 2024 report 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.
• This 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: RetrotopeRT001, 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 PharmaceuticalsOmaveloxolone 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.

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

This 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?

Key Players

• 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

Key Products

• RT 001
• Omaveloxolone
• Vatiquinone
• MIB-626
• DT-216
• CTI-1601
• MIN-102
• EryDex
• BIIB132
• MBM-01
• IB1001
• Troriluzole
• Elamipretide
• GTX-102
• SLS-005
• KPS-0373
• VO 659
• LX2006
• AVB-202
• STRX 110
• Stemchymal
• S-0373
• Allo BM-MSC

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