Spinocerebellar Ataxias - Pipeline Insight, 2024

This “Spinocerebellar Ataxias - Pipeline Insight, 2024” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Spinocerebellar Ataxias 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

Spinocerebellar Ataxias: Understanding

Spinocerebellar Ataxias: Overview

Ataxia is the absence of voluntary muscle coordination and loss of control of movement that affects gait stability, eye movement, and speech. Spinocerebellar ataxia (SCA) is an inherited (autosomal dominant), progressive, neurodegenerative, and heterogeneous disease that mainly affects the cerebellum. SCA is a subset of hereditary cerebellar ataxia and is a rare disease. SCA is a subset of hereditary cerebellar ataxia and is a rare disease. To date, more than 40 distinct genetic SCAs have been identified which are classified according to the genetic loci in order of identification. SCA1 was the first SCA described and then further subtypes are identified sequentially.

The exact pathogenesis of spinocerebellar ataxia is still not known. But many study series promulgated that common mechanisms of SCA are genetic mutations causing abnormal protein products, transcriptional dysregulation, dysfunction of autophagy, channelopathies, mitochondrial dysfunction, toxic RNA gain of function. Ataxins are targeted by ubiquitin-proteasome proteolytic complex in an attempt to degrade a remove and form the aggregations. Also, cellular interactions with abnormal ataxins have some role in pathogenesis. Ataxins bind to other proteins, including the TATA-binding transcription protein and the CREB-binding protein, impairing their functions disrupt the normal transcription regulation, which leads to abnormal and uncontrolled transcription.

Several types of spinocerebellar ataxia are associated with anticipation, in which there is a tendency of gradual expansion of CAG repeats in a consecutive generation. CAG repeat expansion occurs in SCA1, 2, 3, 6, 7, 8, 12, and 17. Similarly, SCA 10 is caused by the expansion of ATTCT (pentanucleotide), SCA 31, 36, 37 involve amplification of TGGAA (pentanucleotide), GGCCTG (hexanucleotide), and ATTTT (pentanucleotide) respectively. Other SCA subtypes are rarer and involve other repeat expansions or single nucleotide variants. SCA5, SCA13, SCA14, and SCA19 engage the missense mutation, and SCA15, SCA20, and SCA39 involve the deletion or duplication of genes. Treatment is mainly symptomatic to alleviate symptoms like seizures, tremors, depression, ataxia, and eye symptoms. Antiepileptic drugs for seizures, botulinum toxin injections for dystonia, beta-blockers, and primidone for tremors, antidepressants for depression, and levodopa in parkinsonism, etc. can be utilized for symptomatic treatment.

"Spinocerebellar Ataxias - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Spinocerebellar Ataxias pipeline landscape is provided which includes the disease overview and Spinocerebellar Ataxias treatment guidelines. The assessment part of the report embraces, in depth Spinocerebellar Ataxias 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, Spinocerebellar Ataxias collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Spinocerebellar Ataxias R&D. The therapies under development are focused on novel approaches to treat/improve Spinocerebellar Ataxias.

Spinocerebellar Ataxias Emerging Drugs Chapters

This segment of the Spinocerebellar Ataxias report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Spinocerebellar Ataxias Emerging Drugs

Troriluzole: Biohaven Pharmaceuticals

Troriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is reducing synaptic levels of glutamate. Troriluzole increases glutamate uptake from the synapse, by augmenting the expression and function of excitatory amino acid transporters located on glial cells that play a key role in clearing glutamate from the synapse. Troriluzole has the potential to be developed in a number of other diseases associated with excessive glutamate. Currently, the drug is in Phase III stage of clinical trial evaluation for the treatment of Spinocerebellar Ataxias.

VO659: Vico Therapeutics B. V.

VO659 is an investigational therapy developed by Vico Therapeutics B.V., specifically designed to treat neurological diseases caused by CAG repeat expansions, such as Huntington's disease (HD) and spinocerebellar ataxia types 1 and 3 (SCA1 and SCA3). This therapy is classified as an allele-preferential antisense oligonucleotide (ASO), which means it selectively targets the mutated allele responsible for these diseases, aiming to inhibit the translation of toxic proteins associated with these conditions. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Spinocerebellar Ataxias.

Spinocerebellar Ataxias: Therapeutic Assessment

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

Major Players in Spinocerebellar Ataxias

There are approx. 8+ key companies which are developing the therapies for Spinocerebellar Ataxias. The companies which have their Spinocerebellar Ataxias drug candidates in the most advanced stage, i.e. Phase III include, Biohaven Pharmaceuticals.

Phases

The report covers around 10+ 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

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as:

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Spinocerebellar Ataxias: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Spinocerebellar Ataxias 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 Spinocerebellar Ataxias drugs.

Spinocerebellar Ataxias Report Insights

• Spinocerebellar Ataxias Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Spinocerebellar Ataxias 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 Spinocerebellar Ataxias drugs?
• How many Spinocerebellar Ataxias drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Spinocerebellar Ataxias?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Spinocerebellar Ataxias 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 Spinocerebellar Ataxias and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Biohaven Pharmaceuticals
• Vico Therapeutics B. V.
• Steminent Biotherapeutics

Key Products

• Troriluzole
• VO659
• Stemchymal

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