Spinocerebellar Ataxias Clinical Trial Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Domain | DelveInsight

DelveInsight's "Spinocerebellar Ataxias Pipeline Insight 2025" report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Spinocerebellar Ataxias pipeline landscape. It covers the Spinocerebellar Ataxias pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Spinocerebellar Ataxias Pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Spinocerebellar Ataxias Pipeline Report

* In February 2026, Biogen conducted a phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years.
* In January 2026, Quince Therapeutics S.p.A announced a study to provide EryDex treatment to ataxia telangiectasia (A-T) patients who complete the IEDAT-04-2022 trial on the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).
* DelveInsight's Spinocerebellar Ataxias Pipeline report depicts a robust space with 8+ active players working to develop 10+ pipeline Spinocerebellar Ataxias drugs.
* The leading Spinocerebellar Ataxias Companies such as Biohaven Pharmaceuticals, Inc., Steminent US, Inc., Vico Therapeutics B. V., Arrowhead Pharmaceuticals, Neurolixis, Latus Bio, AAV-CB and others.
* Promising Spinocerebellar Ataxias Therapies such as Troriluzole, KPS-0373, VO659, Varenicline, Stemchymal Registered , Excipients, N-Acetyl-L-Leucine , and others.

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Spinocerebellar Ataxias Overview

Spinocerebellar ataxia (SCA) is a rare, progressive, inherited neurodegenerative disorder that primarily affects the cerebellum but can involve other parts of the central nervous system. It is classified into over 40 subtypes based on genetic loci, with common types including SCA1, SCA2, SCA3, and SCA6, which account for the majority of cases. SCA is characterized by the loss of voluntary muscle coordination, impacting gait, speech, and eye movement. Although primarily associated with the cerebellum and spinal cord, SCA can also affect other areas such as the basal ganglia, cortex, and peripheral nerves, making it a complex and diverse condition both genetically and clinically. Symptoms typically worsen over time, leading to significant disability. The age of onset and severity can vary widely, even within the same family, making diagnosis and management challenging.

Spinocerebellar Ataxias Emerging Drugs

* Troriluzole: Biohaven Pharmaceuticals, Inc.

Troriluzole is a New Chemical Entity (NCE) and third-generation novel prodrug 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. In February 2025, Biohaven Ltd. announced that the US Food and Drug Administration (FDA) has accepted for review the Company's New Drug Application for troriluzole for the treatment of adult patients with spinocerebellar ataxia.

* Stemchymal: Steminent US, Inc.

Stemchymal is an allogeneic stem cell therapy, meaning it uses stem cells obtained from one patient to treat another. The mesenchymal stem cells (MSCs) used to create Stemchymal are isolated from the adipose, or fatty, tissue of healthy individuals. Once isolated from this primary tissue using a unique cell processing system, the MSCs are administered to recipients by intravenous (IV) infusion. As MSCs display low immunogenicity, they are less likely to cause an immune reaction in patients receiving treatment. These cells have also displayed therapeutic effects in vivo, with infusion shown to partially restore motor function in SCA mouse models. Stemchymal has also been granted orphan drug designation in Japan. Currently, the drug is in Phase II stage of its development for the treatment of Spinocerebellar Ataxias.

* VO659: Vico Therapeutics B. V.

VO659 is the first clinical candidate directly targeting the CAG repeat expansion, the mutation that causes all polyglutamine diseases. VO659 is an ASO that directly targets the CAG repeat expansion and reduces mutant protein in an allele preferential manner. VO659 is designed to bind directly to the CAG repeats, sterically block translation and reduce the mutant protein. The CAG repeat expansion causes nine polyQ diseases, including Huntington's disease and various forms of spinocerebellar ataxia. By focusing on this common mutation, VO659 has the potential to address multiple polyQ disorders with a single therapy. VO659 employs an innovative allele-preferential approach. This precision targeting allows us to reduce the mutant protein while preserving essential normal gene function - a crucial advancement in treating these complex disorders like SCA. Currently, the drug is in Phase I/II stage of its development for the treatment of Spinocerebellar Ataxias.

The Spinocerebellar Ataxias Pipeline Report provides insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Spinocerebellar Ataxias with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinocerebellar Ataxias Treatment.
* Spinocerebellar Ataxias Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Spinocerebellar Ataxias Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinocerebellar Ataxias market.

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Spinocerebellar Ataxias Companies

Biohaven Pharmaceuticals, Inc., Steminent US, Inc., Vico Therapeutics B. V., Arrowhead Pharmaceuticals, Neurolixis, Latus Bio, AAV-CB and others.

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

Spinocerebellar Ataxias Products have been categorized under various Molecule types such as

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

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Scope of the Spinocerebellar Ataxias Pipeline Report

* Coverage- Global
* Spinocerebellar Ataxias Companies- Biohaven Pharmaceuticals, Inc., Steminent US, Inc., Vico Therapeutics B. V., Arrowhead Pharmaceuticals, Neurolixis, Latus Bio, AAV-CB and others.
* Spinocerebellar Ataxias Therapies- Troriluzole, KPS-0373, VO659, Varenicline, Stemchymal Registered , Excipients, N-Acetyl-L-Leucine , and others.
* Spinocerebellar Ataxias Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
* Spinocerebellar Ataxias Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* Introduction
* Executive Summary
* Spinocerebellar Ataxias: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Spinocerebellar Ataxias- DelveInsight's Analytical Perspective
* Late Stage Products (Registration)
* Troriluzole: Biohaven Pharmaceuticals, Inc.
* Mid Stage Products (Phase II)
* Stemchymal: Steminent US, Inc.
* Early Stage Products (Phase I/II)
* VO659: Vico Therapeutics B. V.
* Preclinical and Discovery Stage Products
* Drug Name: Company Name
* Inactive Products
* Spinocerebellar Ataxias Key Companies
* Spinocerebellar Ataxias Key Products
* Spinocerebellar Ataxias- Unmet Needs
* Spinocerebellar Ataxias- Market Drivers and Barriers
* Spinocerebellar Ataxias- Future Perspectives and Conclusion
* Spinocerebellar Ataxias Analyst Views
* Spinocerebellar Ataxias Key Companies
* Appendix

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