DelveInsight’s, “Spinocerebellar Ataxias Pipeline Insight 2024” report provides comprehensive insights about 10+ companies and 12+ 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.
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Key Takeaways from the Spinocerebellar Ataxias Pipeline Report
- In October 2024:- Quince Therapeutics S.p.A.- This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangectasia (A-T).
- DelveInsight’s Spinocerebellar Ataxias pipeline report depicts a robust space with 10+ active players working to develop 12+ pipeline therapies for Spinocerebellar Ataxias treatment.
- The leading Spinocerebellar Ataxias Companies such as Lexeo Therapeutics, Vico Therapeutics, PTC Therapeutics, Minoryx Therapeutics, IntraBio, Acasti Pharma, Design Therapeutics, and others.
- Promising Spinocerebellar Ataxias Therapies such as IB1001, Dexamethasone sodium phosphate, VO659, troriluzole, and others
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Spinocerebellar Ataxias Emerging Drugs Profile
- Vatiquinone: PTC Therapeutics
PTC is developing vatiquinone, a potential treatment for Friedreich ataxia based on our Bio-e platform. Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately preventing ferroptosis and aiding neuronal survival. Vatiquinone has been evaluated in a number of clinical studies and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. Currently the drug is in Phase III stage of its clinical trial for the treatment of spinocerebellar ataxias.
- Leriglitazone: Minoryx Therapeutics
Leriglitazone (MIN-102) is Minoryx’s novel orally bioavailable and selective PPAR gamma agonist with a potential first-in-class and best-in-class profile for CNS diseases. It has demonstrated brain penetration and a favorable safety profile. It showed robust preclinical proof-of-concept in animal models of multiple diseases by modulating pathways leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. In clinical trials, leriglitazone showed clinical benefit both for X-ALD and Friedreich’s Ataxia patients. Leriglitazone has been granted orphan drug status for X-ALD from the FDA and the EMA and fast track and rare pediatric disease designation from the FDA for the treatment of X-ALD. Currently, the drug is in Phase II stage of its clinical trial for the treatment of spinocerebellar ataxias.
- DT-216: Design Therapeutics
DT-216 by Design Therapeutics is a therapeutic candidate under clinical development for the treatment of Friedreich Ataxia. It comprises synthetic transcription elongation factor 1 (Syn-TEF1) and targets frataxin. The drug is administered intravenously and is part of Design Therapeutics’ efforts to develop small-molecule genomic drugs for degenerative genetic disorders. DT-216 has shown promising results in increasing frataxin mRNA levels, with the potential to restore frataxin protein expression, which is crucial in addressing the clinical manifestations of Friedreich Ataxia. Currently the drug is in Phase I stage of its clinical trial.
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Spinocerebellar Ataxias Companies
Lexeo Therapeutics, Vico Therapeutics, PTC Therapeutics, Minoryx Therapeutics, IntraBio, Acasti Pharma, Design Therapeutics, and others
Spinocerebellar Ataxias pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
- 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- Lexeo Therapeutics, Vico Therapeutics, PTC Therapeutics, Minoryx Therapeutics, IntraBio, Acasti Pharma, Design Therapeutics, and others.
- Spinocerebellar Ataxias Therapies- IB1001, Dexamethasone sodium phosphate, VO659, troriluzole, 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
For a detailed overview of our latest research findings and future plans, read the full details of Spinocerebellar Ataxias Pipeline on our website @ Spinocerebellar Ataxias Emerging Drugs and Companies
Table of Content
- Introduction
- Executive Summary
- Spinocerebellar Ataxias: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Spinocerebellar Ataxias– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Vatiquinone: PTC Therapeutics
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Leriglitazone: Minoryx Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- DT-216: Design Therapeutics
- Drug profiles in the detailed report…..
- Preclinical Stage Products
- Drug name: Company name
- Drug profiles in the detailed report…..
- 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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