DelveInsight’s “Friedreich’s Ataxia Pipeline Insight 2025” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Friedreich’s Ataxia pipeline landscape. It covers the Friedreich’s Ataxia pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Friedreich’s Ataxia 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 Friedreich’s Ataxia Pipeline Report

• In April 2025, Reata announced a study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich’s ataxia.
• In April 2025, PTC Therapeutics conducted a study will serve as a continued access study for participants with FA who have previously participated in a vatiquinone study. The purpose of this study is to assess continued safety and efficacy of vatiquinone dosing in previously treated participants. This study addresses the medical need for participants to continue vatiquinone with a planned study duration of 3 years.
• DelveInsight’s Friedreich’s Ataxia pipeline report depicts a robust space with 10+ active players working to develop 10+ pipeline therapies for Friedreich’s Ataxia treatment.
• The leading Friedreich’s Ataxia Companies such as PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics and others.
• Promising Friedreich’s Ataxia Pipeline Therapies such as Vatiquinone, Interferon Gamma-1b, MIB-626, Elamipretide, CTI-1601, MIN-102, TAK-831, EPI-743 and others.

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Friedreich’s Ataxia Emerging Drugs

• RT 001: Retrotope

RT001 is a clinical-stage isotopically stabilized, synthetic linoleic acid (LA) discovered and developed with Retrotope’s novel platform technology. This platform is designed to combat the oxidative stress and cellular degeneration that arises from lipid peroxidation (LPO). It is currently in phase III stage of development for Friedreich’s ataxia and is being developed by Retrotope.

• Leriglitazone: Minoryx Therapeutics

Leriglitazone (MIN-102) is a novel, orally bioavailable and selective PPAR gamma agonist with a potential best-in-class profile indicated for CNS diseases. It is one of the several metabolites of pioglitazone and has a demonstrated sufficient brain penetration and favorable safety profile in humans, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones. 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. It is currently in phase II stage of development and is being developed by Minoryx Therapeutics.

Friedreich’s Ataxia Market Drivers

• Rapid research in the field of ataxia therapeutics
• Increasing collaborations and acquisitions among key players

Friedreich’s Ataxia Market Barriers

• There are currently no approved therapies for the treatment of patients with Friedreich’s ataxia.

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The Friedreich’s Ataxia pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Friedreich’s Ataxia 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 Friedreich’s Ataxia Treatment.
• Friedreich’s Ataxia Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Friedreich’s Ataxia 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 Friedreich’s Ataxia market.

Friedreich’s Ataxia Companies

PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics and others.

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

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Oral
• Parenteral
• Subcutaneous
• Topical
• Molecule Type

Friedreich’s Ataxia Products have been categorized under various Molecule types such as

• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Product Type

Discover the latest advancements in Friedreich’s Ataxia Treatment by visiting our website. Stay informed about how we’re transforming the future of neurology @ Friedreich’s Ataxia Market Drivers and Barriers, and Future Perspectives

Scope of the Friedreich’s Ataxia Pipeline Report

• Coverage- Global
• Friedreich’s Ataxia Companies- PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics and others.
• Friedreich’s Ataxia Pipeline Therapies- Vatiquinone, Interferon Gamma-1b, MIB-626, Elamipretide, CTI-1601, MIN-102, TAK-831, EPI-743 and others.
• Friedreich’s Ataxia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Friedreich’s Ataxia 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 Friedreich’s Ataxia Pipeline on our website @ Friedreich’s Ataxia Emerging Drugs and Companies

Table of Content

1. Introduction
2. Executive Summary
3. Friedreich’s Ataxia: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Friedreich’s Ataxia– DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Friedreich’s Ataxia Collaboration Deals
9. Late Stage Products (Phase III)
10. RT 001: Retrotope
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. Leriglitazone: Minoryx Therapeutics
14. Drug profiles in the detailed report…..
15. Early Stage Products (Phase I)
16. CTI-1601: Larimar Therapeutics
17. Drug profiles in the detailed report…..
18. Preclinical and Discovery Stage Products
19. LX-2006: LEXEO Therapeutics
20. Drug profiles in the detailed report…..
21. Inactive Products
22. Friedreich’s Ataxia Key Companies
23. Friedreich’s Ataxia Key Products
24. Friedreich’s Ataxia- Unmet Needs
25. Friedreich’s Ataxia- Market Drivers and Barriers
26. Friedreich’s Ataxia- Future Perspectives and Conclusion
27. Friedreich’s Ataxia Analyst Views
28. Friedreich’s Ataxia Key Companies
29. Appendix

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