Friedreich Ataxia - Pipeline Insight, 2024

This “Friedreich’s ataxia - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Friedreich’s 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

Friedreich’s ataxia Understanding

Friedreich’s ataxia: Overview

Friedreich’s ataxia (FRDA) is a genetic, progressive, neurodegenerative movement disorder, with a typical age of onset between 10 and 15 years. Initial symptoms may include unsteady posture, frequent falling, and progressive difficulty in walking due to impaired ability to coordinate voluntary movements (ataxia). Affected individuals often develop slurred speech (dysarthria), characteristic foot deformities, and an irregular curvature of the spine (scoliosis).

FRDA is often associated with cardiomyopathy, a disease of cardiac muscle that may lead to heart failure or irregularities in heart rhythm (cardiac arrhythmias). About a third of the people with FRDA develop diabetes mellitus. The symptoms and clinical findings associated with FRDA result primarily from degenerative changes in the sensory nerve fibers at the point where they enter the spinal cord in structures known as dorsal root ganglia. This results in secondary degeneration of nerve fibers in the spinal cord which leads to a deficiency of sensory signals to the cerebellum, the part of the brain that helps to coordinate voluntary movements.

FRDA is caused by abnormalities (mutations) in the FXN gene and people with FRDA inherit a mutation from each parent, so they have mutations in both copies of their FXN gene. This pattern, wherein parents who are clinically unaffected because they only carry a single mutated FXN gene but can have a child who is affected because he / she inherits a mutation from both carrier parents, is called autosomal recessive inheritance.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Friedreich’s ataxia R&D. The therapies under development are focused on novel approaches for Friedreich’s ataxia.

Friedreich’s ataxia Emerging Drugs Chapters

This segment of the Friedreich’s ataxia 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.

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 byRetrotope.

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: Therapeutic Assessment

This segment of the report provides insights about the different Friedreich’s ataxia drugs segregated based on following parameters that define the scope of the report, such as:

Major Players working on Friedreich’s ataxia

There are approx. 10+ key companies which are developing the Friedreich’s ataxia. The companies which have their Friedreich’s ataxia drug candidates in the most advanced stage, i.e. Phase III include, Retrotope.

Phases

This report covers around 10+ products under different phases of clinical development like

• Late-stage products (Phase III and
• Mid-stage products (Phase II and
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

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

Product Type

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

Friedreich’s ataxia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Friedreich’s 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 Friedreich’s ataxia drugs.

Friedreich’s ataxia Report Insights

• Friedreich’s ataxia pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Friedreich’s ataxia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Scenario and Emerging Therapies:

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

Key Players

• PTC Therapeutics
• Retrotope
• Reata Pharmaceuticals
• Minoryx Therapeutics
• Larimar Therapeutics
• LEXEO Therapeutics
• Exicure
• StrideBio
• Voyager Therapeutics
• Lacerta Therapeutics

Key Products

• Vatiquinone
• RT001
• Omaveloxolone
• Leriglitazone
• CTI-1601
• LX-2006
• XCUR FXN
• STRX-110
• VY-FXN01