Spinocerebellar Ataxias - Pipeline Insight, 2024

This “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.

Geography Covered

• Global coverage

Spinocerebellar Ataxias: Understanding

Spinocerebellar Ataxias: Overview

Spinocerebellar ataxia (SCA) is indeed a complex condition characterized by inherited, progressive, neurodegenerative, and heterogeneous features, primarily affecting the cerebellum. As part of the broader spectrum of hereditary cerebellar ataxias, SCA encompasses a range of genetic subtypes, with more than 40 distinct ones identified so far. These subtypes are classified based on the genetic loci in the order of their discovery. SCA1 was the pioneering subtype identified, with subsequent subtypes designated sequentially. While SCA typically manifests with cerebellar involvement, it's important to note that it can extend beyond the cerebellum and spinal cord, affecting various parts of the central nervous system such as pontine nuclei, spinal cord, peripheral nerves, cortex, and basal ganglia. Spinocerebellar ataxia (SCA) pathogenesis involves genetic mutations causing abnormal protein products, transcriptional dysregulation, and dysfunction of autophagy, channelopathies, and mitochondrial dysfunction. Purkinje cell degeneration is central, driven by toxic protein aggregates, impaired cellular processes, and disrupted neuronal signaling. RNA repeats contribute to neurotoxicity, while DNA repair defects may exacerbate CAG expansions. Overall, SCA pathogenesis is multifaceted, involving intricate interactions between genetic, molecular, and cellular mechanisms.

Clinical characterization is crucial prior to genetic analysis, although phenotypic overlap among SCA subtypes often necessitates genetic testing for definitive diagnosis. Advances in molecular genetics facilitate early classification and familial screening, primarily via polymerase chain reaction (PCR) for nucleotide repeats. Initial testing typically targets common SCAs like SCA1, 2, and 3, proceeding to other subtypes if necessary. Neuroimaging reveals cerebellar atrophy, notably in SCA2, and specific regional changes aiding subtype identification. Electrophysiological assessments can detect nerve conduction abnormalities but do not differentiate subtypes. While PET scanning and magnetic resonance spectroscopy may reveal metabolic aberrations, their routine use is limited by availability and cost.

Treatment for spinocerebellar ataxia (SCA) primarily aims at managing symptoms, including seizures, tremors, and depression, ataxia, and eye issues, using various medications such as antiepileptics, botulinum toxin injections, beta-blockers, primidone, antidepressants, and levodopa. N-acetyl cysteine and citalopram show promise in mitigating symptoms by targeting specific mechanisms. Dantrolene and chlorzoxazone offer symptomatic relief by modulating calcium channels and potassium channels, respectively. Emerging therapies focus on clearing misfolded mutant proteins, including chemical chaperones, mTOR inhibitors, and antisense oligonucleotides, which have shown efficacy in decreasing ataxin expression and delaying disease onset. Neurorehabilitation, physical therapy, occupational therapy, and speech therapy play pivotal roles in improving motor functions and enhancing daily living activities for patients with SCA.

"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 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

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.

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. 10+ 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, PTC Therapeutics.

Phases

This report covers around 12+ 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

• Lexeo Therapeutics
• Vico Therapeutics
• PTC Therapeutics
• Minoryx Therapeutics
• IntraBio
• Acasti Pharma
• Design Therapeutics

Key Products

• LX2006
• VO659
• Vatiquinone
• MIN-102
• IB1001
• GTX-102
• DT-216

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