Huntington's Disease - Pipeline Insight, 2024

This “Huntington’s Disease - Pipeline Insight, 2024,” report provides comprehensive insights about 50+ companies and 50+ pipeline drugs in Huntington’s Disease 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

Huntington’s Disease Understanding

Huntington’s Disease: Overview

Huntington's disease is a rare, inherited disease that causes the progressive breakdown (degeneration) of nerve cells in the brain. It is an autosomal dominant disorder caused by a single defective gene on chromosome 4. The symptoms of Huntington’s Disease include involuntary jerking or writhing movements (chorea), muscle problems, such as rigidity or muscle contracture (dystonia), slow or abnormal eye movements, impaired balance, difficulty with speech or swallowing, difficulty organizing, prioritizing or focusing on tasks, and/or fatigue and loss of energy. A diagnosis of Huntington’s disease is based upon a general physical examination, medical history, and thorough neurological and psychiatric examinations. Treatment for Huntington’s disease is focus on managing the symptoms of the disease, includes medications and psychotherapy. Tetrabenazine (Xenazine) and deutetrabenazine (Austedo), which have been specifically approved by the FDA to suppress the involuntary jerking and writhing movements (chorea) associated with Huntington's disease.

Huntington’s Disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Huntington’s Disease pipeline landscape is provided which includes the disease overview and Huntington’s Disease treatment guidelines. The assessment part of the report embraces, in depth Huntington’s Disease 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, Huntington’s Disease 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 Huntington’s Disease R&D. The therapies under development are focused on novel approaches to treat/improve Huntington’s Disease.

Huntington’s Disease Emerging Drugs Chapters

This segment of the Huntington’s Disease 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.

Huntington’s Disease Emerging Drugs

Pridopidine: Prilenia TherapeuticsPridopidine is a highly selective, first in class small molecule which is sigma-1 receptor (S1R) agonist. Pridopidine is currently in late-stage clinical development for Huntington’s disease. Prilenia has an orphan drug designation for pridopidine for the treatment of Huntington’s disease in both the US and Europe.

Tominersen: Ionis PharmaceuticalsTominersen (formerly known as IONIS-HTTRx and RG6042) is an investigational antisense medicine designed to reduce the production of the huntingtin (HTT) protein, which is the genetic cause of Huntington’s disease (HD). The US FDA granted orphan drug designation to Ionis Pharmaceuticals, for tominersen for the treatment of Huntington's disease in January 2016. In August 2018, European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation for the tominersen for the treatment of Huntington's disease (HD). Ionis Pharmaceuticals entered into a collaboration with Roche to develop and commercialize antisense medicines to treat HD in April 2013.

Huntington’s Disease: Therapeutic Assessment

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

Major Players in Huntington’s Disease

There are approx. 50+ key companies which are developing the therapies for Huntington’s Disease. The companies which have their Huntington’s Disease drug candidates in the most advanced stage, i.e. Phase III include, Ionis Pharmaceuticals.

Phases

This report covers around 50+ 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

Huntington’s Disease 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
• Parenteral
• Intravitreal
• Subretinal
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Huntington’s Disease: Pipeline Development Activities

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

Huntington’s Disease Report Insights

• Huntington’s Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Prilenia Therapeutics
• Ionis Pharmaceuticals
• Annexon
• Vaccinex
• Neurocrine Biosciences
• EIP Pharma
• SAGE Therapeutics
• Celon Pharma
• PTC Therapeutics
• WaVe life Sciences
• Amsterdam Molecular Therapeutics
• Novartis
• Retrotope
• Hope Biosciences
• Stealth BioTherapeutics
• Luye Pharma Group
• Neurimmune Therapeutics
• SOLA Biosciences

Key Products

• Pridopidine
• Tominersen
• ANX-005
• Pepinemab
• Valbenazine
• Neflamapimod
• SAGE-718
• PTC-518
• WVE-003
• AMT 130
• Branaplam
• RT001
• HB-adMSC
• NI-302
• SOL-176

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