Huntington Disease Pipeline Assessment: Clinical Trials, Drug Development by Stages, Target, Mechanism of Action (MOA), RoA, Molecule Type and Key Players (2023 Update) by DelveInsight

October 19 21:59 2023
Huntington Disease Pipeline Assessment: Clinical Trials, Drug Development by Stages, Target, Mechanism of Action (MOA), RoA, Molecule Type and Key Players (2023 Update) by DelveInsight
Huntington Disease Pipeline

DelveInsight’s, “Huntington’s Disease Pipeline Insight, 2023,” 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.

In the Huntington’s Disease pipeline 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.

 

To explore more information on the latest breakthroughs in the Huntington’s Disease Pipeline treatment landscape of the report, click here @ Huntington’s Disease Pipeline Outlook

 

Key takeaways from the Huntington’s Disease Pipeline Report

  • DelveInsight’s Huntington’s Disease Pipeline report depicts a robust space with 50+ active players working to develop 50+ pipeline therapies for Huntington’s Disease treatment.
  • The leading Huntington’s Disease Companies includes Prilenia Therapeutics, Ionis Pharmaceuticals, Annexon, Vaccinex, Neurocrine Biosciences, SAGE Therapeutics, PTC Therapeutics, WaVe life Sciences, Novartis, Neurimmune Therapeutics, SOLA Biosciences, UniQure Biopharma, Roche, Annexon, Inc., Azevan Pharmaceuticals, SOM Innovation Biotech SA, Asklepios BioPharmaceutical, Luye Pharma Group Ltd., BrainStorm Cell Therapeutics, Voyager Therapeutics, Passage Bio, Locana Bio, Ceptur Therapeutics, Enzerna Biosciences, AFFiRiS AG, Arvinas, Atalanta Therapeutics, Anima Biotech, and others
  • Promising Huntington’s Disease Pipeline Therapies includes Pridopidine, Tominersen, ANX-005, Pepinemab, Valbenazine, SAGE-718, PTC-518, WVE-003, AMT 130, Branaplam, NI-302, SOL-176, RO7234292, ANX005, SRX246, Bevantolol, SOM3355, BV-101 Gene Therapy, LPM3770164, Passage Bio Research Project, Huntington disease Research Project, BrainStorm Research Project, Locana Huntington disease Research Project,  Ceptur Huntington Research Project, HTT RNA Research project, mHTT Research Project, AFFiRiS Huntington Research Project, HTT Research Project, Anima Biotech mHTT Research Project, and others.
  • The Huntington’s Disease companies and academics are working to assess challenges and seek opportunities that could influence Huntington’s Disease R&D. The Huntington’s Disease pipeline therapies under development are focused on novel approaches to treat/improve Huntington’s Disease.

 

Got Queries? Find out the related information on Huntington’s Disease Mergers and acquisitions, Huntington’s Disease Licensing Activities @ Huntington’s Disease Emerging Drugs, and Recent Trends

 

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.

 

Latest Developmental Activities or News of the Huntington’s Disease Treatment Landscape

  • In August 2022Asklepios BioPharmaceutical, a wholly owned and independently operated subsidiary of Bayer AG,  received clearance to conduct a Phase I/II trial for its novel Huntington’s Disease (HD) gene therapy, BV-101, in France through its subsidiary BrainVectis. This authorization, provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country’s governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge, enables the company to begin recruiting participants
  • In March 2022PTC Therapeutics announced the initiation of the PIVOT-HD Phase  II clinical trial evaluating PTC518 in people with Huntington’s disease (HD). PIVOT-HD is a global trial starting in the United States. PTC518 is an oral, small molecule splicing modifier that was specifically designed to lower huntingtin mRNA and protein selectively. There are no current treatments for the underlying cause of HD
  • In January 2022Ionis Pharmaceuticals announced that its partner Roche is designing a new Phase II trial to evaluate tominersen in Huntington’s disease (HD). After halting dosing in the Phase III GENERATION HD1 study, exploratory posthoc analyses suggest tominersen may benefit younger adult patients with lower disease burden. These results require confirmation in a randomized, placebo-controlled study
  • In September 2021Sage Therapeutics announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation to SAGE-718 for development as a potential treatment for Huntington’s disease (HD).
  • In September 2021, Voyager Therapeutics introduced new programs in Huntington’s disease, a monogenic form of ALS (SOD1), spinal muscular atrophy, and diseases linked to GBA1 mutations, all powered by its proprietary AAV capsids that have demonstrated superior transgene expression in the brain compared to AAV9 delivery in non-human primates.

 

For further information, refer to the detailed Huntington’s Disease Unmet Needs, Huntington’s Disease Market Drivers, and Huntington’s Disease Market Barriers, click here for Huntington’s Disease Ongoing Clinical Trial Analysis

 

Huntington’s Disease Emerging Drugs Profile

Pridopidine: Prilenia Therapeutics

Pridopidine 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 Pharmaceuticals 

Tominersen (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 Pipeline Therapeutics Assessment

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.

 

Request a sample and discover the recent advances in Huntington’s Disease Ongoing Clinical Trial Analysis and Medications, click here @ Huntington’s Disease Treatment Landscape

 

Scope of the Huntington’s Disease Pipeline Insight Report

  • Coverage- Global
  • Huntington’s Disease Companies- Prilenia Therapeutics, Ionis Pharmaceuticals, Annexon, Vaccinex, Neurocrine Biosciences, SAGE Therapeutics, PTC Therapeutics, WaVe life Sciences, Novartis, Neurimmune Therapeutics, SOLA Biosciences, UniQure Biopharma, Roche, Annexon, Inc., Azevan Pharmaceuticals, SOM Innovation Biotech SA, Asklepios BioPharmaceutical, Luye Pharma Group Ltd., BrainStorm Cell Therapeutics, Voyager Therapeutics, Passage Bio, Locana Bio, Ceptur Therapeutics, Enzerna Biosciences, AFFiRiS AG, Arvinas, Atalanta Therapeutics, Anima Biotech, and others
  • Huntington’s Disease Pipeline Therapies- Pridopidine, Tominersen, ANX-005, Pepinemab, Valbenazine, SAGE-718, PTC-518, WVE-003, AMT 130, Branaplam, NI-302, SOL-176, RO7234292, ANX005, SRX246, Bevantolol, SOM3355, BV-101 Gene Therapy, LPM3770164, Passage Bio Research Project, Huntington disease Research Project, BrainStorm Research Project, Locana Huntington disease Research Project,  Ceptur Huntington Research Project, HTT RNA Research project, mHTT Research Project, AFFiRiS Huntington Research Project, HTT Research Project, Anima Biotech mHTT Research Project, and others.
  • Huntington’s Disease Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration

 

Dive deep into rich insights for drugs for Huntington’s Disease Market Drivers and Huntington’s Disease Market Barriers, click here @ Huntington’s Disease Unmet Needs and Analyst Views

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Huntington’s Disease: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Huntington’s Disease – DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Pridopidine: Prilenia Therapeutics
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. Drug Name: Company Name
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I)
  14. Drug Name: Company Name
  15. Drug profiles in the detailed report…..
  16. Preclinical/Discovery Stage Products
  17. Drug Name: Company Name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Huntington’s Disease Key Companies
  21. Huntington’s Disease Key Products
  22. Huntington’s Disease- Unmet Needs
  23. Huntington’s Disease- Market Drivers and Barriers
  24. Huntington’s Disease- Future Perspectives and Conclusion
  25. Huntington’s Disease Analyst Views
  26. Appendix

 

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