New Therapeutics Options in Huntington's Disease Pipeline as 45+ Key Companies are Working on Drug Profiles | DelveInsight

News provided by

Oct 24, 2022, 11:30 ET

Huntington's Disease pipeline constitutes 45+ key companies continuously working towards developing 50+ Huntington's Disease treatment therapies, analyzes DelveInsight

LAS VEGAS, Oct. 24, 2022 /PRNewswire/ -- DelveInsight's 'Huntington's Disease Pipeline Insight – 2022' report provides comprehensive global coverage of available, marketed, and pipeline Huntington's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Huntington's disease pipeline domain.

Key Takeaways from the Huntington's Disease Pipeline Report

DelveInsight's Huntington's disease pipeline report depicts a robust space with 45+ active players working to develop 50 + pipeline therapies for Huntington's disease treatment.
Key Huntington's disease companies such as 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 are evaluating novel Huntington's disease treatment drugs candidate to improve the treatment landscape.
Promising Huntington's disease pipeline therapies in various stages of development include 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.
In August 2022, Asklepios 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 2022, PTC 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 2022, Ionis 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 2021, Sage 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.

Request a sample and discover the recent advances in Huntington's disease treatment @Huntington's Disease Pipeline Report

The Huntington's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Huntington's disease products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Huntington's disease pipeline landscape.

Huntington's Disease Overview

Huntington's disease is a rare, inherited disease that causes the progressive breakdown (degeneration) of brain nerve cells. It's an autosomal dominant condition caused by a single faulty gene on chromosome 4. The common 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 speaking or swallowing, difficulty organizing, prioritizing, or focusing on tasks, and fatigue and loss of energy. A general physical examination, medical history, and thorough neurological and psychiatric examinations are used for Huntington's disease diagnosis.

Huntington's disease treatment focuses on symptom management, which includes medications and psychotherapy. Tetrabenazine (Xenazine) and deutetrabenazine (Austedo) have been specifically approved by the FDA to suppress Huntington's disease-related involuntary jerking and writhing movements (chorea).

Find out more about Huntington's disease treatment @Drug Treatment for Huntington's Disease

A snapshot of the Huntington's Disease Pipeline Drugs mentioned in the report:

Drugs	Company	Phase	MoA	RoA
Pridopidine	Prilenia Therapeutics	Phase III	Dopamine D2 receptor antagonists; Glutamate modulators; Sigma-1 receptor agonists	Oral
RO7234292	Roche	Phase III	RNA interference	Intrathecal
Tominersen	Ionis Pharmaceuticals	Phase III	HD protein inhibitors; RNA interference	Intrathecal
Valbenazine	Neurocrine Biosciences	Phase III	Vesicular monoamine transporter 2 inhibitors	Oral
ANX-005	Annexon	Phase II	Complement C1 inhibitors	Intravenous
Pepinemab	Vaccinex	Phase II	CD100 antigen inhibitors	Intravenous
SRX246	Azevan Pharmaceuticals	Phase II	Vasopressin 1a receptor antagonists	Oral
SAGE-718	SAGE Therapeutics	Phase II	NMDA receptor modulators	Oral
WVE-003	WaVe life Sciences	Phase I/II	RNA interference	Intrathecal
AMT 130	UniQure Biopharma	Phase I/II	Gene silencing	NA
LPM3770164	Luye Pharma Group	Phase I	Vesicular monoamine transporter 2 inhibitors	Oral
SOL-175	SOLA Biosciences	Preclinical	Gene transference	Parenteral

Learn more about the emerging Huntington's disease pipeline therapies @Huntington's Disease Clinical Trials

Huntington's Disease Therapeutics Assessment

The Huntington's disease pipeline report proffers an integral view of Huntington's disease emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Huntington's Disease Pipeline Report

Coverage: Global
Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravitreal, Subretinal, Topical
Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
Therapeutics Assessment By Mechanism of Action: Dopamine D2 receptor antagonists, Glutamate modulators, Sigma-1 receptor agonists, HD protein inhibitors, RNA interference, Complement C1 inhibitors, CD100 antigen inhibitors, Vesicular monoamine transporter 2 inhibitors, P38 mitogen-activated protein kinase inhibitors, NMDA receptor modulators
Key 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
Key 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.

Dive deep into rich insights for FDA-approved drugs for Huntington's disease, visit @Huntington's Disease Drugs

Table of Contents

1.	Huntington's Disease Pipeline Report Introduction
2.	Huntington's Disease Pipeline Report Executive Summary
3.	Huntington's Disease Pipeline: Overview
4.	Analytical Perspective In-depth Commercial Assessment
5.	Huntington's Disease Pipeline Therapeutics
6.	Huntington's Disease Pipeline: Late Stage Products (Phase III)
6.1	RO7234292: Roche
7.	Huntington's Disease Pipeline: Late Stage Products (Phase II)
7.1	SAGE-718: Sage Therapeutics
8.	Huntington's Disease Pipeline: Mid Stage Products (Phase I/II)
8.1	AMT 130: UniQure Biopharma
9.	Huntington's Disease Pipeline: Early Stage Products (Phase I)
9.1	LPM3770164: Luye Pharma Group
10.	Huntington's Disease Pipeline Therapeutic Assessment
11.	Inactive Products in the Huntington's Disease Pipeline
12.	Company-University Collaborations (Licensing/Partnering) Analysis
13.	Key Companies
14.	Key Products in the Huntington's Disease Pipeline
15.	Unmet Needs
16.	Market Drivers and Barriers
17.	Future Perspectives and Conclusion
18.	Analyst Views
19.	Appendix

For further information on the Huntington's disease pipeline therapeutics, reach out @Drugs for Huntington's Disease

Related Reports

Huntington's Disease Market

Huntington's Disease Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Huntington's disease companies including Amsterdam Molecular Therapeutics, Novartis, Retrotope, Hope Biosciences, Stealth BioTherapeutics, among others.

Huntington's Disease Epidemiology Forecast

Huntington's Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Huntington's disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Wilson Disease Pipeline

Wilson Disease Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others.

Wilson Disease Market

Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others.

Bipolar Disorder Pipeline

Bipolar Disorder Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bipolar disorder companies, including Vanda Pharmaceuticals, Pear Therapeutics, Sunovion, Lyndra Therapeutics, among others.

Obsessive-Compulsive Disorder Pipeline

Obsessive-Compulsive Disorder Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key obsessive-compulsive disorder companies, including Ceruvia Lifesciences, Biohaven Pharmaceuticals, Octapharma, among others.

Other Trending Reports