DelveInsight’s “Huntington’s Disease Pipeline Insight 2025” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in the Huntington’s Disease pipeline landscape. It covers the Huntington’s Disease Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Huntington’s Disease pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Huntington’s Disease Pipeline Report

• On 19 September 2025, Azidus Brasil conducted a phase II dose-response study in which participants with HD will receive three intravenous injections of the investigational product or placebo (one every month for three months) a total of three cycles. The subjects will be randomized in 2: 2: 1 ratio for the groups G1: lower dose (1×10^6 cells/weight range), G2: higher dose (2×10^6 cells/weight range), or G3: placebo.
• On 18 September 2025, Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) conducted a study consists of 2 parts: Dose-Finding Part and Expansion Part; each part consists of 3 phases: Screening Phase (8 weeks, with extension to 12 weeks to accommodate scheduling if needed), Treatment and Initial Follow-Up Phase (52 weeks) and Long-Term Follow-Up Phase (4 years). In the Dose-Finding Part, 2 dose titers will be tested in 3-6 subjects in each cohort. Once a dose is selected based on Dose-Limiting Toxicities, an additional 6 subjects will be enrolled into the Dose Expansion Part.
• On 15 September 2025, Supernus Pharmaceuticals Inc. organized a study is to assess the magnitude of the baseline difference between participants with early Huntington’s Disease (HD) and healthy participants (HP) with respect to measures of cognitive performance.
• DelveInsight’s Huntington’s Disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Huntington’s Disease treatment.
• The leading Huntington’s Disease Companies such as Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio, and others.
• Promising Huntington’s Disease Pipeline Therapies such as Dimebon, Pridopidine, SAGE-718, Tominersen 60 mg, AB-1001, ISIS 443139 10 mg, ER2001 injection, and others.

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The Huntington’s Disease Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Huntington’s Disease Pipeline Report also highlights the unmet needs with respect to the Huntington’s Disease.

Huntington’s Disease Overview

Huntington’s disease is a genetic, progressive, neurodegenerative disorder characterized by the gradual development of involuntary muscle movements affecting the hands, feet, face, and trunk and progressive deterioration of cognitive processes and memory (dementia). This disease causes changes in the central area of the brain, which affect movement, mood and thinking skills. Symptoms of Huntington’s disease usually develop between ages 30 and 50, but they can appear as early as age 2 or as late as 80.

Huntington’s Disease Emerging Drugs Profile

• RG6042: Hoffmann-La Roche

Tominersen (ASO-HTT, RG6042) is an antisense drug in development for the treatment of Huntington’s disease. Tominersen is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for Huntington’s disease. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Huntington’s disease.

• PTC518: PTC Therapeutics

PTC518, a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression. The orally bioavailable small molecule penetrates the blood brain barrier, is selective, titratable, and not effluxed. Currently the drug is in Phase II stage of its clinical trial for the treatment of Huntington’s disease.

• ALN-HTT02: Alnylam Pharmaceuticals

ALN-HTT02 is an innovative therapeutic approach for Huntington’s disease developed by Alnylam Pharmaceuticals. This drug is a small interfering RNA (siRNA) designed to reduce the production of the huntingtin protein, which is implicated in the disease’s pathology. It works by targeting a specific region of the HTT gene’s messenger RNA (mRNA), promoting its degradation and thereby preventing the synthesis of both normal and mutant huntingtin proteins. Administered via intrathecal injection. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Huntington’s disease.

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The Huntington’s Disease Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Huntington’s Disease with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Huntington’s Disease Treatment.
• Huntington’s Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Huntington’s Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Huntington’s Disease market

Huntington’s Disease Companies

Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio and others.

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Huntington’s Disease Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

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Scope of the Huntington’s Disease Pipeline Report

• Coverage- Global
• Huntington’s Disease Companies- Hoffmann-La Roche, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, BPG Bio and others.
• Huntington’s Disease Pipeline Therapies- Dimebon, Pridopidine, SAGE-718, Tominersen 60 mg, AB-1001, ISIS 443139 10 mg, ER2001 injection, and others.
• Huntington’s Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Huntington’s Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

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. RG6042: Hoffmann-La Roche
9. Mid Stage Products (Phase II)
10. PTC518: PTC Therapeutics
11. Early Stage Products (Phase I)
12. ALN-HTT02: Alnylam Pharmaceuticals
13. Mid Stage Products (Phase II)
14. Preclinical Stage Products
15. Drug Name: Company Name
16. Inactive Products
17. Huntington’s Disease – Collaborations Assessment- Licensing / Partnering / Funding
18. Huntington’s Disease – Unmet Needs
19. Huntington’s Disease – Market Drivers and Barriers
20. Appendix

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