HDAC Inhibitor - Pipeline Insight, 2025

This “HDAC Inhibitor - Pipeline Insight, 2025” report provides comprehensive insights about 40+ companies and 50+ pipeline drugs in HDAC Inhibitor 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

HDAC Inhibitor: Understanding

HDAC Inhibitor: Overview

Histone deacetylase inhibitors (HDAC inhibitors or HDACi) represent a diverse class of chemical compounds designed to modulate the activity of histone deacetylases (HDACs), enzymes crucial for the dynamic regulation of chromatin structure and gene expression. HDACs typically function by removing acetyl groups from histone proteins, leading to a more condensed chromatin state that results in transcriptional repression. By inhibiting these enzymes, HDAC inhibitors counteract this deacetylation process, promoting a more relaxed chromatin configuration that facilitates enhanced gene transcription and induces profound epigenomic changes.

The therapeutic potential of HDAC inhibitors has been extensively explored, especially in oncology. These compounds have shown promise in treating various malignancies, including peripheral T-cell lymphoma and multiple myeloma. Four HDAC inhibitors - vorinostat, romidepsin, belinostat, and panobinostat - have received approval for clinical use in cancer therapy, highlighting their efficacy in disrupting the cancer cell cycle and inducing cell death.

HDAC inhibitors function by disrupting the normal deacetylation process carried out by histone deacetylases. In a typical cellular environment, acetylation of histones is associated with active gene transcription, while deacetylation leads to gene silencing. By inhibiting HDACs, these compounds increase the acetylation of histones, resulting in a more open chromatin structure that facilitates transcriptional activation

The classification of HDAC inhibitors is based on their selectivity towards different HDAC classes, which can range from broad-spectrum inhibitors affecting multiple HDAC types to those specifically targeting certain HDACs. This specificity can influence their therapeutic efficacy and side effect profiles. Moreover, the impact of HDAC inhibitors extends beyond histones; they also alter the acetylation status of various non-histone proteins. This broader effect can influence critical cellular processes such as proliferation, differentiation, and apoptosis, underscoring their potential as versatile therapeutic agents across multiple medical fields.

“HDAC Inhibitor - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the HDAC Inhibitor pipeline landscape is provided which includes the disease overview and HDAC Inhibitor treatment guidelines. The assessment part of the report embraces, in depth HDAC Inhibitor 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, HDAC Inhibitor 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 HDAC Inhibitor R&D. The therapies under development are focused on novel approaches to treat/improve HDAC Inhibitor.

HDAC Inhibitor Emerging Drugs Chapters

This segment of the HDAC Inhibitor 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.

HDAC Inhibitor Emerging Drugs

Givinostat: Italfarmaco

Givinostat (ITF2357) is an orally bioavailable hydroxymate inhibitor of histone deacetylase (HDAC) with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. Givinostat inhibits class I and class II HDACs, resulting in an accumulation of highly acetylated histones, followed by the induction of chromatin remodeling and an altered pattern of gene expression. At low nonapoptotic concentrations, this agent inhibits the production of pro-inflammatory cytokines such as tumor necrosis factor (TNF), interleukin-1 (IL-1), IL-6, and interferon-gamma. This reduces the expression of tumor necrosis factor (TNF), interleukin 1α and β, and interleukin. It has also been shown to activate the intrinsic apoptotic pathway, inducing apoptosis in hepatoma cells and leukemic cells. This agent may also exhibit anti-angiogenic activity, inhibiting the production of angiogenic factors such as IL-6 and Vascular Endothelial Cell Growth Factor (VEGF) by bone marrow stromal cells. Currently, the drug is in the Phase III stage of its development for the treatment of Polycythemia Vera.

• AMX0035: Amylyx Pharmaceuticals

AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the US). AMX0035 was designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. The company believes that the proprietary combination of PB and TURSO and their complementary mechanisms of action will allow to synergistically target abnormal cell death to better prevent neurodegeneration than treatment targeted at either mechanism of action alone. AMX0035 is being studied as a potential treatment for Wolfram syndrome and progressive supranuclear palsy, two neurodegenerative disease. Currently, the drug is in the Phase III stage of its development for the treatment of Progressive Supranuclear Palsy.

CKD-506: Chong Kun Dang pharmaceuticals

CKD-506 is a novel selective histone deacetylase 6 (HDAC6) inhibitor developed by Chong Kun Dang Pharmaceuticals. It is primarily being investigated for its therapeutic potential in treating autoimmune diseases, including rheumatoid arthritis and systemic lupus erythematosus (SLE). CKD-506 functions by selectively inhibiting HDAC6, an enzyme involved in the regulation of inflammatory responses. This inhibition leads to the suppression of inflammatory cytokines such as TNF-α and type 1 interferons, which are significant contributors to the pathogenesis of various autoimmune conditions. Studies have shown that CKD-506 can improve the function of regulatory T cells (Tregs) and enhance gut barrier function, further supporting its role in reducing inflammation. It is currently in Phase II for Rheumatoid Arthritis.

• CS1: Cereno Scientific

The drug candidate CS1 is an HDAC inhibitor that works through epigenetic modulation, being developed as a treatment for the rare disease PAH. CS1 has the potential to be an effective, safe and disease-modifying drug. CS1’s unique efficacy profile fits well with the pathogenetic mechanisms of PAH and is believed to be able to address today’s major unmet need for better treatment alternatives. The aim of CS1’s development is to offer improved quality of life and prolonged life with CS1 for patients with PAH. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Pulmonary Arterial Hypertension.

CS014: Cereno Scientific

The investigational drug candidate CS014 belongs to Cereno’s HDAC inhibitor program, capitalizing on the principle of epigenetic modulation. The innovative drug candidate represents a novel approach to antithrombotic treatment without an increased risk of bleeding. CS014 is a new chemical entity with a multi-modal mechanism of action as an epigenetic modulator - regulating platelet activity, local fibrinolysis, and clot stability for the prevention of thrombosis without increasing the risk of bleeding, as documented in preclinical studies. Given the potential for the disease-modifying properties seen with HDAC inhibition, additional cardiovascular benefits of CS014 may be expected, including amelioration of inflammation, fibrosis, vascular remodeling and elevated blood pressure. HDAC inhibition as a therapy to avoid thrombosis could fundamentally change the thrombosis prevention landscape and meet a major unmet medical need. It is currently in Phase I for Thrombosis prevention.

HDAC Inhibitor: Therapeutic Assessment

This segment of the report provides insights about the different HDAC Inhibitor drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in HDAC Inhibitor

There are approx. 40+ key companies which are developing the therapies for HDAC Inhibitor. The companies which have their HDAC Inhibitor drug candidates in the most advanced stage, i.e. Phase III include, Italfarmaco and Amylyx Pharmaceuticals.

Phases

The 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

HDAC Inhibitor 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

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

HDAC Inhibitor: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses HDAC Inhibitor 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 HDAC Inhibitor drugs.

HDAC Inhibitor Report Insights

• HDAC Inhibitor Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Italfarmaco
• Amylyx Pharmaceuticals
• Chong Kun Dang pharmaceuticals
• Cereno Scientific
• Xynomic Pharmaceuticals
• HUYA Bioscience
• Recursion Pharmaceuticals Inc.
• Regenacy Pharmaceuticals
• Acrotech Biopharma
• Aphios Corporation

Key Products

• Givinostat
• AMX0035
• CKD-506
• CS1
• CS014
• Abexinostat
• HBI-8000
• REC-2282
• Ricolinostat
• Belinostat
• APH 0812

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