DUX4 protein inhibitors - Pipeline Insight, 2024

This “DUX4 protein inhibitors - Pipeline Insight, 2024” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in DUX4 protein inhibitors 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

DUX4 protein inhibitors Understanding

DUX4 protein inhibitors: Overview

Double homeobox 4 (DUX4) is a transcription factor implicated in zygotic genome activation (ZGA) during the four-cell stage in human embryos where it acts as an activator of repetitive elements and cleavage-specific genes. DUX4 is considered to be epigenetically repressed in most somatic tissues, including in skeletal muscles. DUX4 is an embryonic transcription factor whose misexpression in skeletal muscle causes facioscapulohumeral muscular dystrophy (FSHD). DUX4 induces the transcription of thousands of RNAs and dysregulates multiple pathways that could contribute to FSHD pathophysiology. DUX4 protein is 424 amino acids long. Two homeodomains are situated at the N-terminus. A transcription-activating domain (TAD) and p300-binding domain are situated at the C-terminus. The TAD encompasses a potential nine amino acid TAD (9aaTAD).

DUX4 protein inhibitors - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the DUX4 protein inhibitors pipeline landscape is provided which includes the disease overview and DUX4 protein inhibitors treatment guidelines. The assessment part of the report embraces, in depth DUX4 protein inhibitors 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, DUX4 protein inhibitors 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 DUX4 protein inhibitors R&D. The therapies under development are focused on novel approaches to treat/improve DUX4 protein inhibitors.

DUX4 protein inhibitors Emerging Drugs Chapters

This segment of the DUX4 protein inhibitors report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

DUX4 protein inhibitors Emerging Drugs

Losmapimod: Fulcrum TherapeuticsLosmapimod is a selective p38a/ß mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38a/ß inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38a/ß reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. In 2020, the company received U.S. and European Orphan Drug Designation for losmapimod for the treatment of FSHD. In May 2021, the Food and Drug Administration (FDA) has granted Fast Track designation to losmapimod for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The Company is evaluating the efficacy and safety of losmapimod in patients with FSHD in the randomized, double-blind, placebo-controlled phase IIb ReDUX4 trial.

DUX4 protein inhibitors: Therapeutic Assessment

This segment of the report provides insights about the different DUX4 protein inhibitors drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in DUX4 protein inhibitors

There are approx. 3+ key companies which are developing the therapies for DUX4 protein inhibitors. The companies which have their DUX4 protein inhibitors drug candidates in the most advanced stage, i.e. phase II include Fulcrum therapeutics.

Phases

This report covers around 3+ 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

DUX4 protein inhibitors pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Subcutaneous
• Intravenous
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Peptides
• Polymer
• Small molecule
• Product Type

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

DUX4 protein inhibitors: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses DUX4 protein inhibitors 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 DUX4 protein inhibitors drugs.

DUX4 protein inhibitors Report Insights

• DUX4 protein inhibitors Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

DUX4 protein inhibitors 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 DUX4 protein inhibitors drugs?
• How many DUX4 protein inhibitors drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of DUX4 protein inhibitors?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the DUX4 protein inhibitors 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 DUX4 protein inhibitors and their status?
• What are the key designations that have been granted to the emerging drugs?

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