Facioscapulohumeral Muscular Dystrophy Pipeline Insight

DelveInsight’s, “Facioscapulohumeral Muscular Dystrophy – Pipeline Insight, 2022,” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Facioscapulohumeral Muscular Dystrophy 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

Facioscapulohumeral Muscular Dystrophy Understanding

Facioscapulohumeral Muscular Dystrophy: Overview

Facioscapulohumeral muscular dystrophy (FSHD) is a disorder characterized by muscle weakness and wasting (atrophy). The disorder gets its name from muscles that are affected in the face (facio), around the shoulder blades (scapulo), and in the upper arms (humeral). Hamstring and trunk muscles are affected -early on but are less well recognized. Other arm and leg muscles are frequently eventually affected in the course of the disease. Symptoms usually appear before age 20, but can begin in infancy or later in adulthood. Severity of the condition varies widely and some people with the disease allele remain asymptomatic. FSHD is most typically characterized by relatively slow disease progression. Specific symptoms and findings may also vary in range and severity, including among affected members of the same family. Life expectancy is not shortened. FSHD is usually inherited as an autosomal dominant genetic condition. FSHD may be diagnosed based upon a thorough clinical examination, identification of characteristic physical findings, a complete individual and family history, and genetic testing. In some affected individuals, laboratory studies may reveal elevated levels of a particular enzyme in the fluid portion of the blood (serum creatine kinase). Tests may also be conducted to record electrical activity in voluntary (skeletal) muscles at rest and during muscle contraction (electromyography [EMG]). Surgical removal (biopsy) and microscopic examination of small samples of muscle tissue is generally not informative in FSHD. Treatment of FSH dystrophy involves a multidisciplinary team. A neurologist oversees the various needs of the patient and directs care. Specialists in rehabilitation medicine are present to meet with patients and provide individualized exercise and stretching programs for the treatment of weakness and contractures. Patients can also be evaluated for the need for splints, orthotics, or other equipment to help with hand or foot function. Social and financial needs can also be addressed. Some patients with FSH dystrophy may have hearing loss or abnormalities of the blood vessels in the eye.

 

“Facioscapulohumeral Muscular Dystrophy- Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Facioscapulohumeral Muscular Dystrophy pipeline landscape is provided which includes the disease overview and Facioscapulohumeral Muscular Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Facioscapulohumeral Muscular Dystrophy 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, Facioscapulohumeral Muscular Dystrophy 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 Facioscapulohumeral Muscular Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve Facioscapulohumeral Muscular Dystrophy. 

Facioscapulohumeral Muscular Dystrophy Emerging Drugs Chapters

This segment of the Facioscapulohumeral Muscular Dystrophy 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.

 

Facioscapulohumeral Muscular Dystrophy Emerging Drugs

 

Losmapimod: Fulcrum Therapeutics

Losmapimod is an investigational, selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Results reported from the ReDUX4 trial demonstrated slowed disease progression and improved function, including positive impacts on upper extremity strength, supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, with no safety signals attributed to losmapimod. Losmapimod has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of FSHD. Fulcrum Therapeutics intends to submit regulatory application to the US FDA and EMA in Facioscapulohumeral muscular dystrophy. The drug is currently in Phase III stage of development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

 

ATYR1940: aTyr Pharma

ATYR1940 is based on a protein naturally secreted from muscle (Resokine) that may act to influence T-cell activation at the tissue level to promote healthier muscle. ATYR1940 as a potential first-in-class intravenous protein therapeutic candidate for the treatment of rare myopathies with an immune component. There is potential that ATYR1940 may translate into an innovative therapeutic for rare genetic myopathies with an immune component, including limb-girdle muscular dystrophy (LGMD), facioscapulohumeral muscular dystrophy (FSHD). Currently the product is in Phase I/II stage of development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

 

DYNE-301: Dyne Therapeutics

DYNE-301 is a DUX4L1 protein expression stimulant, which consists of company’s proprietary Fab conjugated with our linker to an ASO that is designed to address the genetic basis of FSHD by reducing DUX4 expression in muscle tissue. Proof-of-concept data showing that DYNE-301 reduced expression of key DUX4 biomarkers in FSHD patient myotubes, a type of muscle cell. Company anticipate submitting an IND for DYNE-301 in the second half of 2022. Currently the product is in preclinical stage of development for the treatment of Facioscapulohumeral Muscular Dystrophy.

Further product details are provided in the report……..

Facioscapulohumeral Muscular Dystrophy: Therapeutic Assessment

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

 

Major Players in Facioscapulohumeral Muscular Dystrophy

There are approx. 12+ key companies which are developing the therapies for Facioscapulohumeral Muscular Dystrophy. The companies which have their Facioscapulohumeral Muscular Dystrophy drug candidates in the most advanced stage, i.e. phase III include, Fulcrum Therapeutics.

 

Phases

DelveInsight’s report covers around 12+ 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
 

Facioscapulohumeral Muscular Dystrophy 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
  • Ophthalmic
  • 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.

Facioscapulohumeral Muscular Dystrophy: Pipeline Development Activities

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

Facioscapulohumeral Muscular Dystrophy Report Insights

  • Facioscapulohumeral Muscular Dystrophy Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugsx

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

Introduction

Executive Summary

Facioscapulohumeral Muscular Dystrophy: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Facioscapulohumeral Muscular Dystrophy– DelveInsight’s Analytical Perspective

Late Stage Products (Phase III)

  • Comparative Analysis

Losmapimod: Fulcrum Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase I/II)

  • Comparative Analysis

ATYR1940: aTyr Pharma

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

Drug name : Company name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

DYNE-301: Dyne Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Facioscapulohumeral Muscular Dystrophy Key Companies

Facioscapulohumeral Muscular Dystrophy Key Products

Facioscapulohumeral Muscular Dystrophy - Unmet Needs

Facioscapulohumeral Muscular Dystrophy - Market Drivers and Barriers

Facioscapulohumeral Muscular Dystrophy - Future Perspectives and Conclusion

Facioscapulohumeral Muscular Dystrophy Analyst Views

Facioscapulohumeral Muscular Dystrophy Key Companies

Appendix

List of Table

Table 1: Total Products for Facioscapulohumeral Muscular Dystrophy

Table 2: Late Stage Products

Table 3: Mid Stage Products

Table 4: Early Stage Products

Table 5: Pre-clinical & Discovery Stage Products

Table 6: Assessment by Product Type

Table 7: Assessment by Stage and Product Type

Table 8: Assessment by Route of Administration

Table 9: Assessment by Stage and Route of Administration

Table 10: Assessment by Molecule Type

Table 11: Assessment by Stage and Molecule Type

Table 12: Inactive Products

List of Figures

Figure 1: Total Products for Facioscapulohumeral Muscular Dystrophy

Figure 2: Late Stage Products                             

Figure 3: Mid Stage Products

Figure 4: Early Stage Products

Figure 5: Preclinical and Discovery Stage Products

Figure 6: Assessment by Product Type

Figure 7: Assessment by Stage and Product Type

Figure 8: Assessment by Route of Administration

Figure 9: Assessment by Stage and Route of Administration

Figure 10: Assessment by Molecule Type

Figure 11: Assessment by Stage and Molecule Type

Figure 12: Inactive Products

• Fulcrum Therapeutics
• aTyr Pharma
• Myocea
• Dyne Therapeutics
• Avidity Biosciences
• Arrowhead Pharmaceuticals
• Healx

 

 

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