Facioscapulohumeral Muscular Dystrophy - Pipeline Insight, 2024

This “Facioscapulohumeral Muscular Dystrophy - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 10+ 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) typically presents with weakness of the facial muscles, the stabilizers of the scapula, or the dorsiflexors of the foot. Severity is highly variable. Weakness is slowly progressive and approximately 20% of affected individuals eventually require a wheelchair. Life expectancy is not shortened.

Two types of FSHD have been described, FSHD1 (95% of those affected) and FSHD2 (5% of those affected). FSHD1 and FSHD2 have the same signs and symptoms but different genetic causes. FSHD1 is inherited in an autosomal dominant manner. Approximately 70%-90% of individuals have inherited the disease-causing deletion from a parent, and approximately 10%-30% of affected individuals have FSHD as the result of a de novo deletion. Offspring of an affected individual have a 50% chance of inheriting the deletion. Prenatal testing for a pregnancy at increased risk is possible if the D4Z4 pathogenic contraction has been identified in the family. FSHD2 is inherited in a digenic manner.

In around 90% of FSHD patients, symptoms usually begin before age 20, with weakness and atrophy of the muscles around the eyes and mouth, shoulders, abdominal muscles, upper arms, and lower legs, usually with asymmetric involvement.

Some experts divide FSHD into adult-onset and infantile-onset forms. The adult-onset (which includes FSHD that begins in adolescence) is far more common. In either type of FSHD, facial weakness can start in childhood. Occasionally, other FSHD symptoms appear in early childhood.

Infantile-onset FSHD generally runs a more pronounced course with regard to muscle weakness, and sometimes also affects hearing and vision. Preliminary evidence suggests that the infantile-onset form is associated with a larger piece of missing DNA.

The diagnosis of FSHD1 is established in a proband with characteristic clinical features by identification of a heterozygous pathogenic contraction of the D4Z4 repeat array in the subtelomeric region of chromosome 4q35 on a chromosome 4 permissive haplotype. The diagnosis of FSHD2 is established in a proband by identification of hypomethylation of the D4Z4 repeat array in the subtelomeric region of chromosome 4q35 on a chromosome 4 permissive haplotype. Hypomethylation of the D4Z4 repeat array can be the result of a heterozygous pathogenic variant in SMCHD1 or DNMT3B.

Consultation with a physical therapist to establish appropriate exercise regimen; ankle/foot orthoses to improve mobility and prevent falls; occupational and speech therapy in individuals with infantile onset; surgical fixation of the scapula to the chest wall may improve range of motion of the arms over the short term; management of chronic pain by physical therapy and medication; monitoring respiratory function; lubricants to prevent drying of the sclera or taping the eyes shut during sleep to treat exposure keratitis; treatment for retinal vasculopathy as per ophthalmologist; standard treatment of sensorineural hearing loss.

Surveillance: Annual physical therapy assessment; Pain should be assessed at regular visits to the primary care physician or physical therapist; screening for hypoventilation in individuals with abnormal PFTs, severe proximal weakness, kyphoscoliosis, wheelchair dependence, or comorbid disease affecting ventilation; pulmonary consultation for FVC < 60%, excessive daytime somnolence or nonrestorative sleep, and prior to surgical procedures requiring anesthesia; annual dilated ophthalmoscopy in individuals with early childhood-onset FSHD with large pathogenic contraction of D4Z4 and adults with visual symptoms; audiometry in infants at each visit and annually in children.

"Facioscapulohumeral Muscular Dystrophy- Pipeline Insight, 2024" report 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).

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. Currently the product is in preclinical stage of development for the disease.

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. 10+ 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

This report covers around 10+ 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 Drugs

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?

Key Players

• Fulcrum Therapeutics
• Dyne Therapeutics.
• Hoffmann-La Roche
• aTyr Pharma, Inc.
• Avidity Biosciences, Inc.

Key Products

• Losmapimod
• DYNE-301
• RO7204239
• ATYR1940
• AOC 1020

This product will be delivered within 2 business days.