Lamellar Icthyosis - Pipeline Insight, 2024

This “Lamellar Icthyosis- Pipeline Insight, 2024” report provides comprehensive insights about 4+ companies and 5+ pipeline drugs in Lamellar Icthyosis 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

Lamellar Icthyosis: Understanding

Lamellar Icthyosis: Overview

Lamellar ichthyosis (LI) is a rare genetic skin disorder that is present at birth. This condition is inherited in an autosomal recessive pattern. Infants with this condition are typically born with a tight, clear sheath covering their skin called a collodion membrane. Other signs and symptoms of the condition may include ectropion, lips that turn outward, hair loss, palmoplantar hyperkeratosis, nail abnormalities, dehydration, and respiratory problems. The diagnosis of lamellar ichthyosis can be made on the basis of medical history, symptoms, physical exam, and laboratory tests. Treatment generally includes topical applications. These creams often contain ceramides or cholesterol. Moisturizers with petrolatum or lanolin may also be used. Sometimes, mild keratolytics or topical retinoids are used as treatment options.

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

Lamellar Icthyosis Emerging Drugs Chapters

This segment of the Lamellar Icthyosis 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.

Lamellar Icthyosis Emerging Drugs

TMB-001: Timber Pharmaceuticals TMB-001 (0.05% and 0.1% isotretinoin) is under clinical development as a topical therapy for the treatment of congenital ichthyosis, including recessive X-linked ichthyosis and autosomal recessive congenital ichthyosis-lamellar ichthyosis subtypes. The US FDA and the EMA granted orphan drug designation to TMB 001 for the treatment of congenital ichthyosis. In 2018, the FDA awarded $1.5 million to support Phase IIa and Phase IIb clinical trials evaluating TMB-001 through its Orphan Products Grant program.

During the fourth quarter of 2021, Timber announced positive top line results for its Phase IIb CONTROL Study. Clinically meaningful efficacy with a favorable safety profile demonstrated the potential of TMB-001 to be an important option for the treatment of CI where the options for standard of care is limited. The Company is currently being evaluating the drug in Phase III stage of its development.

KB105: Krystal Biotech KB105 is a gene therapy-based therapeutic, currently in Phase II clinical study to treat patients with TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI). KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s technology platform to deliver a functional human TGM1 gene directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viralvectors.

Lamellar Icthyosis: Therapeutic Assessment

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

Major Players in Lamellar Icthyosis

• There are approx. 4+ key companies which are developing the therapies for Lamellar Icthyosis. The companies which have their Lamellar Icthyosis drug candidates in the most advanced stage, i.e. phase III include, Timber Pharmaceuticals.

Phases

This report covers around 5+ 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

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Lamellar Icthyosis: Pipeline Development Activities

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

Lamellar Icthyosis Report Insights

• Lamellar Icthyosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Timber Pharmaceuticals
• Krystal Biotech
• Janssen

Key Products

• TMB-001
• KB-105
• Ustekinumab

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