Ichthyosis - Pipeline Insight, 2025

This “Ichthyosis - Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 6+ pipeline drugs in Ichthyosis 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.

Ichthyosis: Understanding

Ichthyosis: Overview

Ichthyosis refers to a group of skin disorders characterized by dry, scaly, rough, and often red skin, with symptoms ranging from mild to severe. These conditions are typically hereditary, caused by genetic mutations passed down from parents, though some cases are acquired due to other medical disorders or medications. The condition affects the skin's ability to shed cells properly, leading to a buildup of scales. While most forms of ichthyosis affect only the skin, some rarer forms can impact internal organs as well. Diagnosis has traditionally relied on clinical evaluation, family history, and occasionally laboratory tests, with molecular genetic analysis providing a clearer understanding of the disease mechanisms. Histopathological examination of skin biopsies has revealed patterns such as hyperkeratosis, parakeratosis, and changes in the epidermis, which aid in distinguishing various types. Although there is no cure for ichthyosis, treatments are available to manage symptoms and improve quality of life. The severity and progression of the disease vary, requiring lifelong management, and research into genetic causes and treatments is ongoing.

The symptoms of ichthyosis vary in severity, but they typically involve dry, scaly skin that can range from mild flakiness to large, thick, armor-like plates. These scales, which can be white, gray, or brown, often appear on the trunk, stomach, buttocks, legs, face, and scalp, and may worsen in cold, dry weather while improving in warmer conditions. Itching, redness, and skin cracking are common, with more severe cases causing blisters, pain, and difficulty moving due to tight, thickened skin. Additional symptoms may include hair loss, fragile hair, dry eyes, trouble closing eyelids, difficulty sweating, and even hearing loss or joint mobility issues. Most individuals with ichthyosis experience a form known as ichthyosis vulgaris, which often presents in childhood and may worsen before improving with age. Many also experience associated eczema, and while the condition usually persists throughout life, treatments are available to help manage the symptoms and improve quality of life.

Ichthyosis is caused by gene mutations that disrupt the normal cycle of skin cell growth and shedding. These mutations lead to one of three main patterns: skin cells may grow faster than they shed, shed more slowly than they grow, or grow and shed at normal rates but in an imbalanced manner. The inheritance of ichthyosis follows various patterns depending on the type. In dominant inheritance, an individual inherits one normal and one mutated gene, with a 50% chance of passing the disorder to their children. Recessive inheritance involves both parents carrying one mutated gene without symptoms, with a 25% chance of passing both mutated genes to their child, resulting in the disorder. X-linked inheritance affects males more often, as the gene mutation is located on the X chromosome, and males only have one X chromosome. In females, who have two X chromosomes, one normal and one mutated X gene may result in carrier status. Finally, spontaneous mutations can occur without a family history, most commonly in dominant and X-linked forms.

The diagnosis of ichthyosis begins with a detailed medical history and physical examination by a dermatologist, assessing the skin, hair, and nails for characteristic signs. If necessary, the doctor may perform a skin biopsy, examining the sample under a microscope to identify abnormal skin cell patterns, and conduct genetic testing on blood or saliva to detect mutations responsible for specific forms of ichthyosis. While there is no cure for ichthyosis, treatments are available to manage the symptoms, primarily aimed at alleviating dryness, scaling, and discomfort. Emollients such as creams, lotions, or ointments containing ingredients like lanolin, urea, alpha hydroxy acids, or ceramides are commonly recommended to improve moisture retention. Applying these moisturizers immediately after bathing, when the skin is still damp, is most effective. Additional measures may include frequent baths to hydrate the skin, using saltwater baths to reduce itching and stinging, and incorporating mild antiseptic solutions, such as bleach, to prevent infections in individuals prone to recurrent skin infections. Mechanical exfoliation with a pumice stone or products containing salicylic acid, glycolic acid, or lactic acid can help remove excess dead skin. Although these treatments help manage symptoms, patients often require lifelong care to maintain skin health and prevent flare-ups.

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

Ichthyosis Emerging Drugs Chapters

This segment of the Ichthyosis 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.

Ichthyosis Emerging Drugs

TMB-001: Timber Pharmaceuticals Inc.

TMB-001 is an investigational topical formulation of isotretinoin developed by Timber Pharmaceuticals, specifically for the treatment of congenital ichthyosis (CI). TMB-001 treats congenital ichthyosis by promoting keratolysis to reduce skin scaling, enhancing hydration to restore the skin barrier, providing lubrication to alleviate discomfort, and modulating gene expression for healthier skin regeneration. The formulation utilizes a patented delivery system designed to enhance the absorption and efficacy of isotretinoin when applied topically. The drug has also been granted the Orphan Drug Designation and Breakthrough therapy status by the FDA. Currently, the drug is in Phase III stage of its development for the treatment of Ichthyosis.

QRX003: Quoin Pharmaceuticals

QRX003 is a once-daily topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary Invisicare delivery technology. When applied daily to the skin, the active ingredient in QRX003 performs the function of the missing LEKTI protein and down regulates, but does not completely stop, the activity of the Kallikreins, leading to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier. The serine protease inhibitor in QRX003 also acts as a potent anti-inflammatory and antioxidant. The Invisicare delivery technology offers immediate protection against TEWL and environmental agents. Uniquely this technology both moisturizes the skin while providing a protective barrier. Currently, the drug is in Phase II/III stage of its clinical trial for the treatment of Ichthyosis.

Ichthyosis: Therapeutic Assessment

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

Major Players in Ichthyosis

• There are approx. 4+ key companies which are developing the therapies for Ichthyosis. The companies which have their Ichthyosis drug candidates in the most advanced stage, i.e. Phase II include, Timber Pharmaceuticals Inc.

Phases

The report covers around 6+ 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

Ichthyosis 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.

Ichthyosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Ichthyosis 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 Ichthyosis drugs.

Ichthyosis Report Insights

• Ichthyosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Timber Pharmaceuticals Inc.

Boehringer Ingelheim

• Quoin Pharmaceuticals
• Krystal Biotech, Inc.

Key Products

• TMB-001
• Spesolimab
• QRX003
• KB105

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