Netherton Syndrome - Pipeline Insight, 2025

This “Netherton Syndrome- Pipeline Insight, 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Netherton Syndrome 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

Netherton Syndrome: Understanding

Netherton Syndrome: Overview

Netherton Syndrome (NS) is a rare genetic disorder characterized by a triad of clinical features: congenital ichthyosis (a form of skin scaling), hair shaft abnormalities (typically known as trichorrhexis invaginata or "bamboo hair"), and a predisposition to atopic manifestations such as eczema, asthma, and allergic rhinitis. First described by Earl Netherton in 1958, this syndrome is part of the group of conditions known as ichthyoses, which primarily affect the skin. The signs and symptoms of Netherton Syndrome are diverse and often severe. Newborns with NS may present with a condition called erythroderma, where the skin is red, inflamed, and scaly over large areas of the body. This can lead to complications such as dehydration, infections, and difficulty regulating body temperature. The characteristic hair abnormality, trichorrhexis invaginata, involves the hair shaft folding into itself, creating a "bamboo-like" appearance under microscopic examination. Patients are also highly prone to atopic dermatitis, presenting with chronic, itchy, and inflamed skin lesions, which often lead to a significant impairment in the quality of life.

The cause of Netherton Syndrome lies in genetic mutations affecting the SPINK5 gene, which encodes the serine protease inhibitor LEKTI. LEKTI is crucial for the regulation of skin barrier function and integrity. Mutations in SPINK5 lead to dysfunctional or absent LEKTI, resulting in unregulated protease activity in the skin. This disrupts the skin barrier, causing increased transepidermal water loss, vulnerability to infections, and abnormal desquamation, which manifests as ichthyosis.

Diagnosing Netherton Syndrome involves a combination of clinical evaluation, histopathological examination, and genetic testing. Clinically, the presence of erythroderma, characteristic hair shaft abnormalities, and atopic features can suggest the diagnosis. A skin biopsy may reveal typical findings such as hyperkeratosis and parakeratosis, while hair shaft analysis under a microscope can identify trichorrhexis invaginata. Confirmatory diagnosis is achieved through genetic testing to identify mutations in the SPINK5 gene. Treatment for Netherton Syndrome is primarily supportive and symptomatic, focusing on managing skin symptoms and preventing complications. Regular emollient application and skin care are crucial to maintaining skin hydration and barrier function. Topical steroids and calcineurin inhibitors may be used to control inflammation and eczema. Antibiotics or antifungal treatments are often necessary to manage secondary skin infections. Additionally, addressing nutritional needs and providing supportive care for growth and development are essential, particularly in severe cases. While there is no cure for NS, ongoing research into gene therapy and other targeted treatments holds promise for the future.

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

Netherton Syndrome Emerging Drugs Chapters

This segment of the Netherton Syndrome 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.

Netherton Syndrome Emerging Drugs

QRX003: Quoin Pharmaceuticals

QRX003 is a topical lotion, formulated with a proprietary Invisicare® delivery technology, and contains a broad- spectrum serine protease inhibitor, whose mechanism of action is intended to perform the function of a specific protein, called LEKTI. The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier. QRX003 is designed to lead to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier. 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. In March 2024, Quoin Pharmaceuticals Ltd announced clearance from the U.S. Food and Drug Administration (FDA) to recruit teen subjects aged 14 years and older into its two ongoing clinical trials for QRX003, which is being developed as a potential treatment for Netherton Syndrome (NS). Both trials are being conducted under Quoin’s open Investigational New Drug Application (IND) for QRX003. Currently, the drug is in Phase II/III stage of its development for the treatment of Netherton Syndrome.

• SXR-1096: Sixera Pharma

SXR1096 is a first-in-class, potent and selective small molecule inhibitor of kallikreins 5, 7 and 14 (KLK5, 7 and 14). The compound is formulated in a proprietary skin cream formulation designed for daily use in patients with rare skin conditions. Due to the inborn lack of the protease inhibitor LEKTI, KLK 5, 7 and 14 are overactive in the outermost layer of the skin in patients with Netherton syndrome. These drug targets can best be reached by applying the pharmaceutical directly on the skin. The disease is manifest from birth and can be life threatening in newborns. In July 2022, SXR-1096 received rare pediatric disease designation from US FDA for the treatment of Netherton syndrome. Currently, the drug is in Phase I/II stage of its development for the treatment of Netherton Syndrome.

Netherton Syndrome: Therapeutic Assessment

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

Major Players in Netherton Syndrome

• There are approx. 5+ key companies which are developing the therapies for Netherton Syndrome. The companies which have their Netherton Syndrome drug candidates in the most advanced stage, i.e. phase II/III include, Quoin Pharmaceuticals.

Phases

The 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

Netherton Syndrome 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.

Netherton Syndrome: Pipeline Development Activities

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

Netherton Syndrome Report Insights

• Netherton Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Quoin Pharmaceuticals
• Sixera Pharma
• Daiichi Sankyo
• Boehringer Ingelheim
• BioCryst Pharmaceuticals

Key Products

• QRX003
• SXR-1096
• DS-2325a
• BI 655130
• BCX17725

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