The Quoin team is relentlessly focused on developing and commercializing innovative treatments for rare and orphan diseases, for which none exists today. Our pipeline comprises four unique products that collectively have the potential to target a broad number of indications, including Netherton Syndrome, Scleroderma, Peeling Skin Syndrome, Palmoplantar Keratoderma, Epidermolysis Bullosa and others. We are committed to continuously expanding our pipeline of products to target additional rare and orphan diseases.

Therapeutic Development Pipeline

Product Candidate Indication Pre-Clinical Phase 1 Phase 2 Phase 3 Approval
  • Netherton Syndrome
  • Peeling Skin Syndrome
  • SAM Syndrome
  • Palmoplantar Keratoderma
  • QRX008
  • Scleroderma
  • QRX007
  • Netherton Syndrome
  • QRX004
  • Epidermolysis Bullosa
  • Netherton Syndrome

    Netherton Syndrome (NS) is a rare and sometimes fatal skin disease for which there is currently no approved treatment, and no cure. NS is caused by a mutation of the SPINK5 gene which leads to severe skin barrier defects and recurring severe infections, as well as a pronounced predisposition to allergies, asthma, skin cancers and eczema. Patients also suffer from severe dehydration, an inability to regulate their body temperature, chronic skin inflammation and stunted growth.

    The only treatment options currently available for Netherton Syndrome are limited to providing minor symptomatic relief. While moisturizers are frequently used, due to patients’ highly compromised skin, those containing lanolin and petrolatum can cause further skin damage due to friction or high shear forces upon application or removal. Topical steroids can further reduce the thickness of the skin barrier and can cause Cushing’s Syndrome. Other standard topical treatments, such as calcineurin inhibitors, can lead to dangerously high systemic blood levels due to the defective skin barrier. There is a huge unmet need for a safe and effective treat for this devastating disease.

    Other Target Indications

    Peeling Skin Syndrome

    Peeling Skin Syndrome (PSS) is a rare, genetic skin disorder characterized by painless, continual skin peeling due to a separation of the stratum corneum from the underlying layers of the Epidermis. PSS may be generalized, affecting skin over the patient’s entire body, or localized, affecting only the skin of the hands and feet. Generalized PSS can be categorized as non-inflammatory, and less severe, or inflammatory, which is typically more severe and involves other organ systems. There is currently no approved treatment for PSS, and patients manage symptoms using over-the-counter emollients.

    SAM Syndrome

    SAM Syndrome, also known as severe skin dermatitis, multiple allergies and metabolic wasting, is a life-threatening rare disease caused by mutations in the desmoglein 1 (DSG1) and desmoplakin (DSP) genes. A compromised skin barrier results in a predisposition to allergies, and other symptoms include severe pruritus, palmoplantar keratoderma, pustular psoriasis and excessive sweating. Quoin is pursuing the development of QRX003 as a potential treatment for SAM Syndrome.

    Palmoplantar Keratoderma

    Palmoplantar Keratoderma (PPK) is a group of rare skin disorders with symptoms including thickened skin on the palms of the hands and the soles of the feet, fragile and blistering skin, and excessive perspiration. In its rarer forms, PPK can affect organs other than the skin. Genetic PPKs result in keratin abnormalities in patients.


    Scleroderma is a rare, autoimmune disease that affects connective tissue of the skin, blood vessels, internal organs and digestive tract. Symptoms of scleroderma include thickening and tightening of the skin, and inflammation that leads to problems in other organs including the lungs, heart and kidneys. There is currently no approved treatment or cure for scleroderma.

    Epidermolysis Bullosa

    Epidermolysis Bullosa (EB) is a group of rare and genetic skin disorders in which the skin is so fragile, even minor trauma or friction can have devastating results, causing severe pain, blistering, scarring, infections, chronic wounds and immobility. In severe cases, the cost of bandaging alone for EB patients can exceed $10,000 per month. One format of the disease, Recessive Dystrophic EB (RDEB) which is caused by the mutation of the COL7A1 gene, is marked by devastating, progressive, painful blistering. RDEB is diagnosed at infancy and accompanied by a high mortality rate – 76% of patients do not live beyond their 30s.

    Patients & Families

    Rare diseases are only rare if you don’t live with one.®

    Quoin was founded with a singular mission of developing safe and effective treatments for patients who live with a rare disease. Put simply, our goal is to provide hope where there is currently none. You are the center of everything we do and we will remain tirelessly at your side, at all times to deliver on our promise of making our products available to ‘every patient, everywhere’.


    FIRST Skin Foundation for Ichthyosis and related skin types

    Supports patients and families affected by Ichthyosis.

    Toll free: 800-545-3286

    NORD National Organization for Rare Disorders

    NORD is leading the fight to improve the lives of patients with rare diseases.

    Genetic and Rare Diseases (GARD) Information Center

    GARD is a program of the National Institutes of Health (NIH) that provides free access to reliable, easy to understand information about genetic and rare diseases.

    Toll free: 888-205-2311

    Molecular Diagnostic Testing for Netherton Syndrome

    Gabriele Richard, MD Associate Professor, Department of Dermatology and Cutaneous Biology and Department of Medicine / Division of Medical Genetics Thomas Jefferson University

    Ichthyosis Support Group

    Committed to the ongoing provision of an information network and support structure for individuals and families affected by ichthyosis.

    Clinical Trials

    Quoin Pharmaceuticals is conducting two concurrent clinical studies to evaluate QRX003 for the treatment of Netherton Syndrome.

    The first is a randomized, double blinded, vehicle-controlled study being conducted under a U.S. Investigational New Drug (IND) application that is assessing two different doses of QRX003 topical lotion versus a placebo lotion in 18 Netherton Syndrome patients. The test materials will be applied once daily over a twelve-week period, to pre-designated areas of the patient’s body. Based on discussions with the U.S. Food and Drug Administration (FDA), a number of different clinical endpoints will be assessed in the study.

    The second study will evaluate the safety and efficacy of the QRX003 as adjuvant treatment with a systemic biologic for the subset of Netherton Syndrome patients who are currently receiving off-label, systemic therapy for symptomatic relief.

    In this multicenter, open label study QRX003 is applied once daily over a twelve-week period, to pre-designated areas of the patient’s body. A number of different clinical endpoints will be assessed in the study.

    For more information about Quoin’s clinical trials, please visit https://www.nethertonsyndromeclinicaltrials.com/.

    For more information about current clinical trials, please visit www.clinicaltrials.gov.