QRX003
QRX003 is under development for a number of indications. The product is a topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary delivery technology. Our lead indication is Netherton Syndrome (NS).
Netherton Syndrome (NS)
Netherton Syndrome – a rare, hereditary skin disorder – is an autosomal recessive genetic disease caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5).
The SPINK5 gene, when behaving normally, encodes a protein, called Lympho-Epithelial Kazal Type Related Inhibitor or LEKTI, that serves to regulate the activity of certain skin proteases, called Kallikreins, that are responsible for the normal everyday process of skin shedding. For patients with NS, the LEKTI protein is absent, resulting in unregulated hyperactivity of the Kallikreins which in turn results in uncontrolled desquamation and leads to a highly porous and defective skin barrier.
People with Netherton Syndrome have too few layers of their outer skin (stratum corneum) so the skin does not perform its primary function as a protective barrier. This leads to the increased risk of infections, warts, skin cancer and irritation by allergens and other environmental microorganisms. The skin is prone to scaling and is accompanied by hair anomalies, along with increased susceptibility to atopic eczema. Pruritus (itching) is a major problem as well as elevated IgE antibody levels. Furthermore, because the skin is so compromised, patients experience trans-epidermal water loss (TEWL), and adults can lose up to 2 quarts of water a day.
Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.
QRX003: Tailored Treatment
QRX003, a topical lotion, offers a combination of a broad-spectrum serine protease inhibitor formulated with a proprietary delivery technology.
When applied 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 hyperactivity of the Kallikreins, leading to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier.
In addition to Netherton Syndrome, QRX003 is being developed for the following additional rare disease indications:
Peeling Skin Syndrome
Due to mutations in the TGM5 gene, inherited in an autosomal recessive pattern, this disorder is characterized by the continuous peeling of the top layer of skin. It can appear as abnormal blistering of the skin anywhere on the body, but most often presents on the hands and feet. Symptoms can include pruritus (itching), hyperpigmentation, and redness. Incidence is less than one in one million.
SAM Syndrome
Presents with severe skin dermatitis, multiple allergies and metabolic wasting (SAM). This is a rare life-threatening inherited condition caused by mutations in the desmoglein1 gene.
Ichthyosis
Ichthyosis refers to a group of more than 30 skin diseases. Typically characterized by dry, scaling, red, itchy skin, the symptoms will vary depending on the type of ichthyosis.