Product Candidate: 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.

Product Candidate: QRX008

VLA-4 inhibition for treatment of Scleroderma

Systemic sclerosis (Scleroderma) is a multisystem, autoimmune disorder defined by progressive vascular, inflammatory, and fibrotic dysfunction. It is characterized by an early inflammatory phase, which is followed by progressive fibrosis of varying extent and distribution, resulting in severe dysfunction of involved organs. Interaction of vascular cell adhesion molecule 1 (VCAM-1) with its ligand, very late antigen 4 (VLA-4), is a key process in inflammatory disease. There is an established genetic and clinical link for VCAM-1 in Scleroderma, with VCAM-1 having a genome-wide association with Scleroderma, and Scleroderma patients having elevated VCAM-1 serum levels. VLA-4, an α1β4 integrin, plays a pivotal role in controlling immune cell migration into inflamed tissue as it directly participates in cell arrest under flow. The VCAM-1:VLA-4 interaction therefore makes an attractive target for therapeutic intervention in Scleroderma. A lead series of small molecule inhibitors has been developed to disrupt the VCAM-1:VLA-4 interaction. Proof of concept has been demonstrated with VLA-4 inhibition in the Tsk1/+ mouse model of Scleroderma. Further preclinical assessment is underway for the selection of a lead compound for entry into the clinic.

Product Candidate: QRX009

Topical Rapamycin Formulation for Rare Vascular and Skin Malformations

QRX009 is a proprietary topical formulation of rapamycin (sirolimus) in development as a potential treatment for a group of rare, disfiguring conditions, including Microcystic Lymphatic Malformations (MLM), Venous Malformations (VM), and Cutaneous Angiofibromas, among others. These diseases can lead to significant cosmetic and functional impairment, and there are currently either none or limited FDA-approved topical therapies for these indications.

QRX009 is designed to optimize local skin penetration and deliver rapamycin directly to the affected dermal layers where it can be most effective. Previous clinical observations strongly suggest that limited drug delivery has contributed to underperformance of other topical rapamycin products. QRX009 is intended to address this challenge by optimizing the retention and absorption of the rapamycin directly at the target site. Quoin is currently evaluating several technologies to determine the optimal delivery system.

Quoin plans to submit Investigational New Drug (IND) applications for at least two of these indications, with clinical development expected to commence in 1H 2026.