QRX003

QRX003 is under development as a potential therapy for the treatment of Netherton Syndrome (NS). The product is a once-daily topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary Invisicare® delivery technology.

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) leading to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma and eczema. Patients also often suffer from severe dehydration, chronic skin inflammation and stunted growth.

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 due to the mutation of the SPINK5 gene. The effect of this absence of the LEKTI protein is unregulated hyperactivity of the Kallikreins which in turn results in uncontrolled desquamation and leads to a highly porous and defective skin barrier.

Netherton Syndrome

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. In infants and children, TEWL can result in a failure to thrive and delayed or stunted growth.

Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments. What is available, such as skin moisturizers, are currently limited to providing symptomatic relief. Due to the highly compromised skin, even the selection of an appropriate moisturizer can be challenging. For example, moisturizers typically used for skin barrier repair, such as those containing lanolin and petrolatum, can cause further skin damage to NS patients due to friction or high shear forces upon application or removal. Topical steroids may have harmful rather than helpful effects and can further reduce the thickness of the skin barrier. Incidences of Cushing’s Syndrome have also been reported from the use of topical steroids. Other standard topical treatments, such as calcineurin inhibitors, can lead to dangerously high systemic blood levels due to the defective skin barrier.

CURRENTLY, THERE IS NO CURE NOR ANY APPROVED PRODUCTS FOR THE TREATMENT OF NETHERTON SYNDROME.

QRX003: Tailored Treatment

QRX003, a topical lotion, offers a combination of a broad-spectrum serine protease inhibitor formulated with the proprietary Invisicare® 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.

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 painless 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.

Palmoplantar Keratoderma

This condition causes thickening of the skin on the hands and feet and can be either inherited or acquired. Patients may experience pain when walking, excessive foot odor and/or sweating, along with reduced sensation of fingers or toes. Incidence is 4.4 cases per 100,000.

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Quoin Pharmaceuticals is an emerging specialty pharmaceutical company dedicated to supporting underserved patients, their families, communities, and care teams around the world. 

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Dr. Michael Myers

Co-Founder Quoin Pharmaceuticals, Inc., Chairman, Chief Executive Officer and Director

Dr. Myers has more than 30 years of industry experience in the drug delivery and specialty pharmaceutical sectors. He has served CEO of Innocoll, Inc. and was responsible for taking that company public in 2014. He has also served as president of the drug delivery division of West Pharmaceutical Services, president of pharmaceutical operations for Fuisz Technologies (Biovail) and has held executive positions in Flamel Technologies and Elan Corporation. Dr. Myers earned his Ph.D. in Chemistry from the University College Cork. Dr. Myers serves on the Board of Directors of Sonoran Biosciences as well as on the Advisory Boards for two Penn State startup companies, Cranial Devices and Gradient T.

Denise Carter

Co-Founder Quoin Pharmaceuticals, Inc., Chief Operating Officer and Director

Ms. Denise Carter has over 30 years of experience in the drug delivery and specialty pharmaceutical industries. Prior to Quoin, Ms. Carter was executive vice president of business development and corporate affairs at Innocoll, Inc., vice president of business development of the drug delivery division of West Pharmaceuticals, and she has held executive positions at Eurand and Fuisz Technologies (Biovail) Ms. Carter earned her MBA from Wharton School of Business, University of Pennsylvania and a B.S. in Chemistry from the College of William and Mary.

Gordon Dunn

Gordon Dunn

Chief Financial Officer

Gordon joined Quoin in November of 2021. He brings over 30 years of finance experience and has served as CFO of both private and publicly traded companies. In addition, Mr. Dunn has deep experience in investment banking and private equity. Over the last five years he has served as Chief Financial Officer for several private companies, most recently with Qured, a UK based healthcare provider, from 2020 to 2021. Mr Dunn also served as CFO of Innocoll Inc. from 2012-2016. Prior to joining Innocoll, Mr. Dunn had 20 years’ experience in investment banking and private equity, including over ten years managing the private equity funds of NewSmith Capital, and nine years at Merrill Lynch where he served as co-head of the European Private Equity Group and Director of Equity Capital Markets. Mr. Dunn received a B.A. from Stanford University and a J.D. from New York University School of Law.

Dennis H. Langer, MD, JD

Director

After graduating from Georgetown School of Medicine and Harvard Law School, Dr. Langer spent over 35 years in the pharmaceutical industry at Eli Lilly, Abbott, Searle, GSK and as President at Dr. Reddy’s, NA. He was a Director at Sirna Therapeutics (acquired by Merck), Ception Therapeutics (acquired by Cephalon), Transkaryotic Therapies (acquired by Shire), Pharmacopeia (acquired by Ligand), Cytogen (acquired by EUSA Pharma) and Delcath Systems. Dr. Langer is a Director at Myriad Genetics, Dicerna Pharmaceuticals and Pernix Therapeutics. He is a Clinical Psychiatry Professor at Georgetown and serves on the Dean’s Advisory Board of Harvard Law School.

Natalie Leong

Director

Ms. Leong has worked across four continents in valuation and transactions in sale, IPO, float, financial modeling, internal controls and raising capital. She has experience analyzing business plans, performing market analyses, preparing financial projections, developing valuation models, advising on equity transactions, mergers, acquisitions and corporate restructurings. Currently, Ms. Leong is Head of Finance for LoanStreet. Previously, she was US lead for the Asset Liability Committee at RBC Capital Markets. At National Australia Bank, Ms. Leong was promoted to VP of Capital Insights. She has an MBA from Wharton, a BCom and a BA from the University of Melbourne.

James Culverwell

Director

Currently, Mr. Culverwell is Chairman of HOX Therapeutics, focusing on prostate cancer research. He is also NED of Safeguard Biosystems, in late stage development of a low cost, high throughput, molecular diagnostic system. Previously, Mr. Culverwell was NED at Atlantic Healthcare, developing treatments for ulcerative colitis. He was NED and Chairman of Audit at Amryt Pharmaceuticals, which focuses on rare diseases including Hypercholesterolemia and Epidermolysis Bullosa. He was also NED at Innocoll AG. Mr. Culverwell’s experience includes Director and Head of European Healthcare and Pharmaceutical Equity Research at Hoare Govett/ABN Amro. And was FVP of Global Healthcare Research at Merrill Lynch/Bank of America.

Joseph Cooper

Director

With 30 years of pharmaceutical industry experience, Mr. Cooper has held roles in operational, corporate development and general management. Currently, he is Chief of Strategy and Corporate Development at Resonea. Formerly, he was President at Boulder Cove and Executive VP of Corporate Development at Medicis Pharmaceutical. Earlier he was with Schein and GD Searle. His leadership experience is in clinical research, product development, supply chain, business development and executive management in a broad range of therapeutic areas including dermatology, aesthetics, allergy, sleep apnea, stroke and orphan drugs. Mr. Cooper currently serves on the board of Sonoran Biosciences, Bioenvision and board observer for several specialty pharmaceutical companies. He serves as a commercial partner of Tech Launch Arizona, the technology advancement arm of the University of Arizona.

Michael Sember

Director

In his 40-year career, Mr. Sember has been COO or CEO of five diverse pharmaceutical companies ranging from drug discovery tool providers, to therapeutically-focused biotechnology companies, to medical devices. For example, at Marion Laboratories (later Marion Merrell Dow) Mr. Sember handled roles including sales, research and R&D program management. As Executive VP of Business Development for Élan Corporation, he was responsible for strategic collaborations, mergers and acquisitions. Mr. Sember has consulted for numerous companies and has extensive public and private board experience. Currently Mr. Sember serves as Chair of the Screening Panel and Board member for the number-one-ranked Desert Angels, a Tucson, AZ-based group of angel investors.

QRX003

A once-daily topical therapy for the treatment of Netherton Syndrome (NS). In NS patients, QRX-003 mimics the activity of the missing LEKTI protein to regulate the shedding of skin. Read more

QRX004

Also formulated with the Invisicare technology, QRX004 is initially being developed as a potential treatment for Recessive Dystrophic Epidermolysis Bullosa ( RDEB).

QRX004 contains two active ingredients. The primary  ingredient induces a read-through of nonsense mutations and leads to creation of robust and sustained type VII collagen. The result is improved wound closure, reduced blistering and stronger skin.

QRX006

Product content to come