Quoin Pharmaceuticals: On Track to Deliver the First FDA-Approved Treatment for Netherton Syndrome
Quoin Pharmaceuticals (QNRX) is developing a potential treatment for a patient population in desperate need. We spoke with CEO, Dr. Michael Myers, to find out more about Netherton Syndrome, a rare and devastating genetic disease.
New York, New York--(Newsfile Corp. - May 17, 2022) - PCG Digital -- People with rare diseases face severe limitations, debilitating discomfort, and a very uncertain future. Children are especially vulnerable: 3 out of 10 children born with a rare disease do not live to see their 5th birthday. Rare diseases can be challenging to diagnose, but even once a diagnosis is reached, effective treatment options can be even more elusive.
Rare diseases are only rare if you don’t live with one
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Specialty pharmaceutical company, Quoin Pharmaceuticals (NASDAQ: QNRX), is relentlessly pursuing the development of therapeutic products for rare and orphan diseases. Quoin's lead product is a potential treatment for Netherton syndrome, a rare and devastating genetic disease that affects the skin, hair, and immune system.
Symptomatic within the first weeks of life, the 1 in 200,000 newborns who are born with the disease experience a lifetime of illness and an enhanced susceptibility to infections, allergies, skin cancer, severe pruritus and other diseases. It is believed that about 6,000-7,000 people in Europe and the US combined suffer from Netherton syndrome. The disease requires lifelong management, with current treatments providing only limited symptomatic relief.
Quoin is now potentially on track to deliver the first FDA approved treatment for this devastating disease. The company's lead candidate, QRX003, is a whole-body, whole life topical treatment that is providing hope to those around the globe suffering from Netherton syndrome.
We spoke with Dr. Michael Myers, co-founder and CEO of Quoin, to find out more about this disease and what drives him to find an effective treatment.
Q. Netherton syndrome is a skin disease that causes dryness and shedding. Is it similar to eczema or psoriasis?
A. Netherton syndrome is most definitely not a skin disorder. It is a debilitating genetic disease. People with Netherton syndrome have too few layers of skin, so the skin cannot perform its primary protective function. It is a devastating disease for patients and their families, requiring lifelong management of painful, scaly, and itchy skin with an increased risk of severe dehydration, infection, allergies and skin cancer.
Q. What have you heard from people with Netherton syndrome?
A. We hear a mix of frustration, desperation, and devastation. Just last week we received an email from parents of a one-month-old baby with NS. She has to be kept in an incubator at 60% humidity because of how severe her dehydration is, and it's unlikely she will survive. Two years ago, the parents lost another child with NS at just nine days old. They are desperate for any sort of treatment that might help.
A dermatologist reached out to us regarding a patient, a woman in her 30s with NS who has struggled terribly her whole life. She's hoping her patient can be involved in our clinical study to give her a chance at living a normal life.
Recently, a father in Germany contacted us because he has a five-year-old daughter with NS who is very ill. He too is hoping his daughter can participate in our clinical study.
Every time we are contacted like this by a parent or a caregiver, it reinforces our determination to progress the development of this product as expeditiously as possible.
Q. What are the current treatments? Can you tell us more about QRX003?
A. There are no approved therapies for Netherton syndrome and current treatment options offer limited benefits, primarily minor symptomatic relief. And even with these treatments, care has to be taken. Emollient moisturizers containing lanolin or petroleum can damage the fragile skin of people with NS because of the friction caused by application and removal. Topical steroids may reduce skin thickness by 70%. Because of their defective skin barrier, people with NS can also develop dangerously high blood levels of the ingredients used in topical treatments. QRX003 is a unique product that targets the cause of the excess shedding that leads to the highly porous skin that Netherton patients have. In addition, if approved, QRX003 may be used on the entire body surface daily to provide a barrier over the skin, limiting the penetration of unwanted environmental agents whilst enabling the architecture of the skin to heal.
Q. When might people with NS be able to use QRX003?
A. Our goal is to obtain the necessary approvals to make our products available to as many people as we can, as quickly as possible. Quoin has received Investigational New Drug clearance from the FDA for QRX003 and will initiate clinical testing in patients in 2Q of 2022. In addition to our planned commercial infrastructure for the US and Europe, we have distribution agreements for QRX003 in almost 60 countries. Many of these regions have early-access programs that could potentially allow QRX003 to be prescribed on a named-patient basis before formal regulatory approval.
Q. What motivates you to work on treatments such as QRX003?
A. People living with Netherton syndrome have been neglected for a long time, waiting and waiting for an effective treatment. The pursuit of unique solutions for rare and orphan diseases has been Quoin's mission right from the very start. Both my co-founder, Denise, and I have seen the impact Netherton syndrome has on patients and their families - and not just physically. There is a heavy emotional price to pay. We at Quoin are 100% dedicated to supporting these underserved people and doing everything we can to develop effective solutions to treat rare diseases that currently have no treatment or cure.
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