By M. Marin
READ THE FULL QNRX RESEARCH REPORT
Positive interim clinical data
Quoin Pharmaceuticals (NASDAQ:QNRX) is a clinical stage, specialty pharmaceutical company that focuses on developing treatments for rare and orphan diseases, initially in the dermatology space. The company is advancing clinical studies to evaluate lead asset QRX003 for the treatment of Netherton Syndrome (NS). Quoin has active NS studies that are being conducted concurrently under an open Investigational New Drug (IND) application with the FDA. The company believes it is the only development company developing treatment for NS that is conducting dual studies concurrently under an open IND application. Two U.S. trials utilize the same investigators and clinical sites, which Quoin expects will result in substantial operational synergies and cost savings. QNRX is also assessing QRX003 in an NS pediatric patient in an Investigator Pediatric NS study, with plans to enroll additional pediatric patients at other sites.
Key takeaways
- QNRX is advancing clinical studies to evaluate lead asset QRX003 for treatment of Netherton Syndrome (NS), a debilitating skin disorder.
- Interim results are positive, with no adverse events reported to-date.
- 4-weeks after QRX003 treatment ended, all benefits of QRX003 treatment were reversed, which QNRX believes indicates the need for continuous treatment.
- Quoin is optimistic that QRX003 can become the 1st approved treatment for NS. As with all of Quoin’s assets, QRX003 is being designed to treat multiple indications.
- To raise awareness of NS, QNRX has launched the NETHERTON NOW campaign.
Quoin reported positive early results from its current study of QRX003 for pediatric NS patients last week. The company noted that dramatic improvement was observed in the patient following treatment, with what it termed “clear visual evidence of skin healing” after two weeks of whole-body application of QRX003. Moreover, no adverse events have been reported to-date.
Following QRX003 treatment, the patient has discontinued alternative NS medications used previously, including all antihistamines, glucocorticoids and antivirals and also has not needed antibiotics. The company also noted that for the first time ever, the pediatric patient is not having any sleep disturbances.
The clinical assessment of QRX003 in a pediatric patient is being led by Dr. Alan Irvine, a professor of dermatology at Trinity College Dublin and a Consultant Dermatologist at Children’s Health Ireland. Dr. Irvine is also the president of the International Eczema Council and an elected member of the Royal Irish Academy. He has authored more than 250 peer-reviewed papers published in medical journals. QNRX expects to broaden the scope of this clinical assessment to include three additional pediatric patients in Spain. Following a recent share offering, it has extended its cash runway to advance clinical studies.
Treating pediatric patients is important, as early treatment is critical to managing the disease. The company also commenced measures to expand clinical activities on QRX003 internationally. Separately, management raised its stake in QNRX shares, which we view as a positive indicator of its optimism about the company’s prospects. Moreover, according to QNRX, an estimated up to 20% of infants born with NS do not survive.
The concurrent open-label study clinical studies have also yielded positive interim results. The results also follow the positive clinical evidence the company had announced earlier regarding QRX003’s potential efficacy in treating NS patients. Quoin believes the data thus far from its ongoing open-label study has shown evidence that QRX003 can contribute to improvement across all measured clinical endpoints.
After treatment ended, QRX003 benefits reversed… possibly indicating need for chronic treatment
Quoin also noted that four weeks after QRX003 treatment ended, all the positive clinical results seen after 12 weeks of QRX003 treatment and testing were fully reversed. The patient’s disease state reverted to the baseline status seen prior to receiving QRX003 treatment (see table below). The company believes the results indicate that ongoing, chronic treatment with QRX003 is needed to treat NS and control patients’ symptoms.
`Assessing assets for multiple indications can help spread costs over expanded base, broaden target markets upon potential commercialization…
The company’s strategy is to design products to treat multiple indications. Reflecting this strategy, QNRX intends to study QRX003 for several conditions and build a database supporting its efficacy and safety as a treatment for a range of dermatological conditions. The company plans to begin a clinical study of QRX003 in Peeling Skin Syndrome (PSS), a rare autosomal disease that has no approved treatment, to evaluate the safety and efficacy of QRX003 in New Zealand in a pediatric patient with PSS. Most forms of PSS present at birth or during infancy, according to NORD. Symptoms include shedding or peeling of the outermost layer of the skin. Quoin believes it has the opportunity to achieve the first regulatory approval for PSS.
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