Strengthening financial position to advance clinical studies…
Quoin Pharmaceuticals (NASDAQ:QNRX) has completed a capital raise to strengthen its financial position in order to continue to move lead asset QRX003 and other candidates in its portfolio forward. As Quoin advances clinical studies to evaluate QRX003 for the treatment of Netherton Syndrome (NS), the offering enhances the company’s financial flexibility to move forward on several fronts. The company has two active NS studies that are being conducted concurrently and recently received FDA clearance to recruit teenage subjects into both clinical studies. In fact, Quoin believes it is the only development company conducting dual NS studies concurrently under an open IND application. Both trials utilize the same investigators and clinical sites, which Quoin expects will result in substantial operational synergies and cost savings.
One study is a randomized, double blinded, placebo -controlled study designed to assess two different doses of QRX003 topical lotion versus a placebo lotion in NS patients. The second study is investigating the safety and efficacy of QRX003 in Netherton Syndrome patients currently receiving off-label systemic therapy, primarily biologic therapy. The trial is a single arm, open label study investigating the safety and efficacy of QRX003 in these Netherton Syndrome patients, who will continue to receive the treatment throughout the trial. This study is not placebo-controlled. All the patients participating in the trial will be tested with a 4% dose of QRX003 applied daily to pre-designated areas of the patient’s body over 12-weeks.
Quoin has announced positive preliminary clinical data from testing. Five of six subjects evaluated had negligible or absent pruritus (itch) following treatment with QRX003, which represents a substantial improvement for these patients compared to before the study even though all of the subjects enrolled have received off-label systemic treatment for at least one year and/or multiple years. In terms of the Investigator assessed skin scoring system, all six subjects showed improvement. Of the six, three exhibited improvement throughout the study and three at various times over the course of the study. Moreover, all six subjects had positive impressions of QRX003 on multiple metrics that were assessed.
The company also implemented several protocol amendments to its clinical program to optimize its clinical effort. The company believes these protocol modifications could potentially result in accelerating and/or expanding regulatory approvals for QRX003. Modifications included eliminating the lower 2% dose in the double blinded study based on the clean safety profile observed for the higher 4% dose across both studies and changing the dosing frequency to twice-daily from once-daily, plus increasing the number of subjects in both studies.
… As recent FDA clearance expected to widen patient population eligible to participate in ongoing NS clinical studies…
Moreover, this week the company received FDA clearance to recruit patients aged 14 years and older into the two ongoing QRX003 clinical trials. The company believes this represents the first time that non-adult Netherton subjects will be tested in clinical studies conducted under an open-IND. FDA guidelines for testing non-adult patients are relatively strict. They indicate that developmental treatments should first be studied in adults before being assessed in pediatric populations, defined for medical purposes as patients up to the age of 18 to 21, depending on the trial.
Treating pediatric patients is important for a variety of reasons, including that early diagnosis and treatment are important to managing the disease. Moreover, according to NIH, life-threatening complications such as severe cutaneous infections up to sepsis and hypernatremic dehydration “are frequent in infancy.” Quoin notes that newborns with NS have skin that is “red and scaly all over” and “trouble gaining weight in infancy and childhood is common and can be severe.”
The company believes this FDA clearance is a milestone. It will enable Quoin to recruit teenage patients who are currently receiving off-label systemic therapy to participate in the open-label study and those who are not receiving such therapy are not eligible to participate in Quoin’s ongoing placebo controlled blinded study. The company is optimistic that this will not only broaden the pool of people eligible to participate in the studies and therefore facilitate recruitment efforts, but also potentially contribute to the growing database to present for potential regulatory approval; QRX003 is being tested both as monotherapy and in conjunction with off-label treatments. Moreover, given the numbers of younger patients with NS, this could also help shorten the timeline to finding a strong treatment option for this younger patient population.
…While company expands / strengthens its IP position …
Separately, to strengthen its IP position in NS and other orphan / rare disease spaces as it pursues R&D efforts of its assets for multiple indications, Quoin recently filed U.S. and International patent applications for a novel combination product as a potential treatment of NS. This new product combines a broad spectrum serine protease inhibitor with an anti-inflammatory agent in a proprietary topical formulation. Quoin expects that if granted, patents from these applications will extend to at least 2044.
There are no currently approved therapeutic treatments for NS and Quoin believes QRX003 could be the first to receive regulatory approval. Currently, some NS patients are being treated off-label with systemic biologics, including systemic therapy, that provide some symptomatic relief but do not address all symptoms or the underlying cause of NS. The company is optimistic that if the studies are successful, QRX003 for NS could potentially become the standard of care for NS patients. Moreover, Quoin has also started to assess several other potential studies internationally for QRX003 in NS and has assets in its pipeline to treat other rare and orphan diseases, as well.
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