US-based Quoin Pharmaceuticals is recruiting a paediatric population into its ongoing Phase II/III trial investigating QRX003 in Netherton syndrome (NS), CEO Dr Michael Myers told Clinical Trials Arena. QRX003 is a broad-spectrum serine protease inhibitor that down-regulates the hyperactivity of skin kallikreins.

The double-blinded trial (NCT05521438) is currently recruiting adults with NS and dosed the first patient in December 2022. Myers explained that the double-blinded study is essentially the first portion of a pivotal trial where the company is testing two different doses of QRX003 versus placebo in three groups of six adult patients. The test material is applied once daily for 12 weeks to pre-designated areas of the body.

After the topline data readout, which is expected later this year, Quoin will start enrolling children into the so-called second portion of the trial. The delayed enrolment of the paediatric population is due to FDA’s requirement to test the investigational treatment in adults first before administering it to children.

The second part of the trial will be a single dose versus placebo with the same format of once-daily application for 12 weeks in two groups of roughly 14 or 15 subjects each, Myers said. “I would imagine that a majority of the subjects would be children, but we won’t be able to say for certain until enrolment is completed.”

The first part of the ongoing trial has five different endpoints, so the topline data readout will guide Quoin on which primary outcome will be the designated measure for the second part, as well as the dosage.

Myers explained that Quoin is the first company to file an Investigational New Drug (IND) application to the FDA for this disease. As such, there were no previously tested endpoints from a randomised clinical trial and the FDA gave the biotech latitude to test five different outcomes without designating one as the primary.

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NS is a rare hereditary disorder characterised by scaling skin, hair anomalies and increased susceptibility to atopic eczema. The NS prognosis in children may be severe, resulting in significant mortality in the first years of life due to fatal complications. Currently, there are no approved therapeutics for NS and patients are prescribed off-label medication.

Quoin is also conducting an open-label trial (NCT05789056) evaluating QRX003 in combination with standard-of-care treatment in NS adult patients. The biotech dosed the first patient last month and expects a topline data readout in H2 2023.

Beyond NS, Quoin plans to investigate QRX003 in other rare skin diseases such as peeling skin syndrome, SAM Syndrome, and palmoplantar keratoderma. “Based on discussions with KOLs and experts in the space, they believe that the mechanism of action of QRX003 could also provide some clinical benefit for these additional indications,” said Myers, adding that the biotech is looking to launch early-stage clinical testing in the second half of 2023.