NEW YORK – Azitra on Tuesday said the first patient with Netherton syndrome is scheduled to receive its investigational topical treatment for the condition this month in a Phase Ib trial.
ATR-12 is Branford, Connecticut-based Azitra's lead candidate for Netherton syndrome, a rare, chronic skin disorder caused by mutations in the LEKTI gene that result in dysfunctional LEKT1 proteins. ATR-12 is a proprietary strain of Staphylococcus epidermidis that's engineered to express therapeutic levels of an active subunit of LEKTI.
In the clinical trial, Azitra plans to enroll about 12 adult patients with Netherton syndrome who will receive twice-daily treatment with ATR-12 for two weeks on one side of their body and a control agent on the other side of their body. Investigators will evaluate the drug's safety and tolerability, as well as secondary and exploratory endpoints assessing efficacy and biomarkers.
"These patients [with Netherton syndrome] suffer from poor quality of life and often debilitating disease," Mary Spellman, acting chief medical officer at Azitra, said in a statement. "This trial will inform future studies for the treatment of Netherton syndrome in pediatric patients and for longer treatment durations."
Azitra expects to report interim safety data from this clinical trial early next year and full results in the second half of 2025.