Azitra Screens First Patient for Phase 1b ATR-12 Trial for Netherton Syndrome

Azitra, Inc., a precision dermatology company, has announced a significant milestone in its clinical research by screening the first patient in its Phase 1b clinical trial for ATR-12, a treatment for Netherton syndrome. The patient will commence treatment by the end of the month. This study aims to enroll approximately 12 adult patients who will receive treatment twice daily for 14 days. The primary focus of the study is to evaluate the safety and tolerability of the treatment, while secondary and exploratory endpoints will assess efficacy signals and relevant biomarkers. Interim safety data is expected to be available in early 2025, with full results anticipated in the latter half of the year.

ATR-12, Azitra’s lead candidate, is a specially engineered strain of Staphylococcus epidermidis designed to express therapeutic levels of an active subunit of the LEKTI protein. This protein is crucial for patients suffering from Netherton syndrome, a severe and chronic genetic skin disease. The disease can significantly impair the quality of life and can even be life-threatening.

Travis Whitfill, founder and COO of Azitra, emphasized the importance of the trial, stating, “Enrolling the first patient in our Phase 1b trial of ATR-12 is a major achievement for Azitra and offers hope for patients with Netherton syndrome, who have had very limited treatment options.” He noted that preclinical data had shown ATR-12’s potential to deliver the active LEKTI subunit effectively into the skin, addressing the underlying mechanisms of the disease.

Mary Spellman, MD, acting Chief Medical Officer of Azitra, expressed her excitement about the trial's commencement. She stated, “We are thrilled to begin this clinical trial of ATR-12 in Netherton syndrome patients. These patients endure a poor quality of life due to their debilitating disease, and this trial will provide essential insights for future studies, including those involving pediatric patients and extended treatment durations.”

The Phase 1b trial, registered under NCT06137157, is a multicenter, randomized, double-blind, vehicle-controlled study involving approximately 12 adult Netherton syndrome patients. The treatment involves applying 10^9 CFU/g of ATR-12 or its vehicle control on the opposite side of the body twice daily for 14 days. The main objective is to assess the safety and tolerability of the topical application of ATR-12. Additionally, the study will evaluate efficacy signals through global assessments conducted by both investigators and patients, as well as skin pharmacokinetics of the LEKTI subunit. Exploratory objectives will examine pharmacodynamic parameters, biomarkers, anti-LEKTI response, and cytokine responses.

The trial's design is bolstered by compelling preclinical data presented at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting. Key findings from these presentations supported the potential efficacy of ATR-12.

ATR-12, also known as ATR12-351, is an engineered strain of S. epidermidis that expresses an active subunit of the human lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which is deficient in patients with Netherton syndrome. This chronic, and sometimes fatal, skin disease affects approximately one to nine individuals per 100,000 people. ATR-12 aims to deliver the missing LEKTI protein through topical application. Azitra has secured clinical sites and identified patients with Netherton syndrome for this 12-patient Phase 1b trial, which will assess safety, tolerability, and efficacy endpoints. Preliminary safety data is expected to be released by the end of the year.

Azitra, Inc., an early-stage clinical biopharmaceutical company, specializes in developing advanced therapies for precision dermatology using engineered proteins and live biotherapeutic products. The company’s proprietary platform includes a microbial library of around 1,500 unique bacterial strains screened for therapeutic properties, supported by AI and machine learning technologies. Azitra’s initial focus is on developing genetically engineered strains of S. epidermidis, considered optimal for dermatologic therapies.