Azitra, Inc. (NYSE American: AZTR) has announced significant advancements in its clinical pipeline, particularly for ATR-12 in Netherton syndrome and ATR-04 in EGFR inhibitor (EGFRi)-associated rash. The company reported these updates alongside its Q3 2024 financial results.
ATR-12 for Netherton Syndrome
Azitra has dosed the first patient in its Phase 1b clinical trial (NCT06137157) evaluating ATR-12 for Netherton syndrome, a rare genetic disorder characterized by severe skin inflammation and barrier dysfunction. ATR-12 is an engineered strain of Staphylococcus epidermidis designed to deliver a fragment of the missing lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein directly to the skin. This Phase 1b trial is an open-label study actively recruiting adult Netherton syndrome patients to assess the safety, tolerability, and efficacy of ATR-12.
Topline data from the Phase 1b trial with ATR-12 in Netherton syndrome patients is expected by the end of 2025, with initial safety data from the first set of patients anticipated in Q1 2025. Netherton syndrome affects approximately 20,000 patients globally, representing a significant unmet medical need.
ATR-04 for EGFRi-Associated Rash
The FDA has granted Fast Track designation to ATR-04 for the treatment of EGFRi-associated rash, a common and debilitating side effect of EGFR inhibitor therapies used in cancer treatment. This designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and fill unmet medical needs. ATR-04, a live biotherapeutic product, is a naturally derived S. epidermidis strain engineered for safety and controlled growth. It addresses EGFRi rash by targeting the suppression of skin immunity and inflammation often associated with elevated levels of IL-36γ and S. aureus.
Azitra has submitted an Investigational New Drug (IND) application to the FDA and received IND clearance for ATR-04. A Phase 1/2 clinical study is planned to commence in early 2025, with the first patient expected to be dosed in Q1 2025. The company presented positive preclinical data and the clinical plan for the Phase 1/2 study at the European Academy of Dermatology and Venereology (EADV) Congress. Approximately 150,000 patients in the United States suffer from EGFRi rash, highlighting the potential impact of ATR-04.
Financial Update
In Q3 2024, Azitra completed a follow-on offering, securing $10 million in gross proceeds. As of September 30, 2024, the company reported cash and cash equivalents of $7.3 million. Research and Development expenses for the quarter were $1.0 million, compared to $0.5 million for the same period in 2023. The net loss for the quarter was $1.0 million, a decrease from the $1.9 million loss reported in Q3 2023.
Management Commentary
"Azitra achieved a number of significant milestones in the third quarter of 2024 to propel our pipeline forward, highlighted by the dosing of the first patient with ATR-12 in our ongoing Netherton syndrome trial," said Francisco Salva, CEO of Azitra. "With a clear roadmap, strong execution on two programs, and a dedicated team, Azitra is well-positioned to execute these milestones, deliver transformative therapies to patients in need, and ultimately maximize shareholder value."
