Azitra Announces First Patient Screened and Scheduled to Enroll in Phase 1b Trial of ATR-12 for Netherton Syndrome
Azitra (NYSE American: AZTR) has screened the first patient for its Phase 1b clinical trial of ATR-12, a potential treatment for Netherton syndrome. The trial aims to enroll 12 adult patients for a 14-day treatment period. Interim safety data is expected in early 2025, with full results anticipated in the second half of 2025.
ATR-12 is a proprietary strain of Staphylococcus epidermidis engineered to express therapeutic levels of an active LEKTI protein subunit. The trial's primary endpoints focus on safety and tolerability, while secondary endpoints assess efficacy signals and biomarkers. Recent preclinical data showed ATR-12's potential to reduce IL-36γ by 93% and significantly decrease protease activity in skin samples.
Azitra (NYSE American: AZTR) ha selezionato il primo paziente per il suo studio clinico di Fase 1b di ATR-12, un potenziale trattamento per la sindrome di Netherton. Lo studio mira a reclutare 12 pazienti adulti per un periodo di trattamento di 14 giorni. I dati di sicurezza provvisori sono attesi per all'inizio del 2025, con i risultati completi previsti nella seconda metà del 2025.
ATR-12 è un ceppo proprietario di Staphylococcus epidermidis progettato per esprimere livelli terapeutici di una subunità proteica attiva LEKTI. Gli obiettivi primari dello studio si concentrano su sicurezza e tollerabilità, mentre gli obiettivi secondari valutano segnali di efficacia e biomarcatori. I recenti dati preclinici hanno mostrato il potenziale di ATR-12 di ridurre IL-36γ del 93% e diminuire significativamente l'attività della proteasi nei campioni di pelle.
Azitra (NYSE American: AZTR) ha seleccionado al primer paciente para su ensayo clínico de Fase 1b de ATR-12, un posible tratamiento para el síndrome de Netherton. El ensayo tiene como objetivo reclutar 12 pacientes adultos para un período de tratamiento de 14 días. Se esperan datos provisionales de seguridad para principios de 2025, con resultados completos anticipados en la segunda mitad de 2025.
ATR-12 es un cepo propietario de Staphylococcus epidermidis diseñado para expresar niveles terapéuticos de una subunidad proteica activa LEKTI. Los objetivos primarios del ensayo se centran en seguridad y tolerabilidad, mientras que los objetivos secundarios evalúan señales de eficacia y biomarcadores. Los datos preclínicos recientes mostraron el potencial de ATR-12 para reducir IL-36γ en un 93% y disminuir significativamente la actividad de proteasas en muestras de piel.
Azitra (NYSE American: AZTR)는 첫 번째 환자를 screening 했다고 ATR-12의 1b상 임상 시험을 발표했다. 이는 Netherton 증후군을 위한 잠재적 치료제이다. 이 시험은 12명의 성인 환자를 14일 치료 기간 동안 등록할 예정이다. 임시 안전 데이터는 2025년 초에 예상되며, 전체 결과는 2025년 하반기에 공개될 예정이다.
ATR-12는 치료 수준의 활성 LEKTI 단위체를 발현하도록 설계된 특허 보유 균주 Staphylococcus epidermidis이다. 시험의 주요 목표는 안전성과 내약성에 중점을 두며, 2차 목표는 효능 신호와 바이오마커를 평가한다. 최근의 전임상 데이터는 ATR-12가 IL-36γ를 93% 감소시키고, 피부 샘플에서 단백분해 효소 활성을 유의미하게 감소시킬 잠재력을 보여주었다.
Azitra (NYSE American: AZTR) a sélectionné le premier patient pour son essai clinique de Phase 1b d'ATR-12, un traitement potentiel pour le syndrome de Netherton. L'essai vise à recruter 12 patients adultes pour une période de traitement de 14 jours. Des données de sécurité provisoires devraient être disponibles début 2025, tandis que les résultats complets sont attendus dans la deuxième moitié de 2025.
ATR-12 est une conçue pour exprimer des niveaux thérapeutiques d'une sous-unité de protéine LEKTI active. Les principaux objectifs de l'essai portent sur la sécurité et la tolérabilité, tandis que les objectifs secondaires évaluent les signaux d'efficacité et les biomarqueurs. Des données précliniques récentes ont montré le potentiel d'ATR-12 pour réduire IL-36γ de 93% et diminuer significativement l'activité des protéases dans des échantillons de peau.
Azitra (NYSE American: AZTR) hat den ersten Patienten ausgewählt für seine Phase 1b-Studie zu ATR-12, einem potenziellen Behandlungsmittel für das Netherton-Syndrom. Die Studie zielt darauf ab, 12 erwachsene Patienten für einen 14-tägigen Behandlungszeitraum zu rekrutieren. Vorläufige Sicherheitsdaten werden für Anfang 2025 erwartet, die vollständigen Ergebnisse sollen in der zweiten Hälfte von 2025 vorliegen.
ATR-12 ist ein proprietärer Stamm von Staphylococcus epidermidis, der so konzipiert ist, dass er therapeutische Mengen einer aktiven LEKTI-Proteineinheit exprimiert. Die primären Endpunkte der Studie konzentrieren sich auf Sicherheit und Verträglichkeit, während die sekundären Endpunkte Effizienzsignale und Biomarker bewerten. Jüngste präklinische Daten zeigten das Potenzial von ATR-12, IL-36γ um 93% zu reduzieren und die Aktivität der Proteasen in Hautproben signifikant zu verringern.
- First patient screened for Phase 1b trial of ATR-12 for Netherton syndrome
- Interim safety data expected in early 2025, full results in second half of 2025
- Preclinical data showed 93% reduction in IL-36γ and significant decrease in protease activity
- ATR-12 produced higher amounts of LEKTI subunit compared to topical application of LEKTI protein alone
- None.
This Phase 1b trial for ATR-12 in Netherton syndrome represents a significant step forward in addressing an unmet medical need. The trial's design, focusing on safety, tolerability and efficacy signals, is important for this novel therapeutic approach. The use of a contralateral design (treatment vs. control on opposite sides of the body) is particularly clever, allowing for direct comparison within each patient.
The preclinical data presented at ASGCT 2024 is promising, showing ATR-12's potential to reduce inflammatory markers and protease activity, key factors in Netherton syndrome pathology. The
However, investors should note that early-stage trials often face challenges in translating preclinical success to human subjects. The small sample size (
From a financial perspective, Azitra's progress with ATR-12 is encouraging for investors. Netherton syndrome, while rare, represents a potentially lucrative market due to the lack of effective treatments. If successful, ATR-12 could become a first-in-class therapy, potentially commanding premium pricing.
However, investors should be aware of the long timeline to potential commercialization. With full Phase 1b results not expected until H2 2025 and likely need for Phase 2 and 3 trials, revenue generation is still years away. This extended timeline means Azitra will likely need additional funding rounds, potentially diluting current shareholders.
The company's focus on precision dermatology and its innovative approach using engineered bacteria could attract partnership interest from larger pharmaceutical companies, potentially providing non-dilutive funding or acquisition opportunities. However, this is speculative and depends on positive clinical results.
Investors should closely monitor cash burn rate and pipeline progress in the coming quarters, as these will be critical factors in Azitra's ability to bring ATR-12 to market without excessive dilution.
ATR-12 is Azitra's lead candidate, a proprietary strain of Staphylococcus epidermidis engineered to express therapeutic levels of an active subunit of the LEKTI protein to treat Netherton syndrome, a chronic genetic skin disease that can be life threatening.
"Enrolling the first patient in our Phase 1b trial of ATR-12 will mark a significant milestone for Azitra and, more importantly, for patients suffering from Netherton syndrome, who currently have limited treatment options," said Travis Whitfill, founder and COO of Azitra. "Our recent preclinical data demonstrate ATR-12's potential to effectively deliver an active LEKTI subunit into the skin and address the underlying mechanisms of Netherton syndrome."
Mary Spellman MD, Azitra’s acting CMO, added, “We are thrilled to begin this clinical trial of ATR-12 in Netherton syndrome patients. These patients suffer from poor quality of life and often debilitating disease, and this trial will inform future studies for the treatment of Netherton syndrome in pediatric patients and for longer treatment durations.”
The Phase 1b trial (NCT06137157) is a multicenter, randomized, double-blind, vehicle-controlled study in approximately 12 adult patients with Netherton syndrome. Patients will be treated with 109 CFU / g twice daily with ATR-12 or its vehicle control on the contralateral side of the body twice daily for 14 days. The primary objective is to assess the safety and tolerability of topical ATR-12 application. Secondary objectives include evaluating efficacy signals (e.g., investigator and patient global assessments) and skin pharmacokinetics of the LEKTI subunit. Additional exploratory objectives include evaluating pharmacodynamic parameters, biomarkers, anti-LEKTI response, and cytokine responses.
The trial design is supported by compelling preclinical data recently presented at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting. Key findings included:
-
Topical application of ATR-12 in preclinical models reduced IL-36γ by
93% compared to skin extracts induced to overexpress IL-36γ - ATR-12 significantly reduced protease activity in skin samples compared to a Netherton syndrome model skin (p<0.01)
- ATR-12 produced higher amounts of LEKTI subunit compared to topical application of LEKTI protein alone (6.0 µg vs. 2.3 µg, respectively, p<0.01) after 24 hours and resulted in deeper skin penetration of LEKTI
About ATR-12
ATR-12 (also known as ATR12-351) is an engineered strain of S. epidermidis that expresses an active subunit of human lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which is missing in patients with Netherton syndrome, a chronic and sometimes fatal disease of the skin estimated to affect approximately one to nine in every 100,000. ATR-12 has been engineered to deliver missing LEKTI protein when applied topically to Netherton syndrome patients. Azitra has an open IND for a Phase 1b clinical trial in adult patients (NCT06137157). Azitra has secured clinical sites and identified Netherton syndrome patients for enrollment in its 12-patient, Phase 1b clinical trial, which will assess safety, tolerability, and efficacy endpoints. Azitra expects to announce initial safety data before year end.
About Azitra, Inc.
Azitra, Inc. is an early-stage clinical biopharmaceutical company focused on developing innovative therapies for precision dermatology using engineered proteins and topical live biotherapeutic products. The Company has built a proprietary platform that includes a microbial library comprised of approximately 1,500 unique bacterial strains that can be screened for unique therapeutic characteristics. The platform is augmented by artificial intelligence and machine learning technology that analyzes, predicts, and helps screen the Company's library of strains for drug like molecules. The Company's initial focus is on the development of genetically engineered strains of Staphylococcus epidermidis, or S. epidermidis, which the Company considers to be an optimal therapeutic candidate species for engineering of dermatologic therapies. For more information, please visit https://azitrainc.com/.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected timing of the presentation of data from the Phase 1b study of ATR-12 and statements about our clinical and preclinical programs, and corporate and clinical/preclinical strategies.
Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that we may fail to successfully complete our Phase 1b trial for ATR-12 and preclinical studies of other product candidates and obtain required approval before commercialization; our product candidates may not be effective; there may be delays in regulatory approval or changes in regulatory framework that are out of our control; our estimation of addressable markets of our product candidates may be inaccurate; we may fail to timely raise additional required funding; more efficient competitors or more effective competing treatment may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier study results may not be predictive of later stage study outcomes; and we are dependent on third-parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks are described in our Form 10-Q filed with the SEC on August 12, 2024. Azitra explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
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Investor Relations Contact:
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staskey@azitrainc.com
Hayden IR
James Carbonara
(646) 755-7412
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Source: Azitra, Inc.
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