Hemogenyx Pharmaceuticals PLC Announces Third Patient Safety
Hemogenyx Pharmaceuticals (HOPHF) has announced successful safety evaluation of the third patient treated with HG-CT-1, their proprietary CAR-T therapy for relapsed/refractory acute myeloid leukemia (R/R AML) in adults. The Phase I clinical trial has shown promising early results, with the treatment being well-tolerated and meeting predefined safety criteria.
Early efficacy signals were observed as AML cells became undetectable using standard testing methods. The Data Safety Monitoring Board (DSMB) will review safety data from the first three patients treated at the lowest dose to determine if dose escalation can proceed. The trial includes secondary endpoints focusing on efficacy, overall survival, progression-free survival, and duration of response.
Hemogenyx Pharmaceuticals (HOPHF) ha annunciato una valutazione di sicurezza riuscita del terzo paziente trattato con HG-CT-1, la loro terapia CAR-T proprietaria per la leucemia mieloide acuta (LMA) in recidiva/refrattaria negli adulti. Lo studio clinico di fase I ha mostrato risultati iniziali promettenti, con il trattamento ben tollerato e conforme ai criteri di sicurezza predeterminati.
Segnali di efficacia precoce si sono osservati quando le cellule AML sono diventate non rilevabili secondo i metodi di test standard. Il Data Safety Monitoring Board (DSMB) esaminerà i dati di sicurezza dei primi tre pazienti trattati alla dose più bassa per determinare se è possibile procedere con l'aumento della dose. Lo studio include endpoint secondari focalizzati sull’efficacia, sulla sopravvivenza globale, sulla sopravvivenza libera da progressione e sulla durata della risposta.
Hemogenyx Pharmaceuticals (HOPHF) ha anunciado la exitosa evaluación de seguridad del tercer paciente tratado con HG-CT-1, su terapia CAR-T propietaria para leucemia mieloide aguda (LMA) en recaída/refractaria en adultos. El ensayo clínico de fase I ha mostrado resultados iniciales prometedores, con el tratamiento bien tolerado y cumpliendo criterios de seguridad predeterminados.
Se observaron señales tempranas de eficacia al hacerse las células AML indetectables mediante métodos de prueba estándar. La Junta de Monitoreo de Seguridad de Datos (DSMB) revisará los datos de seguridad de los primeros tres pacientes tratados en la dosis más baja para determinar si se puede proceder con el escalado de dosis. El ensayo incluye endpoints secundarios centrados en la eficacia, la supervivencia global, la supervivencia libre de progresión y la duración de la respuesta.
Hemogenyx Pharmaceuticals (HOPHF)가 성인에서 재발/난치성 급성 골수성 백혈병(AML)을 대상으로 한 자사 CAR-T 요법 HG-CT-1으로 치료받은 세 번째 환자의 안전성 평가가 성공적으로 끝났다고 발표했습니다. 1상 임상시험은 초기 결과가 유망하며, 치료가 잘 견디고 미리 정의된 안전 기준을 충족했음을 보여주었습니다.
초기 효능 신호는 AML 세포가 검출 불가하게 되었음을 표준 검사 방법으로 확인했습니다. 데이터 안전성 모니터링 위원회(DSMB)는 최저 용량으로 치료된 처음 세 명의 안전 데이터를 검토하여 용량 증가가 진행될 수 있는지 결정할 예정입니다. 이 임상시험은 효과성, 전체 생존, 무진행 생존, 반응 지속 기간에 중점을 둔 2차 종단점을 포함합니다.
Hemogenyx Pharmaceuticals (HOPHF) a annoncé l’évaluation de sécurité réussie du troisième patient traité par HG-CT-1, leur thérapie CAR-T propriétaire pour les leucémies aiguës myéloïdes (LAM) en rechute/réfractaire chez l’adulte. L’essai clinique de phase I a montré des résultats préliminaires prometteurs, le traitement étant bien toléré et répondant aux critères de sécurité prédéfinis.
Des signaux précoces d’efficacité ont été observés lorsque les cellules AML sont devenues indétectables selon les méthodes de test standard. Le Data Safety Monitoring Board (DSMB) examinera les données de sécurité des trois premiers patients traités à la dose la plus basse pour déterminer si une escalade posologique peut être envisagée. L’essai comprend des critères secondaires axés sur l’efficacité, la survie globale, la survie sans progression et la durée de la réponse.
Hemogenyx Pharmaceuticals (HOPHF) hat die erfolgreiche Sicherheitsbewertung des dritten Patienten gemeldet, der mit HG-CT-1, ihrer proprietären CAR-T-Therapie für erwachsene Patienten mit rezidivierender/refraktärer akuter myeloischer Leukämie (R/R AML), behandelt wurde. Die Phase-I-Studie zeigte vielversprechende erste Ergebnisse, die Behandlung wurde gut toleriert und erfüllte die vordefinierten Sicherheitskriterien.
Frühe Wirksamkeitssignale wurden beobachtet, als AML-Zellen durch Standardtests unauffindbar wurden. Das Data Safety Monitoring Board (DSMB) wird die Sicherheitsdaten der ersten drei Patienten, die bei der niedrigsten Dosis behandelt wurden, prüfen, um festzustellen, ob eine Dosiserhöhung fortgesetzt werden kann. Die Studie umfasst sekundäre Endpunkte mit Fokus auf Wirksamkeit, Gesamtüberleben, progressionsfreies Überleben und Dauer der Ansprechrate.
Hemogenyx Pharmaceuticals (HOPHF) أعلنت عن نجاح تقييم السلامة للمريض الثالث الذي عولج بـ HG-CT-1، علاج CAR-T المملوك لها لسرطان الدم اللمفاوي الحاد المتكرر/المقاوم لدى البالغين. أظهرت تجربة المرحلة I نتائج مبكرة واعدة، مع تحمل العلاج جيداً والالتزام بمعايير السلامة المحددة مسبقاً.
لوحظت إشارات مبكرة للفعالية عندما أصبحت خلايا AML غير قابلة للكشف باستخدام طرق الاختبار القياسية. ستراجع لجنة مراقبة سلامة البيانات (DSMB) بيانات السلامة للمرضى الثلاثة الأوائل الذين عولجوا بأقل جرعة لتحديد ما إذا كان يمكن المضي في زيادة الجرعة. تضم Trial endpoints ثانوية تركز على الفعالية، والبقاء على قيد الحياة بشكل عام، والبقاء دون تقدم، ومدة الاستجابة.
Hemogenyx Pharmaceuticals (HOPHF) 已宣布对其用于成人复发/难治性急性髓系白血病（R/R AML）的专有CAR-T治疗 HG-CT-1 第三名患者的安全性评估取得成功。该I期临床试验显示初步结果令人乐观，治疗耐受良好，符合事先设定的安全标准。
初步有效性信号显示为使用标准检测方法后，AML细胞变为不可检测。数据安全监测委员会（DSMB）将评估在最低剂量下治疗的前3位患者的安全数据，以决定是否可以进行剂量递增。试验还设有聚焦于疗效、总体生存、无进展生存及反应持续时间的次要终点。
- Treatment was well-tolerated and met safety criteria in third patient
- Early efficacy signals showed AML cells became undetectable
- Trial progressing as planned with potential for dose escalation
- Currently only testing lowest dose level
- Long-term efficacy and durability data still pending
Third Patient Treated with HG-CT-1 CAR-T Therapy Successfully Passes Initial Safety Evaluation
LONDON, UNITED KINGDOM / ACCESS Newswire / September 17, 2025 / Hemogenyx Pharmaceuticals plc is pleased to announce that the third patient has been successfully treated in the ongoing Phase I clinical trial of HG-CT-1, the Company's proprietary Chimeric Antigen Receptor T-cell (CAR-T) therapy for relapsed/refractory acute myeloid leukemia (R/R AML) in adults.
The treatment was well tolerated and met the trial's predefined initial safety criteria. Importantly, early indications of clinical efficacy have been observed. Preliminary assessment shows that original AML cells were not detectable in the patient using standard testing methods. The patient will continue to be monitored in line with the FDA-approved trial protocol to evaluate the achievement of the study's secondary endpoints described below.
Safety data from the first three patients treated at the lowest dose of HG-CT-1 will be submitted to an independent Data Safety Monitoring Board (DSMB) for review. The DSMB will determine whether dose escalation to the next level may proceed.
The Phase I trial is a dose-escalation study designed to evaluate the safety and tolerability of HG-CT-1. In addition to safety, the trial includes several key secondary endpoints:
Assessing the efficacy of HG-CT-1 based on AML-specific response criteria
Evaluating overall survival
Measuring progression-free survival
Determining duration of response in patients demonstrating clinical benefit
Data related to these secondary endpoints, including efficacy, durability, and overall clinical outcomes, will be collected over time through continued follow-up of the treated patient. These secondary endpoints are critical for assessing the potential clinical impact of HG-CT-1 in a patient population with limited remaining treatment options.
Further updates will be provided as the trial progresses.
Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented:
"The successful treatment of the third patient marks another important milestone for Hemogenyx Pharmaceuticals and for patients battling relapsed or refractory AML. We are encouraged by the favorable safety profile observed to date, together with the early signals of efficacy. These results reinforce the promise of HG-CT-1 as a potential new therapy for one of the most aggressive and intractable forms of leukemia. We remain committed to advancing the clinical development of this therapy to address a critical unmet medical need, while also creating long-term value for our shareholders."
Market Abuse Regulation (MAR) Disclosure
Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement.
Enquiries:
Hemogenyx Pharmaceuticals plc | |
Dr Vladislav Sandler, Chief Executive Officer & Co-Founder | |
Peter Redmond, Director | |
SP Angel Corporate Finance LLP | Tel: +44 (0)20 3470 0470 |
Matthew Johnson, Vadim Alexandre, Adam Cowl | |
Peterhouse Capital Limited | Tel: +44 (0)20 7469 0930 |
Lucy Williams, Duncan Vasey, Charles Goodfellow | |
About Hemogenyx Pharmaceuticals plc
Hemogenyx Pharmaceuticals is a publicly traded company (LSE:HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City.
The Company is a clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as platform technologies that it uses as engines for novel product development.
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SOURCE: Hemogenyx Pharmaceuticals PLC
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