Vor Bio Provides Clinical Update Further Validating Approach of Using Shielded Transplants to Deliver Targeted Therapies; Receives Supportive Feedback from FDA Regarding Registrational Trial Design
Vor Bio has released updated clinical data from its Phase 1/2 VBP101 study of trem-cel followed by Mylotarg in relapsed/refractory AML patients. The study showed promising results with 25 patients treated with trem-cel, of which 15 received Mylotarg. Key findings include improved relapse-free survival, successful blood system shielding, and reliable engraftment with 100% of patients achieving primary neutrophil engraftment (median 9.5 days).
The company received supportive feedback from the FDA regarding their registrational trial design, with agreement on study population, control arm, primary endpoint, stratification factors, and statistical design. The FDA acknowledged trem-cel's neutrophil and platelet engraftment capabilities and its safety profile comparable to unedited CD34+ grafts.
Vor Bio ha pubblicato dati clinici aggiornati dal suo studio di Fase 1/2 VBP101 su trem-cel seguito da Mylotarg in pazienti con AML relapsa/refrattaria. Lo studio ha mostrato risultati promettenti con 25 pazienti trattati con trem-cel, di cui 15 hanno ricevuto Mylotarg. I risultati chiave includono un miglioramento della sopravvivenza senza recidive, un'efficace protezione del sistema ematologico e un'ottima innesto con il 100% dei pazienti che ha raggiunto l'innesto primario dei neutrofili (mediana 9,5 giorni).
L'azienda ha ricevuto feedback favorevole dalla FDA riguardo la progettazione del loro studio registrativo, con concordanza sulla popolazione dello studio, braccio di controllo, obiettivo primario, fattori di stratificazione e design statistico. La FDA ha riconosciuto le capacità di innesto dei neutrofili e delle piastrine di trem-cel e il suo profilo di sicurezza comparabile a innesti CD34+ non modificati.
Vor Bio ha publicado datos clínicos actualizados de su estudio de Fase 1/2 VBP101 sobre trem-cel seguido de Mylotarg en pacientes con LMA en recaída/refractaria. El estudio mostró resultados prometedores con 25 pacientes tratados con trem-cel, de los cuales 15 recibieron Mylotarg. Los hallazgos clave incluyen una mejoría en la supervivencia libre de recaídas, un exitoso blindaje del sistema sanguíneo y un buen injerto con el 100% de los pacientes logrando el injerto primario de neutrófilos (mediana de 9.5 días).
La compañía recibió comentarios de apoyo de la FDA respecto al diseño de su ensayo registral, con acuerdo sobre la población del estudio, el grupo de control, el punto final primario, los factores de estratificación y el diseño estadístico. La FDA reconoció las capacidades de injerto de neutrófilos y plaquetas de trem-cel y su perfil de seguridad comparable a injertos CD34+ no editados.
Vor Bio는 재발/내성 AML 환자에서 trem-cel과 Mylotarg를 이용한 1/2상 VBP101 연구에서 업데이트된 임상 데이터를 발표했습니다. 이 연구는 25명의 환자를 대상으로 한 trem-cel 치료에서 유망한 결과를 보여주었으며, 이중 15명이 Mylotarg를 받았습니다. 주요 발견 사항으로는 재발 없는 생존율 향상, 혈액계 보호 성공, 100% 환자가 1차 neutrophil이식에 성공했으며 (중앙값 9.5일) 신뢰성 있는 이식이 포함됩니다.
회사는 그들의 등록 시험 설계에 대해 FDA로부터 지원적인 피드백을 받았으며, 연구 인구, 대조군, 주요 종결점, 계층화 요인 및 통계 설계에 대한 동의가 있었습니다. FDA는 trem-cel의 중성구 및 혈소판 이식 능력과 편집되지 않은 CD34+ 이식과 유사한 안전성 프로필을 인정했습니다.
Vor Bio a publié des données cliniques mises à jour de son étude de Phase 1/2 VBP101 sur trem-cel suivi de Mylotarg chez des patients atteints de LMA en rechute/réfractaire. L'étude a montré des résultats prometteurs avec 25 patients traités avec trem-cel, dont 15 ont reçu Mylotarg. Les résultats clés incluent une amélioration de la survie sans rechute, une protection efficace du système sanguin et un bon greffage avec 100% des patients atteignant le greffage primaire des neutrophiles (médiane de 9,5 jours).
La société a reçu des retours favorables de la FDA concernant la conception de leur essai d'enregistrement, avec un accord sur la population d'étude, le bras de contrôle, le critère principal, les facteurs de stratification et la conception statistique. La FDA a reconnu les capacités de greffage des neutrophiles et des plaquettes de trem-cel et son profil de sécurité comparable à celui des greffons CD34+ non modifiés.
Vor Bio hat aktualisierte klinische Daten aus seiner Phase 1/2 VBP101-Studie zu trem-cel gefolgt von Mylotarg bei Patienten mit rezidivierender/refraktärer AML veröffentlicht. Die Studie zeigte vielversprechende Ergebnisse mit 25 Patienten, die mit trem-cel behandelt wurden, von denen 15 Mylotarg erhielten. Zu den wichtigsten Ergebnissen gehören eine verbesserte rezidivfreie Überlebensrate, erfolgreicher Schutz des Blutsystems und eine zuverlässige Einpflanzung, wobei 100% der Patienten das primäre Neutrophileinwachsen erreicht haben (Median 9,5 Tage).
Das Unternehmen erhielt positives Feedback von der FDA zu ihrem Registrierungsstudien-Design, wobei eine Einigung über die Studienpopulation, die Kontrollgruppe, den primären Endpunkt, die Stratifikationsfaktoren und das statistische Design erzielt wurde. Die FDA erkannte die Neutrophil- und Thrombozyteneinpflanzungsfähigkeiten von trem-cel sowie sein Sicherheitsprofil, das mit unmodifizierten CD34+-Transplantaten vergleichbar ist, an.
- Preliminary data shows improved relapse-free survival compared to published groups
- 100% of patients achieved primary neutrophil engraftment
- High CD33 editing efficiency achieved (median 90%)
- FDA provided supportive feedback on registrational trial design
- patient sample size (25 patients total, only 15 received Mylotarg)
- Median follow-up duration of only 7.4 months
Insights
The clinical update from Vor Bio represents a significant milestone in AML treatment. The preliminary data showing improved relapse-free survival with a median follow-up of 7.4 months is particularly noteworthy. The 100% neutrophil engraftment rate with rapid recovery (median 9.5 days) and robust platelet recovery (median 16 days) demonstrates strong efficacy of the trem-cel platform.
The high CD33 editing efficiency (median
The FDA's supportive feedback on the registrational trial design is a important development. Agreement on key elements including study population, control arm, primary endpoint and statistical design significantly de-risks the regulatory pathway. This alignment with the FDA on trial design elements typically streamlines the approval process and reduces the likelihood of major protocol amendments.
The agency's acknowledgment of trem-cel's safety profile being similar to unedited CD34+ grafts is particularly important, as it suggests a favorable view of the platform's safety profile. This regulatory clarity provides a well-defined path forward for Vor Bio's pivotal trial program.
- Preliminary data suggests improved relapse-free survival compared to published groups of acute myeloid leukemia (AML) patients at high risk of relapse post-transplant
- Trem-cel + Mylotarg continue to demonstrate engraftment, shielding, and broadened therapeutic window
- Company has received supportive feedback from the FDA regarding a registrational clinical trial design
CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today announced updated clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™. The data, which was presented in a poster at the American Society of Hematology (ASH) Annual Meeting on Sunday, December 8th, demonstrated durable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of improved relapse free survival compared to published high-risk AML comparators.
“With additional maturity, we are even more encouraged by this data and the potential of offering AML and MDS patients the opportunity to receive post-transplant maintenance therapy while still maintaining healthy blood count levels,” said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer.
The data released today included 25 patients treated with trem-cel of which 15 had received Mylotarg (six at the 2 mg/m2 dose) as of the data cut-off date of November 1, 2024. The data demonstrated:
- Preliminary evidence of improved relapse-free survival (median RFS not reached with median follow-up duration of 7.4 months) compared to published groups of AML patients at high risk of relapse post hematopoietic stem cell transplant (HCT)1.
- Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2.
- Broadened therapeutic index for Mylotarg when administered after trem-cel.
- Reliable engraftment, with
100% of patients achieving primary neutrophil engraftment (median 9.5 days), robust platelet recovery (median 16 days), and full myeloid donor chimerism at Day 28. - Trem-cel continues to be manufactured with high CD33 editing efficiency (median
90% , range 71-94% ).
Company received supportive feedback from the FDA in a Type C meeting
The Company had the opportunity to interact with the FDA regarding data from the trem-cel + Mylotarg study alongside a proposed registrational clinical trial synopsis. The FDA agreed that trem-cel engrafts neutrophils and platelets and has a similar safety profile to unedited CD34+ grafts. In addition, there was agreement with the trem-cel + Mylotarg registrational clinical trial design with respect to study population, control arm, primary endpoint, stratification factors, and statistical design. The Company agreed to provide further updates to the FDA alongside submission of the full clinical trial protocol.
Conference Call & Webcast Information
Vor Bio management, joined by Guenther Koehne, MD, PhD, an investigator on the VBP101 study and Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida, will host a live webcast today at 5:00 AM PT / 8:00 AM ET.
Listeners can register for the webcast via this LINK
Analysts wishing to participate in the Q&A session should use this LINK
A replay of the webcast will be available via the investor section of the Company’s website at www.vorbio.com approximately two hours after the call’s conclusion.
About Vor Bio
Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, including potential improvements in relapse-free survival, the timing of initiation of clinical trials, the potential of trem-cel to enable targeted therapies in the post-transplant setting including Mylotarg and CD33-targeted CAR-Ts while maintaining healthy blood count levels and change the standard of care for patients with blood cancers, the safety profile of trem-cel plus Mylotarg, the potential design of a registrational trial for trem-cel and plans for regulatory submissions for trem-cel. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; uncertainties regarding regulatory approvals to conduct trials or to market products; the success of Vor Bio’s in-house manufacturing capabilities and efforts; and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements and Vor Bio’s ability to continue as a going concern. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.
Contact:
Investors & Media
Sarah Spencer
+1 857-242-6076
sspencer@vorbio.com
FAQ
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