Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual Meeting
Editas Medicine (NASDAQ: EDIT) presented updated clinical data from the Phase 1/2/3 RUBY trial of reni-cel in 28 patients with severe sickle cell disease (SCD). The trial demonstrated positive results with 27 of 28 patients remaining free of vaso-occlusive events post-treatment. Key findings include early normalization of total hemoglobin from 9.8 g/dL at baseline to 13.8 g/dL at Month 6, and sustained fetal hemoglobin levels above 40%.
The treatment was well-tolerated with a safety profile consistent with myeloablative busulfan conditioning. Patients showed successful engraftment with median times of 23 days for neutrophil and 25 days for platelet engraftment. Only two serious adverse events possibly related to reni-cel were reported. The median follow-up was 9.5 months, with 11 patients having more than one year of follow-up.
Editas Medicine (NASDAQ: EDIT) ha presentato dati clinici aggiornati dallo studio RUBY di Fase 1/2/3 riguardante reni-cel in 28 pazienti affetti da sickle cell disease (SCD) severa. Lo studio ha mostrato risultati positivi, con 27 pazienti su 28 che sono rimasti liberi da eventi vaso-occlusivi dopo il trattamento. Risultati chiave includono la normalizzazione precoce dell'emoglobina totale, che è passata da 9,8 g/dL al baseline a 13,8 g/dL dopo 6 mesi, e livelli sostenuti di emoglobina fetale superiori al 40%.
Il trattamento è stato ben tollerato, con un profilo di sicurezza coerente con il condizionamento mieloablativo a base di busulfano. I pazienti hanno mostrato un innesto di successo, con tempi mediani di 23 giorni per l'innesto dei neutrofili e 25 giorni per l'innesto delle piastrine. Sono stati riportati solo due eventi avversi seri possibilmente correlati a reni-cel. Il follow-up mediano è stato di 9,5 mesi, con 11 pazienti che hanno avuto più di un anno di follow-up.
Editas Medicine (NASDAQ: EDIT) presentó datos clínicos actualizados del ensayo RUBY de Fase 1/2/3 de reni-cel en 28 pacientes con enfermedad de células falciformes (SCD) severa. El ensayo mostró resultados positivos con 27 de 28 pacientes libres de eventos vaso-oclusivos después del tratamiento. Hallazgos clave incluyen una normalización temprana de la hemoglobina total de 9.8 g/dL en el inicio a 13.8 g/dL al mes 6, y niveles sostenidos de hemoglobina fetal por encima del 40%.
El tratamiento fue bien tolerado, con un perfil de seguridad consistente con la terapia mieloablativa con busulfano. Los pacientes mostraron un injerto exitoso con tiempos medianos de 23 días para el injerto de neutrófilos y 25 días para el injerto de plaquetas. Solo se informaron dos eventos adversos graves posiblemente relacionados con reni-cel. El seguimiento mediano fue de 9.5 meses, con 11 pacientes que tuvieron más de un año de seguimiento.
Editas Medicine (NASDAQ: EDIT)는 중증 겸상세포병(SCD) 환자 28명을 대상으로 하는 1/2/3상 RUBY 시험의 업데이트된 임상 데이터를 발표했습니다. 이 시험은 치료 후 28명의 환자 중 27명이 혈관폐쇄 사건에서 자유로웠던 긍정적인 결과를 보여주었습니다. 주요 발견으로는 기초에서 9.8 g/dL에서 6개월 후 13.8 g/dL로의 총 헤모글로빈의 조기 정상화와 40% 이상의 지속적인 태아 헤모글로빈 수치가 포함됩니다.
치료는 잘 견딘 것으로 나타났으며, 부술파나를 이용한 골수억제와 일관된 안전성 프로필을 가지고 있었습니다. 환자들은 중립구의 이식 평균 23일 및 혈소판의 이식 평균 25일로 성공적인 이식을 보였습니다. reni-cel과 possibly 관련된 serious adverse events는 단 2건만 보고되었습니다. 평균 추적 기간은 9.5개월이었으며, 11명의 환자는 1년 이상의 추적 관찰을 받았습니다.
Editas Medicine (NASDAQ: EDIT) a présenté des données cliniques actualisées de l'essai RUBY de Phase 1/2/3 concernant le reni-cel chez 28 patients atteints de drépanocytose sévère (SCD). L'essai a démontré des résultats positifs, 27 des 28 patients étant restés libres d'événements vaso-occlusifs après le traitement. Résultats clés incluent une normalisation précoce de l'hémoglobine totale passant de 9,8 g/dL au départ à 13,8 g/dL au mois 6, ainsi que des niveaux soutenus d'hémoglobine fœtale au-dessus de 40%.
Le traitement a été bien toléré, avec un profil de sécurité cohérent avec le conditionnement myéloablatif au busulfane. Les patients ont montré un engraftement réussi, avec des temps médians de 23 jours pour l'engraftement des neutrophiles et de 25 jours pour l'engraftement des plaquettes. Seuls deux événements indésirables graves, possiblement liés au reni-cel, ont été rapportés. La durée médiane de suivi était de 9,5 mois, avec 11 patients ayant eu plus d'un an de suivi.
Editas Medicine (NASDAQ: EDIT) präsentierte aktualisierte klinische Daten aus der Phase 1/2/3 RUBY-Studie zu reni-cel bei 28 Patienten mit schwerer Sichelzellenerkrankung (SCD). Die Studie zeigte positive Ergebnisse, da 27 von 28 Patienten nach der Behandlung frei von vaso-okklusiven Ereignissen blieben. Wichtige Ergebnisse umfassen eine frühe Normalisierung des Gesamthämoglobins von 9,8 g/dL beim Ausgangswert auf 13,8 g/dL nach 6 Monaten sowie nachhaltige Fetalhämoglobinwerte über 40%.
Die Behandlung wurde gut vertragen, mit einem Sicherheitsprofil, das mit der myeloablativen Busulfan-Vorbehandlung übereinstimmt. Die Patienten zeigten ein erfolgreiches Engraftment mit medianen Zeiten von 23 Tagen für das Neutrophilengraft und 25 Tagen für das Plättchenengraft. Es wurden nur zwei schwerwiegende unerwünschte Ereignisse berichtet, die möglicherweise mit reni-cel zusammenhängen. Die mediane Nachbeobachtungszeit betrug 9,5 Monate, wobei 11 Patienten mehr als ein Jahr Nachbeobachtung hatten.
- 27 out of 28 patients remained free of vaso-occlusive events post-treatment
- Significant hemoglobin improvement from 9.8 g/dL to 13.8 g/dL at Month 6
- Successful engraftment in all evaluable patients (27/27)
- Sustained fetal hemoglobin levels above anti-sickling threshold
- Markers of hemolysis improved or normalized by Month 6
- Two serious adverse events possibly related to treatment reported
- One patient experienced vaso-occlusive events post-treatment
Insights
Poster presentation at ASH on Monday, December 9 at 6:00 p.m. PT / 9:00 p.m. ET
CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading gene editing company, will present updated safety and efficacy data in 28 patients living with severe sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. The data will be presented by Dr. Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, during a poster presentation at the American Society of Hematology (ASH) Annual Meeting in San Diego, CA, today at 6:00 p.m. PT (9:00 p.m. ET).
In the RUBY trial as of the data cutoff date (October 29, 2024), reni-cel was well-tolerated and continued to demonstrate a safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant by all patients (N=28). Patients were at a median of 9.5 months post-reni-cel infusion, with 11 patients having >1 year follow-up. Since reni-cel treatment, 27 of the 28 patients were free of vaso-occlusive events (VOEs). Patients were observed to have early normalization of total hemoglobin, with a mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 (n=18). Patients were also observed to have rapid and sustained improvements in fetal hemoglobin (HbF) ≥
Efficacy data in Patients with Severe Sickle Cell Disease
Patients were a median (range) of 9.5 (0.7–25.2) months post-reni-cel infusion, with 11 patients having >1 year follow-up. Of 28 patients, 27 were VOE-free post-reni-cel infusion. Reni-cel administration led to early, robust increases and sustained levels of total Hb and HbF. At month 6, the mean total Hb was 13.8 g/dL with a mean HbF percentage of
The mean percentage of F-cells increased early and was sustained at >
Sustained clinically meaningful improvements were observed in pain, physical, and social patient-reported outcome domains following treatment with reni-cel.
Safety data in Patients with Severe Sickle Cell Disease
Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated RUBY trial patients (N=28). After reni-cel infusion, all evaluable patients (n=27) achieved successful engraftment; with median time to neutrophil engraftment of 23 days and median time to platelet engraftment of 25 days, which is important for limiting infection and bleeding risk. Two serious adverse events (SAEs) assessed by the investigators as possibly related to reni-cel treatment have been reported in the RUBY trial.
RUBY Poster Presentation Details:
Title: Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial
Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland, OH, United States
Date/Time: Monday, December 9, 2024; 6:00 p.m. – 8:00 p.m. PT / 9:00 p.m. – 11:00 p.m. ET
Location: San Diego Convention Center, Halls G-H
Session: 801. Gene Therapies: Poster III
The poster can be accessed on the Editas Medicine website in the posters and presentations section.
About renizgamglogene autogedtemcel (reni-cel)
Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.
About the RUBY Trial
The RUBY trial is a single-arm, open-label, multi-center Phase 1/2/3 study designed to assess the safety and efficacy of reni-cel in patients with severe sickle cell disease. Additional details are available on www.clinicaltrials.gov (NCT04853576).
About Editas Medicine
As a leading gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.
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FAQ
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