Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (nex-z)
Intellia Therapeutics (NASDAQ:NTLA) announced a temporary pause of patient dosing and screening in its Phase 3 MAGNITUDE and MAGNITUDE-2 trials of nexiguran ziclumeran (nex-z) for ATTR-CM and ATTR-PN after an October 24, 2025 safety event.
The event involved a patient dosed on September 30, 2025 who developed Grade 4 liver transaminase elevations and increased total bilirubin, met protocol pausing criteria, was hospitalized and is receiving care. The company said it is consulting experts and engaging regulators. As of October 27, 2025, >650 ATTR-CM patients are enrolled in MAGNITUDE, 47 patients in MAGNITUDE-2, and an estimated >450 patients have been dosed with nex-z. A conference call and webcast were scheduled for 8:30 a.m. ET the same day.
Intellia Therapeutics (NASDAQ:NTLA) ha annunciato una temporanea sospensione della somministrazione e dello screening nei suoi studi di fase 3 MAGNITUDE e MAGNITUDE-2 su nexiguran ziclumeran (nex-z) per ATTR-CM e ATTR-PN dopo un evento di sicurezza del 24 ottobre 2025.
L'evento ha coinvolto un paziente trattato il 30 settembre 2025 che ha sviluppato Elevazioni di transaminasi epatiche di grado 4 e aumento della bilirubina totale, ha soddisfatto i criteri di pausa del protocollo, è stato ricoverato ed è in cura. L'azienda ha detto di consultare esperti e di interfacciarsi con i regolatori. A partire dal 27 ottobre 2025, >650 pazienti con ATTR-CM sono arruolati in MAGNITUDE, 47 pazienti in MAGNITUDE-2, e si stima >450 pazienti siano stati dosati con nex-z. È in programma una conference call e una webcast alle 8:30 ET lo stesso giorno.
Intellia Therapeutics (NASDAQ:NTLA) anunció una pausa temporal de la dosificación y del cribado de sus ensayos de fase 3 MAGNITUDE y MAGNITUDE-2 de nexiguran ziclumeran (nex-z) para ATTR-CM y ATTR-PN tras un evento de seguridad el 24 de octubre de 2025.
El evento involucró a un paciente dosificado el 30 de septiembre de 2025 que desarrolló elevaciones de transaminasas hepáticas de grado 4 y bilirrubina total aumentada, cumplió con los criterios de pausa del protocolo, fue hospitalizado y está recibiendo atención. La empresa dijo que está consultando a expertos y manteniendo contacto con los reguladores. A partir del 27 de octubre de 2025, >650 pacientes con ATTR-CM están enrolados en MAGNITUDE, 47 pacientes en MAGNITUDE-2, y se estima que >450 pacientes han sido dosificados con nex-z. Se programó una llamada de conferencia y webcast para las 8:30 a.m. ET del mismo día.
Intellia Therapeutics (NASDAQ:NTLA)는 ATTR-CM 및 ATTR-PN에 대한 3상 MAGNITUDE와 MAGNITUDE-2 연구에서 nexiguran ziclumeran(nex-z)의 투여 및 선별을 2025년 10월 24일의 안전성 사건 후 일시 중단했다고 발표했다.
사건은 2025년 9월 30일 투여된 환자가 간 트랜스아미나제 4등급 상승 및 총 빌리루빈 증가를 보였고, 프로토콜 중지 기준을 충족했으며 입원하여 치료를 받고 있다. 회사는 전문가 자문 및 규제 당국과의 협의를 진행 중이라고 밝혔다. 2025년 10월 27일 기준 MAGNITUDE에는 ATTR-CM 환자 >650명, MAGNITUDE-2에는 47명, nex-z를 투여받은 환자는 추정상 >450명이다. 같은 날 오전 8시 30분 ET에 전화회의 및 웹캐스트가 예정되어 있었다.
Intellia Therapeutics (NASDAQ:NTLA) a annoncé une pause temporaire de l'administration et du dépistage chez ses essais de phase 3 MAGNITUDE et MAGNITUDE-2 de nexiguran ziclumeran (nex-z) pour ATTR-CM et ATTR-PN après un événement de sécurité le 24 octobre 2025.
L'événement concernait un patient dosé le 30 septembre 2025 qui a développé des elevations des transaminases hépatiques de grade 4 et une augmentation de la bilirubine totale, a satisfait les critères de pause du protocole, a été hospitalisé et reçoit des soins. L'entreprise indique qu'elle consulte des experts et dialogue avec les régulateurs. Au 27 octobre 2025, >650 patients ATTR-CM sont enrôlés dans MAGNITUDE, 47 patients dans MAGNITUDE-2, et on estime que >450 patients ont été dosés avec nex-z. Une conférence téléphonique et une webdiffusion étaient prévues à 8h30 heure de l'Est le même jour.
Intellia Therapeutics (NASDAQ:NTLA) gab eine vorübergehende Aussetzung der Verabreichung und der Bildschirmung in seinen Phase-3-Studien MAGNITUDE und MAGNITUDE-2 von nexiguran ziclumeran (nex-z) für ATTR-CM und ATTR-PN nach einem Sicherheitsereignis am 24. Oktober 2025 bekannt.
Das Ereignis betraf einen Patienten, der am 30. September 2025 dosiert wurde und eine Grad-4-Lebertransaminaseerhöhung und einen Anstieg des Gesamtbilirubins entwickelte, die Protokollpausen-Kriterien erfüllte, hospitalisiert wurde und betreut wird. Das Unternehmen sagte, es konsultiere Experten und biete Regulierungskontakte an. Stand 27. Oktober 2025 sind >650 ATTR-CM-Patienten in MAGNITUDE eingeschrieben, 47 Patienten in MAGNITUDE-2, und schätzungsweise >450 Patienten mit nex-z dosiert worden. Eine Telefonkonferenz und ein Webcast waren für denselben Tag um 8:30 Uhr ET terminiert.
Intellia Therapeutics (NASDAQ:NTLA) أعلنت عن توقف مؤقت لإعطاء الجرعة والفحص في تجاربها من المرحلة الثالثة MAGNITUDE و MAGNITUDE-2 من nexiguran ziclumeran (nex-z) لـ ATTR-CM و ATTR-PN بعد حدث أمني في 24 أكتوبر 2025.
اشتمل الحدث على مريض تم إعطاؤه الجرعة في 30 سبتمبر 2025 وطور ارتفاعات من الدرجة 4 لإنزيمات الكبد وارتفاع في البيليروبين الكلي، ووافق على معايير إيقاف البروتوكول، وتم نقله إلى المستشفى وهو يتلقى الرعاية. قالت الشركة إنها تستشير خبراء وتتواصل مع الجهات التنظيمية. حتى 27 أكتوبر 2025، يوجد >650 مريض ATTR-CM مُسجلون في MAGNITUDE، و47 مريضًا في MAGNITUDE-2، ويُقدَّر أن >450 مريضًا قد تم إعطاؤهم nex-z. عُقدت مكالمة مؤتمرات وبث ويب في الساعة 8:30 صباحاً بتوقيت شرق الولايات المتحدة في اليوم نفسه.
Intellia Therapeutics (NASDAQ:NTLA) 宣布暂时暂停其第三阶段 MAGNITUDE 与 MAGNITUDE-2 针对 ATTR-CM 与 ATTR-PN 的 nexiguran ziclumeran (nex-z) 给药与筛查，原因是在 2025 年 10 月 24 日发生的安全事件。
该事件涉及一名在 2025 年 9 月 30 日给药的患者，出现 4 级肝转氨酶升高和总胆红素增高，符合协议暂停标准，已住院治疗。公司表示正咨询专家并与监管机构沟通。截至 2025 年 10 月 27 日，MAGNITUDE 已入组 >650 例 ATTR-CM 患者，MAGNITUDE-2 入组 47 例，且估计已给 Nex-z 给药的患者超过 450 例。同一天上午 8:30 进行电话会议和网络直播。
- More than 650 patients enrolled in MAGNITUDE
- An estimated >450 patients have been dosed with nex-z
- Company paused dosing immediately to prioritize patient safety
- Temporary pause of dosing and screening in MAGNITUDE and MAGNITUDE-2
- Reported Grade 4 liver transaminase elevations with increased total bilirubin
- At least one patient was hospitalized after dosing on September 30, 2025
Insights
Phase 3 dosing paused after a Grade 4 liver event; >450 patients estimated dosed with nex-z.
What happened: On
Dependencies and risks: Safety data in a Phase 3 program with more than 650 enrolled in MAGNITUDE, 47 in MAGNITUDE-2, and an estimated >450 dosed are material facts that directly affect trial timelines and regulatory interactions. The Grade 4 liver event and hospitalization meet protocol pausing criteria, creating immediate regulatory and operational risk until causality and mitigation are clarified.
Concrete items to watch: the patient's clinical outcome and investigator causality assessment; regulator feedback and any formal hold or guidance; details from the company conference call today at 8:30 a.m. ET and subsequent safety update; specific mitigation measures or protocol amendments and their projected timelines for resuming enrollment.
Conference call today at 8:30 a.m. ET
CAMBRIDGE, Mass., Oct. 27, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the company has temporarily paused patient dosing and screening for its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials of nex-z for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively.
This action follows a report on October 24, 2025 of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial on September 30, 2025, meeting the trial’s protocol-defined pausing criteria. The patient was hospitalized, is being closely monitored and is receiving medical intervention. Intellia is consulting with experts, considering potential risk mitigation strategies and engaging with regulatory authorities.
“In line with our commitment to patient safety, we have taken immediate action to temporarily pause enrollment in MAGNITUDE and MAGNITUDE-2 as we investigate this recent event,” said Intellia President and Chief Executive Officer John Leonard, M.D. “As we focus on ensuring the health of this patient, we also are engaging with regulatory authorities and other stakeholders globally to develop a strategy to resume enrollment as soon as appropriate.”
As of today, more than 650 patients with ATTR-CM are enrolled in MAGNITUDE, and 47 patients with ATTR-PN are enrolled in MAGNITUDE-2. Over 450 of these patients are estimated to have been dosed with nex-z.
Conference Call Information
The company will host a conference call and webcast today at 8:30 a.m. ET to discuss this update. To join the webcast, please visit the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com. To join by phone, U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call. A replay of the webcast will be available at intelliatx.com for approximately 90 days.
About Nex-z
Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN). Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and is currently being investigated in MAGNITUDE and MAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTR-PN, respectively. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Nex-z has received an Orphan Drug and RMAT Designation from the U.S. Food and Drug Administration (FDA) and an Orphan Drug Designation (ODD) from the European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc.
About Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.
Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding: the safety, tolerability, efficacy, success and advancement of its clinical programs for “nex-z” (also known as NTLA-2001), including the ability to resume and successfully complete its MAGNITUDE and MAGNITUDE-2 trials for nex-z.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events 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: uncertainties related to Intellia’s ability to resume the MAGNITUDE and MAGNITUDE-2 trials, the implications of this event on the safety and efficacy of nex-z and further development of nex-z; regulatory agencies’ evaluation of regulatory filings and other information related to our product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for our product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; the risk that any one or more of Intellia’s product candidates, including nex-z, will not be successfully developed and commercialized; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies for the same product candidate or Intellia’s other product candidates; and risks related to Intellia’s reliance on collaborations, including that its collaboration with Regeneron Pharmaceuticals, Inc. will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
Intellia Contacts:
Investors:
Jason Fredette
Vice President, Investor Relations and Corporate Communications
Intellia Therapeutics, Inc.
jason.fredette@intelliatx.com
Media:
Matt Crenson
Ten Bridge Communications
media@intelliatx.com
mcrenson@tenbridgecommunications.com
This press release was published by a CLEAR® Verified individual.
FAQ
Why did Intellia (NTLA) pause dosing in the MAGNITUDE Phase 3 trial on October 27, 2025?
How many patients are enrolled and dosed in NTLA's MAGNITUDE programs as of Oct 27, 2025?
What is the status of MAGNITUDE-2 (ATTR-PN) after Intellia's Oct 27, 2025 update?
Will Intellia (NTLA) discuss the MAGNITUDE pause with investors on Oct 27, 2025?
Is the patient who experienced liver lab abnormalities still receiving care after the Oct 24, 2025 report?
