Intellia Therapeutics Completes Enrollment in the Global Phase 3 HAELO Study of Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Intellia Therapeutics (NASDAQ:NTLA) has achieved a significant milestone by completing enrollment in its global Phase 3 HAELO study for lonvoguran ziclumeran (lonvo-z), a CRISPR-based therapy for hereditary angioedema (HAE). The study enrolled over 60 patients within nine months, with nearly half from the U.S.
The company expects to report topline data in H1 2026, followed by a BLA submission in H2 2026, targeting a U.S. launch in H1 2027. The HAELO study is a randomized, double-blind, placebo-controlled trial evaluating lonvo-z's efficacy in patients aged 16 and older with Type I or II HAE. Additional Phase 1/2 study data will be presented in Q4 2025.
Intellia Therapeutics (NASDAQ:NTLA) ha raggiunto una tappa significativa completando l’arruolamento nel proprio studio globale di fase 3 HAELO per lonvoguran ziclumeran (lonvo-z), una terapia CRISPR-based per l’angioedema ereditaria (HAE). Lo studio ha reclutato oltre 60 pazienti in nove mesi, con quasi la metà provenienti dagli Stati Uniti.
L’azienda prevede di pubblicare i dati primari nel primo semestre 2026, seguiti da una presentazione della BLA nel secondo semestre 2026, con l’obiettivo di un lancio negli USA nel primo semestre 2027. Lo studio HAELO è uno studio randomizzato, in doppio cieco, controllato con placebo che valuta l’efficacia di lonvo-z in pazienti di età pari o superiore a 16 anni con HAE di tipo I o II. Ulteriori dati dello studio di fase 1/2 saranno presentati nel quarto trimestre 2025.
Intellia Therapeutics (NASDAQ:NTLA) ha logrado un hito significativo al completar el reclutamiento en su ensayo global de fase 3 HAELO para lonvoguran ziclumeran (lonvo-z), una terapia basada en CRISPR para el angioedema hereditario (HAE). El estudio inscribió a más de 60 pacientes en nueve meses, con casi la mitad provenientes de EE. UU.
La compañía espera reportar los datos principales en el primer semestre de 2026, seguidos de una presentación de la BLA en el segundo semestre de 2026, con miras a un lanzamiento en EE. UU. en el primer semestre de 2027. El estudio HAELO es un ensayo aleatorizado, doble ciego, controlado con placebo que evalúa la eficacia de lonvo-z en pacientes de 16 años o más con HAE tipo I o II. Se presentarán datos adicionales del estudio de fase 1/2 en el cuarto trimestre de 2025.
Intellia Therapeutics (NASDAQ:NTLA)가 롱보르간 지클루메란(lonvoguran ziclumeran, lonvo-z)에 대한 CRISPR 기반의 유전성 혈관부종(HAE) 치료제의 글로벌 3상 HAELO 연구의 등록 완료라는 중요한 이정표를 달성했습니다. 이 연구는 9개월 간 60명 이상을 등록했으며, 그중 거의 절반은 미국에서 왔습니다.
회사는 2026년 상반기에 주요 데이터를 발표하고, 2026년 하반기에 BLA 제출을 따른 뒤 2027년 상반기 미국 출시를 목표로 하고 있습니다. HAELO 연구는 16세 이상 Type I 또는 II HAE를 가진 환자를 대상으로 한 무작위, 이중 맹검, 위약 대조 임상시험으로, lonvo-z의 효능을 평가합니다. 1/2상 연구의 추가 데이터는 2025년 4분기에 발표될 예정입니다.
Intellia Therapeutics (NASDAQ:NTLA) a atteint une étape significative en terminant l’inclusion dans son essai mondial de phase 3 HAELO pour lonvoguran ziclumeran (lonvo-z), une thérapie CRISPR-based pour l’angio-œdème héréditaire (HAE). L’étude a recruté plus de 60 patients en neuf mois, dont près de la moitié proviennent des États‑Unis.
L’entreprise prévoit de communiquer les données primaires au 1er semestre 2026, suivies d’une soumission BLA au 2e semestre 2026, avec pour objectif un lancement aux États‑Unis au 1er semestre 2027. L’essai HAELO est une étude randomisée, en double aveugle, contrôlée par placebo, évaluant l’efficacité de lonvo-z chez des patients âgés de 16 ans et plus atteints de HAE de type I ou II. Des données additionnelles de l’étude de phase 1/2 seront présentées au 4e trimestre 2025.
Intellia Therapeutics (NASDAQ:NTLA) hat einen wichtigen Meilenstein erreicht, indem es die Rekrutierung in der globalen Phase-3-Studie HAELO für lonvoguran ziclumeran (lonvo-z), eine CRISPR-basierte Therapie für hereditäres Angioödem (HAE), abgeschlossen hat. Die Studie rekrutierte in neun Monaten über 60 Patienten, davon fast die Hälfte aus den USA.
Das Unternehmen rechnet damit, Topline-Daten im ersten Halbjahr 2026 zu berichten, gefolgt von einer BLA-Einreichung im zweiten Halbjahr 2026, mit dem Ziel eines US-Verlaufs im ersten Halbjahr 2027. Die HAELO-Studie ist eine randomisierte, doppelblinde, placebokontrollierte Studie, die die Wirksamkeit von lonvo-z bei Patienten ab 16 Jahren mit Typ I oder II HAE bewertet. Weitere Phase-1/2-Daten werden im 4. Quartal 2025 präsentiert.
Intellia Therapeutics (NASDAQ:NTLA) حققت خطوة مهمة بإتمام التوظيف في دراستها العالمية من المرحلة الثالثة HAELO لعلاج lonvoguran ziclumeran (lonvo-z)، وهو علاج يعتمد على CRISPR لمرض وذمة وعائية وراثية (HAE). درست الدراسة أكثر من 60 مريضًا خلال تسعة أشهر، كان نحو نصفهم من الولايات المتحدة.
تتوقع الشركة أن تعلن عن البيانات الأولية في النصف الأول من 2026، تليها تقديم BLA في النصف الثاني من 2026، مع هدف الإطلاق في الولايات المتحدة في النصف الأول من 2027. وتعد دراسة HAELO تجربة عشوائية مزدوجة التعمية خاضعة للتحكم بالدواء الوهمي تقيم فعالية lonvo-z لدى مرضى تتراوح أعمارهم 16 عامًا فما فوق المصابين بـ HAE من النوعين I أو II. ستُعرض بيانات إضافية من دراسة المرحلة 1/2 في الربع الرابع من 2025.
Intellia Therapeutics (NASDAQ:NTLA) 已实现重要里程碑，完成全球3期HAELO研究招募，针对lonvoguran ziclumeran (lonvo-z)，一种用于遗传性血管性肿胀（HAE）的CRISPR基因治疗。该研究在九个月内招募了超过60名患者，且近一半来自美国。
公司预计在2026年上半年公布主要数据，随后在2026年下半年提交BLA，目标在2027年上半年在美国上市。HAELO研究是一个随机、双盲、安慰剂对照试验，评估lonvo-z在16岁及以上、HAE I型或II型患者中的疗效。阶段1/2研究的额外数据将于2025年第四季度公布。
- None.
- Commercial launch not expected until H1 2027
- Phase 3 efficacy and safety data still pending
Insights
Intellia has rapidly completed Phase 3 enrollment for its CRISPR HAE therapy, positioning for potential 2027 commercial launch.
Intellia's announcement represents a significant milestone in the clinical development of lonvoguran ziclumeran (lonvo-z), their CRISPR-based therapy for hereditary angioedema (HAE). The company has completed enrollment for their pivotal Phase 3 HAELO study in just nine months, substantially faster than typical enrollment timelines for rare disease trials, which often take 18-24 months. This accelerated recruitment, with nearly half of patients enrolled from the U.S., strongly indicates high patient and physician interest in this novel therapeutic approach.
The company has outlined a clear regulatory pathway with topline data expected in H1 2026, followed by BLA submission in H2 2026, positioning for a potential U.S. commercial launch in H1 2027. This timeline provides investors with transparent expectations regarding the development trajectory.
The Phase 3 HAELO study design is robust - a randomized, double-blind, placebo-controlled trial with 60+ patients randomized 2:1 to receive either a single 50 mg infusion of lonvo-z or placebo. The primary endpoints focusing on HAE attack frequency and attack-free status from weeks 5-28 are clinically meaningful and align with FDA requirements for this indication. The optional blinded crossover at week 28 also provides an ethical component to the study design, ensuring all participants eventually receive active treatment.
This progress signals that Intellia is executing effectively on its clinical development strategy, with additional Phase 1/2 data expected in Q4 2025 that will likely provide further insights into lonvo-z's durability and safety profile.
- Completed patient enrollment within nine months with nearly half enrolled from the U.S.
- Expect to report Phase 3 topline data in the first half of 2026
- On track to submit BLA in the second half of 2026 for an anticipated U.S. launch in the first half of 2027
- Plan to present additional data from the Phase 1/2 study in the fourth quarter of 2025
CAMBRIDGE, Mass., Sept. 18, 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 it has completed enrollment in the global Phase 3 HAELO study of lonvoguran ziclumeran (lonvo-z) for the treatment of hereditary angioedema (HAE). Topline data are expected in the first half of 2026. Intellia is on track to submit a biologics license application (BLA) in the second half of 2026 to support the company’s plans for a U.S. launch in the first half of 2027.
"Completing HAELO enrollment within nine months since dosing the first patient marks a pivotal moment for the company and reflects the degree of unmet need we are hearing from people living with HAE,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are deeply grateful to the patients, their families and the trial investigators for their enthusiasm and look forward to sharing topline results from the Phase 3 study next year. Our momentum remains strong for a planned BLA submission in 2026 as we seek to make this potentially life-changing therapy available to HAE patients starting in the U.S.”
“Based on results from the Phase 1/2 study, lonvo-z shows great promise to positively transform the HAE treatment paradigm,” said Aleena Banerji, M.D., Professor at Harvard Medical School, Clinical Director of the Allergy and Clinical Immunology Unit at Massachusetts General Hospital, and HAELO principal investigator. “We look forward to seeing the Phase 3 topline data next year.”
Intellia dosed the first patient in the pivotal Phase 3 HAELO study in January 2025. The Company announced today it has completed enrollment, with nearly half of the patients enrolled from the U.S. HAELO is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of lonvo-z in at least 60 adults and adolescents aged 16 years and older with Type I or Type II HAE. Patients are randomized 2:1 to receive a single 50 mg infusion of lonvo-z or placebo. Patients are eligible for optional blinded crossover at week 28. Key endpoints include the number of HAE attacks and the number of patients who achieve attack-free status from week 5 through week 28. For more information on HAELO (NCT06634420), visit clinicaltrials.gov.
Intellia expects to present longer-term data from patients in the Phase 2 portion of the ongoing Phase 1/2 study in the fourth quarter of 2025.
About Lonvo-z
Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. There are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include lifelong therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.
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 concerning: the safety, efficacy, success and advancement of its clinical program for lonvoguran ziclumeran or “lonvo-z” (also known as NTLA-2002) for the treatment of hereditary angioedema (“HAE”) pursuant to its clinical trial applications and investigational new drug application submissions, including its plan to report Phase 3 topline data in the first half of 2026, its plan to submit a BLA in the second half of 2026, its expectation for a U.S. launch in first half of 2027, its plan to present additional data from the Phase 1/2 study in the fourth quarter of 2025, and the ability of lonvo-z to positively transform the HAE treatment paradigm and potentially be a life-changing therapy for patients with HAE.
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: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its contract manufacturers, collaborators, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the conduct of clinical studies and other development requirements for its product candidates, including risks related to the ability to develop and commercialize any one or more of Intellia’s product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related to the potential delay of planned clinical trials due to regulatory feedback or other developments. 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, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission, including its quarterly report on Form 10-Q. 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:
Brittany Chaves
Senior Manager, Investor Relations
brittany.chaves@intelliatx.com
Media:
Matt Crenson
Ten Bridge Communications
media@intelliatx.com
mcrenson@tenbridgecommunications.com
FAQ
When will Intellia (NTLA) report Phase 3 HAELO study results for lonvo-z?
What is the target patient population for Intellia's lonvo-z HAE treatment?
How many patients are enrolled in NTLA's Phase 3 HAELO trial?
When does Intellia plan to launch lonvo-z in the United States?
What is the dosing protocol for lonvo-z in the HAELO Phase 3 trial?
