Intellia Therapeutics Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema
Intellia Therapeutics (NASDAQ:NTLA) has announced the dosing of the first patient in its global Phase 3 HAELO study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). The company expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027.
NTLA-2002 is being developed as a single-dose treatment for this potentially life-threatening disease. The company plans to present longer-term data from the ongoing Phase 1/2 study later this year to demonstrate the treatment's durability of effect.
Intellia Therapeutics (NASDAQ:NTLA) ha annunciato la somministrazione della prima dose al paziente nel suo studio globale Phase 3 HAELO per NTLA-2002, un trattamento sperimentale di editing genetico in vivo tramite CRISPR per l'angioedema ereditario (HAE). L'azienda prevede di completare l'arruolamento nella seconda metà del 2025 e di presentare una domanda di licenza biologica (BLA) nel 2026, con l'obiettivo di lanciare il prodotto negli Stati Uniti nel 2027.
NTLA-2002 è in fase di sviluppo come trattamento monodose per questa malattia potenzialmente letale. L'azienda prevede di presentare dati a lungo termine dallo studio in fase 1/2 più avanti quest'anno per dimostrare la durata dell'effetto del trattamento.
Intellia Therapeutics (NASDAQ:NTLA) ha anunciado la dosificación del primer paciente en su estudio global Phase 3 HAELO de NTLA-2002, un tratamiento experimental de edición genética in vivo mediante CRISPR para el angioedema heredado (HAE). La empresa espera completar la inscripción en la segunda mitad de 2025 y presentar una solicitud de licencia biológica (BLA) en 2026, con el objetivo de un lanzamiento en EE. UU. en 2027.
NTLA-2002 se está desarrollando como un tratamiento de dosis única para esta enfermedad potencialmente mortal. La empresa planea presentar datos a largo plazo del estudio en fase 1/2 más adelante este año para demostrar la durabilidad del efecto del tratamiento.
Intellia Therapeutics (NASDAQ:NTLA)는 유전성 부종(HAE) 치료를 위한 CRISPR 유전자 편집 치료제인 NTLA-2002의 글로벌 Phase 3 HAELO 연구에서 첫 환자에게 투약을 시작했다고 발표했습니다. 이 회사는 2025년 하반기까지 환자 등록을 완료하고 2026년에 생물학적 라이센스 신청(BLA)을 제출할 예정이며, 2027년에는 미국에서 출시할 계획입니다.
NTLA-2002는 이 잠재적으로 생명 위협적인 질병에 대한 단일 투여 치료제로 개발되고 있습니다. 회사는 올해 후반에 진행 중인 Phase 1/2 연구에서 장기적인 데이터를 발표하여 치료의 효과 지속성을 입증할 계획입니다.
Intellia Therapeutics (NASDAQ:NTLA) a annoncé l'administration de la première dose au patient dans son étude mondiale Phase 3 HAELO pour NTLA-2002, un traitement expérimental d'édition génique in vivo par CRISPR pour l'angioedème héréditaire (HAE). La société prévoit de terminer l'enrôlement au cours de la seconde moitié de 2025 et de soumettre une demande de licence biologique (BLA) en 2026, avec pour objectif un lancement aux États-Unis en 2027.
NTLA-2002 est en cours de développement en tant que traitement à dose unique pour cette maladie potentiellement mortelle. La société prévoit de présenter des données à long terme de l'étude en phase 1/2 plus tard cette année pour démontrer la durabilité de l'effet du traitement.
Intellia Therapeutics (NASDAQ:NTLA) hat die Dosisverabreichung des ersten Patienten in seiner globalen Phase 3 HAELO-Studie zu NTLA-2002 angekündigt, einer experimentellen in vivo CRISPR-Genbearbeitung zur Behandlung des hereditären Angioödems (HAE). Das Unternehmen rechnet damit, die Rekrutierung in der zweiten Hälfte des Jahres 2025 abzuschließen und 2026 einen Antrag auf Zulassung biologischer Produkte (BLA) zu stellen, mit dem Ziel, 2027 in den USA auf den Markt zu kommen.
NTLA-2002 wird als Einzeldosisbehandlung für diese potenziell lebensbedrohliche Erkrankung entwickelt. Das Unternehmen plant, später in diesem Jahr langfristige Daten aus der laufenden Phase 1/2-Studie zu präsentieren, um die Nachhaltigkeit der Behandlungseffekte zu demonstrieren.
- Phase 3 clinical trial initiation marks final stage of clinical development
- Clear timeline for BLA submission (2026) and U.S. launch (2027)
- Treatment designed as a single-dose therapy, potentially offering lifelong relief
- Extended timeline to market with launch not expected until 2027
- Enrollment completion not expected until H2 2025
Insights
The initiation of the Phase 3 HAELO study for NTLA-2002 represents a pivotal milestone in the development of CRISPR-based therapeutics for hereditary angioedema (HAE), a rare genetic condition causing dangerous swelling episodes. This advancement is particularly noteworthy as it positions Intellia at the forefront of in vivo CRISPR gene editing treatments.
The study's commencement carries substantial implications for both the company and the broader biotech sector:
- The single-dose treatment approach, if successful, could revolutionize HAE management, potentially replacing current chronic treatment regimens
- The projected timeline, with enrollment completion in H2 2025 and BLA submission in 2026, suggests a well-structured development pathway toward potential commercialization in 2027
- The global HAE therapeutic market, valued in billions, represents a significant commercial opportunity
The progression to Phase 3 following promising earlier trial results indicates strong clinical validation of Intellia's CRISPR platform. For investors, this development reduces clinical risk and strengthens the company's position in the genetic medicine space. The focus on completing enrollment in 2025 and the clear regulatory pathway demonstrate strategic execution toward commercialization.
NTLA-2002's Phase 3 trial initiation marks a transformative moment in HAE treatment development. HAE patients currently face a significant burden with regular prophylactic treatments or on-demand medications for acute attacks. A successful single-dose CRISPR therapy could fundamentally alter this paradigm.
The therapeutic potential is compelling for several reasons:
- Current HAE treatments require frequent administration (weekly to monthly), creating a substantial burden on patients
- A one-time treatment that provides durable protection could significantly improve quality of life and reduce healthcare costs
- The precision of CRISPR gene editing offers the potential for long-term disease modification rather than symptom management
The anticipated presentation of longer-term data from the Phase 1/2 study later this year will be important in validating the durability of treatment effect, a key differentiator for this therapeutic approach. Success in this program could also validate Intellia's broader in vivo CRISPR platform, potentially accelerating development across their pipeline.
CAMBRIDGE, Mass., Jan. 22, 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 the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based therapy in development as a single-dose treatment for this potentially life-threatening disease. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the Company’s plans for a U.S. launch in 2027.
“We are pleased to have initiated dosing in the HAELO Phase 3 study as we are in our final lap of clinical development for NTLA-2002,” said Intellia President and Chief Executive Officer John Leonard, M.D. “With the promising data we’ve presented thus far, we believe patients could achieve independence from both HAE attacks and medications required to treat this disease. We look forward to presenting longer-term data from the ongoing Phase 1/2 study later this year highlighting the durability of effect of NTLA-2002.”
"We are excited to have treated the first patient in the U.S. with a new generation of therapy that could potentially provide patients with lifelong relief from the primary symptoms of HAE,” commented Dr. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc.
About the HAELO Study
The pivotal Phase 3 HAELO clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 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), please visit clinicaltrials.gov.
About NTLA-2002
Based on Nobel-prize winning CRISPR/Cas9 technology, NTLA-2002 has the potential to become the first one-time treatment for hereditary angioedema (HAE). NTLA-2002 is designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, 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 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. Although there is no known cure for 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 life-long 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 regarding: the safety, efficacy, success and advancement of our clinical program for NTLA-2002 for the treatment of hereditary angioedema (HAE), including its ability to be a single dose treatment for HAE and to offer patients independence from both HAE attacks and medications needed to treat HAE; near-term clinical milestones, including completing enrollment in the HAELO Phase 3 study in the second half of 2025; our interactions with regulatory authorities, including submitting a biologics license application for NTLA-2002 in 2026; our commercialization plans, including a U.S. launch of NTLA-2002 in 2027; and the expected timing and contents of future data releases, including presenting longer-term data from the ongoing Phase 1/2 study later this year highlighting the duration of effect of NTLA-2002.
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 licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that any one or more of Intellia’s product candidates, including NTLA-2002, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies, such as the Phase 1/2 study of NTLA-2002, 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 future financial condition and its ability to fund its operations. 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 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.
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FAQ
When will Intellia (NTLA) complete enrollment for the HAELO Phase 3 study?
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What type of treatment is NTLA-2002 for hereditary angioedema?
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