Intellia Therapeutics Announces Initiation of HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE)
Intellia Therapeutics (NASDAQ:NTLA) has initiated HAELO, a global Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema (HAE). NTLA-2002 is designed as a single-dose treatment to prevent potentially life-threatening swelling attacks in HAE patients. The study will evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE, with patients randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo.
The primary endpoint is the change in number of HAE attacks from week 5 through week 28. This initiation follows positive safety and efficacy data from the ongoing Phase 1/2 study, which showed dramatic reductions in attack rate and consistent, deep, and durable reductions in kallikrein levels. Detailed results from the Phase 2 portion will be presented at the 2024 ACAAI Annual Scientific Meeting in October.
Intellia Therapeutics (NASDAQ:NTLA) ha avviato HAELO, uno studio globale di fase 3 su NTLA-2002, un trattamento sperimentale di editing genomico CRISPR in vivo per l'angioedema ereditario (HAE). NTLA-2002 è progettato come un trattamento a dose singola per prevenire attacchi di gonfiore potenzialmente fatali nei pazienti affetti da HAE. Lo studio valuterà l'efficacia e la sicurezza di NTLA-2002 in 60 adulti con HAE di Tipo I o Tipo II, con i pazienti randomizzati in un rapporto di 2:1 per ricevere un'infusione singola di 50 mg di NTLA-2002 o un placebo.
Il criterio primario è il cambiamento nel numero di attacchi di HAE dalla quinta alla ventottesima settimana. Questa iniziativa segue dati positivi di sicurezza ed efficacia dal corrente studio di fase 1/2, che ha mostrato riduzioni drammatiche nel tasso di attacchi e riduzioni costanti, profonde e durature nei livelli di callicreina. I risultati dettagliati della parte di fase 2 saranno presentati alla Riunione Scientifica Annuale ACAAI 2024 in ottobre.
Intellia Therapeutics (NASDAQ:NTLA) ha iniciado HAELO, un estudio global de fase 3 de NTLA-2002, un tratamiento de edición genética CRISPR in vivo en investigación para el angioedema hereditario (HAE). NTLA-2002 está diseñado como un tratamiento de dosis única para prevenir ataques de hinchazón que pueden amenazar la vida en pacientes con HAE. El estudio evaluará la eficacia y seguridad de NTLA-2002 en 60 adultos con HAE Tipo I o Tipo II, con los pacientes randomizados en una proporción de 2:1 para recibir una infusión única de 50 mg de NTLA-2002 o un placebo.
El objetivo principal es el cambio en el número de ataques de HAE desde la semana 5 hasta la semana 28. Esta iniciativa sigue a los datos positivos de seguridad y eficacia del estudio en curso de fase 1/2, que mostró reducciones dramáticas en la tasa de ataques y reducciones consistentes, profundas y duraderas en los niveles de calicreína. Los resultados detallados de la parte de fase 2 se presentarán en la Reunión Científica Anual ACAAI 2024 en octubre.
인텔리아 테라퓨틱스(NASDAQ:NTLA)는 유전성 혈관 부종(HAE)에 대한 인체 내 CRISPR 유전자 편집 치료제인 NTLA-2002에 대한 글로벌 3상 연구인 HAELO를 시작했습니다. NTLA-2002는 HAE 환자에서 치명적인 부종 발작을 예방하기 위해 단일 투여 치료로 설계되었습니다. 이 연구는 I형 또는 II형 HAE를 가진 60명의 성인을 대상으로 NTLA-2002의 효능과 안전성을 평가하며, 환자들은 NTLA-2002 50mg 단일 주사 또는 위약을 투여받기 위해 2:1로 무작위 배정됩니다.
주요 목표는 5주째부터 28주째까지 HAE 발작 수의 변화입니다. 이 연구의 시작은 현재 진행 중인 1/2상 연구에서 안전성과 효능에 대한 긍정적인 데이터를 바탕으로 하며, 이 연구에서는 발작 발생률의 극적인 감소 및 카리클레인 수치의 일관되고 깊으며 지속적인 감소가 나타났습니다. 2상 부분의 자세한 결과는 2024년 10월에 열리는 ACAAI 연례 학술대회에서 발표될 예정입니다.
Intellia Therapeutics (NASDAQ:NTLA) a lancé HAELO, une étude mondiale de phase 3 sur NTLA-2002, un traitement expérimental d'édition génique CRISPR in vivo pour l'angioedème héréditaire (HAE). NTLA-2002 est conçu comme un traitement à dose unique pour prévenir des attaques de gonflement potentiellement mortelles chez les patients atteints de HAE. L'étude évaluera l'efficacité et la sécurité de NTLA-2002 chez 60 adultes atteints de HAE de type I ou II, les patients étant randomisés dans un rapport de 2:1 pour recevoir une infusion unique de 50 mg de NTLA-2002 ou un placebo.
Le critère principal est le changement du nombre d'attaques de HAE de la semaine 5 à la semaine 28. Ce lancement fait suite à des données de sécurité et d'efficacité positives de l'étude en cours de phase 1/2, qui a montré des réductions dramatiques du taux d'attaques et des diminutions constantes, profondes et durables des niveaux de kalikréine. Les résultats détaillés de la partie de phase 2 seront présentés lors de la Réunion Scientifique Annuelle ACAAI 2024 en octobre.
Intellia Therapeutics (NASDAQ:NTLA) hat HAELO gestartet, eine globale Phase 3 Studie zu NTLA-2002, einer experimentellen in vivo CRISPR-Gentechnologiebehandlung für hereditäres Angioödem (HAE). NTLA-2002 ist als Einzel-Dosis-Behandlung konzipiert, um potenziell lebensbedrohliche Schwellungsattacken bei HAE-Patienten zu verhindern. Die Studie bewertet die Wirksamkeit und Sicherheit von NTLA-2002 bei 60 Erwachsenen mit HAE vom Typ I oder II, wobei die Patienten im Verhältnis 2:1 randomisiert wurden, um eine Einzelinfusion von 50 mg NTLA-2002 oder ein Placebo zu erhalten.
Der primäre Endpunkt ist die Veränderung der Anzahl von HAE-Attacken von Woche 5 bis Woche 28. Diese Initiierung folgt auf positive Sicherheits- und Wirksamkeitsdaten aus der laufenden Phase 1/2-Studie, die dramatische Reduktionen der Attackenrate und konsequente, tiefe und dauerhafte Reduktionen der Kallikreinwerte zeigte. Detaillierte Ergebnisse aus dem 2. Teil der Phase werden auf dem ACAAI-Jahrestreffen 2024 im Oktober präsentiert.
- Initiation of Phase 3 HAELO study for NTLA-2002, advancing to final stage of clinical development
- Positive interim Phase 1 data showing dramatic reductions in HAE attack rate
- Consistent, deep, and durable reductions in kallikrein levels observed in Phase 1/2 study
- Potential for NTLA-2002 to be a single-dose treatment for HAE, addressing unmet patient needs
- None.
- NTLA-2002 is a single-dose treatment designed to prevent potentially life-threatening swelling attacks in people with hereditary angioedema (HAE)
- NTLA-2002 is Intellia’s second in vivo candidate to enter late-stage clinical development from its modular gene editing platform
CAMBRIDGE, Mass., Oct. 07, 2024 (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 initiation of HAELO, a global, pivotal Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for this potentially life-threatening disease. Patient screening is active following Intellia’s successful end-of-Phase 2 meeting and submission of an Investigational New Drug Application amendment to the U.S. Food and Drug Administration (FDA).
“Initiation of the HAELO Phase 3 trial is a significant milestone for Intellia as we enter the final stage of clinical development for NTLA-2002 for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Data from the ongoing Phase 1/2 study showed great promise that a single-dose treatment can lead to a complete response – no more attacks and no further treatment required. We are working urgently to bring forward NTLA-2002 to address the real-world needs of people suffering from this disease and, ultimately, believe it will bring significant value to patients, physicians and payors.”
HAELO is a global, 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. The primary endpoint is the change in number of HAE attacks from week 5 through week 28.
Intellia is initiating the Phase 3 study based on positive safety and efficacy data from the ongoing Phase 1/2 study (NCT05120830) of NTLA-2002. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Intellia previously announced positive toplines results from the Phase 2 portion of the study. The Company plans to present the detailed results at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.
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. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. 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 programs for NTLA-2002 for the treatment of hereditary angioedema (“HAE”) pursuant to its clinical trial applications (“CTA”) and investigational new drug (“IND”) submissions, including the expected timing of data releases, regulatory feedback, regulatory filings, and the initiation, enrollment, dosing and completion of clinical trials, the potential of NTLA-2002 to lead to a complete response after a single dose; and its growth as a Company and expectations regarding its uses of capital, expenses, future accumulated deficit and financial results.
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 authorization, initiation and conduct of preclinical and clinical studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; 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; risks related to the potential delay of planned clinical trials due to regulatory feedback or other developments; and risks related to Intellia’s collaborations not continuing or not being 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, 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:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
ian.karp@intelliatx.com
Lina Li
Senior Director, Investor Relations and Corporate Communications
lina.li@intelliatx.com
Media:
Matt Crenson
Ten Bridge Communications
media@intelliatx.com
mcrenson@tenbridgecommunications.com
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FAQ
What is the purpose of Intellia's HAELO Phase 3 study for NTLA-2002?
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