Intellia Therapeutics Presents Additional Positive Clinical and Patient-Reported Data for Lonvoguran Ziclumeran (lonvo-z) at ACAAI 2026

• Among patients previously on long-term prophylaxis (LTP) in HAELO Phase 3 trial, monthly attack rate decreased by 81% following lonvo-z dosing compared with prior LTP
• 100% of patients receiving 50 mg of lonvo-z across Phase 1/2 clinical trial and HAELO were LTP-free as of the data cutoffs
• Among 23 Phase 1/2 patients interviewed, all described their hereditary angioedema (HAE) as well controlled after receiving lonvo-z and 22 (96%) reported lonvo-z as an optimal HAE therapy
• Intellia hosting investor webcast today at 10:00 a.m. ET

CAMBRIDGE, Mass., Nov. 12, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced highlights from its seven presentations at the American College of Allergy, Asthma & Immunology (ACAAI) 2026 Annual Scientific Meeting in Phoenix, Arizona. Intellia will host an investor webcast today at 10:00 a.m. ET to review certain data from the ACAAI presentations, share perspectives from key opinion leaders and discuss the company’s preparations for a planned commercial launch of lonvo-z in HAE.

Oral Presentation Details:

• Title: Four-Year Durability/Safety of Lonvoguran Ziclumeran (Lonvo-z) 50 mg in Patients with Hereditary Angioedema
  Session: Distinguished Industry and Late-Breaking Oral Abstracts – Session 2
  Date and Time: Saturday, November 14, 2026, from 4:43 – 4:53 p.m. MT
  Presenter: Danny Cohn, M.D., Ph.D., Associate Professor, Amsterdam University Medical Center, Department of Vascular Medicine, University of Amsterdam; Phase 1/2 and HAELO principal investigator
  • In the Phase 1/2 study, deep, consistent and durable reductions in plasma kallikrein were observed in all patients, with a mean reduction of 84% at four weeks that was maintained through up to four years of follow-up. Lonvo-z continues to be well tolerated with no new long-term risks identified.
  • Additional biomarkers were reported that support lonvo-z’s intended mechanism of action in which inactivation of the KLKB1 gene and the associated reduction in plasma kallikrein protein leads to reduced activity of the kallikrein-kinin system and less bradykinin production.
    
    
• Title: Attack-Free and Long-Term Prophylaxis-Free Status Following Treatment with Lonvoguran Ziclumeran in Hereditary Angioedema
  Session: Distinguished Industry and Late-Breaking Oral Abstracts – Session 2
  Date and Time: Saturday, November 14, 2026, from 4:53 – 5:03 p.m. MT
  Presenter: Emel Aygören-Pürsün, M.D., Department for Children and Adolescents, Angioedema Centre, University Hospital Frankfurt, Goethe University; Phase 1/2 and HAELO principal investigator
  • In the Phase 1/2 study (n=32), following a one-time 50 mg dose of lonvo-z, the proportion of patients who were attack-free after one year was at least 90% during each month with up to 4 years of follow-up. Additionally, 100% of patients were LTP-free as of the data cutoff.
  • The estimated probability of beginning an attack-free and LTP-free interval lasting >6 months increased from 81% at six months to 93% at 24 months post-dosing.
    
    

Poster Presentation Details: 

• Title: Lonvoguran Ziclumeran Demonstrated Consistent Benefit in All Hereditary Angioedema Patient Subgroups in Ph3 HAELO Study
  Poster Session: Monitor 17
  Date and Time: Friday, November 13, 2026, from 3:05 – 3:20 p.m. MT
  Presenter: Marc Riedl, M.D., Professor of Medicine, Clinical Director of the U.S. Hereditary Angioedema Association (HAEA) Angioedema Center, University of California San Diego; HAELO principal investigator
  • Among the 35 HAELO patients taking LTP at study entry who were randomized to the lonvo-z arm, the mean HAE attack rate decreased by 81% during the efficacy observation period when compared to the attack rate while on prior LTP. Following lonvo-z treatment, 63% of these patients achieved complete control, more than tripling the attack-free rate while on prior LTP.
  • All prespecified subgroups demonstrated consistent benefit in attack rate reduction favoring lonvo-z over placebo regardless of disease severity or prior LTP treatment. All treatment emergent adverse events (TEAEs) were mild or moderate and there were no serious adverse events observed in the lonvo-z arm.
    
    
• Title: Lived Experiences of Patients Treated with Lonvoguran Ziclumeran, a One-Time CRISPR Treatment for Hereditary Angioedema
  Poster Session: Monitor 17
  Date and Time: Friday, November 13, 2026, from 3:20 – 3:35 p.m. MT
  Presenter: Padmalal Gurugama, MBBS, M.S., M.D., MRCP, FRCPath, Consultant, Clinical Immunology and Allergy, Cambridge University Hospitals NHS Foundation Trust; Phase 1/2 and HAELO principal investigator
  • In interviews with 23 Phase 1/2 patients with a median of 2.4 years of follow-up since lonvo-z infusion, all reported their HAE was well controlled following lonvo-z, compared with 48% before lonvo-z. Among the 23 patients, 86% changed their definition of well controlled post-treatment, describing the shift from symptom management and medication dependence to having no attacks and reduced treatment burden.
  • Of the 23 patients, 22 (96%) described lonvo-z as an optimal therapy, citing its perceived effectiveness and its potential to serve as a one-time treatment that frees them from ongoing preventive treatment.
    
    
• Title: Long-Term Prophylaxis for Hereditary Angioedema: Treatment Use, Patterns, and Gaps in a U.S. Claims Database
  Poster Session: Monitor 18
  Date and Time: Friday, November 13, 2026, from 4:05 – 4:20 p.m. MT
  Presenter: Raffi Tachdjian, M.D., M.P.H., Associate Clinical Professor of Medicine and Pediatrics, Division of Allergy and Clinical Immunology, David Geffen School of Medicine, University of Los Angeles; HAELO principal investigator
  • In a U.S. claims analysis of 538 HAE patients taking LTP over a median of 1.2 years, almost 40% were found to be non-adherent. HAE attacks were similar in both adherent and non-adherent groups, indicating breakthrough attacks despite being on LTP treatment.
    
    
• Title: A One-Time Treatment of Lonvoguran Ziclumeran Improves Quality of Life in Patients with Hereditary Angioedema
  Poster Session: Monitor 17
  Date and Time: Friday, November 13, 2026, from 4:35 – 4:50 p.m. MT
  Presenter: William Lumry, M.D., Clinical Professor of Internal Medicine, Allergy and Immunology Division, University of Texas Southwestern Medical School, Medical Director, AARA Research Center; HAELO principal investigator
  • HAELO met its key secondary endpoint of change from baseline to week 28 in Angioedema Quality of Life (AE-QoL) total score. Additionally, lonvo-z demonstrated clinically meaningful improvements across all four domains of AE-QoL vs. placebo, with the improvement of quality of life being sustained over time.
  • Patients who received lonvo-z had a 23.5-point mean reduction in AE-QoL total score from baseline to week 28, compared with a 6.5-point mean reduction for placebo. A 6-point reduction on AE-QoL total score is considered to be the threshold for a clinically important difference.
    
    
• Title: Integrated Analysis of Lonvoguran Ziclumeran Phase 1/2/3 Studies in Hereditary Angioedema: Efficacy, Safety, and Tolerability
  Poster Session: Monitor 16
  Date and Time: Friday, November 13, 2026, from 5:35 – 5:50 p.m. MT
  Presenter: Aleena Banerji, M.D., Professor at Harvard Medical School, Director of Clinical Care, Center for Drug and Vaccine Allergy at Massachusetts General Hospital; HAELO principal investigator
  • Of the 93 patients who received a single one-time 50 mg dose of lonvo-z across the Phase 1/2 and Phase 3 HAELO studies, the overall attack rate reduction from baseline was 89% through up to four years of follow-up, and 97% of patients never initiated LTP after treatment. All patients were LTP-free at the latest follow-up.
  • Favorable safety and tolerability data were observed with the most common (>15% of patients) TEAEs being infusion-related reactions, headache, fatigue and nasopharyngitis. No new long-term risks have been identified.
    
    

All presentations will be made available on www.intelliatx.com on the Scientific Publications & Presentations page at the start of the respective sessions.

Investor Webcast Reminder
Intellia will host a live webcast on Thursday, November 12, 2026, at 8:00 a.m. MT / 10:00 a.m. ET to review data from the ACAAI presentations, share perspectives from key opinion leaders and discuss the company’s preparations for a planned commercial launch of lonvo-z in HAE.

To join the webcast, visit the Events page of the Investors & Media section of the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for approximately 90 days following the call.

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 in vivo CRISPR gene editing candidate that is intended to permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose that is administered in an outpatient setting. Lonvo-z has received five notable regulatory designations: Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) Designations 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. In September 2026, the FDA accepted the Biologics License Application (BLA) for lonvo-z and granted Priority Review with a PDUFA target action date of March 10, 2027.

About 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 may 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 biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies. The company’s mission is to transform the lives of people with severe diseases by developing and commercializing potentially curative treatments. With deep scientific, technical and clinical development experience, Intellia aims to reset the standard for medicine by durably treating the root causes of disease. 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 success and advancement of its program for lonvoguran ziclumeran or “lonvo-z” (also known as NTLA-2002) for the treatment of hereditary angioedema (“HAE”), including its expectations regarding review and approval of its biologic license application for lonvo-z for the treatment of HAE by the Prescription Drug User Fee Act target action date of March 10, 2027, its expectations regarding any planned U.S. launch of lonvo-z, and the potential of lonvo-z to become the first one-time treatment for HAE and to permanently lower kallikrein by inactivating the KLKB1 gene with a single dose that is administered in an outpatient setting.

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 the conduct of clinical studies and other development and commercialization requirements for its product candidates, including lonvo-z, including risks related to the ability to develop and successfully commercialize lonvo-z or any of Intellia’s product candidates; 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; 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.

Investor Contact:
Jason Fredette
Vice President, Investor Relations and Corporate Communications
Intellia Therapeutics, Inc.
jason.fredette@intelliatx.com

Media Contact:
Mike Tattory
Vice President
LifeSci Communications
mtattory@lifescicomms.com