Intellia Therapeutics Presents Longer-Term Clinical Data for Lonvoguran Ziclumeran (lonvo-z); Hereditary Angioedema (HAE) Patient-Focused Research at AAAAI 2026

Rhea-AI Summary

Intellia Therapeutics (Nasdaq: NTLA) presented four posters at AAAAI 2026 on lonvoguran ziclumeran (lonvo-z; NTLA-2002) for hereditary angioedema (HAE). A pooled Phase 1/2 cohort (n=32) given a one-time 50 mg dose showed durable plasma kallikrein reductions and a mean monthly attack rate ≤0.2 through up to three years.

Key readouts: 96% mean reduction in HAE attacks versus baseline, 31/32 (97%) attack-free and LTP-free at data cutoff, and 86% of 28 patients with >6 months follow-up attack-free and LTP-free for >6 months. A patient survey (n=100) highlighted ongoing treatment burden.

Positive

• Mean monthly attack rate ≤0.2 through up to 3 years
• 96% mean reduction in HAE attacks from baseline
• 31 of 32 patients (97%) attack-free and LTP-free at data cutoff
• 86% of 28 patients with >6 months follow-up attack-free and LTP-free >6 months

Negative

• Small pooled sample size (n=32) limits generalizability
• Follow-up duration variable; some patients had as little as 2 months follow-up
• Survey (n=100) shows 34% report ≥1 attack per month, underscoring remaining unmet need
• Durability claims are ongoing and based on interim follow-up, not final long-term outcomes

News Market Reaction – NTLA

-11.88% 2.0x vol

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-11.88% Session close to close

-8.0% Trough in 11 hr 44 min

$1.82B Market Cap

2.0x Rel. Volume

In the Mar 3 session, NTLA declined 11.88%, reflecting a significant negative market reaction. Argus tracked a trough of -8.0% from its starting point during tracking. Our momentum scanner triggered 50 alerts that day, indicating high trading interest and price volatility. Trading volume was elevated at 2.0x the daily average, suggesting increased selling activity.

Data tracked by StockTitan Argus on the day of publication.

Market Context

The stock dropped -11.9% in the session following this news. A negative reaction despite encouraging...

Analysis

The stock dropped -11.9% in the session following this news. A negative reaction despite encouraging lonvo-z durability data would fit a pattern where safety history in related programs and financing considerations weighed on sentiment. Clinical headlines have previously produced both strong downside on adverse safety events and modest upside on positive data. In that context, traders may have focused on broader risk, trial complexity or prior gains rather than the HAE efficacy profile, leaving room for large downside swings around news.

Key Figures

Dose: 50 mg Sample size: 32 patients Attack reduction: 96% reduction +5 more

8 metrics

Dose 50 mg One-time lonvo-z dose in Phase 1/2 HAE analyses

Sample size 32 patients Pooled Phase 1/2 lonvo-z analysis in HAE

Attack reduction 96% reduction Mean reduction in HAE attacks from baseline through last follow-up

Attack-free patients 31 of 32 (97%) Patients both attack-free and LTP-free at data cutoff

Attack rate ≤0.2 attacks/month Mean monthly attack rate across 32 lonvo-z patients

Durable response 2 months to 3 years Range of attack-free and LTP-free periods with ongoing follow-up

6+ month freedom 86% of 28 patients Attack-free and LTP-free for >6 months after one-time 50 mg lonvo-z

Disease burden survey 34% ≥1 attack/month; 20% attack-free Reported attacks in prior year among 100 surveyed HAE patients

Previous Clinical trial Reports

5 past events · Latest: Mar 02 (Positive)

Same Type Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Mar 02	Hold lifted MAGNITUDE	Positive	+12.1%	FDA removed clinical hold on MAGNITUDE Phase 3 ATTR-CM trial with safety changes.
Jan 27	Hold lifted MAG-2	Positive	+6.3%	FDA lifted clinical hold on MAGNITUDE-2 Phase 3 ATTRv-PN trial after protocol changes.
Nov 10	Longer-term nex-z data	Positive	+2.2%	Reported 3-year Phase 1 nex-z data with deep TTR reduction and clinical stability.
Nov 08	Pooled lonvo-z data	Positive	+2.2%	Presented pooled Phase 1/2 lonvo-z HAE data showing high attack-free, LTP-free rates.
Oct 27	MAGNITUDE safety pause	Negative	-42.2%	Paused MAGNITUDE trials after Grade 4 liver enzymes and a serious safety event.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Clinical trial headlines have generally produced directionally consistent moves: positive updates on nex-z and lonvo-z saw gains, while the MAGNITUDE safety pause coincided with a sharp decline.

Recent Company History

Over the past months, Intellia’s key news flow has centered on clinical trial progress for nexiguran ziclumeran and lonvoguran ziclumeran. Events include a safety-driven pause of the MAGNITUDE programs on Oct 27, 2025, followed by positive longer-term Phase 1 data in ATTR-CM and pooled Phase 1/2 data in HAE in Nov 2025. In Jan–Mar 2026, sequential FDA lifts of clinical holds on MAGNITUDE-2 and MAGNITUDE allowed Phase 3 enrollment to resume with enhanced safety monitoring. Today’s HAE durability data extend this clinical narrative around one-time gene-editing treatments.

Key Terms

CRISPR gene editing, Phase 1/2, plasma kallikrein, C1-esterase inhibitor, +4 more

8 terms

CRISPR gene editing technical

"focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies"

CRISPR gene editing is a laboratory technique that uses a guided molecular “scissor” to change DNA at specific locations, allowing scientists to add, remove, or correct genetic code. For investors, it matters because it underpins a new class of therapies and biotech products with high upside if they work, but also carries long development timelines, regulatory uncertainty, and technical risks that can create large swings in company value.

Phase 1/2 medical

"In this pooled Phase 1/2 analysis (n=32), a one-time 50 mg dose"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

plasma kallikrein medical

"led to deep, stable and durable reductions in plasma kallikrein across all patients"

Plasma kallikrein is an enzyme in the blood that acts like a pair of molecular scissors, cutting a larger protein to release bradykinin, a small molecule that widens blood vessels and promotes swelling and fluid movement. Its activity helps regulate inflammation, blood pressure and clotting pathways, so drugs that block or modify it can change disease symptoms and safety profiles—making it an important therapeutic target and risk factor for investors watching drug development or regulatory news.

C1-esterase inhibitor medical

"HAE with significant C1-esterase inhibitor deficiency generates excess plasma kallikrein"

A C1-esterase inhibitor is a naturally occurring protein that acts like a brake on parts of the immune system to prevent excessive swelling and inflammation; therapeutically, it is given as a biologic drug to treat conditions such as hereditary angioedema. Investors care because it is a specialized, often high-cost biologic with complex manufacturing, tight regulation, and clear clinical endpoints—so trial results, approvals, supply and reimbursement decisions can materially affect a company’s revenue and valuation.

prekallikrein medical

"an 85% reduction in prekallikrein was shown to reduce peak free kallikrein"

Prekallikrein is a naturally occurring blood protein that is the inactive precursor to an enzyme involved in blood clotting and inflammation. Think of it as a dormant tool in a first-aid kit that, when activated, helps trigger responses to injury or infection. Investors watch it because changes in its levels or activity can signal disease, serve as a diagnostic biomarker, or be a target for drugs that treat clotting disorders, inflammation, or related cardiovascular conditions.

bradykinin medical

"compared to healthy individuals, directly correlating with bradykinin increases"

Bradykinin is a naturally occurring small protein in the body that signals blood vessels to widen and makes tissues feel tender or swollen, much like a neighborhood alarm that opens water valves and calls attention to damage. Investors care because changes in bradykinin levels can drive drug effects and side effects—such as swelling, low blood pressure, or cough—so therapies that alter it affect safety, labeling, and market value.

long-term prophylaxis medical

"89% of whom were on long-term prophylaxis therapies and 11% on on-demand"

Long-term prophylaxis is an ongoing preventive treatment given regularly to keep a disease or condition from occurring or worsening over months or years, like taking a daily medicine to prevent attacks. For investors, it signals a chronic-use market with predictable, recurring demand, potential for steady revenue, and different regulatory and pricing dynamics than one-time treatments—similar to a subscription service versus a single purchase.

hereditary angioedema medical

"50 mg in Patients with Hereditary AngioedemaSession: Allergic Skin Diseases"

A rare inherited disorder that causes sudden, painful swelling under the skin or in internal tissues, including the airway, because a natural blood‑control protein is missing or not working. Attacks can be unpredictable and sometimes life‑threatening, so people often need ongoing medication or emergency treatment. For investors, hereditary angioedema represents a niche but stable market for specialized therapies, diagnostics, and emergency care solutions.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Presentations include three-year follow-up data from patients receiving a one-time 50 milligram (mg) dose of lonvo-z and survey findings highlighting patients’ continued treatment burden and unmet needs

CAMBRIDGE, Mass., March 03, 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 details about its presentation of four posters at the 2026 American Academy of Allergy, Asthma & Immunology Annual Meeting (AAAAI) that took place this past weekend in Philadelphia, Pennsylvania. All posters are available on intelliatx.com on the Scientific Publications & Presentations page.

Poster Presentation Details:

• Title: Long-Term Durability and Safety of Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) 50 mg in Patients with Hereditary Angioedema
  Session: Allergic Skin Diseases
  Poster Number: 061
  Presenter: Markus Magerl, M.D, Professor, Head of Clinical Trials, Charité - Universitätsmedizin Berlin Institute of Allergology
  • In this pooled Phase 1/2 analysis (n=32), a one-time 50 mg dose of lonvo-z led to deep, stable and durable reductions in plasma kallikrein across all patients with up to three years of follow-up.
  • Across the 32 patients, the mean monthly attack rate was consistently ≤0.2, representing a mean reduction of 96% in HAE attacks from baseline through last follow-up. Of the 32 patients, 31 (97%) were both attack-free and LTP-free as of the data cutoff, with the attack-free and LTP-free periods ranging from 2 months to 3 years with follow-up ongoing.
• Title: Evolving Treatment Goals to Achieve Freedom from Attacks and Long-Term Prophylaxis Following a One-Time Treatment with Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002)
  Session: Allergic Skin Diseases
  Poster Number: 005
  Presenter: Aleena Banerji, M.D., Professor at Harvard Medical School, Clinical Director of the Allergy and Clinical Immunology Unit at Massachusetts General Hospital
  • Recent research shows that achieving an attack-free status and simultaneously minimizing treatment burden are primary treatment goals for HAE experts and patients.
  • Of the 28 patients with >6 months of follow-up after receiving a one-time 50 mg treatment of lonvo-z in a pooled Phase 1/2 analysis, 86% were attack-free and LTP-free for >6 months, a timeframe suggested by patients to be clinically meaningful.
• Title: Quantitative Systems Biology Modeling Estimates Extent of Excessive Kallikrein Generation in Hereditary Angioedema Patients
  Session: Allergic Skin Diseases
  Poster Number: 003
  Presenter: Allen Kaplan, M.D., Professor, Department of Medicine, Medical University of South Carolina
  • This model suggested that HAE with significant C1-esterase inhibitor deficiency generates excess plasma kallikrein compared to healthy individuals, directly correlating with bradykinin increases.
  • In this model, an 85% reduction in prekallikrein was shown to reduce peak free kallikrein and peak bradykin to near normal ranges. This level of reduction in prekallikrein is consistent with what has been observed clinically with a 50 mg dose of lonvo-z.
• Title: Chronic Medications Pose Challenges for People Living with Hereditary Angioedema
  Session: Bridging Evidence for Real World Impact
  Poster Number: 716
  Presenter: Paula Busse, M.D., Professor, Department of Medicine, Division of Clinical Immunology, Mount Sinai Hospital
  • Among 100 surveyed U.S. patients with HAE, 89% of whom were on long-term prophylaxis therapies and 11% of whom were on on-demand therapies only, 34% reported having at least one attack per month and only 20% reported being attack free in the prior year.
  • Most respondents indicated that eliminating lifetime chronic medication use and enhancing efficacy are the most important ways to improve their current therapy.

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 that is currently being investigated in HAELO, a Phase 3 clinical trial in HAE, and is 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 Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage 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.

Contact:

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

FAQ

What were the three-year durability results for lonvo-z (NTLA) presented at AAAAI 2026?

Data showed durable kallikrein reductions and low attack rates through up to three years in a pooled cohort. According to Intellia, a one-time 50 mg dose produced a mean monthly attack rate ≤0.2 and sustained biomarker reductions with follow-up ongoing.

How many patients were attack-free after a one-time 50 mg lonvo-z dose in the NTLA Phase 1/2 pooled analysis?

Of 32 patients, 31 (97%) were attack-free and LTP-free at the data cutoff in the pooled analysis. According to Intellia, attack-free and LTP-free periods ranged from two months to three years, with follow-up ongoing.

What percentage of patients remained attack-free for >6 months after lonvo-z (NTLA) treatment?

Among 28 patients with >6 months follow-up, 86% were attack-free and LTP-free for more than six months. According to Intellia, patients considered that timeframe clinically meaningful for reduced treatment burden.

What safety or biomarker effects did the lonvo-z 50 mg dose show in the NTLA presentation?

The 50 mg dose produced deep, stable reductions in plasma kallikrein and prekallikrein consistent with modeled effects. According to Intellia, modeled 85% prekallikrein reduction aligns with observed reductions and near-normal bradykinin ranges.

What did the AAAAI 2026 patient survey reveal about HAE treatment burden relevant to NTLA (lonvo-z)?

A U.S. survey (n=100) found 34% reported at least one attack per month and only 20% were attack-free in the prior year. According to Intellia, most respondents prioritized eliminating lifetime chronic medication use and improving efficacy.

What are the limitations of the lonvo-z results presented by Intellia (NTLA) at AAAAI 2026?

Key limitations include small pooled sample size (n=32) and variable follow-up durations across patients. According to Intellia, follow-up is ongoing, so longer-term, larger controlled data are needed to confirm durability and safety.