On February 26, 2026 Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, reported business updates and financial results for the fourth quarter and year ended December 31, 2025.

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"2025 was a time of accomplishment and resiliency for Intellia as we presented encouraging longer term Phase 1/2 clinical data for both lonvo-z and nex-z, rapidly enrolled patients in our three Phase 3 trials, commenced activities to prepare for a potential lonvo-z launch in HAE and responded to the clinical holds on our nex-z Phase 3 trials late in the year," said Intellia President and Chief Executive Officer John Leonard, M.D. "We expect the year ahead to be a pivotal one, highlighted by our topline Phase 3 data and planned BLA submission for lonvo-z, which has the potential to transform the HAE treatment paradigm by freeing most patients from both their attacks and chronic therapy. Additionally, we are focused on resuming our forward momentum with nex-z by completing patient enrollment in MAGNITUDE-2 and resolving the clinical hold on MAGNITUDE."

Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE)

Lonvo-z is a wholly owned, investigational in vivo CRISPR-based therapeutic candidate designed to inactivate the KLKB1 gene in the liver, drive consistent, deep and potentially lifelong reduction in kallikrein levels, and dramatically reduce or eliminate HAE attacks via a one-time treatment.

In the fourth quarter at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting, Intellia presented positive clinical data from a pooled analysis of all patients who received a 50 milligram (mg) dose of lonvo-z in the company’s ongoing Phase 1/2 clinical trial in patients with HAE. Observations from the analysis included durable reductions in plasma kallikrein in all patients at month 24, a high percentage of patients achieving prolonged attack-free status (for at least seven months and up to 32 months for patients with the longest follow-up) and a well-tolerated safety profile for lonvo-z.
Intellia sponsored a blinded market research study in late 2025 with 104 U.S. HAE patients and 151 U.S. HAE treating physicians. After reviewing a blinded target product profile aligned with lonvo-z’s Phase 1/2 clinical data, 99% of patients said they would be at least somewhat likely – and 64% said they would be extremely or very likely – to take lonvo-z if prescribed. Additionally, 92% of healthcare providers indicated they would prescribe a product with this profile, estimating they would prescribe it to 54% of the approximately 4,000 patients with HAE under their care.
This weekend, the company will present four posters at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting taking place February 27 – March 2 in Philadelphia, Pennsylvania (poster numbers 003, 005, 061 and 716). The presentations include three-year follow-up data from patients receiving a one-time 50 mg dose of lonvo-z and new survey findings assessing the chronic treatment burden and unmet needs among patients living with HAE.
Dosing in the global HAELO Phase 3 clinical trial was initiated in January 2025 and was completed in September 2025, with 80 patients enrolled. Intellia expects to report HAELO topline data by mid-2026 and, if the data are supportive, submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the second half of 2026.
To prepare for a planned launch in the first half of 2027, the company has expanded its field medical team, strengthened engagement with treating physicians and patient advocacy groups, initiated its payer engagements and advanced its launch strategy. In 2026, the company intends to build its field sales and reimbursement teams, finalize its distribution models, identify U.S. treatment centers and advance its pricing and access planning strategy.
Nexiguran Ziclumeran (Nex-z) for Transthyretin (ATTR) Amyloidosis

Nex-z is an investigational in vivo CRISPR-based therapeutic candidate designed to inactivate the TTR gene in the liver, thereby preventing the production of transthyretin (TTR) protein. Nex-z offers the possibility of halting and reversing disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a one-time treatment. Intellia leads the development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc.

In the fourth quarter at the American Heart Association (AHA) Scientific Sessions, Intellia presented positive follow-up data from the ongoing Phase 1 clinical trial of nex-z in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). Observations from these longer-term data included consistent and durable reductions in serum TTR through up to three years of follow up, stability or improvement in multiple markers of cardiomyopathy for most patients and encouraging mortality data.
As previously reported, on October 29, 2025, the FDA placed a clinical hold on the Investigational New Drug (IND) applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for patients with ATTR-CM and hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), respectively.
On January 27, 2026, the company announced the FDA lifted the clinical hold on the IND for MAGNITUDE-2. The company is in the process of resuming MAGNITUDE-2 enrollment.
Intellia’s engagement with FDA is ongoing regarding the clinical hold on the IND for the MAGNITUDE Phase 3 clinical trial of nex-z for patients with ATTR-CM. The company plans to provide an update once alignment has been achieved on the path forward for this program.
Fourth Quarter and Full-Year 2025 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $605.1 million as of December 31, 2025, compared to $861.7 million as of December 31, 2024. The company’s cash, cash equivalents and marketable securities as of December 31, 2025 are expected to fund operations into the second half of 2027 and through lonvo-z’s anticipated U.S. commercial launch for HAE.

Collaboration Revenue: Collaboration revenue was $23.0 million for the fourth quarter of 2025, compared to $12.9 million for the fourth quarter of 2024. The increase was primarily driven by the recognition of $9.0 million in revenue related to the termination of the license and collaboration agreement with SparingVision SAS and an increase in cost reimbursements related to the company’s collaboration with Regeneron.
R&D Expenses: Research and development (R&D) expenses were $88.7 million for the fourth quarter of 2025, compared to $116.9 million for the fourth quarter of 2024. The $28.2 million decrease was primarily driven by employee-related expenses, stock-based compensation, research materials and contracted services, partially offset by an increase in facility-related expenses as well as clinical trial expenses related to nex-z. Stock-based compensation expense included in R&D expenses was $10.5 million for the fourth quarter of 2025.
G&A Expenses: General and administrative (G&A) expenses were $33.1 million for the fourth quarter of 2025, compared to $32.4 million for the fourth quarter of 2024. Stock-based compensation expense included in G&A expenses was $6.2 million for the fourth quarter of 2025.
Net Loss: Net loss was $95.8 million for the fourth quarter of 2025, compared to $128.9 million for the fourth quarter of 2024.

Conference Call Information
The company will host a conference call and webcast today at 8:00 a.m. ET to discuss recent updates and the company’s fourth quarter and full-year 2025 financial results. To join the webcast, please visit the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com. To join by phone, U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726 approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call. A replay of the webcast will be available for approximately 90 days.

(Press release, Intellia, FEB 26, 2026, View Source [SID1234663078])