Intellia gets FDA priority review for HAE drug

Rhea-AI Filing Summary

Intellia Therapeutics, Inc. (NTLA) reports that the U.S. FDA has accepted its Biologics License Application for lonvoguran ziclumeran (lonvo-z) for hereditary angioedema and granted Priority Review with a Prescription Drug User Fee Act target action date of March 10, 2027. FDA also indicated it is not currently planning to hold an advisory committee meeting on the application. The BLA is supported by a global Phase 3 HAELO trial of 80 patients, where a single 50 mg dose of lonvo-z achieved an 87% reduction in mean monthly attacks versus placebo and 62% of treated patients were attack-free and HAE-therapy-free over six months. All treatment-emergent adverse events in the lonvo-z arm were mild or moderate with no serious adverse events reported. Intellia highlights that, if approved, lonvo-z could become the first in vivo CRISPR-based therapy and the only one-time treatment for HAE.

Positive

• FDA Priority Review for lonvo-z BLA with a PDUFA target action date of March 10, 2027 accelerates potential U.S. market entry for Intellia’s hereditary angioedema therapy.
• Strong Phase 3 efficacy: lonvo-z achieved an 87% reduction in mean monthly HAE attacks versus placebo, with 62% of treated patients attack-free and HAE-therapy-free over six months.
• Favorable safety profile in Phase 3: all treatment-emergent adverse events in the lonvo-z arm were mild or moderate, with no serious adverse events reported.

Negative

• None.

8-K Event Classification

3 items: 7.01, 8.01, 9.01

3 items

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Item 8.01 Other Events Other

Voluntary disclosure of events the company deems important to shareholders but not covered by other items.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, or exhibit attachments filed with this report.

Key Figures

PDUFA target action date: March 10, 2027 Phase 3 HAELO trial enrollment: 80 patients Lonvo-z dose in Phase 3: 50 milligrams +5 more

8 metrics

PDUFA target action date March 10, 2027 FDA Priority Review for lonvo-z BLA for hereditary angioedema

Phase 3 HAELO trial enrollment 80 patients Global Phase 3 HAELO trial fully enrolled in nine months

Lonvo-z dose in Phase 3 50 milligrams One-time dose evaluated in adults and adolescents aged 16+ with Type 1 or Type 2 HAE

Reduction in mean monthly HAE attacks 87% Lonvo-z vs placebo during weeks 5 to 28 efficacy evaluation period

Attack-free and HAE-therapy-free patients (lonvo-z arm) 62% Proportion over six-month efficacy evaluation period in the lonvo-z arm

Attack-free and HAE-therapy-free patients (placebo arm) 11% Proportion over six-month efficacy evaluation period in the placebo arm

Estimated HAE prevalence 1 in 50,000 people Estimated frequency of hereditary angioedema in the general population

Regulatory designations received 5 designations Orphan Drug, RMAT, Innovation Passport, PRIME and EU Orphan Drug Designation for lonvo-z

Key Terms

Priority Review, Biologics License Application, PDUFA target action date, in vivo CRISPR-based therapy, +2 more

6 terms

Priority Review regulatory

"granted the BLA Priority Review with a PDUFA target action date"

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

Biologics License Application regulatory

"FDA has accepted the Biologics License Application (BLA) for lonvo-z"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

PDUFA target action date regulatory

"with a PDUFA target action date of March 10, 2027"

The PDUFA target action date is the deadline set by the U.S. Food and Drug Administration (FDA) by which it aims to decide whether to approve or reject a new drug application. This date helps investors gauge when a company’s new medication might reach the market, potentially influencing sales and revenue expectations. It acts as a key milestone signaling progress in the drug approval process.

in vivo CRISPR-based therapy medical

"would be the world’s first in vivo CRISPR-based therapy"

Regenerative Medicine Advanced Therapy regulatory

"Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) Designations"

Regenerative Medicine Advanced Therapy (RMAT) is a U.S. regulatory designation for cell, gene, and tissue‑based therapies intended to treat serious or life‑threatening conditions; it gives developers a “fast lane” with more frequent agency interaction and eligibility for accelerated review pathways. For investors, an RMAT label signals that a therapy may reach market faster and face less regulatory uncertainty than a standard program, which can raise the potential value and reduce timeline risk—though it is not a guarantee of approval.

Orphan Drug Designation regulatory

"as well as Orphan Drug Designation (ODD) by the European Commission"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did the FDA decide regarding Intellia Therapeutics (NTLA) lonvo-z application?

The FDA accepted the Biologics License Application for Intellia’s lonvo-z for hereditary angioedema and granted Priority Review, with a PDUFA target action date of March 10, 2027. FDA also advised it is not currently planning to hold an advisory committee meeting on the application.

What efficacy did lonvo-z show in Intellia (NTLA) Phase 3 HAELO trial?

In the Phase 3 HAELO trial, lonvo-z demonstrated an 87% reduction in mean monthly hereditary angioedema attacks versus placebo from weeks 5 to 28. 62% of lonvo-z patients were attack-free and HAE-therapy-free over six months, compared with 11% in the placebo arm.

What is the safety profile of lonvo-z reported by Intellia (NTLA)?

As of the February 10, 2026 cutoff, all treatment-emergent adverse events in the lonvo-z arm were mild or moderate, with no serious adverse events. The most common events included infusion-related reactions, headache, fatigue, back pain and upper respiratory tract infection.

How many patients were enrolled in Intellia (NTLA) Phase 3 HAELO study and what dose was used?

The global Phase 3 HAELO study was fully enrolled with 80 patients in nine months. It evaluated the efficacy and safety of a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 years and older with Type 1 or Type 2 hereditary angioedema.

Why is lonvo-z potentially significant for Intellia (NTLA) and HAE treatment?

If approved, lonvo-z would be the world’s first in vivo CRISPR-based therapy and the only one-time treatment for hereditary angioedema. It is designed to permanently lower kallikrein by inactivating the KLKB1 gene using CRISPR/Cas9 technology.

What regulatory designations has lonvo-z received according to Intellia (NTLA)?

Lonvo-z has received five regulatory designations: U.S. Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations, the U.K. MHRA Innovation Passport, EMA PRIME (Priority Medicines) designation, and European Commission Orphan Drug Designation.

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

Understanding 8-K Material Events →

See more from StockTitan in Google Search and AI answers. Adds StockTitan as a preferred source · opens Google

Add on Google Not now

Available on EDGAR 09/08/2026 - 04:05 PM Accepted by SEC EDGAR 09/08/2026 - 04:05 PM Learn about SEC filing dates

0001652130false00016521302026-09-082026-09-08

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): September 08, 2026

INTELLIA THERAPEUTICS, INC.

(Exact name of Registrant as Specified in Its Charter)

Delaware			001-37766	36-4785571
(State or Other Jurisdiction of Incorporation)			(Commission File Number)	(IRS Employer Identification No.)
400 Technology Square, Suite 100
Cambridge, Massachusetts				02139
(Address of Principal Executive Offices)				(Zip Code)

Registrant’s Telephone Number, Including Area Code: 857 285-6200

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class		Trading Symbol(s)		Name of each exchange on which registered
Common Stock, par value $0.0001 per share		NTLA		The Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 7.01 Regulation FD Disclosure.

On September 8, 2026, Intellia Therapeutics, Inc. (the “Company” or “Intellia”) issued a press release titled “Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review for Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE).” A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

The information under this Item 7.01, including Exhibit 99.1 hereto, is being furnished herewith and shall not be deemed “filed” for the purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section, nor shall such information be deemed incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.

Item 8.01 Other Events.

Also on September 8, 2026, the Company announced that the U.S. Food and Drug Administration (“FDA”) has accepted the Biologics License Application (“BLA”) for lonvoguran ziclumeran (“lonvo-z”) and granted the BLA Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of March 10, 2027. Additionally, FDA has advised the Company that it is not currently planning to hold an advisory committee to discuss the application. If approved, lonvo-z would be the world’s first in vivo CRISPR-based therapy and the only one-time treatment for hereditary angioedema (“HAE”).

Forward Looking Statements

This Current Report on Form 8-K and certain of the materials furnished or filed herewith contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding: the success and advancement of its program for lonvo-z (formerly known as NTLA-2002) for the treatment of HAE, including its expectations regarding review and approval of its BLA for lonvo-z, such as whether the FDA will hold an advisory committee to discuss the BLA and the timing of such review and approval based on the PDUFA target action date of March 10, 2027 for the BLA; its belief that lonvo-z could fundamentally change the way HAE is treated and has the potential to become the world's first approved in vivo CRISPR-based therapy; and its expectations regarding its preparations for and the potential success of the commercial launch of lonvo-z, if approved.

Any forward-looking statements in this current report on Form 8-K 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 or regulatory filings 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 and 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 current report on Form 8-K is as of the date of the report, and Intellia undertakes no duty to update this information unless required by law.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.		Description
99.1		Press release dated September 8, 2026 titled “Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review for Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE)”
104		Cover Page Interactive Data File (embedded within the Inline XBRL document)

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

			Intellia Therapeutics, Inc.
Date:	September 8, 2026	By:	/s/ John M. Leonard
			Name: John M. Leonard Title: Chief Executive Officer and President

Exhibit 99.1

Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review for Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE)

•FDA sets Prescription Drug User Fee Act (PDUFA) date of March 10, 2027

•Positions lonvo-z to be the world’s first in vivo CRISPR-based therapy and the only one-time HAE treatment, if approved

CAMBRIDGE, Mass., September 8, 2026 -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lonvo-z and granted the BLA Priority Review with a PDUFA target action date of March 10, 2027. Additionally, FDA has advised the company that it is not currently planning to hold an advisory committee to discuss the application. If approved, lonvo-z would be the world’s first in vivo CRISPR-based therapy and the only one-time treatment for HAE.

“Today marks an important milestone for the patients we are committed to serving and for Intellia’s pioneering work in the field of in vivo gene editing,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “Backed by compelling Phase 3 data, we believe lonvo-z could fundamentally change the way HAE is treated and are excited by its potential to become the world's first approved in vivo CRISPR-based therapy. With the FDA’s Priority Review underway, our team is well prepared to deliver this one-time treatment to patients who are waiting for new options.”

Joshua Jacobs, M.D., Medical Director, Allergy and Asthma Clinical Research, Inc., and a HAELO trial investigator, added, “HAE is an unpredictable disease that can be responsible for profound disability and place patients at risk for fatal attacks. Today’s announcement is exciting because it advances us one step closer to

Page 1 of 4 intelliatx.com

potentially having a one-time treatment option available for patients who continue to be burdened by this chronic disease.”

The BLA is supported by positive data from Intellia’s global Phase 3 HAELO clinical trial, which was fully enrolled with 80 patients in just nine months and was designed to evaluate the efficacy and safety of a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 years and older with Type 1 or Type 2 HAE. HAELO met its primary and all key secondary endpoints, demonstrating an 87% reduction (p<0.0001) in mean monthly attacks for lonvo-z compared with placebo during the efficacy evaluation period (weeks 5 to 28). In addition, 62% of patients in the lonvo-z arm were entirely attack free and HAE therapy free for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm (p<0.0001). As of the February 10, 2026 data cutoff, all patients who received lonvo-z at baseline or in crossover after week 28 remained free from long-term prophylaxis therapy.

Favorable safety and tolerability data were observed for lonvo-z as of the data cutoff. The most common treatment emergent adverse events during the primary observation period (infusion through week 28) that were higher in the lonvo-z group compared to placebo were infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection. All reported treatment emergent adverse events were mild or moderate and there were no serious adverse events observed in the lonvo-z arm.

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.

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

Page 2 of 4 intelliatx.com

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” (formerly known as NTLA-2002) for the treatment of hereditary angioedema (“HAE”), including its expectations regarding review and approval of its biologics license application (“BLA”) for lonvo-z, such as whether the FDA will hold an advisory committee to discuss the BLA and the timing of such review and approval based on the Prescription Drug User Fee Act ("PDUFA") target action date of March 10, 2027 for the BLA; its belief that lonvo-z could fundamentally change the way HAE is treated and has the potential to become the world's first approved in vivo CRISPR-based therapy; and its expectations regarding its preparations for and the potential success of the commercial launch of lonvo-z, if approved.

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 review and approval of the BLA for lonvo-z and 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

Page 3 of 4 intelliatx.com

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 recent 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

Page 4 of 4 intelliatx.com

Filing Exhibits & Attachments

2 documents

Press Releases

• EX-99.1 EX-99.1 34.6 KB

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA WITH EMBEDDED LINKBASES DOCUMENT 24.4 KB