Intellia Therapeutics Completes Enrollment in Phase 3 MAGNITUDE-2 Trial of Nexiguran Ziclumeran (Nex-z) for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)

MAGNITUDE-2 measures nerve impairment at month 18 and blood transthyretin levels at day 29 after treatment.

Rhea-AI Summary

Intellia Therapeutics (Nasdaq: NTLA) completed enrollment in its Phase 3 MAGNITUDE-2 trial of nexiguran ziclumeran (nex-z) for hereditary transthyretin amyloidosis with polyneuropathy.

The randomized, double-blind, placebo-controlled trial was designed to evaluate efficacy and safety in approximately 60 patients. Adults were randomized 1:1 to a single 55 mg infusion of nex-z or placebo. The primary endpoints measure change in a modified score of nerve impairment at month 18 and change in blood transthyretin protein levels at day 29. Nex-z is an investigational gene-editing treatment designed to inactivate the TTR gene in the liver. It is also being evaluated in the Phase 3 MAGNITUDE trial for transthyretin amyloidosis with cardiomyopathy.

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• Moderate pointPhase 3 MAGNITUDE-2 enrollment completed, reaching a clinical development milestone for nex-z in ATTRv-PN.
• Minor pointPhase 3 MAGNITUDE also evaluates nex-z in patients with transthyretin amyloidosis with cardiomyopathy.

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Key Figures

Planned enrollment: Approximately 60 patients Randomization: 1:1 Dose: Single 55 mg infusion +1 more

Planned enrollment

Approximately 60 patients

MAGNITUDE-2 Phase 3 trial

Randomization

1:1

Nex-z or placebo

Dose

Single 55 mg infusion

Nex-z treatment arm

Primary endpoint assessments

Month 18 and day 29

Neuropathy impairment score and serum TTR level, respectively

Key Terms

crispr gene editing, in vivo, placebo-controlled, double-blind

4 terms

crispr gene editing technical

"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.

in vivo technical

"investigational in vivo CRISPR-based therapeutic candidate"

In vivo describes tests or experiments performed inside a living organism, such as an animal or human, to observe how a drug, device or biological process behaves in a real, functioning body. Investors care because in vivo results reveal safety, effectiveness and possible side effects that lab tests cannot, much like road-testing a prototype car in traffic rather than only on a bench — outcomes can strongly influence regulatory approval, clinical success and a company’s valuation.

placebo-controlled medical

"randomized, double-blind, placebo-controlled trial"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

double-blind medical

"randomized, double-blind, placebo-controlled trial"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

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

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CAMBRIDGE, Mass., Nov. 04, 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 the completion of enrollment in MAGNITUDE-2, the Phase 3 clinical trial of nex-z in patients with ATTRv-PN. 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 rapid, deep, consistent and potentially lifelong reduction in TTR protein after a one-time treatment.

“We are grateful to the patients, caregivers and investigators who are participating in MAGNITUDE-2,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “Completing enrollment in this trial is a pivotal step toward bringing a potentially transformative one-time treatment to people living with ATTRv-PN, a debilitating and life-threatening disease with significant unmet need.”

“While disease-modifying therapies have advanced the standard of care for ATTRv-PN, many patients continue to progress,” said John L. Berk, M.D., Professor Emeritus of Medicine, Amyloidosis Center, Boston University School of Medicine. “An agent that can quickly, substantially and consistently reduce TTR after a single dose would be a meaningful advancement for patients. Completing enrollment in the MAGNITUDE-2 trial is an exciting milestone toward that goal.”

MAGNITUDE-2 is a randomized, double-blind, placebo-controlled trial that was designed to evaluate the efficacy and safety of nex-z in approximately 60 patients with ATTRv-PN. Adult patients with ATTRv-PN were randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. The primary endpoints of the trial are the change in modified neuropathy impairment score at month 18 and the change in serum TTR levels at day 29. Patients are eligible for optional blinded crossover following month 18. For more information on MAGNITUDE-2 (NCT06672237), please visit clinicaltrials.gov.

Nex-z is also being investigated in MAGNITUDE, a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of nex-z in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). For more information on MAGNITUDE (NCT06128629), please visit clinicaltrials.gov.

About Nex-z
Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for ATTR-CM and/or ATTRv-PN. Nex-z is designed to inactivate the TTR gene that encodes the TTR protein and is being investigated in MAGNITUDE and MAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTRv-PN, respectively. Nex-z has received an Orphan Drug Designation (ODD) and RMAT Designation from the U.S. Food and Drug Administration (FDA) and an ODD from the European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc.

About Transthyretin Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal TTR protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as ATTRv-PN, which can lead to nerve damage, or ATTRv-CM, which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.

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 safety, tolerability, efficacy, success and advancement of its clinical programs for nexiguran ziclumeran (“nex-z”) for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (“ATTRv-PN”), including the potential of nex-z to halt and reverse ATTRv-PN by driving a rapid, deep, consistent and potentially lifelong reduction in TTR protein after a one-time treatment.

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 nex-z, including risks related to the ability to develop and successfully commercialize nex-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, 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

FAQ

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

What milestone did Intellia Therapeutics reach in the MAGNITUDE-2 nex-z trial?

Intellia completed enrollment in Phase 3 MAGNITUDE-2, which evaluates nex-z in hereditary transthyretin amyloidosis with polyneuropathy. The trial was designed to assess efficacy and safety in approximately 60 patients.

Can patients cross over to nex-z in Intellia's MAGNITUDE-2 trial?

Patients are eligible for optional blinded crossover following month 18 in MAGNITUDE-2.