Intellia Presents Updated Interim Data from the

• Data introduced in late-breaking oral presentation demonstrated deep and constant TTR discount following a single dose of NTLA-2001 in sufferers with ATTR amyloidosis with cardiomyopathy (ATTR-CM)
• Greater than 90% imply serum TTR reductions after a single dose of NTLA-2001 had been sustained at each doses examined, with follow-up now reaching 4 to 6 months
• NTLA-2001 was typically well-tolerated

CAMBRIDGE, Mass., Nov. 05, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a number one clinical-stage genome enhancing firm targeted on growing doubtlessly healing therapeutics leveraging CRISPR-based applied sciences, immediately introduced extra interim outcomes from an ongoing Phase 1 scientific trial of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome enhancing remedy in growth as a single-dose therapy for transthyretin (ATTR) amyloidosis in collaboration with Regeneron Pharmaceuticals. Results had been introduced in a Late-Breaking Science oral presentation at the American Heart Association (AHA) Scientific Sessions 2022, held November 5 – 7 in Chicago, Illinois.

The interim information from the dose-escalation portion of the Phase 1 research embrace 12 grownup sufferers with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III coronary heart failure. The information introduced had been as of an information cutoff date of August 25, 2022. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 had been administered through a single intravenous infusion, and the change from baseline in serum transthyretin (TTR) protein focus was measured for every affected person.

Administration of NTLA-2001 led to deep and sturdy reductions in serum TTR by day 28 as follows:

^{*Mean (min, max) % serum TTR discount by day 28 for 0.7 mg/kg dose degree (n=9) was 93% (91%, 97%).}

These deep reductions in serum TTR had been sustained by means of the commentary interval, with affected person follow-up ranging from 4 to 6 months. These information spotlight NTLA-2001’s potential as a one-time therapy to completely inactivate the TTR gene and cut back the disease-causing protein in folks with ATTR-CM.

“This presentation at the AHA Scientific Sessions marks the first time Intellia has had the opportunity to share interim data from our landmark clinical trial of NTLA-2001 for the treatment of ATTR amyloidosis with the cardiology community,” stated Intellia President and Chief Executive Officer John Leonard, M.D. “We believe a single dose of NTLA-2001 has the potential to halt and potentially reverse this life-threatening disease. With the depth and consistency of TTR protein reduction seen with NTLA-2001 thus far, we envision a future where physicians and patients can make treatment decisions based on a therapy’s ability to achieve specific threshold protein levels. We look forward to progressing this first-ever systemically administered in vivo CRISPR investigational therapy toward completion of the Phase 1 study and subsequent pivotal studies in collaboration with our partners at Regeneron.”

At both dose levels, NTLA-2001 was generally well tolerated. As previously reported, two of 12 patients reported transient infusion reactions, which were the only observed treatment-related adverse events. One patient in the 0.7 mg/kg dose NYHA Class III cohort experienced a Grade 3 infusion-related reaction, which resolved without clinical sequalae. Per the study protocol, this group was subsequently expanded from three to six patients to further characterize safety at this dose level. No additional patients in the 0.7 mg/kg dose NYHA Class III cohort reported a treatment-related adverse event. No clinically significant laboratory abnormalities were observed at either dose level.

The Phase 1 study, run by Intellia as the program’s development and commercialization lead as part of a multi-target collaboration with Regeneron, is evaluating NTLA-2001 in patients with either ATTR-CM or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Dosing at 55 mg, the fixed dose corresponding to 0.7 mg/kg, is ongoing in Part 2, the dose-expansion portion of the study. Intellia remains on track to complete, by the end of this year, planned enrollment of both arms of the Phase 1 study that will inform the dose selection for subsequent pivotal studies.

About NTLA-2001
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Visit clinicaltrials.gov (NCT04601051) for more details.

About Transthyretin (ATTR) 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 transthyretin (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 polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (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.

About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatx.

Intellia Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) inside the which means of the Private Securities Litigation Reform Act of 1995. These forward-looking statements embrace, however are usually not restricted to, specific or implied statements concerning Intellia’s beliefs and expectations concerning: its potential to conduct and full scientific research for NTLA-2001 for the therapy of transthyretin amyloidosis (ATTR); its potential to generate information to show NTLA-2001 as a possible single-dose therapy for ATTR; the perception that NTLA-2001 can halt and doubtlessly even reverse ATTR; its potential to develop its modular platform and full-spectrum method to advance its advanced genome enhancing capabilities, together with to use its proprietary CRISPR/Cas9 know-how platform to extra product candidates; the development and enlargement of its CRISPR/Cas9 know-how to develop human therapeutic merchandise; its potential to take care of and increase its associated mental property portfolio, and keep away from or purchase rights to legitimate mental property of third events; its potential to show its platform’s modularity and replicate or apply outcomes achieved in preclinical research and scientific research, together with these in its NTLA-2001 program, in any future research, together with human scientific trials; its potential to develop different in vivo or ex vivo cell therapeutics of every kind, and NTLA-2001 particularly, utilizing CRISPR/Cas9 know-how; and the timing of regulatory filings and scientific trial execution, together with enrollment and dosing of sufferers.

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: risks related to the successful enrollment of patients in the Phase 1 study for NTLA-2001 for the treatment of ATTRv-PN or ATTR-CM; risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to the authorization, initiation and conduct of studies and other development requirements, including manufacturing, for its in vivo and ex vivo product candidates, including NTLA-2001; the risk that any one or more of Intellia’s product candidates, including NTLA-2001, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies, including for NTLA-2001, will not be predictive of future results in connection with future studies; and the risk that Intellia’s will not be able to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies to develop additional product candidates, including to apply its proprietary CRISPR/Cas9 technology platform successfully to additional product candidates. 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 of 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 (SEC). 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.

Intellia Contacts:

Investors:
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Media:
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Ten Bridge Communications
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