Intellia and Regeneron Announce Initial Data from the

• Interim information from the cardiomyopathy arm of the Phase 1 research of NTLA-2001 confirmed deep and sustained imply serum transthyretin (TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28
• NTLA-2001 was typically well-tolerated at each dose ranges
• Intellia to debate information at investor occasion in the present day, Friday, September 16, at 8:00 a.m. ET

CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Sept. 16, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) in the present day introduced optimistic interim outcomes from an ongoing Phase 1 medical trial of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome enhancing remedy in improvement as a single-dose therapy for transthyretin (ATTR) amyloidosis. The interim information embrace 12 grownup sufferers with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III coronary heart failure. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 have been administered by way of 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 speedy and deep reductions in serum TTR by day 28 as follows:

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

These profound reductions in serum TTR have been sustained all through the commentary interval, with affected person follow-up ranging from two to 6 months as of the information deadline of July 1, 2022. These information assist NTLA-2001’s potential as a one-time therapy to completely inactivate the TTR gene and cut back the disease-causing protein in individuals with ATTR-CM.

“ATTR amyloidosis is a multifaceted disease in need of additional treatment options. These new interim results demonstrate that NTLA-2001 can profoundly reduce serum TTR levels in patients whose condition results in cardiomyopathy,” mentioned Intellia President and Chief Executive Officer John Leonard, M.D. “Together with the previously reported data from the polyneuropathy arm of this landmark study, these results strongly suggest that NTLA-2001 could serve as a single-dose treatment regardless of disease manifestation. At these deep and consistent levels of protein reduction, we believe NTLA-2001 has the potential to halt and even reverse the underlying cause of ATTR amyloidosis. Given the similarly robust TTR reductions observed at the two doses tested, we have selected a fixed dose comparable to the 0.7 mg/kg level for evaluation across both arms in the ongoing dose-expansion portion of the study. We look forward to completing the Phase 1 study as we advance closer to a potential pivotal trial, which we expect will include patients in the U.S.”

“We’re encouraged to see profound and sustained serum TTR reductions in people with cardiomyopathy manifestations of this rare and fatal disease, further bolstering the prospects for a one-time, in vivo treatment for multiple ATTR patient groups,” mentioned George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. “Intellia and Regeneron are working together diligently to advance this potentially groundbreaking application of CRISPR technology, which could one day be used for many different genetic diseases.”

At each dose ranges, NTLA-2001 was typically properly tolerated. Two of 12 sufferers reported transient infusion reactions, which was the solely noticed treatment-related adversarial occasion. One affected person in the 0.7 mg/kg dose NYHA Class III cohort skilled a Grade 3 infusion-related response, which resolved with out medical consequence. Per the research protocol, this group was subsequently expanded from three to 6 sufferers to additional characterize security at this dose stage. No extra sufferers in the 0.7 mg/kg dose NYHA Class III cohort reported a treatment-related adversarial occasion. No clinically vital liver findings have been noticed at both dose stage.

The Phase 1 research, run by Intellia as the program’s improvement and commercialization lead as a part of a multi-target collaboration with Regeneron, is evaluating NTLA-2001 in sufferers with both ATTR-CM or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). A protocol modification has been submitted to judge a hard and fast dose similar to 0.7 mg/kg in the dose-expansion portion, with enrollment throughout each arms anticipated to be accomplished by the finish of 2022, topic to regulatory suggestions.

NTLA-2002 Interim Clinical Results
In a separate press launch issued earlier in the present day, Intellia introduced optimistic interim medical information from an ongoing Phase 1/2 medical research of NTLA-2002, its second in vivo genome enhancing candidate, for the therapy of hereditary angioedema (HAE). Please go to this hyperlink, or the Press Releases part of the firm’s web site at www.intelliatx.com.

Intellia Therapeutics Investor Event and Webcast Information
Intellia will host a dwell webcast in the present day, Friday, September 16, 2022, at 8:00 a.m. ET, to offer a medical replace from its in vivo portfolio, throughout which the firm will assessment these outcomes from NTLA-2001 alongside interim information from NTLA-2002. To be a part of the webcast, please go to this hyperlink, or the Events and Presentations web page of the Investors & Media part of the firm’s web site at www.intelliatx.com. A replay of the webcast shall be out there on Intellia’s web site for at the very least 30 days following the name.

About NTLA-2001
Based on Nobel Prize-winning CRISPR/Cas9 know-how, NTLA-2001 may doubtlessly be the first single-dose therapy for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR remedy candidate to be administered systemically, or by way of a vein, to edit genes inside the human physique. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to ship to the liver a two-part genome enhancing system: information RNA particular to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision enhancing. Robust preclinical information, exhibiting deep and long-lasting transthyretin (TTR) discount following in vivo inactivation of the goal gene, helps NTLA-2001’s potential as a single-administration therapeutic. Intellia leads improvement and commercialization of NTLA-2001 as a part of a multi-target discovery, improvement and commercialization collaboration with Regeneron. The world Phase 1 trial is an open-label, multi-center, two-part research 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 extra particulars.

About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a uncommon, progressive and deadly illness. Hereditary ATTR (ATTRv) amyloidosis happens when an individual is born with mutations in the TTR gene, which causes the liver to provide structurally irregular transthyretin (TTR) protein with a propensity to misfold. These broken proteins construct up as amyloid in the physique, inflicting severe issues in a number of tissues, together with the coronary heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which may result in nerve injury, or cardiomyopathy (ATTRv-CM), which may result in coronary heart failure. Some people with out the genetic mutation produce non-mutated, or wild-type TTR proteins that develop into unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This situation, known as wild-type ATTR (ATTRwt) amyloidosis, primarily impacts the coronary heart. There are an estimated 50,000 individuals worldwide residing with ATTRv amyloidosis and between 200,000 and 500,000 individuals with ATTRwt amyloidosis.

About Intellia Therapeutics
Intellia Therapeutics, a number one clinical-stage genome enhancing firm, is growing novel, doubtlessly healing therapeutics leveraging CRISPR-based applied sciences. To totally notice the transformative potential of CRISPR-based applied sciences, Intellia is pursuing two major approaches. The firm’s in vivo packages use intravenously administered CRISPR as the remedy, wherein proprietary supply know-how allows extremely exact enhancing of disease-causing genes straight inside particular goal tissues. Intellia’s ex vivo packages use CRISPR to create the remedy by utilizing engineered human cells to deal with most cancers and autoimmune ailments. Intellia’s deep scientific, technical and medical improvement expertise, together with its strong mental property portfolio, have enabled the firm to take a management position in harnessing the full potential of genome enhancing to create new lessons of genetic medication. Learn extra at intelliatx.com. Follow us on Twitter @intelliatx.

About Regeneron
Regeneron (NASDAQ: REGN) is a number one biotechnology firm that invents life-transforming medicines for individuals with severe ailments. Founded and led for almost 35 years by physician-scientists, our distinctive capacity to repeatedly and persistently translate science into medication has led to 9 FDA-approved remedies and quite a few product candidates in improvement, nearly all of which have been homegrown in our laboratories. Our medicines and pipeline are designed to assist sufferers with eye ailments, allergic and inflammatory ailments, most cancers, cardiovascular and metabolic ailments, ache, hematologic situations, infectious ailments and uncommon ailments.

Regeneron is accelerating and bettering the conventional drug improvement course of by way of our proprietary VelociSuite^® applied sciences, akin to VelocImmune^®, which makes use of distinctive genetically humanized mice to provide optimized totally human antibodies and bispecific antibodies, and by way of bold analysis initiatives akin to the Regeneron Genetics Center, which is conducting one in all the largest genetics sequencing efforts in the world.

For extra details about the firm, please go to www.regeneron.com or comply with @Regeneron on Twitter.

Intellia Forward-Looking Statements

This press launch incorporates “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 will not be restricted to, specific or implied statements relating to Intellia’s beliefs and expectations relating to: its capacity to conduct and full medical research for NTLA-2001 for the therapy of transtherytin amyloidosis (ATTR); its capacity 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 the underlying reason for ATTR; its capacity 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 capacity to keep up and broaden its associated mental property portfolio, and keep away from or purchase rights to legitimate mental property of third events; its capacity to show its platform’s modularity and replicate or apply outcomes achieved in preclinical research, together with these in its NTLA-2001 program, in any future research, together with human medical trials; its capacity to develop different in vivo or ex vivo cell therapeutics of all kinds, and NTLA-2001 specifically, utilizing CRISPR/Cas9 know-how; and the timing of regulatory filings and medical trial execution, together with enrollment and dosing of sufferers.

Any forward-looking statements on this press launch are based mostly on administration’s present expectations and beliefs of future occasions, and are topic to quite a few dangers and uncertainties that might trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embrace, however will not be restricted to: dangers associated to the profitable enrollment of sufferers in the Phase 1 research for NTLA-2001 for the therapy of ATTRv-PN or ATTR-CM; dangers associated to Intellia’s capacity to guard and keep its mental property place; dangers associated to the authorization, initiation and conduct of research and different improvement necessities, together with manufacturing, for its in vivo and ex vivo product candidates, together with NTLA-2001; the threat that anyone or extra of Intellia’s product candidates, together with NTLA-2001, is not going to be efficiently developed and commercialized; the threat that the outcomes of preclinical research or medical research, together with for NTLA-2001, is not going to be predictive of future ends in reference to future research; and the threat that Intellia’s won’t be able to show its platform’s modularity and replicate or apply outcomes achieved in preclinical research to develop extra product candidates, together with to use its proprietary CRISPR/Cas9 know-how platform efficiently to extra product candidates. For a dialogue of those and different dangers and uncertainties, and different vital elements, any of which may trigger Intellia’s precise outcomes to vary from these contained in the forward-looking statements, see the part entitled “Risk Factors” in Intellia’s most up-to-date annual report on Form 10-Ok and quarterly report of Form 10-Q, in addition to discussions of potential dangers, uncertainties and different vital elements in Intellia’s different filings with the Securities and Exchange Commission (SEC). All info on this press launch is as of the date of the launch, and Intellia undertakes no responsibility to replace this info until required by legislation.

Regeneron Forward-Looking Statements and Use of Digital Media

This press launch consists of forward-looking statements that contain dangers and uncertainties referring to future occasions and the future efficiency of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and precise occasions or outcomes might differ materially from these forward-looking statements. Words akin to “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such phrases, and related expressions are meant to establish such forward-looking statements, though not all forward-looking statements include these figuring out phrases. These statements concern, and these dangers and uncertainties embrace, amongst others, the affect of SARS-CoV-2 (the virus that has brought on the COVID-19 pandemic) on Regeneron’s business and its staff, collaborators, and suppliers and different third events on which Regeneron depends, Regeneron’s and its collaborators’ capacity to proceed to conduct analysis and medical packages, Regeneron’s capacity to handle its provide chain, internet product gross sales of merchandise marketed or in any other case commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”), and the world economy; the nature, timing, and doable success and therapeutic purposes of Regeneron’s Products and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and analysis and medical packages now underway or deliberate, akin to NTLA-2001 (a product candidate being developed for transthyretin (ATTR) amyloidosis underneath a multi-target discovery, improvement, and commercialization collaboration between Regeneron and Intellia Therapeutics, Inc.); the extent to which the outcomes from the analysis and improvement packages performed by Regeneron and/or its collaborators or licensees (together with the Phase 1 medical research evaluating NTLA-2001 mentioned on this press launch) could also be replicated in different research and/or result in development of product candidates to medical trials, therapeutic purposes, or regulatory approval; the potential of the CRISPR/Cas9 genome enhancing know-how mentioned on this press launch for in vivo therapeutic improvement; uncertainty of the utilization, market acceptance, and business success of Regeneron’s Products and Regeneron’s Product Candidates and the affect of research (whether or not performed by Regeneron or others and whether or not mandated or voluntary), together with the research mentioned or referenced on this press launch, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (akin to NTLA-2001); the probability, timing, and scope of doable regulatory approval and business launch of Regeneron’s Product Candidates (akin to NTLA-2001) and new indications for Regeneron’s Products; the capacity of Regeneron’s collaborators, licensees, suppliers, or different third events (as relevant) to carry out manufacturing, filling, ending, packaging, labeling, distribution, and different steps associated to Regeneron’s Products and Regeneron’s Product Candidates; the capacity of Regeneron and/or its collaborators to fabricate and handle provide chains for a number of merchandise and product candidates; questions of safety ensuing from the administration of Regeneron’s Products and Regeneron’s Product Candidates in sufferers, together with severe issues or uncomfortable side effects in reference to the use of Regeneron’s Products and Regeneron’s Product Candidates (akin to NTLA-2001) in medical trials; determinations by regulatory and administrative governmental authorities which can delay or prohibit Regeneron’s capacity to proceed to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, analysis and medical packages, and business, together with these referring to affected person privateness; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, together with personal payer healthcare and insurance packages, well being upkeep organizations, pharmacy profit administration corporations, and authorities packages akin to Medicare and Medicaid; protection and reimbursement determinations by such payers and new insurance policies and procedures adopted by such payers; competing medicine and product candidates which may be superior to, or more economical than, Regeneron’s Products and Regeneron’s Product Candidates; unanticipated bills; the prices of growing, producing, and promoting merchandise; the capacity of Regeneron to fulfill any of its monetary projections or steering and modifications to the assumptions underlying these projections or steering; the potential for any license, collaboration, or provide settlement, together with Regeneron’s agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their respective affiliated corporations, as relevant), in addition to Regeneron’s collaboration with Intellia Therapeutics, Inc. mentioned on this press launch, to be cancelled or terminated; and dangers related to mental property of different events and pending or future litigation relating thereto (together with with out limitation the patent litigation and different associated proceedings referring to EYLEA^® (aflibercept) Injection, Dupixent^® (dupilumab), Praluent^® (alirocumab), and REGEN-COV^® (casirivimab and imdevimab)), different litigation and different proceedings and authorities investigations referring to the Company and/or its operations, the final consequence of any such proceedings and investigations, and the affect any of the foregoing might have on Regeneron’s business, prospects, working outcomes, and monetary situation. A extra full description of those and different materials dangers will be present in Regeneron’s filings with the U.S. Securities and Exchange Commission, together with its Form 10-Ok for the yr ended December 31, 2021 and its Form 10-Q for the quarterly interval ended June 30, 2022. Any forward-looking statements are made based mostly on administration’s present beliefs and judgment, and the reader is cautioned to not depend on any forward-looking statements made by Regeneron. Regeneron doesn’t undertake any obligation to replace (publicly or in any other case) any forward-looking assertion, together with with out limitation any monetary projection or steering, whether or not because of new info, future occasions, or in any other case.

Regeneron makes use of its media and investor relations web site and social media shops to publish vital details about the Company, together with info which may be deemed materials to buyers. Financial and different details about Regeneron is routinely posted and is accessible on Regeneron’s media and investor relations web site (https://newsroom.regeneron.com/) and its Twitter feed (https://twitter.com/regeneron).

Intellia Contacts:

Investors:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
+1-857-449-4175
[email protected]

Lina Li
Senior Director, Investor Relations and Corporate Communications
+1-857-706-1612
[email protected]

Media:
Rebecca Spalding
Ten Bridge Communications
+1-646-509-3831
[email protected]
[email protected]

Regeneron Contacts:

Investors:
Vesna Tosic
+1-914-847-5443
[email protected]

Media:
Alexandra Bowie
+1-914-847-3407
[email protected]