Senile cardiac amyloidosis

CAMBRIDGE, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient dosed in the global pivotal, Phase 3 MAGNITUDE trial of NTLA-2001. NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis. The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy. “Dosing of the first patient in the MAGNITUDE trial of NTLA-2001 puts us one step closer to bringing a potential one-time gene editing treatment to people living with ATTR amyloidosis,” said Intellia President and Chief Executive Officer John Leonard, M.D. “With multiple sites now enrolling patients, including in the U.S., we are off to a great start to rapidly enroll this landmark study. MAGNITUDE was informed by the compelling interim Phase 1 results reported last year. These data showed that a single-dose treatment of NTLA-2001 resulted in deep and durable reductions of the TTR protein responsible for the clinical consequences of the disease. We look forward to evaluating the efficacy and safety of NTLA-2001 in patients with cardiomyopathy in the pivotal Phase 3 trial. Assuming a positive outcome, it will pave the way for future global marketing applications, ultimately supporting our goal to bring forth a groundbreaking therapy for the ATTR amyloidosis community.” “The diagnosis of ATTR amyloidosis, a life-threatening condition leading to heart failure and other complications, is rapidly increasing,” said Julian Gillmore, M.D., Ph.D., Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K., and the Phase 3 study’s national coordinating investigator. “We, along with our colleagues at Richmond Pharmacology, are thrilled to be the first to dose a patient in the pivotal trial of NTLA-2001, an in vivo CRISPR-based treatment that offers the potential to dramatically reset the standard of care for people living with this devastating disease. There is remarkable interest from the ATTR amyloidosis patient community for a potential single-dose treatment, and we look forward to contributing to the advancement of novel treatment approaches.” About the MAGNITUDE Study The pivotal Phase 3 MAGNITUDE clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2001 in approximately 765 patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The primary endpoint of the study is a composite endpoint of cardiovascular (CV)-related mortality and CV-related events. Adult patients with hereditary or wild type ATTR-CM will be randomized 2:1 to receive a single 55 mg infusion of NTLA-2001 or placebo. For more information on MAGNITUDE (NCT06128629), please visit clinicaltrials.gov. About NTLA-2001Based on Nobel prize-winning CRISPR/Cas9 technology, NTLA-2001 has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. NTLA-2001 is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. NTLA-2001 is the first investigational CRISPR therapy to be administered systemically to edit genes inside the human body. Interim Phase 1 clinical data showed the administration of NTLA-2001 led to consistent, deep and long-lasting transthyretin (TTR) reduction. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. About Transthyretin (ATTR) Amyloidosis Transthyretin amyloidosis, or ATTR amyloidosis, is a 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. 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 clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. 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 regarding: the safety, efficacy, success and advancement of its clinical program for NTLA-2001 for transthyretin (“ATTR”) amyloidosis pursuant to its clinical trial applications and investigational new drug submission, including its ability to rapidly enroll and complete the Phase 3 Magnitude study; the success of its Phase 3 Magnitude study; its submission of global marketing applications; and its ability to bring forth NTLA-2001 as a groundbreaking therapy to the ATTR amyloidosis community and dramatically reset the standard of care. 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 Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its 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; and the risk that the results of preclinical studies or clinical studies, such as the Phase 1 clinical study of NTLA-2001, will not be predictive of future results in connection with future studies for the same product candidate or Intellia’s other 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 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 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:Ian KarpSenior Vice President, Investor Relations and Corporate Communicationsian.karp@intelliatx.com Lina LiSenior Director, Investor Relations and Corporate Communications lina.li@intelliatx.com Media:Matt CrensonTen Bridge Communicationsmedia@intelliatx.commcrenson@tenbridgecommunications.com

As part of the partnership, Ultromics will research and secure the US Food and Drug Administration (FDA) approval for its EchoGo Amyloidosis algorithm, which already received the FDA Breakthrough Device Designation for the detection of cardiac amyloidosis Ultromics to expedite development of Echo AI Algorithm. (Credit: PRNeswire/Ultromics ) UK-based health technology company Ultromics has teamed up with US-based pharmaceutical firm Pfizer to support the validation and regulatory approval of its AI-powered cardiac amyloidosis detection tool. As part of the partnership, Ultromics will research and secure the US Food and Drug Administration (FDA) approval for its EchoGo Amyloidosis algorithm. The algorithm received the FDA Breakthrough Device Designation in April last year, as a medical device for the detection of cardiac amyloidosis. Cardiac amyloidosis is a heterogeneous disease caused due to the accumulation of abnormal proteins within the heart, worsening its ability to pump blood. The build-up of proteins may cause the heart muscle to stiffen over time, leading to heart failure and may result in outcomes if not identified in early stages. Ultromics’ EchoGo Amyloidosis algorithm leverages deep learning to analyse routine echocardiograms to detect the disease, which is often undetected during standard testing. Ultromics founder and CEO Ross Upton said: “AI can notify clinicians of disease presence and offer an early warning. Early identification and treatment of cardiac amyloidosis is critical to help slow disease progression. “If cleared, EchoGo Amyloidosis will help physicians identify early signs of this debilitating disease, with the goal of facilitating earlier diagnosis and access to potentially life-saving treatment.” Ultromics is developing an advanced EchoGo platform, which is transforming the way heart failure is diagnosed using artificial intelligence and cardiac ultrasound as a modality. The company’s first AI-powered diagnostic system EchoGo Pro, intended to detect coronary artery disease, was approved by the US FDA in 2021 and is undergoing a clinical trial in the UK. Its algorithm to improve detection of Heart Failure has previously received FDA Breakthrough Device Designation and was approved by the FDA in November 2022. In December last year, Ultromics was selected to participate in the FDA’s Total Product Life Cycle Advisory Program (TAP) Pilot of 15 breakthrough cardiovascular devices. The initiative aims to advance the development and enhance the accessibility of high-quality, safe, effective, and innovative medical devices.

OXFORD, England, Jan. 10, 2024 /PRNewswire/ -- Ultromics, a patient outcomes-focused AI-driven health technology company with roots at the University of Oxford, UK, has entered into an agreement with Pfizer to support the validation and FDA clearance of its AI-based technology for the detection of cardiac amyloidosis. Continue Reading Ultromics Announces Partnership to Expedite Development of Echo AI Algorithm for Early Detection of Cardiac Amyloidosis Under the partnership with Pfizer, Ultromics will research and pursue FDA clearance for its EchoGo® Amyloidosis algorithm that already has Breakthrough Device Designation as a medical device for the detection of cardiac amyloidosis. The algorithm uses deep learning to analyse routine ultrasound scans of the heart (echocardiograms) to detect disease that often goes undetected during standard assessments. "Echocardiography, while fundamental for the early detection of many heart conditions, is hindered by subjectivity and the demand for scarce expertise in the accurate identification of cardiac amyloidosis," commented Prof. Sanjiv J. Shah, MD, the Director of Research for the Bluhm Cardiovascular Institute and Director, Center for Deep Phenotyping and Precision Medicine in the Institute for Augmented Intelligence in Medicine at Northwestern University Feinberg School of Medicine. "AI can help detect cardiac amyloidosis early through routine ultrasound scans of the heart, which is vitally important because current treatments for cardiac amyloidosis work best when implemented early in the course of disease. AI-augmented detection of cardiac amyloidosis may be especially useful in clinical facilities lacking the time and resources needed for comprehensive assessment," he added. Cardiac amyloidosis is a heterogeneous disease that results from the accumulation of abnormal proteins within the heart, impairing its ability to pump blood. Over time, the build-up of these proteins causes the heart muscle to stiffen, eventually leading to heart failure, which can have dire outcomes if not found early.1,2 "AI can notify clinicians of disease presence and offer an early warning," stated Dr. Ross Upton, CEO and Founder of Ultromics. "Early identification and treatment of cardiac amyloidosis is critical to help slow disease progression. If cleared, EchoGo® Amyloidosis will help physicians identify early signs of this debilitating disease, with the goal of facilitating earlier diagnosis and access to potentially life-saving treatment." Ultromics' expertise has been demonstrated through its algorithms for improving the detection of Heart Failure, which previously received FDA Breakthrough Device Designation before being cleared by the FDA in November 2022. FDA Breakthrough Device Designation recognizes novel innovations that demonstrate the potential to provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases. This builds on the history of Echo diagnostic algorithms developed by Ultromics including the first ever AI diagnostic, EchoGo® Pro for the detection of coronary artery disease, which was FDA cleared in 2021 and is undergoing a prospective clinical trial in the UK. In December 2023, Ultromics proudly revealed it had been chosen to participate in the FDA's Total Product Life Cycle Advisory Program (TAP) Pilot, an exclusive cohort comprising of only 15 breakthrough cardiovascular devices. The strategic initiative is geared towards accelerating development and enhancing the accessibility of high-quality, safe, effective, and innovative medical devices. 1 Maurer M, Elliott P, Comenzo R, et al. Addressing common questions encountered in the diagnosis and management of cardiac amyloidosis. Circulation. 2017. 135(14):1357-1377. 2Siddiqi O., Ruberg F. 2018. Cardiac Amyloidosis: An Update on Pathophysiology, Diagnosis, and Treatment. Trends Cardiovasc Med. DOI:10.1016/j.tcm.2017.07.004 EchoGo® Amyloidosis pending 510(k) clearance About Ultromics: Ultromics is a pioneer in precision heart failure detection. Our ground-breaking platform, EchoGo®, is transforming the way heart failure is diagnosed using artificial intelligence and cardiac ultrasound as a modality. The technology empowers clinicians to make precise, efficient, and accurate assessments of heart failure, leveraging the largest known heart disease dataset in echocardiography. The model was trained on thousands of patients to accurately detect disease and was validated against 5-year patient outcomes. The technology has been built in collaboration with Mayo Clinic and the NHS England. Ultromics' mission is to stop heart failure in its tracks with its precision detection platform. Photo - Logo - SOURCE Ultromics