A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

The primary objective of this study is to assess the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the composite endpoint of all-cause mortality (ACM) and total cardiovascular (CV) clinical events.

Start Date11 Jan 2024

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

JPRN-jRCT2031230517 / SuspendedPhase 3

A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) - DepleTTR-CM

Start Date04 Jan 2024

Sponsor / Collaborator-

NL-OMON52809 / CompletedNot Applicable

A Phase 1, First-in-Human, Double-Blind, Placebo-Controlled, Multicenter, Single and Multiple Ascending Dose Study of NI006 in Patients with Amyloid Transthyretin Cardiomyopathy Followed by an Open-Label Extension - NI006 in Patients with Amyloid Transthyretin Cardiomyopathy

Start Date08 Oct 2020

Sponsor / Collaborator

Neurimmune AG

100 Clinical Results associated with NI-006

100 Translational Medicine associated with NI-006

100 Patents (Medical) associated with NI-006

Literatures (Medical) associated with NI-006

20 Jul 2023·The New England journal of medicineQ1 · MEDICINE

Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid

Q1 · MEDICINE

Article

Author: Frost, Robert J A ; Nitsch, Roger M ; Michalon, Aubin ; Hock, Christoph ; Grimm, Jan ; Aus dem Siepen, Fabian ; Nitsch, Roger M. ; van der Meer, Peter ; Mercuri, Michele F. ; Garcia-Pavia, Pablo ; Kristen, Arnt V. ; Lairez, Olivier ; Frost, Robert J.A. ; Kahr, Peter C. ; Donal, Erwan ; Damy, Thibaud ; aus dem Siepen, Fabian ; Kristen, Arnt V

BACKGROUND:

Transthyretin amyloid (ATTR) cardiomyopathy is a progressive and fatal disease caused by misfolded transthyretin. Despite advances in slowing disease progression, there is no available treatment that depletes ATTR from the heart for the amelioration of cardiac dysfunction. NI006 is a recombinant human anti-ATTR antibody that was developed for the removal of ATTR by phagocytic immune cells.

METHODS:

In this phase 1, double-blind trial, we randomly assigned (in a 2:1 ratio) 40 patients with wild-type or variant ATTR cardiomyopathy and chronic heart failure to receive intravenous infusions of either NI006 or placebo every 4 weeks for 4 months. Patients were sequentially enrolled in six cohorts that received ascending doses (ranging from 0.3 to 60 mg per kilogram of body weight). After four infusions, patients were enrolled in an open-label extension phase in which they received eight infusions of NI006 with stepwise increases in the dose. The safety and pharmacokinetic profiles of NI006 were assessed, and cardiac imaging studies were performed.

RESULTS:

The use of NI006 was associated with no apparent drug-related serious adverse events. The pharmacokinetic profile of NI006 was consistent with that of an IgG antibody, and no antidrug antibodies were detected. At doses of at least 10 mg per kilogram, cardiac tracer uptake on scintigraphy and extracellular volume on cardiac magnetic resonance imaging, both of which are imaging-based surrogate markers of cardiac amyloid load, appeared to be reduced over a period of 12 months. The median N-terminal pro-B-type natriuretic peptide and troponin T levels also seemed to be reduced.

CONCLUSIONS:

In this phase 1 trial of the recombinant human antibody NI006 for the treatment of patients with ATTR cardiomyopathy and heart failure, the use of NI006 was associated with no apparent drug-related serious adverse events. (Funded by Neurimmune; NI006-101 ClinicalTrials.gov number, NCT04360434.).

News (Medical) associated with NI-006

23 May 2024

·www.biospace.com

AstraZeneca demonstrates commitment to patients living with amyloidosis at the 2024 International Symposium on Amyloidosis (ISA)

Additional clinical and real-world data from the industry’s largest amyloidosis pipeline will advance understanding of underlying mechanisms of disease and unmet needs WILMINGTON, Del.--(BUSINESS WIRE)-- AstraZeneca and Alexion, AstraZeneca Rare Disease, will showcase 14 studies, including real-world evidence (RWE), from their portfolio and pipeline of investigational amyloidosis therapies at the International Symposium on Amyloidosis (ISA), in Rochester, MN from May 26–30, 2024. Presentations will include a new subgroup analyses of the Phase 3 NEURO-TTRansform study of WAINUATM (eplontersen), which was approved by the US Food and Drug Administration (FDA) in December 2023 for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.1 Additionally, clinical data will be presented on ALXN2220 and anselamimab, which are being evaluated in Phase III clinical trials for ATTR and light chain (AL) amyloidosis, respectively. Sarah Walters, Vice President, US Cardiovascular, Renal & Metabolic Diseases, AstraZeneca, said: “AstraZeneca is dedicated to advancing the science and improving the lives of patients that are impacted by this devastating disease. Our data at the 2024 International Symposium on Amyloidosis demonstrate the leadership commitment AstraZeneca, Alexion and our partners Ionis and Neuroimmune have to develop best in class treatment options to serve a broad range of amyloidosis patients.” Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “With the largest and fastest-growing pipeline of investigational amyloidosis therapies, we are working to advance multiple modalities with the potential to halt, reduce or reverse organ damage. Key presentations at the ISA Annual Meeting include live-cell imaging and Phase I clinical data evaluating the ability of ALXN2220 to remove amyloid from cardiac tissue, to support its further study as a potential treatment for advanced ATTR cardiomyopathy. Additionally, findings on epidemiology and patient renal outcomes will reinforce the urgency for differentiated amyloidosis diagnosis and additional treatment options in AL amyloidosis.” Robust evidence program increases understanding of amyloidosis patient characteristics and treatment effectiveness. In addition to sub-analyses of the NEURO-TTRansform data, AstraZeneca will present US patient characteristics data, preliminary data from the OverTTuRe study measuring prevalence and characteristics of phenotypes in patients with ATTR amyloidosis in the United States and Japan; as well as the MaesTTRo study design, part of a global, multi-study, real-world evidence program designed to fill the evidence gaps of the effectiveness of treatments for ATTR. WAINUA is currently being evaluated in the Phase 3 CARDIO-TTRansform study for adults with cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset.2-5 The CARDIO-TTRansform Phase 3 study is fully enrolled with more than 1,400 patients–making it the largest study in this patient population to date.6 As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing WAINUA for the treatment of hATTR-PN in the US and are seeking regulatory approval in Europe and other parts of the world.6 Novel investigational amyloid depleter assets demonstrate potential to improve outcomes for amyloidosis patients High-resolution live-cell imaging will offer new insight into the underlying cellular mechanism of antibody-mediated amyloid removal with ALXN2220. Results will show the addition of ALXN2220 to ATTR patient-derived cardiac tissue triggered phagocytosis, including recruitment of macrophages to amyloid deposits, detachment, internalization and degradation of ATTR amyloid. An encore poster presentation will report results from the first-in-human study of ALXN2220. Initial treatment with ALXN2220 was well tolerated with a transient increase in C-reactive proteins, suggesting initial targeted immune activation and recruitment of phagocytic immune cells. Additionally, an encore poster presentation will describe the pharmacokinetic and pharmacodynamic (PK/PD) model used for the selection of the dose range, dose escalation, dosing interval and study duration. Further, a poster will outline the baseline demographics and characteristics of the 406 participants of the Alexion CARES clinical program evaluating anselamimab in patients with Mayo Stage IIIa and IIIb AL amyloidosis, which includes the largest prospective AL amyloidosis study to date in advanced stage AL amyloidosis. Advancing understanding of the amyloidosis landscape A retrospective chart analysis from the US EMR TriNetX database will report outcomes in AL amyloidosis patients with renal involvement receiving bortezomib or daratumumab. Only half of patients were renal-responders and few achieved renal complete response (renCR, proteinuria ≤200 mg/day), underscoring the unmet medical need for this patient subset. Retrospective analyses will also be presented across three posters on the incidence and prevalence of AL and ATTR amyloidosis, reinforcing the importance of timely diagnosis of amyloidosis and continued monitoring. A comprehensive list of AstraZeneca key abstracts to be presented at the 2024 International Symposium on Amyloidosis includes7: Lead author Abstract title Presentation details Poster presentation session 1: 10:00-10:30 Poster presentation session 2: 14:45-15:45 Eplontersen Gillmore JD, Davis M, Hahn K, Smith G, Shivappa N, Papas M, Folkvaljon F, Pao C, Sundin AK, L Wittbrodt E, Grogan M Prospective, Real-World Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin Amyloidosis: Design of the MaesTTRo Study Date: Wednesday, May 29 Abstract ID: 204 Waddington Cruz M, Berk J, Parman Y, Gertz M, Khella S, Weiler M, Kwoh J, Chen J, Reicher B, Nåtman J, Dasgupta N Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients Date: Wednesday, May 29 Abstract ID: 202 Gillmore JD, Adams D, Weiler M, Masri A, Obici L, Kwoh J, Reicher B, Chen J, Waddington Cruz M, Natman J, Gertz M Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis in Patients With the V30M TTR variant and Early-onset or Late-onset Disease Date: Wednesday, May 29 Abstract ID: 230 Wixner J, Conceição I, Berk J, Adams D, Polydefkis M, Attarian S, Gillmore J, Dyck J, Coelho T, Chen J, Hardy E, Kwoh J, Nåtman J, Waddington Cruz M Neuropathy Impairment and Nutritional Status With Eplontersen in Patients With Hereditary Transthyretin-Mediated Amyloidosis Date: Wednesday, May 29 Abstract ID: 174 Alexander K, Alvarez C, Kumar N, Pao C, Wittbrodt E, Kohsaka S Prevalence and Descriptive Characteristics of Clinical Phenotypes in Patients With ATTR amyloidosis in the United States and Japan: Preliminary Results from the OverTTuRe Study Date: Monday, May 27 Abstract ID: 198 Bazell C, Alston M, Kumar N, Huang J, Venditto J, Grillis D, Eisenberg S, Nativi-Nicolau J Descriptive Characteristics of Patients Diagnosed With Transthyretin Amyloidosis in the Commercial and Medicare Populations Date: Wednesday, May 29 Abstract ID: 518 ALXN 2220 Michalon A, Huy, Mercuri M, Kahr P, Hock C, Grimm J ALXN2220: High-resolution Live-cell Imaging of Antibody-mediated Cardiac ATTR Amyloid Depletion Date: Tuesday, May 28 Abstract ID: 226 Michalon A, Renaud L, Machacek M, Cortijo C, Udaata C, Mercuri M, Buller F, Hock C, Nitsch R, Kahr P, Grimm J Prediction of Cardiac ATTR Depletion by NI006 Using Mechanistic PK/PD Modeling Date: Wednesday, May 29 Abstract ID: 291 Aus dem Siepen F, Van der Meer P, Damy T, Garcia-Pavia P, Donal E, Lairez O, Blank A, Kristen A, Quarta CC, Buchele G, Mercuri M, Hock C, Michalon A, Kahr P, Rey S, Tichy M ALXN2220: Targeted Immune Activation in Patients Undergoing Antibody-mediated Cardiac ATTR Amyloid Depletion Date: Wednesday, May 29 Abstract ID: 227 Amyloidosis Incidence and Analyses Laires PA, Evans J, Thompson J, Manwani R, Mudumby P, Field M, Fang S Prevalence, Incidence, and Characterization of Light Chain Amyloidosis in the USA: A Real-World Analysis Utilizing Electronic Health Records (EHR) Date: Monday, May 27 Abstract ID: 65 Manwani R, Yang F, Zhang Y, Laires PA Outcomes in Patients With AL Amyloidosis With Renal Involvement: Findings from the TriNetX Database Date: Monday, May 27 Abstract ID: 113 Laires PA, Li SXL, Uday A, Kumar P, Silva AM, Quarta C Prevalence and Incidence of ATTR Amyloidosis in the United States: Insights from Claims Database and Electronic Health Records Date: Tuesday, May 28 Abstract ID: 68 Laires PA, Zhang Y, Manwani R, Silva AM, Catini J, Thompson J, Dozier M, Yang F Subtype Distribution of Amyloidosis in the United States: Insights from an Electronic Health Records Database Analysis Date: Tuesday, May 28 Abstract ID: 112 The full list of ISA 2024 congress abstracts are available online. INDICATION for WAINUATM (eplontersen) WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. IMPORTANT SAFETY INFORMATION for WAINUATM (eplontersen) WARNINGS AND PRECAUTIONS Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ADVERSE REACTIONS Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%). Please see link to US Full Prescribing Information for WAINUA. Notes NEURO-TTRansform NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with hATTR-PN.8,9 The trial enrolled adult patients with hATTR-PN Stage 1 or Stage 2 compared to the external placebo group from the NEURO-TTR registrational trial that Ionis completed in 2017.8,9 The comparison of efficacy and safety for WAINUA versus external placebo was based on data up to week 66, and all patients were followed on treatment until week 85, when they had the option to transition into an open-label extension study, which is still ongoing.8,9 WAINUA WAINUATM (eplontersen) is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of transthyretin, or TTR protein.9,10 WAINUA has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (also referred to as hATTR-PN).1 AstraZeneca in amyloidosis Amyloidosis is a group of complex rare diseases caused by abnormal proteins that misfold and clump together to form toxic amyloids that deposit in tissues or organs, including the heart, kidneys and peripheral nerves.11-12 The build-up of these toxic amyloids can result in significant organ damage and organ failure that can severely impact quality of life and ultimately be fatal.11-12 AstraZeneca and its Rare Disease Unit, Alexion, are developing and evaluating multiple modalities with the potential to halt and reduce organ damage across various types of amyloidosis. The Company is uniquely positioned to lead therapeutic and diagnostic advances for people living with amyloidosis with the largest and fastest-growing pipeline of investigational amyloidosis therapies to address the spectrum of patient needs. Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, MA, US. About AstraZeneca AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries, and its innovative medicines are used by millions of patients worldwide. For more information, please visit and follow us on social media @AstraZeneca. References WAINUATM (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2023. Viney N, et al. Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data. ESC Heart Failure. 2021;8:652-661. Rintell D, et al. Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups. Orphanet J Rare Dis. 2021;16:70. Columbia University Irving Medical Center [Internet]. Drug Reduces Death from Underdiagnosed Form of Heart Failure [last accessed 11 December 2023]. Available from: . ClinicalTrials.gov. CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) [last accessed 21 May 2024] Available from: . Ionis Pharmaceuticals [Internet]. WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis [last accessed 16 May 2024]. Available from: . Mayo Clinic. International Symposium on Amyloidosis: Abstracts/Posters [last accessed 21 May 2024] Available from: . Coelho T, et al. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx(ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Nerol Ther. 2021 Jun;10(1):375-389. Coelho T, et al. Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy. JAMA. 2023;330(15):1448–1458. Coelho T, et al. Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen. Neurol Ther. 2023;12:267–287. Cleveland Clinic. Amyloidosis. Cleveland Clinic website. Accessed May 21, 2024. . Mayo Clinic. Amyloidosis. Mayo Clinic website. Accessed May 21, 2024. . US-89473 Last Updated 5/24

Phase 3Clinical ResultPhase 1Drug Approval

11 Jan 2024

·www.pharmaceutical-technology.com

BridgeBio gears up for FDA review of acoramidis following Phase III results

ATTR-CM is caused by TTR protein build-up in the heart muscle causing thickening and stiffening of the muscle. Image Credit: Chinnapong / Shutterstock. BridgeBio Pharma has published results from the Phase III ATTRibute-CM trial evaluating acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The trial results were published , which were published in the New England Journal of Medicine , show a better hierarchal outcome in the acoramidis treatment arm compared to the placebo. The hierarchical outcome was measured using the Finkelstein and Schoenfeld (FS) test, which measures composite endpoints including mortality, morbidity, and function in the order of clinical importance, and was reported as a win ratio . The composite endpoints order of priority as part of the primary hierarchal outcome was all-cause mortality (ACM), frequency of cardiovascular-related hospitalization (CVH), change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and change from baseline in 6-minute walk distance (6MWD). The analysis favoured the treatment arm with a win ratio of 1.8, with 63.7% pairwise comparisons favouring acoramidis and 35.9% favouring placebo. The ACM and CHV outcomes also contributed to the win ratio in favour of acoramidis, with 58% of all pairwise comparisons favouring the treatment. The overall incidence of adverse events was 98.1% and 97.6% in the treatment and placebo groups, respectively. The incidence of serious adverse events in the treatment and placebo groups was 54.6% and 64.9%, respectively. A genetic mutation of the gene for transthyretin (TTR) protein causes ATTR, which leads to the production of an abnormal form of the protein causing protein build-up in the organs. ATTR-CM is caused by TTR protein build-up in the heart muscle causing thickening and stiffening of the muscle. This impairs the heart’s ability to function properly and can lead to stroke or death. Acoramidis is a TTR stabiliser and works by mimicking disease protective action of the TTR-stabilising mutation T119M. The randomised, double-blind placebo-controlled Phase III ATTRibute-CM trial (NCT03860935) evaluated acoramidis in 632 patients with ATTR-CM. The US-based company had previously reported that patients in the acoramidis treatment arm demonstrated an 81% absolute survival rate and a 0.29 observed mean annual CVH frequency. BridgeBio submitted the new drug application (NDA) with the US Food and Drug Administration (FDA) on 5 December 2023. The agency has a 60-day filing review period to determine whether the NDA is complete and accepted for review. The company plans to file additional marketing authorisation applications to global health authorities this year. ATTR-CM treatment landscape Multiple companies are developing therapies for ATTR-CM. One of the more clinically advanced therapy programmes includes Alnylam Pharmaceuticals ’s Onpattro (patisiran). The Phase III trial (NCT03997383) for the RNA interference (RNAi) therapy met its primary endpoint but the FDA refused to approve the therapy despite positive results. In October 2023, the agency issued a complete response letter (CRL) stating that the drug “could not be approved in its present form”. Following the CRL, Alnylam stated that it would no longer seek US approval for the therapy in ATTR-CM indication. Gene therapies are also being developed to treat ATTR-CM. Intellia Therapeutics and Regeneron Pharmaceuticals ’ CRISPR/Cas9 genome editing therapy NTLA-2001 is currently in Phase I trial (NCT04601051). Neurimmune’s monoclonal antibody therapy NI006 demonstrated a positive safety profile in the Phase I trial. NI006 is being developed in partnership with AstraZeneca .

Clinical ResultPhase 3Phase 1NDA

20 May 2023

·www.biospace.com

Neurimmune: Promising Phase 1 Results for Antibody NI006 in ATTR Cardiomyopathy Published in the New England Journal of Medicine

Results also presented during late-breaking session at ESC Heart Failure congress 2023 ZURICH, SWITZERLAND / ACCESSWIRE / May 20, 2023 / Neurimmune announced today that primary results of its Phase 1 proof-of-concept study of NI006, a recombinant human antibody to deplete amyloid deposits in ATTR cardiomyopathy, have been presented in Prague at Heart Failure 2023, a scientific congress of the European Society of Cardiology (ESC), and published in The New England Journal of Medicine (DOI: 10.1056/NEJMoa2303765). Amyloid transthyretin cardiomyopathy (ATTR-CM) is an underdiagnosed, systemic condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis.1,2 Progressive ATTR amyloid depositions characterize the disease, causing heart failure and death. Despite recent advances and approved therapies, there remains significant unmet medical need in treating moderate to severe ATTR-CM, and amyloid depletion may constitute an important new mechanism to achieve further effectiveness. NI006 has been designed to target the pathology of ATTR-CM by enabling depletion of amyloid fibrils in the heart. This Phase 1 study investigated safety, tolerability, pharmacology, and efficacy at 12 months of NI006 treatment in ascending doses. Results indicate that the safety pro NI006 is favorable up to the highest dose tested. No apparent dose-limiting toxic effects or drug-related serious adverse reactions were observed. The pharmacokinetic pro consistent with that of an IgG antibody, and no antidrug antibodies were detected. At doses of at least 10 mg per kilogram, cardiac amyloid deposition (detected by either scintigraphy or cardiac magnetic resonance imaging) was substantially reduced over a period of 12 months. Reductions were also seen in levels of biomarkers measuring cardiac stress and cardiomyocyte death, N-terminal pro-B-type natriuretic peptide and troponin T. "The depletion of the cardiac ATTR deposits is a rational therapeutic target to revert disease pathology and restore organ function", said Dr. Pablo Garcia-Pavia from the Hospital Universitario Puerta de Hierro and CNIC in Madrid, Spain, and principal investigator of the study. "The results of this study are very promising and show initial evidence that NI006 acts as a depleter of cardiac amyloid load with potential to improve cardiac structure, function and outcomes in ATTR cardiomyopathy." "ATTR cardiomyopathy is a rare but serious condition that is progressive, systemic and potentially fatal," said Gianluca Pirozzi, SVP, Head of Development, Regulatory and Safety, Alexion. "These early results are encouraging, and we look forward to continuing to evaluate NI006 as a potential therapeutic option to address the high unmet medical need." "We thank all patients, their families and investigators, study staff and collaborators for the participation in the NI006 first-in-human study," said Prof. Christoph Hock, Chief Medical Officer of Neurimmune. "The NI006 results warrant further development of this drug candidate with the potential of reverting disease progression in ATTR amyloidosis." In 2022, Neurimmune entered into an exclusive global collaboration and license agreement with Alexion, AstraZeneca's Rare Disease group, for NI006. Neurimmune will continue to be responsible for completion of the current Phase 1 clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Following the Phase 1 trial, Alexion will be responsible for further clinical development, manufacturing, and commercialization. 1. Lauppe RE, et al. Nationwide prevalence and characteristics of transthyretin amyloid cardiomyopathy in Sweden. Open Heart. 2021 Oct;8(2):e001755. doi: 10.1136/openhrt-2021-001755. 2. González-Duarte A, et al. Impact of non-cardiac clinicopathologic characteristics on survival in transthyretin amyloid polyneuropathy. Neurol Ther. 2020;9(1):135-149. doi:10.1007/s40120-020-00183-7. NI006 Published Article in The New England Journal of Medicine: Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid Pablo Garcia-Pavia, MD PhD1; Fabian aus dem Siepen, MD2; Erwan Donal, MD PhD3; Olivier Lairez, MD4; Peter van der Meer, MD PhD5; Arnt V. Kristen, MD6; Michele F. Mercuri, MD PhD7; Aubin Michalon, PhD8; Robert J. A. Frost, MD PhD8; Jan Grimm, PhD8; Roger M. Nitsch, MD8,9; Christoph Hock, MD8,9; Peter C. Kahr, MD8,10; Thibaud Damy, MD PhD11. N Engl J Med 2023; DOI: 10.1056/NEJMoa2303765 The authors' affiliations are as follows: 1) Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, Madrid, Spain, Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain, Universidad Francisco de Vitoria (UFV), Pozuelo de Alarcon, Spain 2) University Hospital Heidelberg, Department of Cardiology, Im Neuenheimer Feld 410, 69120 Heidelberg, Germany 3) Department of Cardiology, University of Rennes, CHU Rennes, INSERM, LTSI - UMR 1099, Rennes, France 4) CHU de Toulouse - Hôpital Rangueil, Service de Cardiologie, 1 Avenue du Pr. Jean Poulhès, 31059 Toulouse, France 5) Department of Cardiology, University Medical Center Groningen, University of Groningen, Hanzeplein 1, 9713 GZ Groningen, The Netherlandsn 6) Cardiovascular Center Darmstadt, Dieburger Straße 31c, 64287 Darmstadt, Germany 7) Alexion/AstraZeneca Rare Disease, 121 Seaport Blvd, Boston, MA 02210, USA 8) Neurimmune AG, Wagistrasse 18, 8952 Schlieren, Switzerland 9) Institute for Regenerative Medicine, University of Zurich, Wagistrasse 13, 8952 Schlieren, Switzerland 10) Center for Molecular Cardiology, University of Zurich, Wagistrasse 13, 8952 Schlieren, Switzerland 11) Cardiology Department and French National Reference Centre for Cardiac Amyloidosis, at Hôpitaux Universitaires Henri Mondor, AP-HP, and IMRB, INSERM, Université Paris Est Creteil, 94010 Créteil, France. Contact Information Martin Meier-Pfister Neurimmune Media Team media@neurimmune.com SOURCE: Neurimmune View source version on accesswire.com:

Phase 1License out/in

100 Deals associated with NI-006

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	US	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	CN	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	JP	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	AR	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	AU	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	AT	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	BE	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	BR	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	CA	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024
Amyloidosis, Hereditary, Transthyretin-Related	Phase 3	CZ	AstraZeneca Global R&D (China) Co., Ltd.	11 Jan 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04360434 (NI006-101, Literature) Manual	Phase 1	Transthyretin Amyloid Cardiomyopathy	40	NI006	ojhfttkllh(nzcfhotmuk) = The use of NI006 was associated with no apparent drug-related serious adverse events. augjaoyckw (vmfnvwnmby )	Positive	30 Jul 2023
				placebo

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