Skip to content
Medical Health Aged Care

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

IRF Reputation AG 4 mins read

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).

IRF Reputation AG, Saturday, May 20, 2023, Press release picture

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 profile of 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 profile was 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

SOURCE: Neurimmune


View source version on

More from this category

  • Medical Health Aged Care, Property Real Estate
  • 19/06/2024
  • 19:54
Kurraba Group

Kurraba launches Australia’s first-commercial life sciences campus

Sydney, Australia: Leading Australian life sciences and bio-medical property investment and development manager, the Kurraba Group (‘Kurraba’), is excited to announce the launch of Australia’s first commercial life sciences Campus, a $490-million development in the heart of Sydney’s emerging health and innovation precinct. Just 2kms from the CBD, the strategically-located 26,000sqm development site at 100 Botany Road, Waterloo, will house hi-tech laboratories, knowledge workspaces, and the necessary infrastructure and facilities for professionals working in Australia’s rapidly-evolving life sciences sector. The Campus will drive investment, productivity and high-growth across advanced medical and health sciences industries, creating a robust platform for local…

  • Medical Health Aged Care
  • 19/06/2024
  • 15:15
Royal Australian College of GPs

RACGP thanks outgoing Rural Health Commissioner for her service and advocacy

The Royal Australian College of GPs (RACGP) has thanked outgoing National Rural Health Commissioner Associate Professor Ruth Stewart for her service and welcomed the appointment of Professor Jenny May AM to the role. Professor May’s appointment was announced by the Minister for Health and Aged Care the Hon Mark Butler MP today, commencing 2 September 2024. RACGP President Dr Nicole Higgins thanked Associate Professor Stewart for her service and leadership. “Thanks to Ruth Stewart for her fierce advocacy, her service, and her leadership in advancing healthcare for rural Australians,” she said. "The RACGP worked with Associate Professor Stewart on progressing…

  • Contains:
  • CharitiesAidWelfare, Medical Health Aged Care
  • 19/06/2024
  • 12:51
National Rural Health Alliance

Congratulations to our long-term ally Prof Jenny May AO on her new role as National Rural Health Commissioner

The National Rural Health Alliance (the Alliance) congratulates Professor Jenny May AO, a long-term ally of the Alliance, on her appointment as the National…

  • Contains:

Media Outreach made fast, easy, simple.

Feature your press release on Medianet's News Hub every time you distribute with Medianet. Pay per release or save with a subscription.