Initiation of a Phase 3 Study of ALXN2220 for the Treatment of Transthyretin-mediated Amyloid Cardiomyopathy

Neurimmune today announced that its collaboration partner Alexion, AstraZeneca’s Rare Disease group, initiated the Phase 3 DepleTTR-CM clinical study to assess the efficacy and safety of ALXN2220 (formerly NI006) for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) (NCT06183931). ALXN2220 is an investigational human monoclonal antibody designed to deplete amyloid deposits in ATTR-CM in combination with standard of care.^1,2

Amyloid transthyretin cardiomyopathy (ATTR-CM) is increasingly recognized as a treatable cause of heart failure. Progressive ATTR amyloid depositions characterize the disease, leading to progressive heart failure and a high rate of fatality if left untreated. Despite recent advances in slowing disease progression, there is no treatment available to deplete cardiac ATTR amyloid and revert cardiac dysfunction.^3,4,5

“We are excited about the opportunity to advance, together with our collaboration partner Alexion, AstraZeneca Rare Disease, the cardiac amyloid depleting antibody ALXN2220 to Phase 3 clinical development,” said Roger M. Nitsch, President and CEO of Neurimmune. “The DepleTTR-CM study is the first study designed to demonstrate that depletion of cardiac amyloid can lead to improved cardiovascular health in patients with ATTR-CM. The initiation of Phase 3 is an important milestone. We thank the Alexion team, the clinical investigators and the study participants for their contributions and commitment to this program.”

The Phase 3 DepleTTR-CM study is randomized, double-blind, international multicenter clinical study to evaluate the efficacy and safety of ALXN2220 for the treatment of ATTR-CM. Participants will receive either ALXN2220 or placebo via intravenous infusion every four weeks. The primary endpoint is a composite endpoint to assess both survival and cardiovascular health over the treatment period of up to 48 months.

“Given the progressive nature of ATTR and the substantial impact on quality of life, there is an urgent need for additional treatment options for patients across all stages of the disease,” said Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion. “We are encouraged to see ALXN2220 advance to Phase 3, a significant milestone as we work collectively to bring new innovation to patients.”

In 2022, Neurimmune entered into an exclusive global collaboration and license agreement with Alexion, AstraZeneca’s Rare Disease group, for ALXN2220 (formerly NI006). Neurimmune is responsible for completion of the Phase 1b clinical study on behalf of Alexion, with Alexion incurring certain trial costs. Aside from the Phase 1b trial, Alexion is responsible for further clinical development, manufacturing, and commercialization as ALXN2220.

For additional information, please visit clinicaltrials.gov: NCT06183931

¹Garcia-Pavia et al., N Engl J Med 2023; 389(3):239.
²Michalon et al., Nat Commun 2021; 12(1):3142.
³Garcia-Pavia et al., Eur Heart J 2021; 42(16):1554.
⁴Kittleson et al., Circulation 2020; 142(1):e7.
⁵Kittleson et al., J Am Coll Cardiol 2023; 81(11):1076.