Clinical success for Neurimmune

Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed, systemic clinical condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis. Despite recent advances in slowing disease progression, there is currently no treatment available to deplete cardiac ATTR amyloid and revert cardiac dysfunction. Schlieren-based startup, Neurimmune, which develops drug candidates for central nervous system (CNS) and related protein aggregation diseases including Alzheimer’s disease, amyotrophic lateral sclerosis, frontotemporal dementia and ATTR cardiomyopathy, is currently testing a drug candidate, ALXN2220 (formerly NI006), a human antibody targeting ATTR Cardiomyopathy.  

In 2022, Neurimmune entered into an exclusive global collaboration and license agreement with Alexion, AstraZeneca’s Rare Disease group, for ALXN2220. 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. The two companies have now initiated the phase 3 of the DepleTTR-CM clinical study to assess the efficacy and safety of ALXN2220 for the treatment of ATTR-CM. 

The DepleTTR-CM clinical study was started in early 2020. It is a 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. 

Roger M. Nitsch, President and CEO of Neurimmune, is very excited about this development and states, “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.” 
 
(Press release / SR / RAN)