AstraZeneca Expands Amyloidosis Collaboration with Deal Potentially Reaching $780 Million
Alexion unit licenses preclinical monoclonal antibody NI009 targeting AL amyloidosis
Alexion Pharmaceuticals, the rare disease unit of AstraZeneca, has expanded its partnership with biotech company Neurimmune by licensing a second drug candidate. The new agreement involves NI009, a monoclonal antibody in advanced preclinical development, with a total potential value reaching up to $780 million.
Deal Structure and Funding:
In exchange for exclusive worldwide rights to NI009, Alexion is responsible for a combination of an upfront payment and potential payments tied to clinical development, regulatory milestones, and commercialization goals.
NI009 is designed to target and clear lambda light chain fibrils and deposits from affected tissues and organs in light chain (AL) amyloidosis. The biotech partner will oversee the first clinical trial of the drug before transitioning the development and commercialization process entirely to Alexion. Should the drug successfully reach the market, the biotech company will also be eligible for tiered royalties on net sales worldwide.
AL amyloidosis is a rare, progressive disorder caused by the build-up of toxic amyloid proteins produced by faulty plasma cells. These deposits, primarily in the heart and kidneys, lead to severe organ damage and dysfunction.
Post-Challenge R&D Strategy
This latest deal follows the partners’ initial collaboration four years ago in 2022, which licensed the ATTR amyloidosis candidate NI006. Subsequently, Phase 1 data for NI006 showed substantial reductions in cardiac amyloid deposition at higher doses.
However, AstraZeneca has faced challenges with its existing AL amyloidosis candidate, anselamimab. In July of this year, the Phase 3 CARES study for anselamimab failed to meet its primary endpoint of reducing deaths and hospitalizations.
A spokesperson for Alexion stated that they plan to apply key learnings derived from the CARES program to the development of NI009. The goal is to develop a complementary fibril depleter, potentially capitalizing on NI009’s broad activity against diverse lambda light chain subtypes – a feature noted by Neurimmune’s CEO as significant despite the high clonal heterogeneity of the disease. This expanded partnership reinforces the corporation’s commitment to delivering new therapies that may improve survival and cardiac outcomes for patients with AL amyloidosis.
