Almost four years after penning its first amyloidosis collaboration with Neurimmune, AstraZeneca has returned to pick up a second asset.
The latest licensing deal involves NI009, a preclinical monoclonal antibody that targets lambda light chain fibrils and deposits from affected tissues and organs in light chain (AL) amyloidosis. In return for the exclusive rights, AstraZeneca’s Alexion Pharmaceuticals rare disease unit is liable to pay up to $780 million in a combination of upfront and potential development, regulatory and commercial milestone payments.
NI009 is currently in “advanced preclinical development,” according to Neurimmune, which will also oversee the first clinical trial of the drug before handing the process over to Alexion.
Should NI009 make it to market, Neurimmune will also be in line for tiered royalties on net sales.
AL amyloidosis is a rare, progressive disorder caused by defective plasma cells in the bone marrow. The accumulation of these toxic amyloid deposits, notably in the heart and kidneys, can cause progressive organ damage and dysfunction, Neurimmune explained in a Dec. 4 release.
NI009 has so far exhibited “broad activity against amyloids of diverse lambda light chain subtypes across patients despite the high clonal heterogeneity of the disease,” Neurimmune’s CEO Roger Nitsch said in the release.
This morning’s deal follows AstraZeneca’s $30 million payment to the biotech at the start of 2022 to license the ATTR amyloidosis candidate NI006. Since then, Neurimmune has read out data from a phase 1 study of NI006 that it claimed showed “substantial” reductions in cardiac amyloid deposition at higher doses.
AstraZeneca already has a more advanced AL amyloidosis candidate in its pipeline in the form of anselamimab, which it acquired when it bought out Caelum Biosciences back in 2021. However, the pharma’s hopes of turning anselamimab into a potential blockbuster appeared dampened in July of this year, when the phase 3 Cares study showed the light chain depleter antibody failed to reduce deaths and hospitalizations.
The U.K.-based drugmaker claimed at the time that anselamimab had demonstrated a “highly clinically meaningful improvement” in an unspecified subgroup of patients, and the upbeat rhetoric was maintained in this morning’s release.
“This announcement follows the high-level results from Alexion's CARES phase 3 clinical program which demonstrated anselamimab's potential as a first-in-class fibril depleter to improve survival and cardiac outcomes in patients living with advanced AL-kappa amyloidosis, a debilitating progressive rare disease,” Gianluca Pirozzi, head of development, regulatory and safety at Alexion, said.
“In expanding our collaboration with Neurimmune, we aim to apply key learnings from the CARES program to develop NI009 as a complementary fibril depleter to potentially benefit more patients living with AL amyloidosis,” Pirozzi added.