Planting another flag on Pfizer and Alnylam's turf, AstraZeneca grabs amyloidosis drug from the biotech that invented Aduhelm

07 Jan 2022
endpts.com
CollaborateAntibody
AstraZeneca — or, more specifically, its rare disease subsidiary Alexion — is serious about getting into ATTR amyloidosis. Just weeks after licensing a late-stage antisense candidate from Ionis, AstraZeneca has struck another deal to pick up a Phase Ib antibody hitting the same target, this time from Swiss biotech Neurimmune.
The upfront from Alexion comes in at $30 million, with the potential to add up to $730 million in milestones. Alexion is hoping the program would tackle transthyretintransthyretin amyloid cardiomyopathy, or ATTR-CM, which is characterized by cardiac buildup of toxic amyloid fibrils.
As ATTR-CM can lead to progressive heart failure and be fatal, the deal fits into AstraZeneca’s broader ambitions to become the leader in heart failure, integrating into a portfolio that also features the SGLT2 inhibitor Farxiga. Eplontersen, the antisense compound it scored from Ionis weeks ago for $200 million in cash, is also being tested in ATTR cardiomyopathy (in addition to ATTR polyneuropathy).
“With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed,” CEO Marc Dunoyer said. Dubbed NI006, the Neurimmune drug specifically targets misfolded transthyretin and is designed to spur the removal of amyloid fibril deposits in the heart.
Neurimmune is known for its other amyloid-clearing antibody, the FDA-approved Alzheimer’s drug Aduhelm, which was still known as aducanumab when it was licensed to Biogen.
It will be responsible for completing the current Phase Ib trial, with Alexion shouldering part of the costs before taking over clinical development, manufacturing and commercialization.
But Alexion will have plenty of catching up to do. Pfizer’s Vyndaqel and Vyndamax (two formulations of the same transthyretin-stabilizer, tafamidis) were approved two and a half years ago for ATTR-CM, and together they brought in more than $1 billion in sales just over the first nine months of 2021. Alnylam is also far ahead in testing its suite of RNAi therapies for the indication.
In a statement, Alexion noted that “multiple mechanisms of action” will likely be required to address the needs of patients with various types and levels of severity of amyloidosis.
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