AstraZeneca is stepping up its pursuit of a rare disease market targeted by Alnylam and Pfizer. Having entered the ATTR space through its takeover of Alexion, AstraZeneca is paying $30 million for worldwide rights to Neurimmune’s NI006 in its second deal in the indication in quick succession.
NI006 targets misfolded transthyretin, the protein at the heart of ATTR. Changes to the structure of the protein impair its ability to bind to other molecules, leading to deposits in the heart or the nerves. The resulting cardiomyopathy and polyneuropathy, known respectively as ATTR-CM and ATTR-PN, are treated today with Pfizer’s Vyndaqel and Vyndamax and Alnylam’s Onpattro.