AstraZeneca picks up new weapon in rare disease fight with Alnylam and Pfizer, paying $30M for rights to an early-phase asset

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.

While treatments are now available, AstraZeneca still spies an opportunity—and is advancing on several fronts to seize it. Alexion already had rights to a late-phase ATTR-CM candidate in Japan at the time of its takeover by AstraZeneca, although the recent failure of BridgeBio Pharma’s phase 3 trial of the prospect has taken some of the shine off that asset. 

Last month, AstraZeneca licensed a phase 3 antisense drug for use in ATTR-CM and ATTR-PN from Ionis Pharmaceuticals for $200 million upfront, giving it another shot on goal. The latest deal sees AstraZeneca move into earlier development. In return for $30 million upfront and up to $730 million in milestones, AstraZeneca has secured a global license to an antibody Neurimmune is studying in patients with wild-type and hereditary ATTR-CM in a phase 1b clinical trial. 

RELATED: AstraZeneca wades deeper into rare diseases with Ionis tie-up

Neurimmune has designed NI006 to remove the amyloid that builds up in the hearts of patients with ATTR-CM. By looking at samples from healthy elderly subjects, Neurimmune hit upon an antibody that it expects to target the disease-associated amyloid conformation with high affinity and leave physiological forms of transthyretin alone. The antibody cleared pathological ATTR in preclinical models.

NI006 entered a 36-subject phase 1 clinical trial early in 2020. lists the completion date as June 2022, suggesting that AstraZeneca will soon be in a position to decide on the next steps for the  program.

The drug is targeting a competitive area. It is less than three years since Pfizer became the first company to win FDA approval for a drug to treat wild-type or hereditary ATTR-CM. But with Alnylam targeting a mid-2022 data drop for a phase 3 trial of Onpattro in ATTR-CM—while moving its follow-up vutrisiran through a pivotal study—and BridgeBio still pushing ahead with its program, AstraZeneca could face multiple competitors.