AstraZeneca Enters Two Agreements for Transthyretin Amyloid Cardiomyopathy and Transthyretin Amyloidosis

AstraZeneca has entered into two collaborative agreements which will improve research for two rare diseases: transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloidosis (ATTR).


The first collaboration is with Neurimmune AG to work on the development and commercialization of N1006 for ATTR-CM.

N1006 is an ATTR depleter that has previously been studied as a treatment for eliminating cardiac amyloid fibril deposits. It has also demonstrated an ability to improve cardiac function in ATTR-CM patients.

ATTR-CM is a rare, progressive, and ultimately fatal disease that ends in heart failure. Therefore, a treatment that could improve cardiac function would be life-changing.

This therapy is currently being studied in a Phase 1b clinical trial, led by Neurimmune, and paid for (in part) by AstraZeneca.

The new collaboration will include a 30 million dollar payment from AstraZeneca to Neurimmune as well as further milestone payments. Neurimmune will run the clinical trial and AstraZeneca will lead the clinical development, commercialization, and manufacturing of the therapy.


Back in December, an agreement was closed between AstraZeneca and Ionis Pharmaceuticals surrounding the development and commercialization of the therapy eplontersen for ATTR amyloidosis.

Through this collaboration, Ionis is responsible for the manufacturing and the supply of the treatment for current clinical trials. AstraZeneca is responsible for the entire commercial supply. Additionally, AstraZeneca will be in charge of recording all sales of the therapy.

You can read more about these two agreements and the rare disease treatments they involve here.

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