AstraZeneca buys into protein drugs for $200M+

AstraZeneca has signed two separate drug development and licensing deals in the past month to treat a rare protein disease. | DBT PHOTO BY JACOB OWENS

WILMINGTON – Pharmaceutical giant AstraZeneca has signed two separate global development and commercialization agreements for drugs that treat a protein disease, signaling that the firm sees the patient segment as a potential growth area.

AstraZeneca, which has its U.S. headquarters in the Wilmington suburbs, signed the first deal with Ionis Pharmaceuticals, a California-based biotech firm that specializes in RNA-targeted therapeutics, on Dec. 29. The two firms will jointly develop and commercialize the drug eplontersen in the U.S., while AstraZeneca obtained the rights to do so overseas, aside from Latin America.

AstraZeneca paid Ionis $200 million upfront in the cash deal, while agreeing to additional conditional payments of up to $485 million following regulatory approvals. It will also pay up to $2.9 billion of sales-related milestones based on sales thresholds between $500 million and $6 billion, plus royalties in the range of low double-digit to mid-20s percentage depending on the region.

Ionis will continue to manufacture and supply eplontersen for the existing clinical studies and process qualification, while AstraZeneca will eventually be responsible for commercial supply.

Then on Jan. 7, AstraZeneca’s rare disease group, Alexion, announced that it had reached an exclusive global collaboration and license agreement with Neurimmune AG, a Swiss biopharma firm, for its monoclonal antibody. The deal grants Alexion an exclusive worldwide license to develop, manufacture and commercialize the drug known only as NI006 so far. The AstraZeneca subsidiary will pay $30 million upfront, with the potential for additional milestone payments of up to $730 million after certain development, regulatory and commercial milestones, as well as low-to-mid teen royalties on net sales of any approved medicine resulting from the collaboration.

Neurimmune will continue to be responsible for completion of the current Phase 1B clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing and commercialization.

Both eplontersen and NI006 are used to treat forms of amyloidosis, a rare protein disease. Eplontersen is an antisense investigational medicine currently in Phase 3 clinical trials that reduces the production of a troublesome protein, transthyretin (TTR), which can build up and cause the stiffening of heart muscles and other tissues and organs. NI006 targets certain transthyretin, allowing for its removal from the heart.

If untreated, the amyloidosis disease is progressive and fatal within a few years of diagnosis. It can be both hereditary and non-hereditary in nature. It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms, the firms reported. Worldwide, there are an estimated 300,000 to 500,000 patients with the heart-related disease, transthyretin amyloid cardiomyopathy (ATTR-CM), and 10,000 to 40,000 patients with the nerve-related disease, amyloid transthyretin polyneuropathy (ATTR-PN).

“Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, irrespective of whether it’s caused by genetic mutations or aging. Thanks to its precise liver-targeting properties, it also has the potential to be a best-in-class treatment for patients suffering from this devastating disease and who currently have limited options,” said Mene Pangalos, executive vice president of biopharmaceuticals R&D at AstraZeneca, in a statement.

Hereditary ATTR-PN is expected to be the first indication for which AstraZeneca and Ionis will seek regulatory approval for eplontersen, with the potential to file a new drug application with the U.S. Food and Drug Administration by the end of 2022. Meanwhile, Alexion and Neurimmune are advancing work on NI006 in treatment of ATTR-CM.