AstraZeneca pumps US$ 200 million to acquire rights into Ionis’ rare disease drug

UNITED KINGDOM – AstraZeneca has announced that it would pay Ionis Pharmaceuticals, the market leader in RNA-targeted therapies, US$200 million for the rights to its investigational medicine, eplontersen.

Eplontersen is a ligand-conjugated antisense investigational medicine that is currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy and polyneuropathy.

It is intended to reduce transthyretin (TTR) production in order to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

ATTR amyloidosis is a very rare type of amyloidosis caused by misfolding TTR proteins, which interferes with normal function in peripheral nerves, the heart, the gastrointestinal system, the eyes, the kidneys, the central nervous system, the thyroid, or bone marrow tissues.

According to a company press release, it is a progressive, systematic, and ultimately fatal disease that typically results in progressive heart failure within four years of diagnosis.

Eplontersen is intended to treat ATTR, a systemic, progressive, and fatal disease, by reducing the production of transthyretin, or TTR protein. It makes use of Ionis’ cutting-edge LIgand-Conjugated Antisense (LICA) technology.

The companies will work together to create a global strategy for developing, manufacturing, and commercializing eplontersen.

Ionis will receive a US$200 million upfront payment, up to US$485 million in development and approval milestone payments, and up to US$2.9 billion in sales-related milestone payments under the terms of the agreement.

The collaboration includes cost-sharing provisions for territory-specific development, commercial, and medical affairs. Ionis is also eligible for royalties ranging from the low double digits to the mid-20s, depending on region.

Ionis worked with GlaxoSmithKline for many years to develop Tegsedi. However, the pharma company declined an option to acquire the drug in 2017 after concerning side effects were discovered in a Phase 3 trial.

Eplontersen’s competitors

Onpattro, a competitor medicine from Alnylam Pharmaceuticals that did not have the same safety concerns, had previously reached the US market.

Tegsedi was approved by the Food and Drug Administration soon after, but it hasn’t sold as well as Onpattro.

One of the reasons Ionis chose to acquire and restructure Akcea Therapeutics, a former spin-off tasked with selling Tegsedi and other Ionis medicines, was the drug’s poor performance. Sobi, a Swedish company, now sells the majority of Tegsedi.

Ionis has been hoping for better results with eplontersen, a second-generation Tegsedi that is supposed to be more precise and address a larger portion of the population with ATTR.

Unlike Tegsedi, eplontersen is intended for patients with both inherited and acquired forms of the disease, whereas Tegsedi only treats the former.

Tegsedi is also only approved for ATTR patients who have nerve damage, whereas eplontersen is in late-stage testing for those who also have heart damage.

Intellia Therapeutics’ gene editing drug has also shown promise in early testing. And, like eplontersen, Novo Nordisk recently acquired a drug from Prothena that is intended for both inherited and acquired forms of ATTR.

The agreement expands AstraZeneca’s focus on rare disease drugs, specifically amyloidosis. In July, AstraZeneca completed its nearly US$40 billion acquisition of Alexion Pharmaceuticals, and a few months later, it purchased Caelum Biosciences, which is developing a drug for a different type of amyloidosis.

The AstraZeneca-Ionis agreement grants AstraZeneca partial U.S. rights to Ionis’ drug and full ownership elsewhere.

Ionis anticipates Phase 3 results in ATTR patients with nerve damage in the middle of 2022, and if successful, it may file for approval by the end of the year.

In other related news, Lonza and Bioqube Ventures, a European venture capital firm, have signed a framework agreement for a strategic collaboration in which Lonza will assist Bioqube Ventures’ portfolio companies in developing and manufacturing biologics and small molecules.

According to a recent press release, Lonza will assist Bioqube Ventures during the due diligence of candidate biotechs and will provide a tailored offering of advice and services to those portfolio companies.

Lonza provides expertise and technology to accelerate timelines while mitigating risks associated with the development and manufacturing of molecules ranging from monoclonal antibodies, complex proteins, small molecules, and antibody-drug conjugates.

The company’s approach to drug substance and drug product development and manufacturing streamlines the supply chain, reduces process complexity, and shortens development timelines.

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