Offense rare disease field!Aslikon United Ionis developed EPLONTERSEN

Compiling 丨 Fan Dongdong

Upon completion of the acquisition of Alexion's only a few months, AstraZeneca has recently signed a new global eplontersen on development and commercialization agreement with Ionis Pharmaceuticals. The two companies will jointly develop eplontersen representation in the US market and commercialized, while AstraZeneca will be responsible alone in the rest of the world (except Latin America) to develop and commercialize the drug.

Ionis AstraZeneca will pay $ 200 million upfront payment and pay up to $ 485 million in additional payments conditional upon regulatory approval. According to US $ 500 billion to $ 6 billion sales threshold, AstraZeneca will also be costs associated with sales milestones of up to $ 2.9 million, depending on the region as well as payment of royalties and given the low double digits to mid-1920s use fee, also includes cooperation for the development of specific areas, commercial and medical affairs cost-sharing provisions.

The partnership will enable Pfizer and AstraZeneca join the competition of Alnylam. The previously known pharmaceutical IONIS-TTR-LRX, research is an antisense drug to the ligand coupling, eplontersen currently in treating transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloid polyneuropathy (ATTR-PN) phase III clinical trials. The therapy by reducing the generation transthyretin (TTR protein) to treat genetic and non-genetic form of TTR amyloidosis (ATTR).

After the start of cooperation, Ionis will continue to process existing clinical studies and certified manufacturing and supply eplontersen. AstraZeneca will be responsible for commercial supply, the transition period agreed upon by both parties. AstraZeneca will register all sales generated according to the protocol. According to customary closing conditions and regulatory approval, the transaction is expected in the fourth quarter will be completed in 2021. AstraZeneca said the deal Ionis and cooperation will not affect the 2021 financial guidance.

Hereditary ATTR-PN is expected to become the first two companies to seek regulatory approval eplontersen indications, and is likely to submit a new drug application to the US Food and Drug Administration (FDA) by the end of 2022. If eplontersen ultimately approved, the drug could be competition in the market and Alnylam RNA interference therapy Onpattro and Pfizer rapid growth Vyndamax. Mani Foroohar SVB Leerink analyst in a report to clients said in October that Wall Street believes eplontersen can generate $ 1.3 billion in sales in 2030.

ATTR-CM is a systemic, progressive and fatal disease that can lead to progressive heart failure and death within four years after diagnosis. Due to lack of awareness and heterogeneity of the symptoms of the disease, its diagnosis is still insufficient, and its prevalence is thought to be underestimated. The hereditary ATTR-PN is a debilitating disease that can cause peripheral nerve damage and movement disorders within five years after diagnosis, if left untreated, is usually fatal within a decade.

According to Ionis that global estimated 300,000 to 500,000 patients with ATTR. Although the disease is classified as a rare disease, but ATTR may not be so rare as people think. Pfizer has said ATTR may be more prevalent disease that has not been fully diagnosed. Therefore, to promote awareness and diagnosis of the disease has been a key strategy of Pfizer launched Vyndamax, this strategy is working. 2021 first nine months, Vyndamax and Vyndaqel brought in $ 1.45 billion in global sales, an increase of 66%.

It is worth noting that recent actions by AstraZeneca point of view, the company seems to have a tendency to develop orphan drugs. AstraZeneca has just blocked an investment group for $ 8.0 billion privatization deal Sobi, because this competition as well as the British pharmaceutical company Sobi consider buying some assets. Sobi has the right to AstraZeneca respiratory syncytial virus drugs Synagis in the US market, but AstraZeneca has just recently announced the acquisition of Alexion. In ATTR indications, AstraZeneca entered into a more crowded competitive space. In addition to Vyndamax and Onpattro, Alnylam is also developing a second-generation RNAi therapeutics called vutrisiran, according to Wall Street estimates, by 2030, this therapy could generate $ 3.6 billion in revenue.

Reference Source:

1.Watch out, Pfizer and Alnylam. AstraZeneca wades deeper into rare diseases with Ionis tie-up

2.AstraZeneca and Ionis sign deal to develop and commercialise eplontersen

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