Ionis Pharmaceuticals has signed a licence agreement under which Otsuka Pharmaceutical will gain sole rights to market the ligand-conjugated antisense therapy, donidalorsen, for hereditary angioedema (HAE) in Europe.
Donidalorsen is an investigational prophylactic treatment for HAE.
Ionis will be responsible for the non-clinical and clinical development of the product while Otsuka will oversee its regulatory filings and marketing in Europe.
Otsuka will make a $65m upfront payment and milestone payments to Ionis on meeting regulatory and sales-based goals.
Ionis is also entitled to receive tiered royalty payments for annual net product sales from Otsuka.
Otsuka Pharmaceutical president and representative director Makoto Inoue stated: “Otsuka has developed drugs in Europe for rare diseases such as autosomal dominant polycystic kidney disease.
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“Through this collaboration with Ionis, a leader in RNA-targeted therapy, if regulatory approval is received, we look forward to bringing donidalorsen to patients in Europe to address the unmet medical needs of patients with HAE.”
According to two-year data from the Phase II open-label extension clinical trial of donidalorsen, a 96% overall sustained mean decline was observed in HAE attack rates from baseline.
In the trial, discolouration and reaction at the injection site were the only drug-associated treatment-emergent adverse events observed, with serious adverse events seen.
Ionis will publish data from the Phase III study of the product as a prophylactic treatment of HAE in the first half of 2024.
The US Food and Drug Administration recently granted orphan drug designation for donidalorsen.
Following the product launch in Europe, Ionis also intends to introduce donidalorsen in the US.
Ionis CEO Brett Monia stated: “We are excited to collaborate with Otsuka given their proven results in bringing rare disease medicines to patients in Europe.
“This agreement is aligned with our strategy to initially focus our commercialisation efforts on the US market.
“We are encouraged by the strong product profile of donidalorsen to date and look forward to reporting pivotal topline Phase III donidalorsen results in HAE in the first half of next year.”
