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Ionis’ HAE Opportunity Boosted By Positive Phase II Data

Wholly Owned PKK-LRx HAE Candidate Hit All Endpoints

 
• By Sten Stovall

Positive topline Phase II data for Ionis’ antisense hereditary angioedema candidate has boosted the biotech’s chances of developing PKK-LRx as a treatment for the disease.    

HAE
Positive HAE data lifts investor sentiment towards Ionis stock • Source: Shutterstock

Ionis Pharmaceuticals, Inc.'s investigational antisense medicine IONIS-PKK-LRx produced impressive topline results in a Phase II trial in hereditary angioedema, easily hitting primary and secondary endpoints and producing a 90% reduction in the number of monthly HAE attacks.

Although the trial was small – comprising just 20 patients – the administered antisense oligo (ASO), designed to reduce the...

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