New Investigative Therapy for Hereditary Angioedema Found Efficacious in Study

Ionis Pharmaceuticals has just released their findings from a study investigating the effect of IONIS-PKK-LRx and IONIS-PKKRx in hereditary angioedema (HAE) patients with different forms of the disease. These therapies work to reduce production of PKK which is responsible for activating inflammatory mediators in HAE.

This study was published in the New England Journal of Medicine.


HAE is a rare disease that causes recurrent swelling attacks throughout the body. This swelling can be life threatening without treatment.

The disease is caused by either a deficiency in C1-INH (Type 1 HAE) or a dysfunction of that inhibitor (Type 2 HAE). C1-INH is responsible for regulating a few different pathways in the body.

Type 3 HAE is a particularly rare presentation of the disease, whose cause is still unknown. This form of the disease mostly presents in women. This form is also associated with a higher incidence of swelling in the tongue, face, and pharyngeal region.

The Study

This investigation included one patient who had Type 1 and one patient who had Type 3 HAE. Each patient was given IONIS-PKKRx for 12-16 weeks and then IONIS-PKK-LRx every 3 to 4 weeks for a total of 7-8 months.

PKK was found to be significantly reduced by the therapies. In addition, both patients had fewer HAE attacks each month. One patient with Type 1 HAE had complete resolution of attacks.

IONIS-PKK-LRx is additionally beneficial because it only has to be administered once each month by subcutaneous injection. This provides greater convenience to patients.

Researchers are encouraged by the results of this study. Since not all HAE patients are the same, we need to work to find therapies that will be efficacious for each subset. This treatment could be beneficial for those who don’t respond well to current treatment options.

You can read more about this investigation in HAE here.

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