According to a story from PR Newswire, the biotechnology company Ionis Pharmaceuticals recently announced promising findings from its phase 2 clinical trial. This clinical trial was evaluating the company’s experimental antisense therapy called IONIS-PKK-LRx as a treatment for hereditary angioedema, a rare disease. In this study, the treatment was able to successfully achieve the primary and secondary endpoints.
About Hereditary Angioedema (HAE)
Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the SERPING1 gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attacks as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.
Trial Findings
In this trial, twenty people living with either type 1 or type 2 hereditary angioedema received either a placebo or an 80 mg dose of the experimental treatment. The treatment was administered once per month for a period of 17 weeks. The primary endpoint for this study was to bring down the number of monthly symptom episodes, and patients that received IONIS-PKK-LRx saw a 90 percent mean reduction in episodes. From weeks five to 17, the number of attacks was reduced by a mean of 97 percent; in this period, 92 percent of patients experienced no symptoms. Zero percent of patients in the placebo group were free of symptoms. Adverse effects in the study were mostly minor and included nausea and headaches.
IONIS-PKK-LRx was developed using the company’s ligand-conjugated antisense technology. Its mechanism of action is designed to bring down the production of prekallikrein (PKK), which is known to trigger inflammatory mediators that are known to play a role in symptom attacks in hereditary angioedema. This therapy is meant as a preventative treatment and is not designed to stop symptoms once an attack has begun.
The findings from this trial bode well for the continued development of IONIS-PKK-LRx. If trials continue to go smoothly, patients living with hereditary angioedema may soon have a new and more effective preventative therapy that can prevent symptom attacks.
