Ionis Pharma's genetic disease drug succeeds in late-stage study

Ionis Pharmaceuticals said on Monday its experimental drug reduced the occurrence of painful attacks in patients with a rare genetic disease that triggers frequent episodes of swelling.

The life-threatening genetic disease called hereditary angioedema causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat.

Shares of the drugmaker rose as much as 3% in premarket trading.

Stifel analyst Paul Matteis said questions remain over the commercial opportunity for donidalorsen, given competition from both existing drugs made by Takeda Pharmaceuticals and BioCryst Pharmaceuticals and other experimental treatments.

He added that the results from the late-stage study represent a clear win for Ionis, but would await for more data on the Ionis drug, expected to be presented in the future, to fully understand its differentiation versus rivals.

Takeda and BioCryst's drugs combined brought in sales of about $1.5 billion in 2023. Other companies such as CSL are also developing drugs to treat the condition.

In the study, patients received an 80 milligram dose of the Ionis drug, donidalorsen, through subcutaneous or under-the-skin injection every four weeks or every eight weeks.

Meeting the main goal of the trial, the drug showed statistical significant reduction in the rate of the attacks when compared to placebo.

Ionis said it is preparing to submit a marketing application with the U.S. Food and Drug Administration based on the data.

Japan-based Otsuka, which has exclusive rights to commercialize donidalorsen in Europe, is preparing to submit an application to the European Medicines Agency, the company said. (Reporting by Sriparna Roy and Puyaan Singh in Bengaluru; Editing by Devika Syamnath and Shailesh Kuber)