Ionis Reports Positive Topline Data From The HALOS Phase 1/2a Study In Angelman Syndrome

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) Thursday reported positive topline data from the HALOS Phase 1/2a study of ION582 in Angelman syndrome, a neurodevelopmental disorder.

ION582 was safe and well tolerated in the study and showed consistent improvements in key areas of functioning including cognition, communication and motor function in Angelman syndrome patients.

At 6 months, 65 percent of patients achieved improvement in cognition and motor skills, and 70 percent of patients showed improvement in communication on the Bayley-4, an objective physician assessment of clinical functioning. Ionis said it plans to move ION582 into pivotal trial.