Isis Pharmaceuticals (ISIS) outlines data from a Phase 2 trial of the antisense drug ISIS-APOCIIIRx in familial chylomicronemia syndrome.
All three patients saw "substantial reductions" (average was 69%) in triglyceride levels.
The disease only affects 3-5K patients worldwide, so the trial was quite small, but the data "are ... compelling and demonstrate the potential of ISIS-APOCIIIRx to produce dramatic triglyceride lowering in patients with FCS," Dr. Daniel Gaudet notes.
The company says "the significant unmet medical need [associated with FCS] should enable [ISIS to] rapidly move forward." (PR)