Isis Moves SMA Drug Forward As Market Interest For The Rare Disease Expands

Early efficacy results for Ionis Pharmaceuticals Inc.’s spinal muscular atrophy (SMA) drug seem promising, but the biotech needs to run more trials before it can prove the antisense drug will be worthy of partner Biogen Inc.’s attention. The company is moving forward cautiously in a space that is expected to become more crowded in the next five years as compounds from Roche and Pfizer Inc., among others, enter the clinic.

Isis announced results from a Phase I study of ISIS-SMrx, an antisense compound meant to treat SMA. The disease, which tends to manifest in children as young as six-months old, is caused by a defect of the SMN1 gene that results in the body failing to make a protein needed for muscle development