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 Moves SMA Drug Forward As Market Interest For The Rare Disease Expands
Spinal muscular atrophy is a rare disease that affects a small patient population of children. Isis is ready to move its compound into mid-stage testing after positive Phase I results, but the biotech isn’t the only company exploring the field.
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