Isis Pharmaceuticals, the California-based pharmaceutical company that specializes in antisense based drug development, has announced the initiation of a Phase III trial of ISIS-SMNRx in 120 non-ambulatory children with Spinal Muscular Atrophy (SMA). Patients with SMA express low levels of the survival of motor neuron protein (SMN) due to a mutated SMN1 gene, and ISIS-SMNRx functions by increasing production of SMN through a closely related gene, SMN2. In parallel to this study, Isis is conducting a Phase III study in infants with the disease. Isis will receive a $27M milestone payment from its development partner, Biogen Idec, for launching this trial.
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