Two giants of the pharmaceutical industry, Biogen and Ionis Pharmaceuticals, said their investigational treatment for infantile-onset spinal muscular atrophy demonstrated statistically significant improvement in certain motor functions, according to an interim analysis of late-stage study data. The treatment was developed from discoveries made at UMass Medical School, with grant support from Cure SMA, a leading nonprofit that funds research into spinal muscular atrophy.
The companies announced the results on Aug. 1, noting that they had stopped the trial earlier than planned because of the promising results.
Spinal muscular atrophy is a genetic disease that results in the loss of motor neurons in the spinal cord that leads to paralysis and difficulty in basic functions such as swallowing and breathing. There currently is no approved treatment for the disease. Ionis Pharmaceuticals was formerly known as Isis Pharmaceuticals, which licensed rights to develop discoveries made at UMass Medical School that used “antisense” technology to increase the availability of a protein crucial for normal neuron function.
According to the Wall Street Journal, Biogen will exercise an option to develop and commercialize the treatment, known as nusinersen, and pay Ionis a licensing fee. Biogen also plans to submit regulatory filings seeking approval of the therapy globally in coming months. United States and European regulators have granted special status to nusinersen in an effort to expedite the review process, including orphan drug status and fast track designation in the United States and orphan drug designation in the European Union.
The companies said that studies of nusinersen in presymptomatic infants and later-onset spinal muscular atrophy will continue.