Isis Rises on Trial Data for Babies With Spinal Muscular Atrophy

Isis Pharmaceuticals Inc. rose after reporting encouraging results in a small trial aimed at helping babies with a rare spinal disease.

Most of the babies with spinal muscular atrophy who took the treatment, known as ISIS-SMN-RX, have lived longer without permanent ventilation and increased their ability to use their muscles, the Carlsbad, California-based company said in a statement Thursday. Only one of the 19 infants who completed the treatment required permanent ventilation, the company said.

The drug, being developed with Biogen Inc., could target a $1 billion market, according to Michael Yee, an analyst at RBC Capital Markets. The company may consider filing with the U.S. Food and Drug Administration and European Medical Agency for approval early, he said.

“We are impressed by the fact that no additional infants have succumbed to their disease since the last follow-up over seven months ago,” Eric Schmidt, a senior research analyst at Cowen & Co., wrote in a note to clients. “In addition, many infants appear to be showing significant gains in muscle function that stand in contrast to the natural history of this disease.”

Shares rose 4.1 percent to $69.35 at 10:24 a.m. in New York. They reached as high as $71.50 earlier, the highest since March. In February 2014 Isis reported that a baby had died from pneumonia while being treated with the drug. A Phase 3 study, which is designed to determine the drug’s effectiveness, is in under way.

Spinal muscular atrophy affects 30,000 to 50,000 patients in the U.S., Europe and Japan, according to Isis. The disease is caused by a defect in the production of a protein that helps nerve cells in the spinal cord. Isis’s drug aims to increase the body’s production of that protein.

In the study, the median age without permanent ventilation or death increased to 19.9 months from 16.3 months for four infants on one level of dosage and to 16.7 months from 11.6 months on a higher dosage. Most babies with spinal muscular atrophy type 1, the variety Isis aimed to treat, die within the first two years of life, according to the National Institutes of Health. Mechanical respiration can be used to prolong the infants’ lives.

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