Spinal Atrophy Drug From Isis Pharmaceuticals, Inc. Showing Promise, Says UBS

Published: Oct 13, 2014

Spinal Atrophy Drug From Isis Showing Promise, Says UBS

October 13, 2014

By Riley McDermid, BioSpace.com Breaking News Sr. Editor

Biogen Idec, Inc. partner Isis Pharmaceuticals, Inc. has reported significant progress with its spinal atrophy drug and is likely to keep traders bullish on the company’s stock in the near-term, said Matthew Rogen, a biotech analyst with UBS, on Monday.

Stanley Crooke, chairman and chief executive of Isis, told attendees at the World Muscle Society Congress this week that a Phase II study has shown infants given the drug, ISIS-SMNRx, have seen increased muscle function and have survived longer than usual. That’s a marked departure from a disease which typically sees most patients die before age two.

ISIS-SMNRx is injected into the spine in an attempt to increase production of a protein called SMN that has been shown to prolong the life of spinal motor neurons. Analysts have been keeping a close eye of how the drug performs and are pleased so far.

Sixteen infants who were given two doses, injected multiple times, during the Phase II study Crooke cited. Dosing varied, with 16 infants receiving 12 milligrams of dosage, and four receiving 6 milligrams. A total of 16 infants saw improved muscle function scores by an average of 9.3 points on a 64-point test from the Children's Hospital of Philadelphia.

“Data remain encouraging in both studies…We continue to find the observations of dose- and time dependent responses positive for the study in children,” wrote Rogen in a note to investors. “Although four patients have died in the infant study (one due to an accident), infants with Type 1 SMA have the most severe form of the disease. While the infant data with 12 milligrams are not as mature as with 6 milligrams, we remain encouraged by the greater increases in muscle function and changes in motor milestones with the higher dose.”

A Phase III trial of ISIS-SMNRx is currently underway, with a trial for older children slated for the fourth quarter of 2014, Crooke said.

“[We see] incremental positive with more mature data,” concluded Rogen. “We think the data support the bull case on the program, although we think randomized controlled data are required for it to be considered de-risked.”

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