BioCentury
ARTICLE | Top Story

Isis jumps on interim SMA data

February 22, 2014 1:46 AM UTC

RNAi play Isis Pharmaceuticals Inc. (NASDAQ:ISIS) added $920.5 million in market cap on Friday after reporting top-line data from a Phase Ib/IIa trial evaluating the company's ISIS-SMNRx to treat spinal muscular atrophy (SMA) in patients ages 2-15. In 24 patients in the first three cohorts of the open-label, dose-escalation trial, 3, 6 and 9 mg doses of intrathecal ISIS-SMNRx were well tolerated and led to dose-dependent increases in Hammersmith Functional Motor Scale-Expanded (HFSME) scores. HFSME measures muscle function in SMA patients. Isis jumped $7.93 (16%) to $59 on the day for a closing valuation of $6.8 billion.

After market close, Isis released data from a separate Phase II trial evaluating ISIS-SMNRx in infants. The company plans to present detailed data from both trials at the American Academy of Neurology meeting in April. Mid-year, Isis plans to start a Phase III trial with ISIS-SMNRx in infants, with a Phase III trial in children with SMA also slated to start this year. ISIS-SMNRx is an antisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA. ...