ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS), announced that the FOCUS FH phase 3 study of KYNAMRO® (mipomersen sodium) in patients with severe heterozygous familial hypercholesterolemia (severe HeFH) met its primary efficacy endpoint, a statistically significant reduction in LDL-cholesterol after 60 weeks of treatment of once weekly injections of 200 mg of KYNAMRO compared to placebo. LDL-cholesterol reduction was similar to that observed in previous phase 3 studies. In addition, based on the data available for review, the safety profile of KYNAMRO observed in the FOCUS FH trial was similar to the safety profile reported in previous phase 3 studies. Genzyme will provide a more in depth review of the safety and efficacy data at a future medical meeting.

“We are pleased with the outcome of this fifth KYNAMRO randomized placebo controlled phase 3 trial, which shows statistically significant reductions in LDL-cholesterol in severe HeFH,” said Richard Geary, Ph.D., senior vice president of development at Isis Pharmaceuticals. “It is encouraging to see sustained LDL-C reductions over 60 weeks that were similar to what we’ve observed in previous phase 3 studies and a safety profile consistent with previous phase 3 and long-term extension studies. We look forward to the full analysis and presentation of this investigational data at an upcoming scientific meeting.”

“These investigational data provide additional information about KYNAMRO in the treatment of patients with severely elevated LDL-cholesterol and we look forward to further analysis of these data,” said Genzyme’s President and CEO,David Meeker, M.D. (Original Source)

Shares of Isis Pharmaceuticals closed today at $53.96, down $0.97 or 1.77%. ISIS has a 1-year high of $77.80 and a 1-year low of $29.17. The stock’s 50-day moving average is $57.20 and its 200-day moving average is $63.70.

On the ratings front, Isis Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on June 23, Needham analyst Chad Messer reiterated a Buy rating on ISIS, with a price target of $91, which implies an upside of 68.6% from current levels. Separately, on June 22, Deutsche Bank’s Alethia Young reiterated a Buy rating on the stock and has a price target of $78.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Chad Messer and Alethia Young have a total average return of 7.2% and 3.9% respectively. Messer has a success rate of 37.0% and is ranked #968 out of 3724 analysts, while Young has a success rate of 44.4% and is ranked #1500.

Isis Pharmaceuticals Inc is engaged in antisense drug discovery and development, exploiting a novel drug discovery platform it created to generate a broad pipeline of first-in-class drugs.