ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) announced encouraging preliminary results from an investigator-sponsored study in patients with transthyretin amyloid-related cardiomyopathy that was presented yesterday by Dr. Merrill Benson at the 20th World Congress on Heart Disease (WCHD) in Vancouver, Canada.

“I am encouraged by the safety, tolerability, TTR lowering and apparent stabilization of cardiac disease progression we have observed in the study to date,” said Merrill Benson, M.D., professor of pathology and medical genetics at Indiana University.  “In this open-label study, measures of TTR amyloid cardiac disease, including echocardiographic and strain imaging evaluation, indicated little to no cardiac disease progression after six months of dosing with ISIS-TTRRx. These data compare favorably to our previously published work1, which showed that patients with TTR-related cardiomyopathy exhibit disease progression at six months using similar echocardiographic measurements.”

“These new data on cardiac measures in patients with TTR-cardiomyopathy give us additional confidence that the TTR-lowering we have observed in this and other clinical studies has the potential to provide therapeutic benefit to patients with TTR amyloidosis,” said Brett Monia, Ph.D., senior vice president of drug discovery at Isis Pharmaceuticals.

In a presentation titled, “Transthyretin Amyloid Cardiomyopathy Treatment with an Antisense Oligonucleotide Inhibitor of TTR (ISIS-TTRRx)”, Dr. Benson reported on preliminary results from his investigator-sponsored open-label study in patients with TTR-related cardiomyopathy.  In patients who completed nine months of weekly dosing (n=3) with 300 mg of ISIS-TTRRx, reductions in TTR protein of up to 88% were observed with a mean reduction of 78%.  In addition, patients who completed six months of dosing (n=5) were evaluated by echocardiography and showed no increase in left ventricular wall thickness.  In this study, patients receive 300 mg of ISIS-TTRRx once weekly for a total of 24 months with a three month follow up period. (Original Source)

Shares of Isis Pharmaceuticals closed last Friday at $53.15. 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 $59.06 and its 200-day moving average is $64.37.

On the ratings front, Isis 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 represents a potential upside of 71.2% from where the stock is currently trading. 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 4.7% and 6.2% respectively. Messer has a success rate of 36.8% and is ranked #1243 out of 3712 analysts, while Young has a success rate of 48.9% and is ranked #1179.

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.