ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) announced the initiation of an investigator-sponsored, open-label Phase 2 study evaluating ISIS-TTRRx in patients with wild-type transthyretin amyloidosis (wt-TTR amyloidosis, previously referred to as senile systemic amyloidosis, or SSA).  The Phase 2 study will be conducted by Dr. Rodney Falk, the director of the Cardiac Amyloidosis Program and a cardiovascular medicine specialist at the Brigham and Women’s Hospital in Boston.

“Together with GSK, we are conducting a robust development plan designed to establish ISIS-TTRRx as a best-in-class treatment for all patients with TTR amyloidosis.  Already, we have demonstrated that ISIS-TTRRx, given as one, once a week, self-administered, at home injection, can robustly reduce TTR levels in patients with both the polyneuropathy and cardiomyopathy forms of the disease, and we have preliminary evidence of disease stabilization in patients with TTR-related cardiomyopathy,” said Brett Monia, Ph.D., senior vice president of drug discovery and franchise leader for oncology and rare diseases at Isis Pharmaceuticals.  “Dr. Falk is a leading expert in TTR amyloidosis and has extensive knowledge of wt-TTR cardiomyopathy.  As such, Dr. Falk is an ideal physician to evaluate the effects of ISIS-TTRRx in these patients, who have the same type of disease as those who are represented in his large natural history database.”

The Phase 2, investigator-initiated study will evaluate treatment with ISIS-TTRRx in approximately 50 patients with wt-TTR amyloidosis.  Patients must have evidence of cardiac disease as indicated by an interventricular septum thickness (IVS) of >1.3cm with a positive cardiac biopsy.  The effects of ISIS-TTRRx on cardiac disease stabilization will be measured by cardiac imaging at 6, 12 and 18 months.  Data from this study will provide additional information on ISIS-TTRRx and can also be included in the robust data package Isis and GSK are generating to support regulatory filings for ISIS-TTRRx in all patients with TTR amyloidosis.

In addition to the Phase 2 study initiated today, ISIS-TTRRx is being evaluated in the following studies:

  • Isis is evaluating ISIS-TTRRx in a Phase 3 randomized, double-blind, placebo-controlled, international study (NEURO-TTR) in patients with familial amyloid polyneuropathy (FAP). The study is designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life.
  • Isis is evaluating ISIS-TTRRx in an open-label extension study that is eligible to patients with FAP who have completed the NEURO-TTR Phase 3 study. To date, all eligible patients have enrolled in the open-label extension study.
  • GSK plans to initiate a small Phase 3 study evaluating ISIS-TTRRx in patients with FAP in Japan.
  • GSK plans to initiate a Phase 3 randomized, double-blind, placebo-controlled, multicenter, international study (CARDIO-TTR) in patients with familial amyloid cardiomyopathy (FAC) and wt-TTR amyloidosis who also have a history of heart failure. The study will measure the effects of ISIS-TTRRx on a clinical composite outcome that includes mortality, cardiac transplant and cardiovascular hospitalization.
  • Dr. Merrill D. Benson, professor of pathology and lab medicine and molecular genetics at Indiana University School of Medicine, is evaluating ISIS-TTRRx in an open-label, investigator-sponsored, Phase 2 study in patients with TTR amyloid cardiomyopathy, which includes patients with FAC and patients with wt-TTR amyloidosis. (Original Source)

Shares of Isis Pharmaceuticals closed yesterday at $59.36, up $2.94 or 5.21%. ISIS has a 1-year high of $77.80 and a 1-year low of $37.38. The stock’s 50-day moving average is $58.21 and its 200-day moving average is $54.08.

On the ratings front, Isis Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on November 18, Goldman Sachs analyst Navdeep Singh assigned a Hold rating on ISIS, with a price target of $65, which represents a potential upside of 9.5% from where the stock is currently trading. Separately, on November 5, Piper Jaffray’s Joshua Schimmer upgraded the stock to Buy and has a price target of $80.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Navdeep Singh and Joshua Schimmer have a total average return of 15.7% and -1.9% respectively. Singh has a success rate of 66.7% and is ranked #460 out of 3645 analysts, while Schimmer has a success rate of 44.9% and is ranked #3111.

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.