Ionis Pharmaceuticals Inc (NASDAQ:IONS) announced positive data from a Phase 2 placebo-controlled study evaluating IONIS-FXIRx in patients with end-stage renal disease (ESRD) on hemodialysis. In this Phase 2 study patients treated with IONIS-FXIRx achieved statistically significant, dose-dependent reductions in Factor XI activity. There were no clinically meaningful reductions in platelet levels and no treatment-related major or clinically relevant non-major bleeding events.

“IONIS-FXIRx is the first and only antithrombotic in development to have demonstrated potential to separate antithrombotic activity from bleeding risk. In this Phase 2 study in patients with ESRD on hemodialysis, it was important to evaluate the safety profile of IONIS-FXIRx in these patients who are at high-risk for bleeding. These new data are consistent with those of our previous Phase 2 comparator-controlled study evaluating the incidence of venous thromboembolic events (VTE) in patients treated with IONIS-FXIRx undergoing total knee replacement surgery. In that Phase 2 study, patients treated with 300 mg IONIS-FXIRx had a 7-fold lower incidence of VTE with no increase in bleeding events compared with patients treated with enoxaparin,” said Sanjay Bhanot, M.D., Ph.D., vice president of development at Ionis.

The Phase 2 study of IONIS-FXIRx was a double-blinded, randomized, placebo-controlled study in 43 patients with ESRD receiving hemodialysis. The primary purpose of the study was to understand the drug’s behavior and activity in patients with severe kidney disease on dialysis. Patients received 200 mg or 300 mg of IONIS-FXIRx or placebo for 12 weeks. In patients treated with 200 mg and 300 mg of IONIS-FXIRx, a mean percent reduction in FXI activity of 56% (p=<0.001) and 71% (p=<0.001), respectively, was achieved at week 13, compared to a mean percent reduction of 4% for placebo-treated patients. Furthermore, a decrease in severe clotting events in the dialysis circuit after six weeks compared to baseline was observed.  IONIS-FXIRxdisplayed a favorable safety and tolerability profile. In this small 3-month study, there were no clinically meaningful reductions in platelets and no treatment-related major or clinically relevant non-major bleeding events. There were no treatment-related serious adverse events. An increase in minor bleeding events was observed in patients receiving the 300 mg dose.  In addition, there were no clinically meaningful changes in laboratory values, including those related to liver function. IONIS-FXIRx was well tolerated in the study with no flu-like or injection site reactions.

“IONIS-FXIRx is a prime example of the power of antisense technology to selectively target a key component of the complex antithrombotic pathway that is not easy to target with other therapeutic modalities,” said Richard Geary, Ph.D., senior vice president of development at Ionis. “Patients with ESRD are at high risk of thrombotic and bleeding events and have defective platelet function.  Therefore, to see such a good safety profile in this study gives us additional confidence that antisense drugs might be safely used in patients with severe kidney disease. We were also encouraged by the absence of clinically meaningful platelet declines in this study.  Even though this was a small 3-month study, it provided further evidence that 2’MOE antisense oligonucleotides, as a class, are not associated with clinically meaningful platelet declines.”

In May 2015, Ionis entered into an exclusive license agreement with Bayer to develop and commercialize IONIS-FXIRx for the prevention of clotting disorders. Upon review of the Phase 2 data and potential advancement of the program by Bayer, Ionis will be eligible to receive a $55 million payment. (Original Source)

Shares of Ionis Pharmaceuticals are up nearly 2% to $26.40 in pre-market trading Tuesday. IONS has a 1-year high of $62.68 and a 1-year low of $19.59. The stock’s 50-day moving average is $32.93 and its 200-day moving average is $31.03.

On the ratings front, IONS has been the subject of a number of recent research reports. In a report issued on October 10, Wells Fargo analyst Jim Birchenough maintained a Buy rating on IONS. Separately, on October 9, BMO’s Do Kim reiterated a Buy rating on the stock and has a price target of $48.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Jim Birchenough and Do Kim have a yearly average return of 18.1% and 8.6% respectively. Birchenough has a success rate of 43% and is ranked #108 out of 4178 analysts, while Kim has a success rate of 39% and is ranked #903.

Overall, 3 research analysts have assigned a Hold rating and 4 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $42.00 which is 62% above where the stock closed yesterday.

Ionis Pharmaceuticals, Inc. engages in the development and commercialization of antisense drug discovery. It operates its business through the Ionis Core and Akea Therapeutics segments. The Ionis Core segment involves a a novel drug discovery platform generate a broad pipeline of drugs. The Akea Therapeutics segment develops and commercializes drugs for cardiometabolic disorders.