Company Update (NASDAQ:ISIS): Akcea Therapeutics Receives Orphan Drug Designation from the US FDA for Volanesorsen

Akcea Therapeutics, a wholly-owned subsidiary of ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS), announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS).  FCS is a rare genetic disease characterized by extremely high triglyceride levels and risk of pancreatitis. In a Phase 2 study published in the NEJM in December 2014, patients with FCS treated with volanesorsen achieved substantial reductions in apoC-III, triglycerides, chylomicrons and apoC-III-associated very low density lipoprotein-cholesterol (VLDL-C) particles.1  Akcea is currently conducting an international multi-center, randomized, double-blind, placebo-controlled Phase 3 study in patients with FCS.

“FCS is a rare and very serious genetic disorder associated with very high triglyceride levels that put patients at risk of potentially life-threatening pancreatitis.  People with FCS cope with daily consequences of their disease, including persistent abdominal pain and life-altering food restrictions. Unfortunately, current treatment options do not reduce triglyceride levels enough to reduce the risk of serious illness in patients with FCS,” said Paula Soteropoulos, President and Chief Executive Officer at Akcea Therapeutics.  “Orphan drug designation for FCS underscores the need for improved therapies to treat patients with FCS and is an important benchmark as we complete the ongoing Phase 3 study in patients with FCS and prepare for commercialization.”

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the United States.  Orphan drug designation entitles Akcea Therapeutics to seven years of market exclusivity in the United States if market approval is granted for volanesorsen for the treatment of patients with FCS.  Additional incentives include tax credits related to clinical trial expenses, an exemption from theFDA-user fee, and FDA assistance in clinical trial design. (Original Source)

Shares of Isis Pharmaceuticals closed last Thursday at $56.13 . ISIS has a 1-year high of $77.80 and a 1-year low of $27.37. The stock’s 50-day moving average is $64.01 and its 200-day moving average is $65.28.

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 implies an upside of 62.1% 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 2.9% and 2.2% respectively. Messer has a success rate of 38.2% and is ranked #1519 out of 3689 analysts, while Young has a success rate of 42.2% and is ranked #1782.

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.

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