About

We are the leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. We have created an efficient and broadly applicable drug discovery platform. Using this platform, we believe that we are revolutionizing medicine with the goal to improve the quality of, and save, lives.

Using our proprietary antisense technology, we have created a large pipeline of first-in-class or best-in-class drugs, with over forty drugs in development. The three most advanced drugs in our pipeline include SPINRAZA® (nusinersen), TEGSEDI™ (inotersen) and WAYLIVRA™ (volanesorsen).

SPINRAZA is approved in global markets for the treatment of patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA and is now responsible for all development, regulatory and commercialization activities and costs for SPINRAZA. In 2017, Ionis and Biogen were awarded the prestigious Prix Galien USA Award for Best Biotechnology Product for SPINRAZA. In addition, the launch of SPINRAZA is one of the most successful rare disease launches of all time. For more information about SPINRAZA, please visit www.SPINRAZA.com. 

We designed TEGSEDI to treat patients with transthyretin amyloidosis, or TTR amyloidosis (ATTR), a fatal disease in which patients experience progressive buildup of amyloid plaque deposits in tissues throughout the body, including peripheral nerves, heart, intestinal tract, kidney, and bladder. TEGSEDI is currently under regulatory review in the U.S. and EU. The U.S. Food and Drug Administration (FDA) accepted the TEGSEDI New Drug Application (NDA) for Priority Review and set a Prescription Drug User Fee Act (PDUFA) date of October 6, 2018. Priority Review is granted by the FDA to drugs with the potential to address a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The FDA previously granted TEGSEDI Orphan Drug Designation and Fast Track Status. We also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), which has granted Accelerated Assessment and Orphan Drug Designation to TEGSEDI. An Early Access Program (EAP) for TEGSEDI is currently enrolling patients with ATTR in the U.S. and EU. Our affiliate Akcea Therapeutics Inc., or Akcea, licensed the global rights to commercialize TEGSEDI and is now responsible for all commercialization activities. Akcea is planning to launch TEGSEDI globally in 2018, if approved.

We designed WAYLIVRA to treat patients with two severe and rare genetic conditions called familial chylomicronemia syndrome, or FCS, and familial partial lipodystrophy, or FPL. WAYLIVRA is currently under regulatory review in the U.S., EU and Canada for the treatment of patients with FCS. If approved, WAYLIVRA would be the first therapy indicated for people with FCS. In the U.S., the FDA set a PDUFA date of August 30, 2018. WAYLIVRA has been granted priority review in Canada. The U.S. and European regulatory agencies have granted Orphan Drug Designation to WAYLIVRA for the treatment of patients with FCS. In the U.K., WAYLIVRA has been granted a Promising Innovative Medicine (PIM) Designation. An EAP for WAYLIVRA is currently enrolling patients with FCS in the U.S. and EU. Our affiliate, Akcea, licensed the global rights to develop, manufacture and commercialize WAYLIVRA and is now responsible for all development, regulatory and commercialization activities and costs for WAYLIVRA. Akcea is planning to launch WAYLIVRA globally in 2018, if approved.

We are currently conducting a Phase 3 study in patients with FPL. The European regulatory agency has also granted Orphan Drug Designation to WAYLIVRA for the treatment of FPL.

Akcea is a biopharmaceutical company we established to focus on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs licensed from us, including TEGSEDI, WAYLIVRA, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx (previously IONIS-TTR-LRx), all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed by Ionis, and are based on our proprietary antisense technology. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Cambridge, MA with offices in Carlsbad, CA and across the EU and Canada. Additional information about Akcea is available at akceatx.com.