Akcea Completes Deal That Gives It Marketing Rights to FAP Treatment Inotersen

Jose Marques Lopes PhD avatar

by Jose Marques Lopes PhD |

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Akcea Therapeutics has completed a deal giving it the global marketing rights to its affiliate  Ionis Pharmaceuticals’ familial amyloid polyneuropathy therapy inotersen.

The deal also gives Akcea rights to a follow-up treatment known as AKCEA-TTR-L Rx, formerly known as IONIS-TTR-L Rx.

Inotersen was designed for hereditary forms of FAP, which are also known as hereditary transthyretin amyloidosis, or hATTR.

The therapy decreases the production of the TTR protein, which clumps in hATTR patients who have a mutation of the TTR gene.

TTR’s build-up occurs in several tissues and organs. In peripheral nerves, the clumping causes damage that leads to loss of movement function.

Inotersen is composed of small strands of nucleic acids that bind to a patient’s RNA, turning off the production of both normal and faulty versions of TTR protein.

The  molecule is going through regulatory reviews in the U.S. and E.U. Akcea and Ionis expect it to hit the market by mid-2018.

Inotersen has received orphan drug status and fast track designation from the U.S. Food and Drug Administration, and orphan drug status from the European Medicines Agency.

In July 2017, Ionis announced results of the 15-month Phase 3 NEURO-TTR clinical trial (NCT01737398), which evaluated the safety and effectiveness of a once-a-week injection of inotersen in 172 FAP patients. Inotersen reduced the worsening of patients’ nerve damage and improved their quality of life, researchers said.

A Phase 3 open-label extension trial (NCT02175004) that is still under way is assessing the safety and effectiveness of long-term inotersen treatment.

Akcea and Ionis are developing AKCEA-TTR-L Rx for both hereditary and non-hereditary ATTR. They plan to start clinical trials of it in 2018.

Under their agreement, Akcea gave Ionis a $150 million up-front licensing payment for the two therapies. The full rights to inotersen cost it $1.7 billion plus profit-sharing payments.

Regulatory approval of the treatments will trigger milestone payments to Ionis.

The agreement also stipulated that Sarah Boyce, Ionis’s chief business officer, join Akcea as president and member of its board.