Rare Daily Staff

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, entered into an exclusive, worldwide agreement to license Ionis’ experimental therapy inotersen and a preclinical candidate for the rare disease hATTR for $150 million in stock and milestones that could bring the total value to $1.7 billion.

For Akcea, the deal accelerates its transition to a commercial company while strengthening its pipeline. For Ionis, the agreement allows it to retain significant value in the commercialization of inotersen, increases its stake in Akcea, and allows it to remain focused on its core strength of drug development.

In November, Ionis submitted inotersen to the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval as a treatment for hereditary transthyretin amyloidosis, or hATTR, a progressive, systemic, and fatal genetic disease caused by the formation and aggregation of TTR amyloid deposits in various tissues and organs throughout the body.

Inotersen is an antisense drug designed to reduce the production of transthyretin, or TTR protein. The U.S. Food and Drug Administration has granted Orphan Drug Designation and Fast Track Status to inotersen for the treatment of patients with polyneuropathy due to hereditary TTR amyloidosis, and the European Medicines Agency has granted Orphan Drug Designation to inotersen for the treatment of patients with ATTR.

The agreement comes as Akcea has been ramping up its commercial capabilities in anticipation of approval of volanesorsen, which is under regulatory review in the United States and European Union as a treatment of familial chylomicronemia syndrome, or FCS, a rare genetic lipid disorder.

“This collaboration is transformational for Akcea,” said Paula Soteropoulos, CEO of Akcea Therapeutics. “Adding two potentially life-changing therapies, inotersen and AKCEA-TTR-LRx, expands our pipeline of drugs to treat people with serious and under-served rare diseases. Through our launch preparations for volanesorsen, we have rapidly expanded our capabilities and team to become a unique and leading presence in rare diseases.”

As part of the collaboration, the inotersen commercial team from Ionis is joining Akcea, and will transition the ongoing launch preparations for inotersen to the company. Sarah Boyce, currently Ionis’ chief business officer, will join Akcea as president and take a seat on the Akcea board of directors upon closing. Boyce has been leading the inotersen launch and will bring extensive, global experience in the life sciences industry to Akcea. The newly combined Akcea team is preparing to launch inotersen in the United States and European Union following expected approvals in mid-2018

Stanley Crooke, chairman and CEO of Ionis, said the company in its partnering discussions had several options and decided that the partnership with Akcea would provide it with the opportunity to maximize the commercial value of inotersen.

“This collaboration reflects our ever-increasing confidence in the value of inotersen and exemplifies our strategy to use commercial affiliates to commercialize our drugs, keeping the core of Ionis focused on innovation and our antisense pipeline,” said Crooke. “This collaboration will allow the combined Ionis-Akcea team to rapidly deliver inotersen to the patients who desperately need this treatment.”

The companies will also develop AKCEA-TTR-LRx for hereditary and wild-type forms of ATTR. AKCEA-TTR-LRx is planned to enter clinical development in 2018.

Under the terms of the agreement, Akcea will pay Ionis an upfront licensing fee of $150 million in common stock priced by reference to a recent trading average.

Akcea will have rights to commercialize inotersen and AKCEA-TTR-LRx globally. To support commercialization of inotersen, Ionis will purchase $200 million of Akcea common stock priced by reference to a recent trading average. Upon closing the transaction, Ionis’ ownership in Akcea will increase to 75 percent from 68 percent today.

Regulatory approval of inotersen and AKCEA-TTR-LRx in the United States will trigger a $50 million milestone payment to Ionis and approval in the European Union will trigger a $40 million milestone payment to Ionis. Approval in other areas will trigger additional milestone payments.

Commercial profits and losses from inotersen will be split 60 percent to Ionis and 40 percent to Akcea until the first commercial sales of AKCEA-TTR-LRx, after which the profits and losses will be shared 50/50.

The costs of the development of AKCEA-TTR-LRx and the profits from its commercialization will be shared 50/50. The license for the two drugs also includes various sales milestone payments of up to nearly $1.3 billion.

March 15, 2018
Photo: 

X