After GSK snub, Ionis finds new commercialization partner—its own spinout

Handshake business deal executives
Ionis is handing Akcea the worldwide rights to inotersen and IONIS-TTR-LRx, which is designed to treat hereditary and wild-type forms of ATTR, and is slated to enter the clinic this year. (rawpixel)
Last August, GlaxoSmithKline ditched Ionis Pharma as the two were closing in on a regulatory filing for the rare disease drug inotersen. Now, the biotech is pushing forward with new partner—Akcea Therapeutics, a subsidiary it spun out to develop and commercialize four antisense drugs.

Ionis expects inotersen to bag U.S. and EU approvals by midyear for the treatment of hereditary transthyretin amyloidosis (hATTR). Under the deal, it is handing Akcea the worldwide rights to inotersen and IONIS-TTR-LRx, which is designed to treat hereditary and wild-type forms of ATTR and is slated to enter the clinic this year, according to a statement. 

Akcea, which priced its IPO at $125 million last summer, will fork over an upfront licensing fee of $150 million. And "to support commercialization of inotersen, Ionis will purchase $200 million of Akcea common stock priced by reference to a recent trading average," boosting its stake in Akcea to 75% from 68%, Ionis said in the statement. Ionis stands to reap a total of $90 million in milestones on the U.S. and EU approvals of inotersen and IONIS-TTR-LRx, now dubbed AKCEA-TTR-LRx, with the potential for more if they're approved in other markets. And the duo will split inotersen profits 60/40, with Ionis taking the lion's share, until they launch AKCEA-TTR-LRx, when they will share them equally. 

As part of the deal, Ionis' chief business officer, Sarah Boyce, will join Akcea as president and a board member.

"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 Stanley Crooke, M.D., chief executive officer and chairman of Ionis. 

Partnerships are par for the course for Ionis, which is working with Roche on a Huntington's disease treatment, and has, just in the past year, licensed antisense drugs to Janssen and AstraZeneca. But it must be asked—why has Ionis essentially farmed inotersen out to itself rather than signing on a pharma like GSK? 

“Our goals when deciding on the ideal partner for inotersen were to be launch-ready upon approval to rapidly deliver inotersen to the patients who desperately need this treatment, to maximize the commercial success of inotersen and to optimize our commercial participation in our TTR franchise," said an Ionis spokesperson via email.

"Ionis conducted a thorough process to evaluate potential partners and types of transaction for inotersen. We had significant interest from a range of companies and received multiple offers. After a thoughtful evaluation of all the possibilities, we arrived at the conclusion that this transaction with Akcea met all three of our key partnering goals. … We believe this transaction is a win for patients, a win for shareholders, and a win for both companies.”

Although Akcea has not yet commercialized a drug, the collab positions it to launch two this year—inotersen and volanesorsen, the drug developed for the treatment of familial chylomicronemia syndrome or familial partial lipodystrophy that it picked up when it spun out of Ionis. 

"This collaboration is transformational for Akcea," said Akcea CEO Paula Soteropoulos. "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." 

It's the right time for Akcea to add inotersen to its stable, as it has built up its "capabilities and team" in its preparations to debut volanesorsen, she said.

"This commercial build-up, combined with our highly accomplished team, allows us to accelerate readiness for the upcoming inotersen launch. Further, it increases our options to scale our business given the expansion of our call points and sales force as we work with this new physician community"

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