- Six months after losing big pharma partner GlaxoSmithKline plc, Ionis Pharmaceuticals Inc. will tap its majority-owned affiliate Akcea Therapeutics Inc. to commercialize its rare disease drug inotersen, which is currently under review for approval by the Food and Drug Administration.
- Essentially dealing with itself, Ionis will license inotersen and a preclinical drug candidate to Akcea in exchange for a $150 million upfront fee paid in Akcea stock. Concurrently, Ionis will buy $200 million worth of shares in Akcea, taking its ownership of the Cambridge-based biotech up to 75% from 68% previously.
- Akcea is currently in the midst of preparations to launch volanesorsen, an antisense drug also licensed from Ionis that is awaiting approval in the U.S. and Europe. The companies aim to build upon that existing field force in order to more quickly commercialize inotersen should it win approval as expected.
Motivating Ionis to ensure a rapid launch of inotersen is the looming competitive threat from rival drugmaker Alnylam Pharmaceuticals Inc.
Both Inotersen and Alnylam's drug candidate patisiran are awaiting an approval decision from the FDA as treatments for a progressive genetic disease known as hereditary transthyretin amyloidosis (hATTR). The condition, thought to affect approximately 50,000 patients worldwide, is caused by the accumulation of TTR amyloid deposits in organs which leads declining sensory and motor function over time.
Approval of inotersen could come as soon as early July. Yet Alnylam won't be far behind, having secured a target decision date for patisiran of Aug. 11.
Launching quickly, then, is crucial for Ionis to secure an initial foothold on the market.
Data from Alnylam's pivotal study of patisiran showed treatment with the drug reduced the progression of neuropathy and improved patient quality of life with a clean safety profile.
Inotersen, which succeeded in its study but came up short of patisiran's efficacy mark, offers a potentially more convenient subcutaneous dosing option. Problems with low platelet counts and kidney function seen in inotersen's study could require greater patient monitoring, however.
"The conclusion we came to was that no partner other than the combined Ionis inotersen team and the Akcea team could guarantee being launch-ready in Europe and the U.S.," said Ionis CEO Stanley Crooke on a March 15 call with analysts.
"As you might expect, interest in inotersen took a variety of forms from larger companies who wanted global rights with no Ionis participation to more regional transactions, from companies with substantial rare disease expertise and those with less."
By licensing inotersen to its affiliate Akcea, Ionis could piggy back on the biotech's ongoing launch preparations for volanesorsen.
Ionis' commercial team for inotersen will transfer to Akcea, and Sarah Boyce, currently Ionis' chief business officer, will join Akcea as president. Akcea already has a commercial team of about 50 people in place in the U.S. and is building out its staff in Europe as well.
In addition, by paying the license fee to Ionis in stock, Akcea preserves its cash on hand to fund its commercialization efforts. The biotech reported roughly $260 million in cash and equivalents on its balance sheet as of Dec. 31.
On top of the licensing fee, Akcea will owe Ionis $90 million in milestone payments should inotersen win approval in the U.S. and Europe. The companies will initially split any sales of the drug on 60/40 basis, with Ionis taking home the larger share. All told, sales milestones could total up to $1.1 billion.