- AstraZeneca plc has handed over a $30 million license fee to Ionis Pharmaceuticals Inc. for IONIS-AZ6-2.5-LRx, an antisense agent developed to block an unknown target for treatment of nonalcoholic steatohepatitis (NASH).
- The drug combines two antisense technologies that Ionis says improves the targeting and potentially enables lower or less frequent dosing.
- Further development and commercialization will be carried out by AstraZeneca, which could pay as much as $300 million in additional development and regulatory milestone payments tied to the drug's clinical progress.
The NASH deal is the third under the terms of the agreement signed between Ionis and AstraZeneca back in 2015. At the time, the British pharma paid an upfront fee of $65 million to Ionis to expand the companies' collaborative work into cardiovascular, metabolic and renal diseases.
That therapeutic area is one of AstraZeneca's core areas of focus, and Ionis' NASH candidate joins the big pharma's portfolio of drugs in type 2 diabetes and heart disease, which have some overlap with the fatty liver disease.
Ionis' business strategy has centered on partnering with bigger biopharma companies to advance development of its large slate of roughly two dozen experimental antisense therapeutics.
A spinal muscular atrophy treatment called Spinraza (nusinersen) is the best example of that model succeeding. Partner company Biogen Inc. won approval for the drug in December 2017 and recorded $884 million in first-year sales of the drug.
Ionis and Biogen are conducting further R&D to expand their SMA portfolio, and Biogen has licenses or options for seven different candidates from Ionis.