- Ionis Pharmaceuticals Inc. received a $30 million license fee from AstraZeneca plc on Tuesday, when the big pharma opted in for development of IONIS-AZ5-2.5Rx (AZD2373) for kidney disease.
- AstraZeneca will be responsible for developing and commercializing the antisense drug. And Ionis is eligible for another $300 million in development and commercialization milestones, as well as tiered, low double-digit royalties.
- The drug is the second compound that the British pharma has opted into developing in an ongoing collaboration centered around cardiovascular, metabolic and renal diseases.
Ionis no longer has to prove its antisense technology works. The company already gained approval, with partner Biogen, for the spinal muscular atrophy (SMA) drug Spinraza (nusinersen).
The SMA drug has been performing well beyond expectations since its launch at the beginning of 2017 and is likely the best rare disease drug launch in recent memory.
The biotech has built its business by making platform deals for its antisense technology – now in what Ionis calls its Generation 2.5 iteration. Typically, Ionis discovers and then develops compounds through early stages, before kicking off the drug to a big pharma or big biotech partner. Although there have been instances where Ionis had to develop compounds on its own when a partner didn't opt into a program.
For instance, the company announced in 2016 that partner Sanofi Genzyme handed back the rights to the injectable cholesterol drug Kynamro (mipomersen) after slow sales. Ionis has since sold off the compound.
Previously known as Isis Pharma, Ionis inked its first collaboration with AstraZeneca in 2012, centered on oncology assets. But the British pharma discontinued one of the previously licensed compounds from Ionis, AZD5312, which was in early studies for prostate cancer.
Before the setback, AstraZeneca expanded its work with Ionis in August 2015, tapping the biotech for development of cardiovascular, metabolic and renal compounds. AZD2373 is the first compound that AstraZeneca has opted into from that collaboration so far.