2007 - 2022
In 2007, Ionis and Alnylam Pharmaceuticals formed a 50/50 joint venture, Regulus Therapeutics, to research oligomer biotherapeutics to micro-RNA targets.[24] In 2008, Ionis won a $175 million contract to develop a cholesterol drug then-called mipomersen and later branded as Kynamro, in partnership with Genzyme Corporation.[8] Genzyme paid $325 million in a stock and cash deal, plus a $175 million licensing fee and 30-50% royalty on sales. It was Ionis' first "notable success".[25] Mipomersen was rejected by the European Medicines Agency in 2012,[26] but approved by the FDA in 2013.[27] Ionis terminated the agreement with Genzyme in 2016[28] and sold licensing rights to Kastle Therapeutics.[29]
By this time, Ionis had 26 drugs under development and had just opened a new research and development center in 2011.[8][30] Ionis earned $325 million from Kynamro that year, which was enough to keep the company solvent.[9] This was followed by a series of more profitable deals.[9] In 2015, the company changed its name from "ISIS" to "IONIS" to avoid confusion with the terrorist group Islamic State of Iraq and the Levant (ISIS).[31][32]
The cardiovascular division of Ionis was spun-off into a separate company called Akcea in 2017, to help fund IONIS' own research and development.[33] However, after many of its treatments demonstrated early results, Ionis bought Akcea back in 2020.[34] In 2018, Tegsedi was approved by the FDA as a treatment for polyneuropathy. However, a competing drug Onpattro had fewer side effects and took the market from Ionis. Ionis later replaced Tegsedi with a drug called Wainua, which was approved in December 2023.[34] Another Ionis drug, Qalsody, was also approved that year.[34]
In 2021, Ionis signed a deal with AstraZeneca to develop and commercialize eplontersen. The deal involved a $200 million upfront payment and several billion depending on hitting development and sales milestones.[35] In 2022, Ionis entered the field of gene editing for the first time in a partnership with Metagenomi. Ionis paid $80 million for the two to work together on four gene editing projects.[36]
Ionis' first major commercial success was Spinraza,[34] which was approved by the FDA in 2016 to treat spinal muscular atrophy. A clinical trial showed Spinraza could save the lives of babies that would otherwise die before turning two years-old, or at least give them more years of life. Ionis made $327 million from the drug by the following year.[9] However, Ionis only got about 12-18 percent of the billions in revenue from the drug's sales, under its agreement with commercialization partner Biogen.[37] This was criticized by many investors. Ionis had also experienced a series of setbacks when commercialization partners decided not to take viable drugs to market based on corporate decisions by the commercialization partner.
