Under the terms of an extended collaboration agreement to develop novel antisense drug candidates for a broad range of neurological diseases, Biogen has exercised an option to license IONIS-MAPTRx. The ten-year collaboration agreement, which was signed last year, saw Biogen pay Ionis US$1 bn in cash, including US$625 m to purchase Ionis stock at a 25% premium, and a US$375 m upfront.
IONIS-MAPTRx is an antisense oligonucleotide therapy developed by IONIS in collaboration with Timothy Miller at Washington University. Like Spinraza, it is administered as a once-monthly intrathecal injection directly into the cerebral spinal fluid. The drug selectively decreases the production of microtubule-associated protein tau (MAPT) in the CNS. Tau is a protein implicated in multiple CNS conditions including Alzheimer’s, chronic traumatic encephalopathy (CTE), frontotemporal dementia, Parkinson’s and progressive supranuclear palsy (PSP
In preclinical research, IONIS-MAPTRx showed prevention and reversal of pathology associated with tau. Ionis commenced a Phase 1 trial of MAPTRx in mild Alzheimer’s in 2017, for which it received a US$10 m milestone payment from Biogen, and is currently conducting a follow-up one-year extension trial.
Under the terms of this latest deal, Ionis will complete the current extension study with all further development and commercialization to be the responsibility of Biogen.
Biogen is paying Ionis US$45 million upfront for IONIS-MAPTRx. Ionis will also be eligible for up to $155 million in milestone payments as well as royalties in the low- to mid-teens on any future sales of the drug.
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