Biogen has agreed a deal to license a brain disease candidate from Karyopharm for $10m upfront, and up to $217m in milestone payments.
The orally-active candidate, called KPT-350, is a selective inhibitor of nuclear export (SINE) compound that binds to and inhibits a protein called XPO1 which in turn mediates proteins that are believed to play a role in neurological and inflammatory processes.
It’s the first deal of the year for Biogen but stops short of the big transaction that some analysts have been expecting from the cash-rich company, which spun off its haemophilia and other rare blood disorders business into a new company called Bioverativ last year (since acquired by Sanofi).
Some shareholders have been calling for a large-scale deal – akin to the recent announcements from the likes of Celgene – to help drive near-term growth and diversify Biogen’s pipeline, and the company’s chief executive Michel Vounatsos has indicated that it is looking at opportunities among companies with mid- to late-stage clinical candidates.
On a conference call to discuss Biogen’s fourth quarter results, R&D head Michael Ehlers said that KPT-350 is a “first-in-class phase I ready investigational oral compound KPT-350 for certain neurological and neurodegenerative conditions, primarily focused on amyotrophic lateral sclerosis”.
“We plan on moving this asset into a phase I study by the end of the year,” he added. Biogen is already focusing on ALS with one of its other collaborations, a pair-up with antisense specialist Ionis targeting genetic subsets of ALS such as patients with the SOD1 mutation.
Biogen reported 2017 revenues of $12.3bn, up 15% on the prior year, thanks to gains for its multiple sclerosis drugs, particularly Tecfidera (dimethyl fumarate) and rising royalties from Ocrevus (ocrelizumab) – licensed to Roche, as well as spinal muscular atrophy therapy Spinraza (nusinersen).
Spinraza – licensed from Ionis – brought in a whopping $363m for Biogen in the quarter, ahead of analyst expectations, thanks to its $750,000 first-year price (which drops to $375,000 in subsequent years).