Bristol-Myers Squibb has licensed rights to gene therapy specialist uniQure’s lead heart failure candidate in a deal valued at up to $1bn.
Netherlands-based uniQure will receive $50m upfront for rights to the lead candidate for heart failure – which uses a virus to deliver a calcium-binding protein (S100A1) that regulates cardiac function – and another $15m within three months for three other cardiovascular candidates.
BMS will also take a 4.9% equity stake in uniQure which will cost around $32m, with another 5% ownership scheduled before the end of the year.
uniQure also stands to receive research, development and regulatory milestones, including up to $254m for the lead project and up to $217m for each subsequent product. In total, the companies may collaborate on 10 targets.
uniQure claimed the distinction of being the first company to bring a gene therapy through to regulatory approval in the western world, getting EMA backing for its Glybera (alipogene tiparvovec) treatment for the inherited disease lipoprotein lipase deficiency (LPLD) in 2012, although four others are sold in markets outside the US and EU.
The approval – after decades of research held back by serious safety challenges – prompted a resurgence of interest in gene therapy among big pharma companies and a series of other deals involving the likes of Pfizer, Novartis, Bayer and Sanofi/Genzyme as the technology looks increasingly likely to develop into a $10bn-plus market.
Simultaneously, Glybera‘s $1m cost per one-off treatment has raised questions about how healthcare systems will be able to afford this new, highly-targeted form of therapy, particularly when it start to become available for common diseases such as heart failure and cancer.
There are almost 500 candidates in development, according to market research firm Roots Analysis, with more than 40% targeted at cancer and one in five in late-stage clinical development.
uniQure will head the discovery effort, with responsibility for manufacturing clinical and commercial supplies using its vector technologies and insect cell-based manufacturing platform, while BMS will handle and fund development and regulatory activities for all programmes , with sole responsibility for the sale of products arising from the alliance.
Other gene therapy players include Bluebird bio and Spark Therapeutics, which both completed listings on the Nasdaq exchange last year.
This is also the second billion-dollar deal for BMS this year after its $1.2bn takeover of immunotherapy specialist Flexus in February.