Bristol-Myers sends uniQure soaring with $1B cardiovascular gene therapy deal
- Bristol-Myers Squibb and Netherlands-based uniQure have struck a $1 billion collaboration deal in which the pharma giant will have exclusive access to uniQure's gene therapy technology platform in order to develop treatments for cardiovascular diseases.
- The deal allows the companies to collaborate on up to ten targets, mostly concentrating on (but not limited to) cardiovascular therapies, such as a treatment to reinvigorate congestive heart failure patients' ability to synthesize S100A1.
- The gene therapy company will lead discovery efforts and clinical manufacturing, while BMS will fund the research, lead development and regulatory activities, and have sole commercialization rights under the collaboration. uniQure's stock is up more than 45% on the news in morning trading.
After gene therapy's heyday about a decade ago, biopharma's enthusiasm for the technology diminished significantly in the absence of tangible breakthroughs.
But a slew of new companies exploring targeted genetic therapies and the success of drugs like Vertex's Kalydeco—not to mention a high profile endorsement for gene therapies from President Barack Obama during his most recent State of the Union address—has resurrected the enthusiasm, and big companies are feeling confident enough to put their money where their mouths are.
BMS will pay $50 million upfront and $100 million total in the near-term, and the company will gain a sizeable stake in uniQure with options to but even more equity (at a premium) as the companies determine more targets. uniQure will receive R&D and milestone payments of $254 million for the first main therapy and up to $217 million for each of the other therapies.