Bristol-Myers bets big on gene therapy with a $1B uniQure deal

uniQure CEO Joern Aldag

Bristol-Myers Squibb ($BMY) is the latest pharma heavyweight to buy into gene therapy's renaissance, signing a deal with uniQure ($QURE) worth as much as $1 billion and getting its hands a novel cardiovascular treatment.

Under the agreement, Bristol-Myers will hand over $100 million in up-front payments, licensing fees and equity investments in exchange for the commercial rights to uniQure's lead candidate and three more potential therapies. The Holland headquartered-uniQure will handle all discovery and manufacturing in the collaboration, eligible for up to $254 million tied to top prospect S100A1 and $217 million for each of the three other assets. Bristol-Myers is on the hook for all R&D costs, promising its partner a royalty on future sales.

UniQure's lead candidate, picked up in an acquisition of Germany's InoCard last year, is a gene therapy that uses a harmless virus to deliver the calcium-binding protein S100A1, a master regulator of cardiac function. By boosting S100A1, the treatment has the potential to relieve symptoms of congestive heart failure, according to uniQure, and Bristol-Myers is betting its cardio expertise can push the program forward.

Beyond the top prospect, each of the pair's subsequent collaborations will use uniQure's in-house gene therapy platform, which uses adeno-associated viruses (AAV) to shuttle corrective genes to their targets and relies on insect-cell-based manufacturing technology to produce doses at an industrial scale. UniQure has long been a leader among the new generation of gene therapy companies, making history in 2012 when its Glybera became the first such treatment to win a global approval.

To date, the majority of gene therapy work has focused on rare genetic diseases--including Glybera, a treatment for familial lipoprotein lipase deficiency. S100A1 stands out as one of the few targeting a major indication with a huge patient population, and success could further bolster the resurgent gene therapy space, uniQure CEO Joern Aldag has said.

"Bristol-Myers Squibb's strength in the cardiovascular area and its commitment to gene therapy will allow them to leverage the full breadth and capacity of our platform for cardiovascular diseases," Aldag said in a statement. "This collaboration will accelerate the application of gene therapy for large patient populations suffering from heart diseases and will complement the further development of uniQure's internal pipeline in two focus areas: liver diseases, including hemophilia; and CNS, including lysosomal storage diseases."

News of the agreement sent uniQure's shares soaring up 50% on Monday morning.

With the deal, Bristol-Myers joins a long line of traditional biopharma powers taking the plunge on gene therapy, a field long dogged by safety and efficacy concerns. Over the past year, Pfizer ($PFE), Sanofi ($SNY), Bayer and others have inked deals with pioneering biotechs to get some irons in the fire, while Biogen ($BIIB) has made gene therapy a standalone R&D vertical.

- read the announcement