Bristol-Myers Squibb (BMS) will gain exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases, under a collaboration that could net the Dutch gene therapy developer more than $1 billion, the companies said today.

The companies agreed to collaborate on up to 10 targets—including uniQure’s lead gene therapy program for congestive heart failure. The program is intended to improve clinical outcomes for patients with reduced ejection fraction by restoring the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function.

BMS and uniQure may also pursue target-exclusive collaborations in other disease areas under their collaboration, expected to close during the second quarter subject to customary closing conditions, including clearance under the Hart-Scott-Rodino Antitrust Improvements Act.

“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,” uniQure CEO Joern Aldag said in a statement.

uniQure will lead discovery efforts and be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, insect-cell based manufacturing platform. The parties have also inked a supply contract, under which uniQure will undertake manufacturing of all gene therapy products under the collaboration.

BMS will lead development and regulatory activities across all programs, shoulder all R&D costs, and will be solely responsible for commercialization of all products from the collaboration.

In return, BMS agreed to pay uniQure about $100 million short-term; as well as payments tied to research, development, and regulatory milestones that equal up to $254 million for S100A1, and up to $217 million for each other gene therapy product to be developed through the collaboration.

The short-term payments will consist of:

  • An upfront payment of $50 million at the close of the deal;
  • Another $15 million payment for the selection of three collaboration targets in addition to S100A1, to be made within three months of the closing;
  • An initial equity investment by BMS in uniQure for up to 4.9% of the total number of shares outstanding, at a purchase price of $33.84 per share, or at least $32 million.  The initial stock deal is also set to close during Q2 2015, subject to approval by uniQure shareholders.
  • An additional 5.0% of outstanding shares to be acquired by BMS before December 31, 2015, at a 10% premium. The pharma giant will be granted two warrants to acquire up to an additional 10% equity interest, at a premium, based on additional targets being introduced into the collaboration.

Aldag said uniQure partnered with BMS because one of its eight areas of disease focus is cardiovascular disease. The pharma giant markets Eliquis® (apixaban), a factor Xa inhibitor anticoagulant with a variety of cardiovascular indications that generated $774 million in worldwide revenues last year, up more than four-fold from $146 million in 2013.

According to its website, BMS has a pipeline of three small-molecule cardiovascular compounds that includes an IKur inhibitor in phase II, a Factor Xia inhibitor in Phase I, and PAR4 antagonists also in Phase I.

“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 predicted.








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