BMS, uniQure Enter Cardiovascular R&D Pact

Bristol-Myers Squibb and uniQure entered an agreement that provides BMS with exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular disease. The collaboration includes uniQure’s gene therapy program for congestive heart failure that aims to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function. The agreement also includes the potential for target-exclusive collaboration in other disease areas on ten targets, including S100A1.

uniQure will lead discovery efforts and will be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial insect-cell based manufacturing platform. BMS will lead development and regulatory activities across all programs and will be responsible for all R&D costs, as well as commercialization of any resulting products.

BMS will make near-term payments of approximately $100 million, including an upfront payment of $50 million, a $15 million payment for the selection of three collaboration targets, in addition to S100A1, and an initial equity investment in uniQure for 4.9% of outstanding shares. The companies also entered a supply contract, under which uniQure will manufacture all gene therapy products under the collaboration.

uniQure will be eligible to receive research, development, and regulatory milestone payments, including as much as to $254 million for S100A1 and as much as $217 million for each other gene therapy product developed, as well as royalties on product sales.

“Bristol-Myers Squibb has an excellent and long-standing track record of success in discovering and developing treatments for cardiovascular diseases and in embracing advancing technologies for the treatment of human diseases,” said Carl Decicco, Ph.D., head of Discovery, R&D, Bristol-Myers Squibb. “Collaborating with uniQure, a clear leader in the field with an innovative and validated gene therapy platform, further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases, including cardiovascular diseases such as heart failure.”

“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,” said Joern Aldag, chief executive officer of uniQure. “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.”