European gene therapy pioneer lands $2B-plus partnership deal

08 Apr 2015 | News
uniqure NV, the Dutch company which won the first approval for a gene therapy, is taking its technology into the mainstream in a collaboration with Bristol-Myers Squibb to develop treatments for heart diseases

uniQure NV sealed a $2 billion-plus deal with Bristol-Myers Squibb, giving the pharma company exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases.

The collaboration includes uniQure’s proprietary gene therapy programme for congestive heart failure, in which it is intended to restore the heart’s ability to synthesise the protein S100A1, a calcium sensor and master regulator of heart function.

Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas. In total, the companies may collaborate on ten targets, including S100A1.

The Amsterdam-based company will be eligible to receive research, development and regulatory milestone payments, including up to $254 million for the S100A1 programme and up to $217 million for each other gene therapy product potentially developed under the collaboration. uniQure is also eligible to receive net sales based milestone payments and tiered single to double-digit royalties on product sales.

uniQure will lead discovery efforts and be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, proprietary insect-cell based manufacturing platform. Bristol-Myers Squibb will lead development and regulatory activities across all programmes and be responsible for all research and development costs. Bristol-Myers Squibb will be solely responsible for commercialisation of any products from the collaboration.

Carl Decicco, Head of Discover at Bristol-Myers Squibb said, “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.”

Joern Aldag, Chief Executive Officer of uniQure, said, “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.”

Under the terms of the agreement, Bristol-Myers Squibb will make near-term payments of approximately $100 million, including an upfront payment of $50 million to be made at the closing of the transaction, a $15 million payment for the selection of three collaboration targets, in addition to S100A1, to be made within three months of the closing and an initial equity investment in uniQure for a number of shares that will equal 4.9 per cent of the total number of shares outstanding following such issuance, at a purchase price of  $33.84 per share, or at least $32 million in total.

Bristol-Myers-Squibb will acquire an additional 5 per cent ownership before December 31, 2015, at a 10 per cent premium, and will be granted two warrants to acquire up to an additional 10 per cent equity interest, at a premium, based on additional targets being introduced into the collaboration.

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