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BMS and UniQure in $1bn gene therapy pact

Posted on 07 April 2015

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Agreement provides Bristol-Myers Squibb with exclusive access to uniQure’s gene therapy technology platform for multiple targets in cardiovascular diseases.

The collaboration includes uniQure’s proprietary gene therapy program for congestive heart failure that is intended to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction.

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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.

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 programs and be responsible for all research and development costs.

Bristol-Myers Squibb will be solely responsible for commercialization of all products from the collaboration.

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% 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 5.0% ownership before December 31, 2015, at a 10% premium, and 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.

The parties have also agreed to enter into a supply contract, under which uniQure will undertake manufacturing of all gene therapy products under the collaboration.

uniQure will be eligible to receive research, development and regulatory milestone payments, including up to $254 million for the lead S100A1 therapeutic 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.

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