Bristol-Myers Squibb Enters Gene Therapy Deal with uniQure

Today, Bristol-Myers Squibb (BMS) announced that it has entered into a deal with uniQure for its gene therapy technology platform.

The companies said that they have entered into an agreement that provides BMS with exclusive access to uniQure’s technology platform for multiple targets in cardiovascular diseases, as well as the potential for target-exclusive collaboration in other disease areas.

The collaboration includes uniQure’s proprietary gene therapy 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. The agreement also includes the potential for targets in other diseases. In total, the companies may collaborate on ten targets, including S100A1.

Discovery efforts will be led by uniQure, which will be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, proprietary insect-cell based manufacturing platform. Development and regulatory activities across all programs will be led by BMS, which will be responsible for all research and development costs. Additionally, BMS will be solely responsible for commercialization of all products from the collaboration.

“Bristol-Myers Squibb has an excellent and long-standing track record of success in discovering and developing treatments for cardiovascular disease and in embracing advancing technologies for the treatment of human diseases,” said Carl Decicco, PhD, 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.”

BMS will pay about $100 million, including an upfront payment of $50 million, a $15 million payment for selecting two collaboration targets, and a $32 investment in uniQure that represents a 4.9 percent stake in the company, at a purchase price of $33.84 per share, or at least $32 million in total. BMS will acquire an additional 5 percent ownership before December 31, 2015, at a 10 percent 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.

uniQure will be eligible to receive 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. The company is also eligible to receive net sales based milestone payments and tiered single to double-digit royalties on product sales.

“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 disease 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.”

Following the news, shares of uniQure surged 42 percent in premarket trading.

The companies anticipate the collaboration to be effective during the second quarter of 2015. The transaction, which is subject to customary closing conditions, is anticipated to close in the second quarter of this year.

Source: Bristol-Myers Squibb Company

Last updated: 4/6/15; 9:55am EST

 

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