Last month, Bristol-Myers Squibb (BMS) and UniQure, the Dutch pioneer in human gene therapy, were at the forefront of the scene after having agreed on two collaborations, respectively, an agreement between Bavarian Nordic & BMS and a collaboration between Sympromics & UniQure. A few days ago, they announced an exclusive strategic collaboration to develop gene therapies for cardiovascular diseases.

The collaboration which should be effective as of the second quarter of 2015 includes uniQure’s proprietary gene therapy program for congestive heart failure that intends 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. 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 clinical and commercial supplies using its vector technologies and its industrial, proprietary insect-cell based manufacturing platform. Bristol-Not only will Myers Squibb lead development and regulatory activities across all programs and be responsible for all research and development costs, the company will also solely be in charge of the commercialization of all products from the collaboration.

Carl Decicco

Carl Decicco

Carl Decicco, Ph.D., Head of Discovery, R&D, Bristol-Myers Squibb explained: “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 will make near-term payments of approximately €92.2M. Nearly €13.8M of this investment are destine to the selection of three collaboration targets, in addition to S100A1, and to be made within the three months of the closing. BMS will acquire an additional 5% ownership before the end of 2015, and will be granted two warrants to acquire up to an additional 10% equity interest, on additional targets being introduced into the collaboration. UniQure will be eligible to receive research, development and regulatory milestone payments, including up to €234.1M for the lead S100A1 therapeutic and up to €200M for each other gene therapy product potentially developed under the collaboration. UniQure is also eligible to receive net sales based milestone payments and to single to double-digit royalties on product sales.

Jörn Aldag

Jörn Aldag

Jörn Aldag, Chief Executive Officer of UniQure declared: “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.

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