Bristol-Myers invests in uniQure, gaining rights to gene therapy

Dutch biotech firm uniQure (Nasdaq: QURE) saw its shares rocket more than 52% to $34.42 this morning, after it revealed a multi-million agreement with US pharma major Bristol-Myers Squibb (NYSE: BMY) that could result in payments in excess of $2.2 billion for uniQure.

B-MS will acquire 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. Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas. In total, the companies may collaborate on 10 targets, including S100A1.

uniQure acquired s100A1 last year when it bought Germany’s InoCard GmbH, an innovative, early-stage biotechnology company focused on the development of gene therapy approaches for cardiac disease, in a deal involving an upfront payment of just around $4 million (The Pharma Letter August 12, 2014).