BMS acquires from uniQure N.V. exclusive rights to S100 A1, a calcium sensor in early development for heart failure

Bristol-Myers Squibb Company and uniQure N.V. announced an agreement that 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 S100 A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction.

Comment:The therapy was developed based on a fundamental discovery that S100 A1 is downregulated in CHF and administration of S100 A1 has demonstrated in vivo beneficial effects on contractile force, growth control of heart muscle cells and rhythm stability of the heart and is also able to adapt the heart�s energy supply to increased cardiac output. In a porcine heart failure model,treatment with AAV-S100 A1 demonstrated a 12-month survival rate of 90%. S100 A1 was originally developed by InoCard GmbH which was acquired by uniQure. uniQure and Bristol-Myers Squibb anticipate their collaboration to be effective during the second quarter of 2015.