uniQure Acquires InoCard, Gene Therapy For CHF

uniQure, a company focused on developing human gene therapy, announced that it has acquired biotech firm InoCard and its novel gene therapy for the one-time treatment of congestive heart failure (CHF).

InoCard is an early stage biotech company that specializes in developing gene therapy approaches for the treatment of cardiac diseases. The company’s lead program centers on the discovery of a gene therapy for the expression of the calcium-binding protein S100A1, shown to be a key regulator of myocardial function and found to be downregulated in CHF. Administration of the protein has demonstrated in vivo benefits on heart muscle cells growth control, contractile force, and heart rhythm stability. It can also adapt the heart’s energy supply to level up cardiac output. The company reported that in an animal model of heart failure, the AAV-S100A1 gene therapy demonstrated a one-year survival rate of 90 percent.

As part of the agreement, uniQure will pay InoCard shareholders €1.5 million in cash and €1.5 million in uniQure stock as upfront payment. InoCard will also receive milestones and royalties based on pre-defined achievements.

Prof. Patrick Most and Prof. Hugo Katus, InoCard’s founders, will join the leadership team of UniQure. Jörn Aldag, uniQure's CEO, said, “There is strong scientific rationale that addressing calcium dysregulation leads to an astounding effect in congestive heart failure. We are very excited to have Patrick Most and Hugo Katus join us. …We believe that together we will deliver the best-in-class treatment for congestive heart failure.” The company also recently welcomed former Insmed CEO Will Lewis as its new board member.

Prof. Most commented, “We believe that combining our promising S100A1 therapy with uniQure's capabilities in innovating safe and effective gene therapies has the potential to transform the treatment of cardiovascular diseases.”

Congestive heart failure is characterized by the disability of the cardiac muscle to supply adequate circulatory support to the body both in active and resting states. The disease afflicts 26 million patients around the world. Patients faced with severe heart failure have a 5-year mortality rate of more than 50 percent. Prevalence of the disease is expected to jump two to three times more by 2030. There is currently no effective causative or long-term therapy for CHF.

The company stated that the first human trial for AAV-S100A1 is anticipated to launch in 2016.