UniQure bets on a gene therapy for heart failure

After making international headlines with the West's first approval for a gene therapy, pioneering biotech uniQure ($QURE) is working to flesh out its pipeline of one-time treatments, buying into an early-stage shot at heart failure that could put its technology on the biggest stage yet.

The Netherlands-headquartered company has acquired Germany's InoCard for its lead asset, a gene therapy that turns up the calcium-binding protein S100A1 in hopes of relieving congestive heart failure (CHF). InoCard founders Patrick Most and Hugo Katus discovered that the protein served as a master regulator of cardiac function, noting that it was downregulated in CHF patients and that upping its expression resulted in perceptible benefits.

So, the researchers attached S100A1 to a harmless adeno-associated virus (AAV) and crafted a gene therapy to combat heart failure. The resulting treatment, dubbed AAV-S100A1, demonstrated a 90% survival rate in a proof-of-concept study on an animal heart failure model, the company said, setting the table for further preclinical study.

That's where uniQure comes in. The biotech is trading €3 million ($4 million) up front in cash and stock, plus undisclosed milestones, to pick up where Most and Katus left off. The two professors will join uniQure's team as it works to get AAV-S100A1 ready for first-in-human trials by 2016, hoping to develop a one-shot therapy for the world's more than 20 million CHF sufferers.

Thanks to some maturing technology and renewed investor confidence, gene therapy is having something of a second honeymoon, factors that gave a "best-in-class" company like InoCard more than a few options when looking for a deal, uniQure CEO Jörn Aldag said. But his company's lengthy résumé in the field and deep investments in AAV manufacturing made it stand alone, he said.

"We believe uniQure, with its unique and scalable manufacturing process, is in the best position to push this program forward," Aldag told FierceBiotech. "InoCard's founders wanted to partner with us because of the proven expertise we have in gene therapy."

The company is at work on a 55,000-square-foot production plant in Lexington, MA, scheduled to start churning out commercial-grade AAV products next year. The facility will complement an EMA-approved plant in Amsterdam, and all that capacity allows uniQure to quickly scale up R&D efforts without having to rely on outside contractors, Aldag said, a major selling point for potential partners.

To date, the majority of gene therapy work has focused on rare genetic diseases--including that of Glybera, uniQure's EU-approved treatment for familial lipoprotein lipase deficiency. InoCard's treatment stands out as one of the few targeting a major indication with a huge patient population, and success could further bolster the resurgent gene therapy space, Aldag said.

"Now you see new companies are being founded on a monthly basis, and suddenly public markets are interested--private markets too," he said, adding that even Big Pharma has begun to make some noise in the field. "In a way it resembles what you may have seen with silencing technology and with antibodies."

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Special Report: FierceBiotech's 2013 Fierce 15 - UniQure

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