UniQure beats a path to Phase III after a 'promising' gene therapy trial

UniQure CEO Joern Aldag

UniQure ($QURE) is pressing forward with its gene therapy for a rare and deadly disease after charting a positive signal in a small yearlong trial, news that sent the Dutch company's share price soaring.

The drug, AMT-110, is designed to treat Sanfilippo B syndrome, a rare disorder that results from a lack of the NaGlu protein and is generally fatal for the children who inherit it. In a four-patient trial conducted by three French research institutions, a single dose of AMT-110 increased NaGlu activity from 0% at baseline to as much as 17% after three months, uniQure said, adding that the effect was "persistent" through 12 months.

The news, released over the weekend, sent uniQure's shares up as much as 20% in premarket trading on Monday.

The company is disclosing only top-line results from the Phase I/II trial for now but is heralding AMT-110's effect as a promising first. No prior gene therapy has ever demonstrated a consistent restoration of NaGlu over 12 months, lead investigator Marc Tardieu said, and past studies suggest that boosting protein activity by even 5% to 10% is associated with longer life spans and better quality of life for children with Sanfilippo B.

UniQure believes it has seen enough early signs of efficacy in AMT-110's four-person trial to merit further development, and the company is now negotiating with its French partners to take the reins on the gene therapy, planning a pivotal trial in Sanfilippo B.

"We are gratified that our pursuit of a gene therapy targeting CNS diseases has found this early and promising success," uniQure CEO Joern Aldag said in a statement. "We acknowledge the tremendous support we have received from the consortium that was paramount for generating this early proof in patients."

Meanwhile, uniQure is advancing on multiple fronts in gene therapy. Its lead asset, Glybera, has run into regulatory delays in the U.S., as the FDA wants to see data from a second Phase III trial before considering approval. But uniQure is moving right along with in-house clinical programs for hemophilia B and Parkinson's disease, at the same time working with Bristol-Myers Squibb ($BMY) under a cardiovascular-focused partnership worth up to $1 billion for the company.

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