uniQure 'intensifying efforts to further expand' gene therapy product pipeline
uniQure announced its near-term objectives following a transformational year of achievement and corporate execution. Key business accomplishments during 2017 included uniQure's alignment with regulatory authorities to advance AMT-061 into late-stage clinical development in hemophilia B, progress with its research pipeline including AMT-130 in Huntington's disease, further enhancement of the company's manufacturing capabilities, and the strengthening of its financial position. "We ended 2017 with significant momentum across all of our programs and the cash to fund operations into 2020," stated Matthew Kapusta, chief executive officer of uniQure. "Now that we have established robust, commercial-scale manufacturing in our Lexington facility, we are highly focused on the near-term initiation of our pivotal study of AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in Huntington's disease. We also are intensifying efforts to further expand our gene therapy product pipeline. In addition to AMT-126, our BMS-partnered gene therapy candidate for congestive heart failure, we have a number of promising early-stage projects focused on liver-directed and CNS disorders. We expect multiple new INDs to be filed over the next two years, further leveraging our industry-leading manufacturing and technology platform." As of December 31, 2017, the company had approximately $159M in cash and cash equivalents, and expects cash on hand will be sufficient to fund operations into 2020.