Uniqure NV (NASDAQ:QURE) investors are overwhelmingly excited today after the drug maker announced advancement of AMT-061, a Padua variant FIX AAV5 gene therapy, in hemophilia B, into a pivotal phase 3 trial in 3Q18.
Cowen analyst Ritu Baral commented, “Surprisingly to us, this payload change (Padua FIX variant has a 2 nucleotide change leading to a single amino acid substitution) will be allowed to start clinical trials under the same IND and maintain FDA Breakthrough Designation and EMA PRIME Designation granted to AMT-060. While this is entirely logical and rational, we are still surprised by the relative flexibility demonstrated by both agencies particularly given historical conservatism and nascent experience with gene therapy both agencies have had.”
“We do note other than the 2 nucleotide substitution, AMT-060 and AMT-061 are otherwise structurally identical, which we find encouraging given the predictability and duration of response seen with AMT-060 as well as excellent safety and low population seropositivity to date. QURE indicated the allowance, a remarkably positive development in our view, was not the result of any particular aggressive or extended regulatory negotiation on their part, and that the agencies main remaining focus in defining the potency of the new construct versus the old ‘060,” the analyst continued.
As such, Baral reiterates an Outperform rating on QURE stock without suggesting a price target. (To watch Baral’s track record, click here)
Wall Street is torn when it comes to whether to sing this biotech firm’s praises or assess with an apprehensive gaze, as according to TipRanks, out of 7 analysts polled in the last 12 months, four are bullish on Uniqure stock while 3 remain sidelined. With a potential downside of 17%, the stock’s consensus target price stands at $11.90.
uniQure NV engages in the research, development, and commercialization of gene therapies. Its discoveries intend to treat hemophilia, Huntington’s disease, glybera, and cardiovascular problems.