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Uniqure NV (QURE) Is Getting the Street All Fired Up on Its Gene Therapy Candidate in Hemophilia B; Oppenheimer Spotlights Efficacy/Safety Strength

Hartaj Singh believes QURE trots out an upper hand advantage with AMT-061.


Uniqure NV (NASDAQ:QURE) shares vaulted 60% yesterday and continue to zoom almost 10% today on back of a clinical trial update on the biotech firm’s hemophilia B- targeting gene therapy Phase III designs for AMT-061. Investors can now look for a pivotal trial to kick-start in 2018 evaluating an asset that has already garnered a Breakthrough Therapy Designation from the FDA and PRIME designation from the EMA.

Oppenheimer analyst Hartaj Singh takes the news under bullish wing, making a point to note that such positive sentiment from the FDA and EMA alike “possibly speaks to the strength of the efficacy/safety data for AMT-061.”

In reaction to the share-popping news, the analyst reiterates an Outperform rating on QURE stock with a $17 price target, which represents a 2% increase from where the stock is currently trading. (To watch Singh’s track record, click here)

The biotech firm is stepping forward with “two competitive advantages” with its gene therapy candidate in hemophilia B candidate, writes Singh, noting that the treatment “has not demonstrated any immune-mediated responses in AMT-060 trials with little loss of FIX activity and has the lowest prevalence of preexisting neutralizing antibodies (NAb) compared to other AAV vectors,” while the firm likewise boasts ” a commercial grade facility already operating to scale up manufacturing of AMT-061 as clinical trials progress.”

Singh highlights: “While we have been waiting for an update to the clinical trial plan for the first hemophilia B product candidate AMT-060, QURE has astutely—and stealthily—brought another product candidate (AMT-061) into the clinic. This product has potentially 6x to 7x the well-known FIX expression levels of AMT-060 and may lead to mean FIX activity of approximately 30% to 50% of normal; a potentially significant benefit to moderate to severe hemophilia B patients. While we await further visibility on the pivotal Phase 3 design, we reiterate our bullish stance.”

Additionally, according to TipRanks, cautious optimism is circling the biotech firm when looking at Wall Street sentiment, considering that based on 6 analysts polled in the 12 months, half rate a Buy on Uniqure stock while half maintain a Hold. The 12-month average price target stands at $10.25.QURE Analyst ratings chart