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There Could Be 109% Upside In uniQure As Company Advances Gene Therapy Into Clinical Trials

There Could Be 109% Upside In uniQure As Company Advances Gene Therapy Into Clinical Trials
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Following its meeting with the FDA and the EMA, Uniqure NV (NASDAQ: QURE) announced it would expeditiously advance its adeno-associated virus, or AAV-based gene therapy, AMT-061 into pivotal trials in severe and moderately severe hemophilia B.

Reacting to the announcement, Chardan maintains its Buy rating on uniQure and raised its price target to $35, attributing the revision to Hemophilia B and the return of tangible pipeline value. The price target suggests a 109 percent gain from $16.75 at which the shares opened today.

At last check, shares of uniQure were skyrocketing 25.33 percent to $19.

Analyst Gbola Amusa said AMT-061 will use AAV5 and the FIX-Padua mutant, which provides six to nine times more FIX activity than the naturally-occurring FIX. The analyst also noted that the company announced it had obtained exclusive use of a patent family that broadly covers the FIX-Padua variant.

FIX is a serine proteases of the coagulation system, the deficiency of which causes hemophilia B.

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Amusa pointed out that AMT-061 will be included under existing Breakthrough Therapy and PRIME designations under the existing IND for AMT-060, which was shelved in favor of AMT-061.

The company will conduct a short dose-confirmatory study in the third quarter, which will run for six weeks, with the pivotal trial also set to start in 2018. Additional pre-clinical data for AMT-061 will be announced at the American Society of Hematology conference taking place between Dec. 9 and 12 in Atlanta, Georgia.


Chardan believes uniQure can achieve hemophilia B segment leadership in vector gene therapy, with the view premised on the company's leading manufacturing capabilities, and its utilization of the AAV5 capsid and the FIX-Padua variant.

The firm said the company's product likely enables broader use in hemophilia B than other vector gene therapy. Additionally, the firm said Padua should enable best-in-class efficacy for AMT-061, similar to that for Spark Therapeutics Inc (NASDAQ: ONCE)'s SPK-9001.

"UniQure is confident that AMT-061 could lead to mean FIX activity of ~30-50% of normal, which we think is consistent," the firm added.

Latest Ratings for QURE

Jan 2018Janney CapitalInitiates Coverage OnBuy
Dec 2017H.C. WainwrightInitiates Coverage OnBuy
Nov 2017Evercore ISI GroupInitiates Coverage OnOutperform

View More Analyst Ratings for QURE
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