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

Following its meeting with the FDA and the EMA, Uniqure NV 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.

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 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.