Nov. 20 Quick Takes: uniQure hemophilia therapy hits pivotal endpoint; plus data from Gilead in HIV, Aivita  in GBM and a setback for Poxel 

uniQure hemophilia therapy hits co-primary Phase III endpoint
uniQure N.V. (NASDAQ:QURE) said gene therapy etranacogene dezaparvovec met one primary endpoint of the Phase III HOPE-B trial to treat hemophilia B. The adeno-associated viral serotype 5 (AAV5) vector carrying the Factor IX gene with FIX-Padua (R338L) mutation led to significant increases in Factor IX activity, reaching a mean of 37.2% at 26 weeks. The company plans to incorporate 52-week co-primary endpoints measuring Factor IX activity and bleeding rates, and intends to hold a pre-BLA submission meeting with FDA next quarter. 

Gilead’s lenacapavir meets in Phase II/III multidrug-resistant HIV study
Gilead Sciences Inc. (NASDAQ:GILD) said its long-acting HIV-1 capsid inhibitor lenacapavir
met the primary endpoint of a viral load reduction of at least 0.5 log10 copies/mL from baseline compared with those receiving placebo at the end of a 14-day monotherapy period. In the Phase II/III CAPELLA trial, 21 out of 24 heavily pretreated HIV patients in the treatment group reached the viral load reduction threshold, versus two out of 12 patients in the placebo group (88% vs. 17%, p<0.0001). ...