Spark sees path forward on Phase III results
Spark Therapeutics Inc. (NASDAQ:ONCE) jumped $9.09 (21%) to $53.02 on Monday after its SPK-RPE65 met the primary endpoint and two of three secondary endpoints in a Phase III trial to treat retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated inherited retinal dystrophies (IRDs). Spark plans to submit a BLA next year for SPK-RPE65.
The adeno-associated virus (AAV) carrying the RPE65 gene met the primary endpoint of improving functional vision vs. the control group (p=0.001). The endpoint was measured by change in bilateral mobility testing from baseline to one year; Spark co-founder and CEO Jeffrey Marrazzo said Spark developed the novel endpoint internally. On a conference call Monday, Marrazzo said two-thirds of patients in the treatment group were able to navigate the mobility course in the lowest lighting conditions vs. zero patients in the control group. ...