Gene therapy for choroideremia fails in late-stage clinical trial
The phase 3 STAR study investigating timrepigene emparvovec (BIIB111/AAV2-REP1; Biogen), an investigational gene therapy for the treatment of choroideremia, did not meet its primary endpoint of proportion of participants with a ≥15 letter improvement from baseline in best-corrected visual acuity (BCVA) at 12 months, according to a press release. Key secondary endpoints were also not met.
“We extend our deepest gratitude to all those who contributed to the STAR study, including the participants, investigators, site staff, and the broader choroideremia community,” Katherine Dawson, MD, head of the Therapeutics Development Unit at Biogen, said in a company news release. “While we are disappointed by the results of the STAR study, we are hopeful that the clinical insights gleaned from this study may help to shape therapeutic innovation for inherited retinal diseases including choroideremia, so that in the future there may be treatment options for the community affected by these debilitating disorders.”
Read the full press release here.