Beacon Therapeutics reported positive topline results from its pivotal VISTA trial of laruparetigene zovaparvovec (laru-zova) in male patients with X-linked retinitis pigmentosa.
The randomized, controlled Phase 2/3 study enrolled 85 males aged 12 to 48 and evaluated two subretinal doses (6.8 E+11 vg/eye high, 3.7 E+11 vg/eye low) over 12 months, the company says.
At month 12, a statistically significant proportion of treated participants achieved a ≥15-letter improvement in low luminance visual acuity: 31.0% in the high-dose group (p=0.0019) and 24.1% in the low-dose group (p=0.0106) versus none in the untreated control group.
Secondary measures showed supportive trends, including improvements in mean macular sensitivity by microperimetry and higher proportions of participants achieving ≥10-letter LLVA gains.
Laru-zova demonstrated a favorable safety and tolerability profile with predominantly mild to moderate ocular treatment-emergent adverse events attributed largely to the surgical procedure; treatment-related TEAEs were reported in 25% of participants in the high-dose group and 38% in the low-dose group, and two ocular serious adverse events in the low-dose arm were attributed to surgery.
Based on the topline results, Beacon plans pre-submission engagement with global regulatory authorities, a rolling BLA submission later this year, and additional data will be presented at the AAO Annual Meeting in October.
