The primary evidence of efficacy for LUXTURNA is derived from the open-label, randomized, controlled Phase 3 study (Study 301). A total of 31 subjects with confirmed biallelic RPE65 mutation-associated retinal dystrophy were randomized 2:1 to the treatment group (n=21) or control group (n=10). The Intent-to-Treat (ITT) population included all 31 subjects.

Primary Endpoint: The primary endpoint was the change from baseline to Year 1 in MLMT score, using both eyes. The MLMT measures functional vision across 7 levels of illumination, from 1 lux (moonless night) to 400 lux (office), with a passing score ranging from -1 (fail at 400 lux) to 6 (pass at 1 lux). The treatment group demonstrated a mean MLMT score improvement of 1.8 (SD 1.1) compared to 0.2 (SD 1.0) in the control group. The difference of 1.6 was highly statistically significant (p=0.001).

Clinical Meaningfulness & Responder Analysis: An MLMT score change of ≥2 is considered clinically meaningful. In the treatment group, 11 of 21 subjects (52%) achieved an MLMT score change of ≥2, compared to only 1 of 10 subjects (10%) in the control group.

Secondary & Supportive Endpoints:
- FST Testing: The treatment group had a >100-fold improvement in light sensitivity, which was statistically significant compared to controls (p<0.001).
- Visual Acuity (VA): While the change in VA was not statistically significant, 11 of 20 subjects (55%) in the treatment group had a clinically meaningful improvement of ≥0.3 LogMAR in the first-treated eye, versus 0% in the control group.

Durability of Effect: The improvement in MLMT score was observed by Day 30 and was sustained through Year 2 in the treatment group. The Phase 1 study data show a persistence of effect for at least three years. The ongoing long-term follow-up study will continue to monitor durability for 15 years.