Lumevoq gene therapy shows sustained efficacy, safety at 3 years in phase 3 LHON trial
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Key takeaways:
- Seventy-three percent of bilateral subjects had a clinically meaningful improvement of at least –0.3 logMAR relative to their observed nadir.
- No serious ocular adverse events were reported at 3 years.
A phase 3 clinical trial demonstrated positive 3-year results for bilateral intravitreal injection of Lumevoq gene therapy, with statistically significant visual acuity improvements from baseline in both treated eyes.
Of the 98 participants in the randomized, double-masked, placebo-controlled REFLECT trial with vision loss due to Leber hereditary optic neuropathy (LHON) caused by a mutated ND4 mitochondrial gene, 48 were randomly assigned to receive Lumevoq (GS010; lenadogene nolparvovec) bilateral treatment while 50 received unilateral treatment and placebo, according to a press release from GenSight Biologics.
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Data at year 3 showed a difference in mean visual acuity improvement between treatment arms of 6.5 ETDRS letters in favor of bilaterally treated subjects, with 73% of bilateral subjects experiencing a clinically meaningful improvement of at least –0.3 logMAR (+15 ETDRS letters) relative to their observed nadir.
Placebo eyes in subjects treated with unilateral Lumevoq also showed visual acuity improvement due to contralateral treatment effect, according to the release. Additionally, among patients who were not able to read any letters on a screen at baseline, 62% are now “back to on-chart territory.”
The 3-year results reinforce those reported at 2 years.
The favorable safety profile of the treatment was confirmed, with no serious ocular adverse events reported.
“The REFLECT trial has delivered a further set of results demonstrating the sustained benefit of lenadogene nolparvovec, while reconfirming its safety profile,” Nancy J. Newman, MD, international principal investigator of REFLECT said in the release. “In addition, the additional benefit among bilaterally treated patients suggests that injection of both eyes may be the best option for LHON patients with the ND4 mutation.”
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