Lumevoq for LHON yields sustained visual acuity improvements at 5 years in phase 3 trial
Key takeaways:
- Visual acuity improvements were sustained through 5 years after one intravitreal injection of the gene therapy.
- There were no serious ocular adverse events reported.
A one-time injection of Lumevoq demonstrated sustained visual acuity improvements with a favorable safety profile at 5 years in patients with Leber hereditary optic neuropathy, according to a press release from GenSight Biologics.
“The REFLECT trial confirms the durability of the treatment benefit from a one-time intravitreal injection of Lumevoq, which will address a frequent question raised by regulatory authorities regarding gene therapies: Will the treatment effect fade over time?” Magali Taiel, MD, chief medical officer of GenSight Biologics, told Healio.
The phase 3 randomized, double-masked, placebo-controlled REFLECT clinical trial enrolled 98 patients with vision loss due to Leber hereditary optic neuropathy (LHON) who received an intravitreal injection of Lumevoq (lenadogene nolparvovec) in one eye. The second eye was randomly assigned to receive either Lumevoq (48 patients) or placebo (50 patients), with patients being followed up to 5 years after injection.
According to the release, there was a clinically meaningful improvement in visual acuity in eyes treated with the gene therapy, with the average visual acuity increasing beyond the 15 ETDRS letter threshold “that conventionally defines clinically meaningful improvement” relative to the observed nadir, which was defined as the worst best corrected visual acuity from baseline to year 5. In addition, 75% of those who were treated bilaterally with Lumevoq “experienced a higher rate of clinically relevant recovery from their nadir” compared with 60% of those treated unilaterally.
At 5 years, bilateral Lumevoq yielded a mean improvement in BCVA of 19 ETDRS letters in the first eye and 18 ETDRS letters in the second eye vs. nadir. Patients who received unilateral Lumevoq experienced a mean improvement of 20 ETDRS letters in the Lumevoq eye and 14 ETDRS letters in the placebo eye.
Seventy-nine percent of bilaterally treated patients and 72% of patients treated in only one eye were able to read letters on a screen, according to the release.
The safety profile was favorable overall and comparable in bilaterally and unilaterally treated patients, with no serious ocular adverse events reported.
“REFLECT provides 5 years of evidence on the safety and efficacy of bilateral injections in LHON, which is important information for a disease that blinds both eyes of patients affected by this rare condition,” Taiel said.
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