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GenSight Biologics announces publication of results from Lumevoq REVERSE pivotal phase III trial in Science Translational Medicine.

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Published:19th Dec 2020
GenSight Biologics announced that the journal Science Translational Medicine has published results from the REVERSE pivotal Phase III clinical trial of Lumevoq gene therapy in ND4 Leber Hereditary Optic Neuropathy (LHON) subjects along with key results from a non-human primate study investigating the contralateral effect of the gene therapy. The paper, published in the December issue under the title “Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy”, is the first peer-reviewed article based on Phase III clinical trial data to document sustained and clinically meaningful bilateral improvement in visual outcomes from a unilateral injection of a gene therapy. The findings from the REVERSE trial and the non-human primate study were key components of the data package submitted by GenSight Biologics in September 2020 to the European Medicines Agency when it applied for marketing authorization for Lumevoq as treatment for patients with visual loss due to LHON caused by a confirmed mutation in the ND4 mitochondrial gene. The agency’s decision is expected in Q4 2021. REVERSE Trial outcomes : 37 ND4 LHON subjects, who experienced onset of vision loss from 6 months to one year before enrollment, participated in the REVERSE trial. The results show a clinically meaningful improvement over baseline of +15 ETDRS letters (?0.308 LogMAR) in the average best-corrected visual acuity (BCVA) of injected eyes of the 37 REVERSE patients 96 weeks after treatment. The patients’ other eye, which received a sham injection, experienced an average visual acuity gain over baseline of +13 letters equivalent (?0.259 LogMAR). Against nadir, or the worst recorded BCVA, the gains were even more impressive, at +28.5 ETDRS letters for the Lumevoq-injected eyes and +24.5 ETDRS letters for sham-injected eyes. 81% of subjects showed a clinically relevant recovery (CRR) from the nadir in one or both eyes. CRR, a measure of treatment response established by an international consensus meeting on the management of LHON1, is defined as either an improvement from off-chart BCVA to on-chart, or an on-chart improvement BCVA of at least -0.2 LogMAR, or +10 ETDRS letters. The improvement in quality of life metrics relative to baseline values taken before treatment, which were evaluated using the well-established National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25), was compelling and largely above the thresholds of clinical relevance. The composite NEI VFQ-25 score showed a mean improvement of 9.5 points, exceeding the clinically relevant threshold of +3.9-+4.3 points. See- "Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy"-Patrick Yu-Wai-Man,, Nancy J. Newman,Valerio Carelli, Mark L. Moster, Valerie Biousse, Alfredo A. Sadun, Thomas Klopstock, Catherine Vignal-Clermont.,et al. Science Translational Medicine 09 Dec 2020: Vol. 12, Issue 573, eaaz7423 DOI: 10.1126/scitranslmed.aaz7423.
Condition: Leber's Hereditary Optic Neuropathy
Type: drug

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