{"id":3920,"date":"2022-01-24T06:30:40","date_gmt":"2022-01-24T11:30:40","guid":{"rendered":"https:\/\/www.gensight-biologics.com\/gensight-biologics-annonce-le-maintien-de-lamelioration-cliniquement-significative-de-la-vision-4-ans-apres-une-seule-injection-de-la-therapie-genique-lumevoq\/"},"modified":"2026-11-04T04:33:04","modified_gmt":"2026-11-04T03:33:04","slug":"gensight-biologics-annonce-le-maintien-de-lamelioration-cliniquement-significative-de-la-vision-4-ans-apres-une-seule-injection-de-la-therapie-genique-lumevoq","status":"publish","type":"post","link":"https:\/\/www.gensight-biologics.com\/fr\/2022\/01\/24\/gensight-biologics-annonce-le-maintien-de-lamelioration-cliniquement-significative-de-la-vision-4-ans-apres-une-seule-injection-de-la-therapie-genique-lumevoq\/","title":{"rendered":"GenSight Biologics annonce le maintien de l\u2019am\u00e9lioration cliniquement significative de la vision, 4 ans apr\u00e8s une seule injection de la th\u00e9rapie g\u00e9nique LUMEVOQ\u00ae"},"content":{"rendered":"\n<strong>Paris, France, le lundi 24 janvier 2022<\/strong>, <strong>7h30 CET<\/strong> \u2013 GenSight Biologics (Euronext : SIGHT, ISIN : FR0013183985, \u00e9ligible PEA-PME), soci\u00e9t\u00e9 biopharmaceutique d\u00e9di\u00e9e au d\u00e9veloppement et \u00e0 la commercialisation de th\u00e9rapies g\u00e9niques innovantes pour les maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine et du syst\u00e8me nerveux central, annonce aujourd&rsquo;hui que des sujets atteints de Neuropathie Optique H\u00e9r\u00e9ditaire de Leber (NOHL) trait\u00e9s avec LUMEVOQ<sup>\u00ae<\/sup> continuent \u00e0 b\u00e9n\u00e9ficier d&rsquo;une nette am\u00e9lioration de leur vision quatre ans apr\u00e8s une seule injection de la th\u00e9rapie g\u00e9nique. Les r\u00e9sultats proviennent de RESTORE (CLIN06), l&rsquo;\u00e9tude de suivi \u00e0 long terme \u00e0 laquelle les participants aux \u00e9tudes pivotales de Phase III RESCUE<sup>1<\/sup> et REVERSE<sup>2<\/sup> ont \u00e9t\u00e9 invit\u00e9s.\n\n&nbsp;\n\nLorsque les sujets ont int\u00e9gr\u00e9 l&rsquo;\u00e9tude RESTORE, 2 ans apr\u00e8s l&rsquo;injection unique, ils pr\u00e9sentaient d\u00e9j\u00e0 des am\u00e9liorations cliniquement significatives par rapport au point le plus bas (le \u00ab nadir \u00bb) de leur acuit\u00e9 visuelle (BCVA) : +18,8 lettres ETDRS* dans les yeux trait\u00e9s par LUMEVOQ<sup>\u00ae<\/sup> et +17,3 lettres dans les yeux non-trait\u00e9s (injection simul\u00e9e ou <em>sham<\/em>). Quatre ans apr\u00e8s le traitement, l&rsquo;am\u00e9lioration bilat\u00e9rale \u00e0 partir du nadir a \u00e9t\u00e9 maintenue, les yeux trait\u00e9s par LUMEVOQ<sup>\u00ae<\/sup> obtenant une am\u00e9lioration moyenne par rapport au nadir de +22,5 lettres et les yeux <em>sham<\/em> montrant une am\u00e9lioration moyenne de +20,5 lettres.\n\n&nbsp;\n\nL&rsquo;impact de ces r\u00e9sultats sur les patients est d\u00e9montr\u00e9 par une am\u00e9lioration des scores de qualit\u00e9 de vie (QoL) autod\u00e9clar\u00e9s en Ann\u00e9e 4 par rapport \u00e0 la <em>baseline<\/em>. La qualit\u00e9 de vie globale moyenne a progress\u00e9 de mani\u00e8re cliniquement significative par rapport \u00e0 la <em>baseline<\/em>, port\u00e9e par des am\u00e9liorations cliniquement significatives des sous-scores pertinents correspondant \u00e0 la sant\u00e9 mentale et \u00e0 la capacit\u00e9 \u00e0 mener des activit\u00e9s de mani\u00e8re autonome (par exemple, limites fonctionnelles, d\u00e9pendance, activit\u00e9s faisant intervenir la vision de pr\u00e8s et de loin, vision g\u00e9n\u00e9rale).\n\n&nbsp;\n\n\u00ab\u00a0<em>Le suivi \u00e0 4 ans de l\u2019\u00e9tude RESTORE fournit aux patients atteints de neuropathie optique h\u00e9r\u00e9ditaire de Leber et \u00e0 leurs familles, ainsi qu&rsquo;\u00e0 la communaut\u00e9 neuro-ophtalmologique, des donn\u00e9es particuli\u00e8rement int\u00e9ressantes sur l&rsquo;efficacit\u00e9 et l&rsquo;innocuit\u00e9 de la th\u00e9rapie intravitr\u00e9enne LUMEVOQ <\/em>\u00bb, a comment\u00e9 le <strong>Dr Robert Sergott<\/strong>, Directeur du service de Neuro-Ophtalmologie, Wills Eye Hospital, et Fondateur et Directeur, William H. Annesley, Jr, EyeBrain Center, Thomas Jefferson University, Philadelphie, Etats-Unis. \u00ab\u00a0<em>Compar\u00e9 \u00e0 l&rsquo;histoire naturelle de la NOHL, les donn\u00e9es observ\u00e9es sur 4 ans valident et \u00e9tendent les observations \u00e0 3 ans, en confirmant que l&rsquo;am\u00e9lioration objective de l&rsquo;acuit\u00e9 visuelle est soutenue et associ\u00e9e \u00e0 une meilleure qualit\u00e9 de vie, sans aucun probl\u00e8me de s\u00e9curit\u00e9 \u00e0 long terme. <\/em>\u00bb\n\n&nbsp;\n\n\u00ab\u2009<em>Les r\u00e9sultats de RESTORE mettent en \u00e9vidence le b\u00e9n\u00e9fice th\u00e9rapeutique du traitement innovant de GenSight pour la NOHL, apr\u00e8s une seule injection : am\u00e9lioration durable et cliniquement significative de la fonction visuelle, associ\u00e9e \u00e0 une s\u00e9curit\u00e9 remarquable<\/em>\u2009\u00bb, a comment\u00e9 <strong>Bernard Gilly<\/strong>, Co-fondateur et Directeur G\u00e9n\u00e9ral de GenSight Biologics. \u00ab\u2009<em>L&rsquo;ensemble des preuves que nous avons maintenant accumul\u00e9es est une excellente nouvelle pour les patients, dans l\u2019attente urgente d&rsquo;un traitement pour cette maladie provoquant une c\u00e9cit\u00e9 brutale. Nous continuons \u00e0 travailler avec diligence, en \u00e9troite collaboration avec les autorit\u00e9s comp\u00e9tentes, pour conduire le processus d&rsquo;examen r\u00e9glementaire et rendre LUMEVOQ disponible pour tous les patients<\/em>.\u2009\u00bb\n\n&nbsp;\n\nRESTORE est l&rsquo;une des plus importantes \u00e9tudes de suivi \u00e0 long terme pour le traitement d&rsquo;une maladie rare, avec 62 sujets acceptant d\u2019\u00eatre inclus et suivis. Tous les sujets atteints de NOHL caus\u00e9e par un g\u00e8ne mitochondrial <em>ND4<\/em> mut\u00e9 ont \u00e9t\u00e9 trait\u00e9s par une injection intravitr\u00e9enne unique de LUMEVOQ<sup>\u00ae<\/sup> dans un \u0153il et par une injection simul\u00e9e (<em>sham<\/em>) dans l&rsquo;autre.\n\n&nbsp;\n\n<strong>Table 1. Am\u00e9lioration de l\u2019acuit\u00e9 visuelle (BCVA) par rapport au Nadir* dans l\u2019\u00e9tude de suivi \u00e0 long terme de LUMEVOQ<sup>\u00ae<\/sup> (RESTORE)<\/strong>\n\n<strong>\u00a0<\/strong>\n<table>\n<tbody>\n<tr>\n<td rowspan=\"2\" width=\"94\"><\/td>\n<td colspan=\"2\" width=\"173\"><strong><em>2 ans post injection<\/em><\/strong><\/td>\n<td colspan=\"2\" width=\"177\"><strong><em>3 ans post injection<sup>3<\/sup><\/em><\/strong><\/td>\n<td colspan=\"2\" width=\"173\"><strong><em>4 ans post injection<\/em><\/strong><\/td>\n<\/tr>\n<tr>\n<td width=\"76\"><strong>LogMAR<\/strong>\n\n<em>(Erreur Standard)<\/em><\/td>\n<td width=\"98\"><strong>Lettres ETDRS**<\/strong><\/td>\n<td width=\"82\"><strong>LogMAR<\/strong>\n\n<em>(Erreur Standard)<\/em><\/td>\n<td width=\"95\"><strong>Lettres ETDRS **<\/strong><\/td>\n<td width=\"76\"><strong>LogMAR <\/strong><em>(Erreur Standard)<\/em><\/td>\n<td width=\"98\"><strong>Lettres ETDRS **<\/strong><\/td>\n<\/tr>\n<tr>\n<td width=\"94\">Yeux trait\u00e9s avec LUMEVOQ<sup>\u00ae<\/sup><\/td>\n<td width=\"76\">-0,375\n\n<em>(0,306)<\/em><\/td>\n<td width=\"98\"><strong>+18,8<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<td width=\"82\">-0,410\n\n<em>(0,365)<\/em><\/td>\n<td width=\"95\"><strong>+20,5<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<td width=\"76\">-0,453\n\n<em>(0,440)<\/em><\/td>\n<td width=\"98\"><strong>+22,5<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<\/tr>\n<tr>\n<td width=\"94\">Yeux <em>Sham<\/em><\/td>\n<td width=\"76\">-0,346\n\n<em>(0,291)<\/em><\/td>\n<td width=\"98\"><strong>+17,3<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<td width=\"82\">-0,387\n\n<em>(0,369)<\/em><\/td>\n<td width=\"95\"><strong>+19,4<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<td width=\"76\">-0,406\n\n<em>(0,361)<\/em><\/td>\n<td width=\"98\"><strong>+20,5<\/strong>\n\n<strong>\u00a0<\/strong><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<em>Note <\/em><strong>: <\/strong>L&rsquo;\u00e9chantillon RESTORE est compos\u00e9 des participants aux \u00e9tudes RESCUE et REVERSE qui ont accept\u00e9 d&rsquo;\u00eatre suivis dans le cadre de l&rsquo;\u00e9tude de suivi \u00e0 long terme. Les valeurs de l&rsquo;ann\u00e9e 4 \u00e9taient les mesures exprim\u00e9es en LogMAR les plus proches de 1461 jours apr\u00e8s le traitement enregistr\u00e9es \u00e0 1461 +\/- 273 jours apr\u00e8s le traitement. Les valeurs manquantes ont \u00e9t\u00e9 imput\u00e9es \u00e0 l&rsquo;aide de la m\u00e9thode de la derni\u00e8re observation report\u00e9e (LOCF). *Nadir = acuit\u00e9 visuelle la plus basse enregistr\u00e9e entre la baseline et l&rsquo;ann\u00e9e 4. ** Les mesures d\u2019acuit\u00e9 visuelle ont \u00e9t\u00e9 enregistr\u00e9es en LogMAR. Le changement depuis le nadir en LogMAR a \u00e9t\u00e9 converti en \u00a0\u00ab \u00e9quivalent lettres ETDRS \u00bb en multipliant le LogMAR par -50 (r\u00e9f. J.T. Holladay, J Refrac Surgery, 1997 ;13, 388-391).\n\n&nbsp;\n\nLes analyses des r\u00e9pondeurs \u00e0 4 ans indiquent qu\u2019une proportion importante des patients suivis ont pr\u00e9sent\u00e9 une am\u00e9lioration de l\u2019acuit\u00e9 visuelle. 71,0% des sujets RESTORE ont ainsi obtenu une r\u00e9cup\u00e9ration cliniquement pertinente (<em>Clinically Relevant Recovery<\/em> ou CRR)<sup>4<\/sup> par rapport au nadir quatre ans apr\u00e8s le traitement, et 80,7% d&rsquo;entre eux avaient une vision \u00ab\u00a0<em>on chart<\/em>\u00a0\u00bb (acuit\u00e9 visuelle \u2264 1,6 LogMAR, correspondant \u00e0 la facult\u00e9 de lire a minima 5 lettres ETDRS \u00e0 1m de distance) dans un ou les deux yeux.\n\n&nbsp;\n\nMis en perspective avec l\u2019\u00e9volution naturelle de la vision g\u00e9n\u00e9ralement observ\u00e9e chez les patients non trait\u00e9s<sup>5<\/sup>, ces r\u00e9sultats repr\u00e9sentent une diff\u00e9rence significative par rapport \u00e0 l\u2019histoire naturelle de la NOHL.\n\n&nbsp;\n\n<strong>Figure 1. Evolution de l\u2019acuit\u00e9 visuelle chez les patients trait\u00e9s avec LUMEVOQ<sup>\u00ae<\/sup> (RESCUE\/REVERSE\/RESTORE) compar\u00e9 aux patients non trait\u00e9s<\/strong>\n\n<img loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-8590\" src=\"\/wp-content\/uploads\/2022\/01\/LOESS-curves-RESTORE-Year-4-500x433.jpg\" alt=\"\" width=\"500\" height=\"433\" \/>\n\n<em>Note <\/em><strong>: <\/strong>Les courbes obtenues selon la m\u00e9thode <em>Locally Estimated Scatterplot Smoothing<\/em> (LOESS) montrent l&rsquo;\u00e9volution, de 12 \u00e0 52 mois apr\u00e8s le d\u00e9but de la perte de vision, de l\u2019acuit\u00e9 visuelle (BCVA) moyenne de tous les yeux (LUMEVOQ<sup>\u00ae<\/sup> et <em>Sham<\/em>) issus des \u00e9tudes REVERSE \/ RESCUE \/ RESTORE, et de tous yeux d&rsquo;une cohorte comparable de patients non trait\u00e9s par LUMEVOQ<sup>\u00ae<\/sup>. Les zones color\u00e9es repr\u00e9sentent l&rsquo;intervalle de confiance \u00e0 95% pour la BCVA moyenne. Les valeurs &gt;52 mois ont \u00e9t\u00e9 fix\u00e9es \u00e0 52 mois. La courbe commence 12 mois apr\u00e8s le d\u00e9but de la maladie lorsque 92,7% des yeux des \u00e9tudes RESCUE et REVERSE ont re\u00e7u un traitement, soit avec LUMEVOQ<sup>\u00ae<\/sup>, soit avec une injection <em>Sham<\/em>. La cohorte non trait\u00e9e \u00e9tait compos\u00e9e de 208 patients <em>ND4<\/em>-LHON qui ont \u00e9t\u00e9 suivis dans le registre REALITY et issus de deux \u00e9tudes d&rsquo;histoire naturelle prospectives et huit r\u00e9trospectives.<sup>6<\/sup>\n\n&nbsp;\n\nLes r\u00e9sultats de s\u00e9curit\u00e9 4 ans apr\u00e8s l&rsquo;injection sont coh\u00e9rents avec les informations pr\u00e9c\u00e9demment rapport\u00e9es, qui concluaient que LUMEVOQ<sup>\u00ae<\/sup> est bien tol\u00e9r\u00e9 : aucun \u00e9v\u00e9nement ind\u00e9sirable grave n&rsquo;a \u00e9t\u00e9 enregistr\u00e9 parmi les yeux trait\u00e9s par LUMEVOQ<sup>\u00ae<\/sup>, et aucune sortie de l\u2019\u00e9tude n&rsquo;est survenue en raison d&rsquo;\u00e9v\u00e9nements oculaires. Il n&rsquo;y a pas eu d&rsquo;\u00e9v\u00e9nements ind\u00e9sirables graves syst\u00e9miques ou de sorties li\u00e9s au traitement ou \u00e0 la proc\u00e9dure de l&rsquo;\u00e9tude.\n\n&nbsp;\n\nL&rsquo;examen de la demande d&rsquo;Autorisation de Mise sur le March\u00e9 (AMM) europ\u00e9enne de LUMEVOQ<sup>\u00ae<\/sup> est en cours, la d\u00e9cision du CHMP \u00e9tant attendue au T4 2022.\n\n&nbsp;\n\n<strong>R\u00e9f\u00e9rences et notes:<\/strong>\n<ol>\n \t<li>Newman NJ, Yu-Wai-Man P, Carelli V, et al. Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset. <em>Ophthalmology<\/em> (2021) 128:649\u201360. doi: 10.1016\/j.ophtha.2020.12.012.<\/li>\n \t<li>Yu-Wai-Man P, Newman NJ, Carelli V, et al. Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy. <em>Sci Transl Med.<\/em> (2020) 12:eaaz7423. doi: 10.1126\/scitranslmed.aaz7423.<\/li>\n \t<li>Biousse, V, Newman, N, Yu-Wai-Man P, et al. Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study, <em>J Neuroophthalmol.<\/em> (2021) 41: 309-315. doi: 10.1097\/WNO.0000000000001367.<\/li>\n \t<li>Une r\u00e9cuperation cliniquement pertinente (<em>Clinically Relevant Recovery<\/em> ou CRR) correspond \u00e0 une am\u00e9lioration d\u2019au moins 0,2\u00a0LogMAR\u00a0(pour les yeux \u201con chart\u201d) ou \u00e0 un passage de \u00ab\u00a0off chart\u00a0\u00bb \u00e0 \u00ab\u00a0on chart\u00a0\u00bb (pour les yeux \u00ab\u00a0off chart\u00a0\u00bb).<\/li>\n \t<li>Newman NJ, Carelli V, Taiel M, Yu-Wai-Man P. Visual outcomes in Leber hereditary optic neuropathy subjects with the m.11778G&gt;A (MTND4) mitochondrial DNA mutation. J Neuroophthalmol. (2020) 40:547\u201357. doi: 10.1097\/WNO.0000000000001045.<\/li>\n \t<li>Newman NJ, Yu-Wai-Man P, Carelli V, et al.,. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G&gt;A ND4 Mutation: Systematic Review and Indirect Comparison. <em> Neurol.<\/em> (2021) 12:662838. doi: 10.3389\/fneur.2021.662838.<\/li>\n<\/ol>\n","protected":false},"excerpt":{"rendered":"<p>Paris, France, le lundi 24 janvier 2022, 7h30 CET \u2013 GenSight Biologics (Euronext : SIGHT, ISIN : FR0013183985, \u00e9ligible PEA-PME), soci\u00e9t\u00e9 biopharmaceutique d\u00e9di\u00e9e au d\u00e9veloppement et \u00e0 la commercialisation de th\u00e9rapies g\u00e9niques innovantes pour les maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine et du syst\u00e8me nerveux central, annonce aujourd&rsquo;hui que des sujets atteints de Neuropathie Optique H\u00e9r\u00e9ditaire [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":5367,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[54],"tags":[],"featured-area":[],"news-topic":[73],"news-type":[75],"post-year":[109],"class_list":["post-3920","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-communiques-de-presse-financiers","news-topic-gs010-lumevoq","news-type-clinical","post-year-109"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>GenSight Biologics annonce le maintien de l\u2019am\u00e9lioration cliniquement significative de la vision, 4 ans apr\u00e8s une seule injection de la th\u00e9rapie g\u00e9nique LUMEVOQ\u00ae | GenSight Biologics<\/title>\n<meta name=\"robots\" content=\"index, follow, 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