{"id":15645,"date":"2019-10-10T05:19:43","date_gmt":"2019-10-10T03:19:43","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=15645"},"modified":"2019-10-16T05:30:38","modified_gmt":"2019-10-16T03:30:38","slug":"proqr-announces-positive-top-line-results-from-the-phase-1-2-study-of-sepofarsen-in-lca10-patients","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/proqr-announces-positive-top-line-results-from-the-phase-1-2-study-of-sepofarsen-in-lca10-patients\/","title":{"rendered":"ProQR Announces Positive Top-Line Results from the Phase 1\/2 Study of Sepofarsen in LCA10 Patients"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced positive top-line results from the PQ-110-001 study, a Phase 1\/2 dose range finding, first-in-human trial of <g class=\"gr_ gr_4 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling\" id=\"4\" data-gr-id=\"4\">sepofarsen<\/g> (QR-110) in patients with Leber\u2019s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the <em>CEP290 <\/em>gene.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cWe reported today that patients receiving sepofarsen had a clinically\nmeaningful improvement in vision, and in some cases the patient\u2019s vision\nimproved to a level that could be deemed life changing. This is very\nencouraging for the LCA10 community and the Inherited Retinal Disease community\nas a whole,\u201d said Stephen R. Russell, MD, Schrage Professor of Ophthalmology\nand Visual Sciences and Principal Investigator at the University of Iowa.\n\u201cLCA10 is a severe inherited retinal disease that leads to blindness, and for\nwhich there is currently no treatment.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">David Rodman, M.D., Executive Vice President of Research &amp; Development\nof ProQR, said, \u201cWe are very pleased with the data reported from the Phase 1\/2\nstudy, in which LCA10 patients treated with sepofarsen experienced a rapid and\ndurable improvement in vision. The top-line data from this study strengthen our\nconfidence in the design of the ongoing Phase 2\/3 trial, which could be the\nsole registration trial for the sepofarsen program. We appreciate the patients\u2019\nand medical communities\u2019 ongoing support for the sepofarsen clinical studies,\nand we will continue to work with the regulators to advance this program as\nefficiently as possible.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Top-line results from\nthe Phase 1\/2 trial<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Based on positive 3-month interim results from the Phase 1\/2 trial (Nature\nMedicine 2018) the Phase 2\/3<em>\nIlluminate<\/em> trial was initiated earlier this year. The 12-month\ntop-line results from the Phase 1\/2 trial that will be presented today confirm\ndurable activity of sepofarsen for up to one year in patients with LCA10.\nFurthermore, the results support the assumptions used in the design and\npowering of <em>Illuminate<\/em>,\nincluding:<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li>the target registration dose (80 \u00b5g with a 160 \u00b5g\n     loading dose) was associated with a clinically meaningful and statistically\n     significant improvement in vision and had a favorable benefit\/risk\n     profile,<\/li><li>a six-month dosing frequency, as used in <em>Illuminate,<\/em> was\n     associated with durable improvements in vision,<\/li><li>the response observed at 12 months in the target\n     registration dose was equal to or greater than the response at the 3-month\n     interim analysis,<\/li><li>and subjects with better vision than light perception\n     (BCVA&gt; LogMAR 3.0) at baseline, the study population in <em>Illuminate,<\/em> were more\n     likely to respond to treatment with sepofarsen.<\/li><\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Table: Change from\nbaseline in treated eye<\/strong><\/p>\n\n\n\n<table class=\"wp-block-table\"><tbody><tr><td>\n  <strong>Endpoint<\/strong>\n  <\/td><td>\n  <strong>Direction of improvement<\/strong>\n  <\/td><td>\n  <strong>Pooled analysis, all dose groups\n  (SEM)<\/strong>\n  <\/td><td>\n  <strong>n<\/strong>\n  <\/td><td>\n  <strong>Target registration dose group,\n  160\/80&nbsp;\u00b5g (SEM)<\/strong>\n  <\/td><td>\n  <strong>n<\/strong>\n  <\/td><\/tr><tr><td>\n  BCVA <br>\n  in LogMAR\n  <\/td><td>\n  Down \u2193\n  <\/td><td>\n  -0.55 (0.26)<br>\n  p&lt;0.05\n  <\/td><td>\n  11\n  <\/td><td>\n  -0.93 (0.43)<br>\n  p&lt;0.01\n  <\/td><td>\n  6\n  <\/td><\/tr><tr><td>\n  FST red <br>\n  in Log(cd\/m<sup>2<\/sup>)\n  <\/td><td>\n  Down \u2193\n  <\/td><td>\n  -0.92 (0.18)<br>\n  p&lt;0.01\n  <\/td><td>\n  10\n  <\/td><td>\n  -0.66 (0.14)<br>\n  p&lt;0.01\n  <\/td><td>\n  6\n  <\/td><\/tr><tr><td>\n  FST blue <br>\n  in Log(cd\/m<sup>2<\/sup>)\n  <\/td><td>\n  Down \u2193\n  <\/td><td>\n  -0.79 (0.23)<br>\n  p&lt;0.02\n  <\/td><td>\n  10\n  <\/td><td>\n  -0.63 (0.31)<br>\n  p&lt;0.01\n  <\/td><td>\n  6\n  <\/td><\/tr><tr><td>\n  Mobility course <br>\n  in Levels\n  <\/td><td>\n  Up \u2191\n  <\/td><td>\n  +2.5 (0.99)<br>\n  p=0.1\n  <\/td><td>\n  10\n  <\/td><td>\n  +4.0 (1.27)<br>\n  p&lt;0.01\n  <\/td><td>\n  6\n  <\/td><\/tr><tr><td>\n  &nbsp;\n  <\/td><td>\n  &nbsp;\n  <\/td><td>\n  &nbsp;\n  <\/td><td>\n  &nbsp;\n  <\/td><td>\n  &nbsp;\n  <\/td><td>\n  &nbsp;\n  <\/td><\/tr><\/tbody><\/table>\n\n\n\n<p class=\"wp-block-paragraph\">Analysis:&nbsp;Pooled\nanalysis: Unpaired t-test, between group change from baseline versus\nuntreated. Target registration dose group: MMRM with repeated measures,\nwithin group change from baseline versus baseline<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Visual acuity<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Statistically significant improvement from baseline in <g class=\"gr_ gr_5 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling ins-del multiReplace\" id=\"5\" data-gr-id=\"5\">best corrected<\/g> visual acuity (BCVA) was observed as assessed by the Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart and the Berkeley Rudimentary Vision Test (BRVT). In the pooled analysis with all patients after twelve months of treatment, the mean improvement (and standard error of <g class=\"gr_ gr_6 gr-alert gr_gramm gr_inline_cards gr_run_anim Grammar only-ins replaceWithoutSep\" id=\"6\" data-gr-id=\"6\">mean<\/g>, SEM) was -0.55 LogMAR (SEM 0.26). The mean change in the untreated contralateral eye was -0.11 LogMAR (SEM 0.07).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In the target registration dose group, the mean change from baseline at\ntwelve months was -0.93 LogMAR (SEM 0.43) with four of six subjects showing an\nimprovement of more than -0.3 LogMAR from baseline equivalent to 3 lines, or 15\nletters, on ETDRS chart (generally considered clinically meaningful by US\nregulators). Five of six subjects showed an improvement greater than -0.2\nLogMAR (generally considered clinically meaningful by EU regulators). The mean\nchange in the untreated contralateral eye in this group was -0.22 LogMAR (SEM\n0.11). In all five subjects in this group, in which a six-month dosing\nfrequency was explored, the observed benefit in visual acuity was maintained\nduring the twelve-month follow up.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong><g class=\"gr_ gr_5 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling ins-del multiReplace\" id=\"5\" data-gr-id=\"5\">Full field<\/g> stimulus threshold test (FST)<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Improvements in visual function were supported by a meaningful increase in the ability to detect flashes of red or blue light as determined by the FST test. In the pooled analysis after twelve months, mean improvement in red light sensitivity was -0.92 log Cd\/m<sup>2<\/sup> (SEM 0.18) and improvement in blue light sensitivity was -0.79 log Cd\/m<sup>2<\/sup> (SEM 0.23). The mean change in the untreated contralateral eye was -0.16 log Cd\/m<sup>2<\/sup> (SEM 0.16) for red light and 0.02 log Cd\/m<sup>2<\/sup> (SEM 0.11) for blue light.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In the target registration dose group, the mean change from baseline at\ntwelve months in red light sensitivity was -0.66 log Cd\/m<sup>2<\/sup> (SEM\n0.14) and improvement in blue light sensitivity was -0.63 log Cd\/m<sup>2<\/sup>\n(SEM 0.31). Three of six subjects showed an improvement of greater than -0.5\nlog Cd\/m<sup>2<\/sup> for blue light, which can be regarded as clinically\nmeaningful. Five of six showed a clinically meaningful improvement for red\nlight. The mean change in the untreated contralateral eye in this group was\n0.05 log Cd\/m<sup>2<\/sup> (SEM 0.17) for red light and -0.12 log Cd\/m<sup>2<\/sup>\n(SEM 0.16) for blue light.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Mobility <g class=\"gr_ gr_7 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling ins-del multiReplace\" id=\"7\" data-gr-id=\"7\">course<\/g><\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><g class=\"gr_ gr_7 gr-alert gr_spell gr_inline_cards gr_disable_anim_appear ContextualSpelling ins-del multiReplace\" id=\"7\" data-gr-id=\"7\">Most<\/g> patients demonstrated improvement in functional vision, as assessed using a series of mobility courses at <g class=\"gr_ gr_8 gr-alert gr_gramm gr_inline_cards gr_run_anim Grammar only-ins doubleReplace replaceWithoutSep\" id=\"8\" data-gr-id=\"8\">increasing<\/g> difficulty and multiple light intensities. In the pooled analysis the mean improvement for patients navigating the mobility course after twelve months of treatment was 2.5 levels (SEM 0.99). The mean change in the untreated contralateral eye was 1.75 (SEM 0.75). This increase was likely due to a training effect. An adjusted mobility course endpoint is being validated in parallel with the <em>Illuminate<\/em> trial.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In the target registration dose group, the mean change at twelve months of\ntreatment was 4.0 levels (SEM 1.27) with five of six subjects improving by more\nthan 2.0 levels, which can be regarded as clinically meaningful. The mean\nchange in the untreated contralateral eye was 2.7 levels (SEM 1.11).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Safety<\/strong>Subjects received up to four doses of <g class=\"gr_ gr_9 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling\" id=\"9\" data-gr-id=\"9\">sepofarsen<\/g> (range 1-4), and all eleven subjects in the study completed twelve months of follow up. This represents data equivalent to over 4,000 treatment days. Sepofarsen was observed to be well-tolerated with manageable safety findings. In total, eight cases of lens opacities (cataract) were observed (three in the target registration dose cohort and five in high dose cohort). All six of the subjects that had lens replacement surgery regained their pre-cataract vision. Four cases (in three subjects) of retinal findings were observed in the <g class=\"gr_ gr_8 gr-alert gr_spell gr_inline_cards gr_run_anim ContextualSpelling ins-del multiReplace\" id=\"8\" data-gr-id=\"8\">now retired<\/g> 320\/160 \u00b5g dose group: Two incidences of mild cystoid macular edema were resolved with topical treatment and two incidences of subclinical retinal thinning stabilized within two months of <g class=\"gr_ gr_13 gr-alert gr_gramm gr_inline_cards gr_run_anim Grammar only-ins replaceWithoutSep\" id=\"13\" data-gr-id=\"13\">last<\/g> dose without additional treatment.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Source <strong><a href=\"http:\/\/ir.proqr.com\/press-releases\">ProQR<\/a><\/strong> (press release)<\/p>\n","protected":false},"excerpt":{"rendered":"<p>ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced positive top-line results from the PQ-110-001 study, a Phase 1\/2 dose range finding, first-in-human trial of sepofarsen (QR-110) in patients with Leber\u2019s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-15645","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - 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