REGENXBIO Inc. e phatlalalitse lintlha tse ling tse ntle tsa nakoana ho tsoa tekong e tsoelang pele ea Phase II ALTITUDE™ ea RGX-314 bakeng sa phekolo ea lefu la tsoekere la retinopathy (DR) ntle le setsi sa diabetic macular edema (CI-DME) se nang le setsi se sebelisoang ka ofising ea suprachoroidal pelehi. Lintlha li hlahisoa kopanong ea Angiogenesis, Exudation, and Degeneration 2022 ke Michael A. Klufas, MD, Retina Service, Wills Eye Hospital, Motlatsi oa Moprofesa oa Ophthalmology, Thomas Jefferson University. RGX-314 e ntse e batlisisoa e le phekolo ea nako e le 'ngoe ea liphatsa tsa lefutso bakeng sa phekolo ea ho senyeha ha macular e amanang le lilemo le DR.
Ntlafatso ea Moralo oa Boithuto le Ts'ireletseho ea Phase II ALTITUDE Teko ea RGX-314 bakeng sa Phekolo ea DR Ho Sebelisa Suprachoroidal Delivery
ALTITUDE ke teko e nang le lits'ebeletso tse ngata, e bulehileng, e sa tloaelehang, e laoloang ea ho eketseha ha lethal dose ho hlahloba katleho, ts'ireletseho le mamello ea ho fana ka suprachoroidal ea RGX-314 ho sebelisa SCS Microinjector® ho bakuli ba nang le lefu la tsoekere la DR ba nang le lefu la tsoekere le itekanetseng kapa le matla le se nang proliferative. retinopathy (NPDR) kapa mild proliferative diabetesic retinopathy (PDR). Bakuli ba mashome a mabeli ba Cohort 1 ba ne ba sa lebelloa ho fumana RGX-314 ka tekanyo ea tekanyo ea 2.5 × 1011 likopi tsa genomic ka leihlo (GC / leihlo) khahlanong le taolo ea ho shebella ka tekanyo ea 3: 1. Sehlopha sa 2 se tla kenyelletsa bakuli ba 20 ba sa tloaelehang ho amohela RGX-314 ka tekanyo e eketsehileng ea tekanyo ea 5 × 1011 GC / leihlo khahlanong le taolo ea ho shebella ka tekanyo ea 3: 1. Cohort 3 e etselitsoe ho hlahloba RGX-314 ka tekanyo ea tekanyo e lekanang le ea Cohort 2 ho bakuli ba 20 ba nang le neutralizing antibody (NAb) positive. Ho ngolisoa ho ntse ho tsoela pele ho Cohorts 2 le 3. Bakuli ba tekong ena ha ba fumane phekolo ea prophylactic immune suppressive corticosteroid pele kapa ka mor'a tsamaiso ea RGX-314.
Ho tloha ka January 18, 2022, RGX-314 e tlalehoa e mamelloa hantle ho Sehlopha sa 1. Liketsahalo tse peli tse mpe tse tebileng li tlalehiloe ho bakuli ba babeli, bao ka bobeli ba neng ba sa nkoa e le ba amanang le lithethefatsi. Har'a bakuli ba Cohort 1 ba fuoeng RGX-314, ha ho ho ruruha ha intraocular ho ileng ha bonoa. Joalokaha ho tlalehiloe pejana, mokuli e mong o ile a ba le bothata bo bobebe ba episcleritis bo ileng ba rarolloa ka topical corticosteroids. Kalafo e tloaelehileng ea mahlo e hlahileng liketsahalo tse mpe leihlong la thuto ho fihlela likhoeling tse tšeletseng li ne li sa nkoe li amana le lithethefatsi 'me hangata li ne li le bobebe. Tsena li ne li kenyelletsa ho tsoa mali ho tsoa ho conjunctival hyperemia le conjunctival hyperemia.
Kakaretso ea Lintlha tsa Sehlopha sa 1 ka Likhoeli tse Tšeletseng
Likhoeling tse ts'eletseng, ho bakuli ba 15 ba fuoeng RGX-314 ho Cohort 1, bakuli ba supileng (47%) ba bonts'itse ntlafatso ea mehato e 'meli kapa e kholoanyane ho tloha qalong ea Phekolo ea Pele ea Diabetesic Retinopathy Study-Diabetic Retinopathy Severity Scale (DRSS), Qetello ea mantlha ea thuto, ha e bapisoa le zero ho bakuli ba bahlano (0%) sehlopheng sa taolo ea tlhokomeliso. Mokuli a le mong (7%) ea nang le RGX-314 o tsoela pele ho bontša ntlafatso ea mehato e mene. Liphesente tsa bakuli ba Cohort 1 ba nang le RGX-314 ho finyella bonyane ntlafatso ea mehato e 'meli ka likhoeli tse tšeletseng ho RGX-314 mahlo a phekoloang (47%) e eketsehile ho tloha liphellong tse tlalehiloeng pele tsa likhoeli tse tharo (33%). Ntlafatso ea mehato e 'meli ho DRSS e amohetsoe e le pheletso ea bohlokoa ke FDA bakeng sa liteko tsa bongaka tsa DR.
Ho bakuli ba supileng ba neng ba e-na le NPDR (DR severity level 47-53) qalong, 57% ea bakuli ba bontšitse ntlafatso ea mehato e 'meli kapa e kholoanyane ho tloha qalong ea DRSS likhoeli tse tšeletseng ka mor'a tsamaiso ea RGX-314. Ho bakuli ba robeli ba neng ba e-na le PDR (DR severity level ≥ 61) qalong, 38% ea bakuli ba bontšitse ntlafatso ea mehato e 'meli kapa e kholoanyane likhoeling tse tšeletseng ka mor'a tsamaiso ea RGX-314.
Likhoeli tse tšeletseng ka mor'a tsamaiso ea RGX-314, bakuli ba Cohort 1 ba ile ba bontša phetoho e tsitsitseng ea moelelo ho BCVA ea + 0.3 litlhaku ha li bapisoa le tsa motheo, ha bakuli ba bahlano ka letsoho la taolo ea ho shebella ba bontšitse phetoho e tsitsitseng ea moelelo ho BCVA ea -2.0 litlhaku ha li bapisoa le tsa motheo.
