Results from Phase 1/2 MGT009 study demonstrate safety profile of investigational gene therapy botaretigene sparoparvovec (AAV-RPGR) and suggest sustained vision improvement in patients with X-linked retinitis pigmentosa (XLRP)
Data from a separate Phase 1 study show all three doses of investigational gene therapy JNJ-1887 met the primary endpoint for safety in adults with geographic atrophy (GA)
The Janssen Pharmaceutical Companies of Johnson & Johnson blazoned moment the primary results from the Phase1/2 study assessing the investigational gene remedy botaretigene sparoparvovec( formerly AAV- RPGR) in cases with the inherited retinal complaintX-linked retinitis pigmentosa( XLRP) associated with the retinitis pigmentosa GTPase controller( RPGR) gene. Treatment with botaretigene sparoparvovec was set up to have an respectable safety profile, and efficacity assessments in this evidence- of- conception study demonstrated encouraging advancements in retinal perceptivity, visual function and functional vision.1 These findings and fresh updates, including data from a Phase 1 trial of investigational gene remedy JNJ- 81201887( JNJ- 1887) for cases with geographic atrophy( GA), a late- stage and severe form of age- related macular degeneration( AMD), were presented in late- breaking oral donations moment at the Retina Subspecialty Day program of the American Academy of Ophthalmology( AAO) 2022 Annual Meeting( objectifications# 30071754 and# 30071749).
XLRP is a rare condition estimated to impact one in,000 people encyclopedically.2, 3 People with XLRP have progressive vision loss, starting in nonage with night blindness.4 Over time, they lose their supplemental vision leading to legal blindness by middle age.4 Botaretigene sparoparvovec is being delved in collaboration with MeiraGTx effects plc to treat cases with XLRP caused by complaint- causing variants in the eye-specific form of the RPGR( RPGR ORF15) gene. Through a one- time administration, botaretigene sparoparvovec is designed to deliver functional clones of the RPGR gene to offset the loss of retinal cells with the thing of conserving and potentially restoring vision for those living with XLRP. presently, there are no approved treatments forXLRP.4
As part of the study, cases performed a functional vision assessment using a visual mobility maze to assess their capability to navigate through dissembled real- life obstacles across a broad range of controlled light. At week 26, enhancement in walk time was observed between the treated eyes in the low and intermediate cure cohorts and the undressed eyes in the randomized concurrent control arm at low illumination situations( full analysis nominal p- value<0.05 at lux 1 and lux 16; in the perceptivity analysis when applying the Phase 3 criteria nominal p- value<0.01 at lux 1, lux 4 and lux 16).1
The safety profile of botaretigene sparoparvovec observed in MGT009 was harmonious with former reports.1 Botaretigene sparoparvovec demonstrated an adverse event( AE) profile that was anticipated and manageable.1 utmost AEs were related to the surgical delivery procedure, were flash and resolved without intervention.1 There were no cure- limiting events.1 A aggregate of three serious adverse events( SAEs) were observed in the overall Phase1/2 MGT009 clinical study; two SAEs, which were preliminarily reported, were observed in the cure- escalation phase of the study( n = 10; one retinal detachment and one panuveitis in the low cure cohort), and a single fresh SAE of increased intraocular pressure was observed in the cure escalation phase and resolved with treatment.1
” Without an approved treatment option available, people with XLRP are faced with the ineluctable fate of going eyeless in the florescence of life,” said James List,M.D.,Ph.D., Global Therapeutic Area Head, Cardiovascular, Metabolism, Retina & Pulmonary Hypertension, Janssen Research & Development, LLC.” We are in a race to save sight for these cases and are encouraged by the strength of the data that we have participated so far. We look forward to advancing the clinical development of botaretigene sparoparvovec as part of our charge to save and potentially restore vision for these cases.”
farther perceptivity analysis was conducted on study actors by applying the Phase 3 LUMEOS( NCT04671433) study eligibility criteria that corroborated the endpoints named for the Phase 3 study.1 presently, the LUMEOS study of botaretigene sparoparvovec for the treatment of cases with XLRP with complaint- causing variants in the RPGR gene is laboriously dosing cases.
About the Phase 1 JNJ- 1887 Trial and JNJ- 1887
JNJ- 81201887( JNJ- 1887), formerly appertained to as AAVCAGsCD59, is an investigational gene remedy for the treatment of people with geographic atrophy( GA) secondary to dry age- related macular degeneration( AMD). JNJ- 1887 is designed to increase the expression of a answerable form of CD59( sCD59) intended to cover retinal cells to decelerate and help complaint progression. JNJ- 1887 was estimated in a Phase 1 clinical trial( NCT03144999), an open- marker, single- center cure escalation study to determine the safety of JNJ- 1887 in grown-ups 50 or aged with advanced dry AMD with GA. The cases were treated at three raising boluses of JNJ- 1887 without steroid prophylaxis through a single intravitreal injection in one eye.
This Phase 1 study met its primary endpoint of safety in all boluses of JNJ- 1887( n = 17), with probative efficacity measures including evaluation of GA lesion growth rates, which showed a continual decline in lesion growth over six- month supplements.
JNJ- 1887 has been granted Fast Track designation by theU.S. Food and Drug Administration( FDA) and Advanced Therapy Medicinal Product( ATMP) designation by the European Medicines Agency( EMA).
About the Janssen Pharmaceutical Companies of Johnson & JohnsonAt Janssen, we are creating a future where complaint is a thing of the history. We are the Pharmaceutical Companies of Johnson & Johnson, working lifelessly to make that future a reality for cases far and wide by fighting sickness with wisdom, perfecting access with imagination, and healing forlornness with heart. We concentrate on areas of drug where we can make the biggest difference Cardiovascular, Metabolism, & Retina; Immunology; contagious conditions & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
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