A Phase I/II trial in patients with the inherited retinal disease X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene, and a Phase I trial in patients with geographical atrophy (GA).
Janssens’s presentation centered around the announcement of data from its Phase I/II trial (MGT009), in collaboration with clinical-stage gene therapy company MeiraGTx Holdings plc, evaluating the investigational gene therapy bortaretigene sparoparvovec in patients with XLRP associated with the eye-specific form of the RPGR gene (RPGR ORF15). Administrated as a one-time therapy bortaretigene sparoparvovec is designed to deliver functional copies of RPGR genes, with the aim to potentially preserve or restore patient vision.
XLRP causes progressive vision loss. Starting with night blindness in childhood, patients then experience a loss of peripheral vison, leading to a diagnosis of legal blindness by middle age. This rare and debilitating disease impacts an estimated 40,000 people worldwide, yet there are currently no approved treatment options.
In MGT009’s dose escalation and expansion stages those treated with botaretigene sparoparvovec, compared to the randomized untreated control arm of the study, demonstrated significantly sustained, or, increased functional improvements in the three vision domains – retinal sensitivity, visual function and functional vision, at six months’ post-treatment.
The study assessed patients’ functional vision in a visual mobility maze to determine their ability to navigate through simulated real-life obstacles across a range of controlled lights. At a 26-week follow-up greater improvements were observed in the intermediate dose cohorts, compared to the low-dose and untreated cohorts, at low levels of light.
Botaretigene sparoparvovec’s safety profile in the MGT009 study was reported as anticipated and manageable, with no does limiting events. Three serious adverse events (SAE) were reported throughout the study.
Michel Michaelides, Consultant Ophthalmologist, Moorfields Eye Hospital, Professor of Ophthalmology, University College London and lead investigator, commented that; “Individuals living with XLRP often begin to experience symptoms in childhood, and as retinal degeneration progresses toward blindness, they can start to feel a sense of hopelessness as there are no treatments to turn to. These results from the MGT009 study are promising, as they represent the potential for botaretigene sparoparvovec to preserve vision and ultimately restore hope for these patients.”
“We look forward to advancing the clinical development of botaretigene sparoparvovec as part of our mission to preserve and potentially restore vision for these patients,” added James List, Global Therapeutic Area Head, Cardiovascular, Metabolism, Retina & Pulmonary Hypertension, Janssen Research & Development.
Janssen and MeiraGTx are currently enrolling eligible participants in a Phase III study – Lumeos – to further evaluate the impact of botaretigene sparoparvovec on patients with XLRP.
Alongside this news, Janssen also presented data from a Phase I open-label, multicenter, does-escalation study NCT03144999, evaluating the safety and tolerability of a single-dose intravitreal injection of the gene therapy JNJ-1887 in patients with advanced non-exudative age-related macular degeneration (AMD) with GA.
GA is an irreversible ophthalmological condition affecting more than 5 million people globally. The progressive vision loss experienced with GA limits a patient’s ability to perform many day-to-day activities and ultimately affects their quality of life.
Study results demonstrated that patients in all three cohorts – low, intermediate, and high doses of JNJ-1887 – met the primary end-points for safety at the two-year follow-up. Additionally, data showed GA lesion growth rates to continually decline over six-month increments in patients’ treatment with JNJ-1887.
These results are the first results to be shared from Janssen’s common eye disease portfolio.
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