MeiraGTx Announces Positive Clinical Data Demonstrating Treatment with AAV-RPGR Investigational Gene Therapy Improves Vision in X-Linked Retinitis Pigmentosa Patients - Seite 2

“XLRP is characterized by early-onset visual field loss, with most patients progressing to blindness and associated loss of independence by young adulthood,” said Michel Michaelides¹, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. “Six-month data demonstrate AAV-RPGR may improve visual function in XLRP patients. Initial data also suggest treatment with AAV-RPGR has the potential to stabilize or slow progressive vision loss. These results support AAV-RPGR as an important advancement in the treatment of XLRP, for which there is no currently available therapeutic option.” 

Based on the encouraging safety and efficacy data demonstrated in the MGT009 trial to date, MeiraGTx and Janssen expect to advance AAV-RPGR into the Phase 3 Lumeos clinical trial for the treatment of patients with XLRP caused by mutations in RPGR gene.

“We are pleased to share these encouraging initial results from our XLRP gene therapy trial and look forward to advancing this program into a Phase 3 trial,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “These early data suggest AAV-RPGR has the potential to address some of the key functional manifestations of this severe disease for which there is no currently available therapy. I’d like to thank the investigators, patients and families who dedicate their time to our clinical trials and who continue to support us in our efforts to develop therapies that have the potential to make a meaningful difference and improve the lives of people with serious diseases.”

Data Summary:
Data obtained to date suggest AAV-RPGR is generally well-tolerated. Most adverse events (AEs) were related to the surgical delivery procedure, were transient and resolved without intervention. There were no dose-limiting events. Inflammatory responses to therapy were observed in two out of three patients in the high dose cohort, which may have been associated with decreased activity of AAV-RPGR in these patients. Inflammation was effectively managed with an extended steroid protocol.