Applied Genetic Technologies Corporation issued the following announcement on April 18.
Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has dosed the first patient in the Company’s Phase 1/2 clinical trial evaluating the safety and efficacy of an investigational AAV-based gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP).
The multicenter study will assess AGTC’s novel recombinant AAV vector expressing a human RPGR gene (the rAAV2tYF-GRK1-RPGR) in patients with XLRP. The patient is being followed by Dr. David Birch of Retina Foundation of the Southwest and the surgery was performed by Dr. Andreas Lauer of Oregon Health & Science University.
“Dosing the first patient in our Phase 1/2 clinical study is an important step forward in advancing a new gene therapy in individuals with XLRP, a condition with no approved treatment options,” said Sue Washer, president and CEO of AGTC. “We are committed to advancing our clinical programs to deliver novel gene-based therapies for inherited orphan diseases and would like to express our deep appreciation to those patients participating in this clinical trial and their contributions to finding a potential treatment.”
The Phase 1/2 trial is an open-label, dose escalation study designed to assess the safety and efficacy of subretinal administration of the AAV-based gene therapy in approximately 15 patients diagnosed with XLRP. Trial participants will be enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 will receive a low, middle and high dose of the investigational study agent, respectively. Patients in Group 4 will receive the maximum tolerated dose as determined by the first three groups. The primary focus of the study will be to assess the safety of the vector through analysis of local (ocular) or systemic treatment-emergent adverse events. Efficacy will be measured by evaluation of changes in retinal structure, function and quality of life.
Along with the XLRS program for which we announced the completion of enrollment last week, the XLRP program is part of AGTC’s collaboration with Biogen. Under the terms of the collaboration, AGTC will receive a milestone payment of $2.5 million as a result of enrollment of the first patient in the XLRP trial.
“This is an exciting clinical milestone for one of the first potential treatments of XLRP,” said Stephen Rose, Ph.D., chief scientific officer for the Foundation Fighting Blindness (FFB). “The Foundation’s registry, My Retina Tracker, which is supported in part by a grant from AGTC, continues to provide invaluable support in identifying patients with XLRP, and other inherited retinal diseases. It allows us to inform them of clinical trials, such as this gene therapy in XLRP, that have the potential to transform their lives.”
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. Preclinical data in a canine model of XLRP caused by mutations in the RPGR gene indicate that treatment with an AAV-based gene therapy product slowed the loss of visual function.
My Retina Tracker® is an online, confidential patient registry for people affected by inherited retinal diseases. The registry collects data with the intention of using the aggregated knowledge to advance research and to help accelerate clinical trial enrollment by providing a source of information about people impacted by retinal diseases. The registry is free for people affected by inherited orphan retinal diseases, and is designed with state-of-the-art technology to protect user privacy. To learn more about My Retina Tracker, visit www.MyRetinaTracker.org or contact the registry’s coordinator at coordinator@MyRetinaTracker.org.
AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene. Orphan drug designation, covered by the U.S. Orphan Drug Act of 1983, is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 individuals. Products receiving orphan drug designation are eligible to receive market exclusivity for a period of seven years, an exemption from certain taxes and user fees and additional clinical support from FDA.
For more information on AGTC and its pipeline of AAV-based gene therapy candidates in rare disease, please visit www.agtc.com/programs.
Original source can be found here.
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