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AGTC Treats First Patient of Second Cohort in Phase 1/2 Clinical XLRP Study; Earns $10 Million Enrollment MilestoneCompany Provides Pipeline Enrollment Update for Clinical Trials in Inherited Retinal Diseases GAINESVILLE, Fla. and CAMBRIDGE, Mass., July 23, 2018 (GLOBE NEWSWIRE) -- 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 enrolled the first patient of the second cohort 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). Under the terms of its collaboration with Biogen (NASDAQ: BIIB), AGTC will receive a milestone payment of $10 million. "We are pleased to announce this important milestone under our collaboration with Biogen and remain on track to complete the dose escalation portion of the trial in the first quarter of 2019," said Sue Washer, President and CEO of AGTC. 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 patients diagnosed with XLRP caused by mutations in the RPGR gene. The primary focus of the study will be to assess the safety of the vector and subretinal delivery procedure through analysis of focal (ocular) and systemic treatment-emergent adverse events. Potential efficacy will be measured by evaluation of changes in retinal structure, visual function and quality of life. Ms. Washer continued, “In addition to achieving this milestone for our XLRP study, we are improving execution through enhanced clinical operations, clinical outreach and patient advocacy efforts to support enrollment of our three additional clinical trials. We remain grateful to our team, the investigators and the patients who have volunteered for these studies for their commitment to accelerating the development of gene therapies for inherited retinal diseases.” In addition to the XLRP study, the company has ongoing clinical trials in X-linked retinoschisis (XLRS), and achromatopsia (ACHM). XLRS Phase 1/2 Clinical Trial ACHM Phase 1/2 Clinical Trials For moe information on AGTC and its pipeline of AAV-based gene therapy candidates in rare disease, please visit www.agtc.com/programs or contact [email protected]. About X-linked Retinitis Pigmentosa (XLRP) 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. 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. About Achromatopsia (ACHM) About X-linked Retinoschisis (XLRS) About AGTC AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), and otology. The clinical-stage XLRS and XLRP programs, the discovery program in ALD and two additional ophthalmology programs are being developed in collaboration with Biogen, and the optogenetics program is being developed in collaboration with Bionic Sight. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products. Forward Looking Statements IR/PR CONTACTS: Corporate Contact: Stephen Potter |