PARIS, Oct. 7, 2015 (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 eye diseases, today announced that data from a study evaluating the natural history of X-linked retinoschisis (XLRS) will be presented at The Retina Society 48th Annual Scientific Meeting, taking place in Paris, France from October 7-11.
"These results provided useful information to inform the design of AGTC's ongoing phase 1/2 clinical trial to evaluate a candidate AAV-based gene therapy product for treating XLRS, a rare inherited retinal disease that can lead to a significant reduction in visual acuity and other serious complications," said Sue Washer, President and CEO of AGTC. "We recently reached an enrollment milestone in our dose escalation study and plan to share initial topline safety data from the first treatment cohorts in the coming months."
The presentation, entitled "A Natural History Study of Subjects with X-linked Retinoschisis in Anticipation of a Phase I/II Gene Therapy Trial," included results from 55 participants with XLRS and confirmed mutations in the retinoschisin (RS1) gene. Patients were evaluated on cross sectional and longitudinal measures of visual acuity, visual field sensitivity, microperimetry, cyst volume and electroretinography (ERG). A subset of 20 patients were treated with a topical carbonic anhydrase inhibitor (CAI) and monitored for maculoschisis (splitting of the layers of the macula).
Preliminary data suggest that patients with XLRS show a spectrum of visual function, with baseline visual acuity ranging from 0 to 82 ETDRS letters (average = 55.5; approximately 20/60). There was a weak correlation between decreased visual acuity and age (r = -0.09). The study also found that visual field and microperimetry testing with a Goldmann Size V target were not useful because most patients showed a ceiling effect, but a smaller size III target was more effective in revealing defects. The subset of patients receiving CAI demonstrated variable individual responses, ranging from dramatic decreases in cyst volume, little change and increases in cyst volume. Changes in visual acuity were minor and weakly correlated to changes in cyst volume.
"XLRS is a rare but serious disease and there is currently incomplete information about its natural course and few validated endpoints for determining response to therapeutic intervention," said Mark E. Pennesi, M.D., Ph.D., primary study author and an ophthalmologist at Oregon Health & Science University specializing in retinal dystrophies and genetic disease. "These baseline findings are important for understanding the variability of the disease prior to any therapeutic treatment." In XLRS gene therapy studies, additional tests such as ERG assessment may be beneficial in evaluating therapeutic effect."
AGTC filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in March to conduct a Phase I/II clinical trial of the company's gene therapy product candidate for the treatment of XLRS. The XLRS clinical study is the first planned in a broad collaboration with Biogen focused on the development of gene-based therapies for orphan diseases of the retina. The collaboration also covers a development program in XLRP as well as options for three early stage discovery programs in two ophthalmic and one non-ophthalmic condition.
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases in ophthalmology. AGTC's lead product candidates focus on inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments.
AGTC has a robust product pipeline, including five named ophthalmology development programs across four targets (XLRS, XLRP, achromatopsia and wet age-related macular degeneration), one non-ophthalmology program (alpha-1 antitrypsin deficiency) and proof-of-concept data in multiple additional indications. The Company employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation and physical delivery of gene therapy products.
Forward Looking Statements
This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: uncertainty inherent in the regulatory review process; uncertainty regarding the ability to achieve the expected benefits from the proposed collaboration, including as a result of risks and uncertainties associated with drug development and commercialization, reliance on third parties over which AGTC may not always have full control and other risks associated with collaborations; and other risks and uncertainties described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2014, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.
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Source:Applied Genetic Technologies