GAINESVILLE, Fla., March 10, 2015 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq:AGTC), a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to conduct a Phase I/II clinical trial of the company's gene therapy product candidate for the treatment of X-linked retinoschisis (XLRS). Utilizing technology licensed from the University of Florida, AGTC's XLRS product candidate uses an AAV capsid with surface residues that have been specifically engineered for better penetration to the back of the eye.
"This filing is a significant milestone for AGTC and represents an opportunity to further explore the clinical utility of our novel AAV-based gene therapy for the treatment of XLRS," said Sue Washer, President and CEO. "Following favorable preclinical study results, we are excited to advance our mission to introduce new treatment options for patients with critical unmet medical needs."
Pending the FDA's acceptance of the IND application, the Company plans to initiate a clinical study evaluating the safety and efficacy of AGTC's proprietary gene therapy for treating XLRS during the second quarter of 2015 and expects to have initial data during the second half of 2015.
XLRS is an inherited retinal disease caused by mutations in the RS1 gene, which encodes the retinoschisin protein. XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual acuity in young boys, which can worsen as the patients age. There are currently no approved treatments for XLRS.
About AGTC
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 X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC is also pursuing pre-clinical development of treatments for wet AMD using the company's experience in ophthalmology to expand into disease indications with larger markets.
Forward Looking Statements
Certain statements in this press release may constitute "forward-looking statements." Such statements relate to a variety of matters, including but not limited to: the anticipated timing of clinical trials for the company's XLRS product candidate and the company's expectations regarding its ability to execute its clinical and business strategies. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of the management of the company and are subject to significant risks and uncertainty. Investors are cautioned not to place undue reliance on any such forward-looking statements. All such forward-looking statements speak only as of the date they are made, and the company undertakes no obligation to update or revise these statements, whether as a result of new information, future events or otherwise, except as required by law.
Factors that could cause actual results to differ materially from the forward-looking statements contained herein include, but are not limited to the following: clinical drug development is expensive, time consuming and uncertain; no gene therapy products have been approved in the United States and we cannot predict when or if we will obtain regulatory approval to commercialize a product candidate; we may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities; and we rely on third parties to conduct and supervise our clinical trials and to conduct certain aspects of our product manufacturing and protocol development;. Additional factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Item 1A—Risk Factors" in the company's Annual Report on Form 10-K for the fiscal year ended June 30, 2014, which was filed with the SEC on September 26, 2014.