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AGTC Announces Orphan Medicinal Product Designation in the European Union for Gene Therapy to Treat

Tuesday, June 07, 2016   (0 Comments)
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GAINESVILLE, Fla., and CAMBRIDGE, Mass., June 07, 2016 (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 the European Commission (EC) has granted an orphan medicinal product designation to its investigational gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

XLRP is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision. Affected men become legally blind at an average of about 45 years of age. The most common form of XLRP is caused by mutations in the RPGR gene. Preclinical data indicate that treatment with a gene therapy product slowed the loss of visual function in canines with XLRP caused by mutations in the RPGR gene. XLRP is one of two lead development programs within AGTC’s collaboration agreement with Biogen, announced in July 2015, along with X-linked retinoschisis.

“Receiving orphan medicinal product designation from the EC for our XLRP gene therapy candidate is an important milestone for AGTC, and a positive step forward in our ongoing efforts to develop effective treatments for rare inherited retinal diseases,” said Sue Washer, President and CEO of AGTC. “With many of our programs in or nearing clinical trials, this is an exciting time for the company and patients affected with ocular diseases with no currently available treatments.”

AGTC has already been granted orphan drug designation from the EC and the U.S. Food and Drug Administration (FDA) for its gene therapy product candidates for the treatment of X-linked retinoschisis and for the treatment of achromatopsia caused by mutations in the CNGA3 and CNGB3 genes.

Orphan medicinal product designation is granted by the European Commission, following a positive opinion from the Committee for Orphan Medicinal Products (COMP), to a medicinal product that is intended for the diagnosis, prevention or treatment of a life-threatening or a chronically debilitating condition affecting not more than five in 10,000 persons in the European Community when the application for designation is submitted. An orphan designation allows a company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market.

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, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments.

AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications.  The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years.  AGTC 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 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

This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. These statements relate to a variety of matters, including but not limited to, the anticipated utility of AGTC's product candidates in the treatment of XLRP and other therapeutic indications and the anticipated development of additional product candidates for the treatment of genetic defects in the cells in the inner ear. 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, which include, but are not limited to, the following: no gene therapy products have been approved in the United States and AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; AGTC relies on third parties to conduct research, conduct, supervise and monitor its clinical trials and to conduct certain aspects of its product manufacturing and protocol development; and increased regulatory scrutiny of gene therapy and genetic research could damage public perception of AGTC's product candidates or adversely affect AGTC's ability to conduct its business. 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 AGTC's Annual Report on Form 10-K for the fiscal year ended June 30, 2015, 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, AGTC assumes 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.

IR/PR CONTACTS: David Carey (IR) or Danielle Lewis (PR) 
Lazar Partners Ltd. T: (212) 867-1768 or (212) 843-0211 or 
Chief Financial Officer 
Applied Genetic Technologies Corporation 
T: (386) 462-2204 
Stephen Potter 
Chief Business Officer 
Applied Genetic Technologies Corporation 
T: (617) 413-2754

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