AGTC Files Investigational New Drug Application for the Treatment of Achromatopsia...
Monday, November 2, 2015
Nov. 2, 2015
AGTC Files Investigational New Drug Application for the Treatment of Achromatopsia Caused by Mutations in the CNGB3 Gene
GAINESVILLE, Fla., Nov. 2, 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 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 achromatopsia caused by mutations in the CNGB3 gene.
"This is another significant milestone for AGTC during what has been a transformative year for the company," said Sue Washer, President and CEO. "In addition to finalizing our collaboration agreement with Biogen, initiating our phase 1/2 clinical trial for XLRS, and expanding our product pipeline, we remain on track to have a second product candidate in clinic shortly, pending the FDA's acceptance of the IND application and approval from the sites' Institutional Review Boards."
The Company plans to initiate a clinical study evaluating the safety and efficacy of AGTC's proprietary gene therapy for treating achromatopsia caused by mutations in the CNGB3 gene at four sites in the United States.
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind. The incidence rate for achromatopsia is approximately one in 30,000 people, and it is estimated that there are approximately 10,000 people in the United States and 17,000 people in Europe with achromatopsia. Mutations in the CNGB3 gene cause the disease in approximately 50% of patients with achromatopsia.
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.
CONTACT: David Carey (IR) or Danielle Lewis (PR) Lazar Partners Ltd.
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Larry Bullock, Chief Financial Officer, Applied Genetic Technologies Corporation
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